Perceptive Advisors

Merida Biosciences is a biotechnology company specializing in the development of precision therapies for autoimmune diseases. It employs a protein engineering platform to create novel Fc therapeutics, which selectively target and neutralize specific antibodies, aiming to treat the root cause of antibody-driven diseases with high precision and durability.

Caris Life Sciences, Inc. is a biotechnology company specializing in molecular profiling and advanced diagnostic technologies aimed at improving cancer patient outcomes. Founded in 1987 and headquartered in Irving, Texas, with additional offices in Phoenix and Basel, the company develops solutions that leverage artificial intelligence and machine learning to analyze the molecular complexity of diseases. Caris offers a range of services, including anatomic pathology, drug development, and molecular analyses of patient samples, which include genomic, transcriptomic, and proteomic profiling. Their Comprehensive Oncology Data Explorer serves as a clinico-genomic data platform. By providing insights into DNA, RNA, and protein profiles, Caris enables healthcare professionals to make more precise and personalized treatment decisions for oncology and other complex diseases. The company operates through distributors across the United States, Europe, Australia, and globally, and has established strategic collaborations to enhance its offerings.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

RAPT Therapeutics, Inc., established in 2015 and headquartered in South San Francisco, California, is a clinical-stage biopharmaceutical company specializing in immunology-based therapies. The company focuses on developing oral small molecule treatments for patients with unmet needs in oncology and inflammatory diseases. Its lead drug candidates are FLX475, an oral C-C motif chemokine receptor 4 antagonist in Phase 2 clinical trials for various cancer types, and RPT193, which selectively inhibits type 2 T helper cell migration into inflamed tissues. Additionally, RAPT is exploring other targets like general control nonderepressible 2 and hematopoietic progenitor kinase 1 in the discovery phase.

Inventiva Pharma is a clinical-stage biopharmaceutical company based in France, dedicated to developing oral small molecule therapies for patients with significant unmet medical needs, particularly in fibrosis, lysosomal storage disorders, and oncology. The company specializes in drugs that target nuclear receptors, transcription factors, and epigenetic modulation. Its primary product candidate, lanifibranor, is being developed for the treatment of non-alcoholic steatohepatitis (NASH), a condition currently lacking approved therapies. Inventiva has established a robust pipeline supported by a proprietary discovery engine, a dedicated research and development facility, and a skilled team with extensive experience in drug development. In addition to advancing lanifibranor, the company is also working on several earlier-stage programs aimed at addressing various medical conditions. All operations are conducted in France.

Bicycle Therapeutics is a clinical-stage biopharmaceutical company focused on developing a novel class of medicines known as Bicycles, which are synthetic short peptides designed to stabilize their structural geometry through two loops. The company primarily targets oncology indications with significant unmet medical needs. Its lead product candidate, BT1718, is a Bicycle Toxin Conjugate currently undergoing Phase I/IIa clinical trials aimed at tumors expressing Membrane Type 1 matrix metalloprotease. Other oncology candidates include BT5528, also in Phase I/IIa trials targeting EphA2, and BT8009, which is in preclinical studies for Nectin-4. Additionally, Bicycle Therapeutics is developing THR-149, a plasma kallikrein inhibitor that has completed Phase I trials for diabetic macular edema. The company collaborates with various biopharmaceutical organizations to advance programs in areas like anti-infectives and cardiovascular health. Founded in 2009, Bicycle Therapeutics is headquartered in Cambridge, United Kingdom.

Endeavor BioMedicines is a clinical-stage company based in Solana Beach, California, that focuses on developing innovative treatments for pulmonary fibrosis, particularly idiopathic pulmonary fibrosis (IPF). Founded in 2018, the company is working on a small-molecule inhibitor designed to target the underlying causes of this progressive lung disease. The treatment aims to not only halt the progression of IPF but also has the potential to reverse its effects, offering healthcare professionals a new avenue for precision treatments in managing terminal conditions. Endeavor BioMedicines strives to improve patient outcomes and enhance quality of life for those affected by this challenging illness.

TORL BioTherapeutics is a clinical-stage biopharmaceutical company dedicated to developing innovative antibody-based medicines aimed at improving outcomes for cancer patients. The company specializes in creating antibody-drug conjugates (ADCs) and monoclonal antibodies (mAbs) to address oncologic diseases that exhibit significant unmet medical needs. TORL BioTherapeutics employs a strategic approach that includes target identification and early discovery work, allowing them to license promising drug programs and develop proprietary therapies with optimized characteristics. Through this pipeline, TORL BioTherapeutics is positioned to advance novel treatments that could significantly enhance patient care in oncology.

Zevra Therapeutics is a development-stage biopharmaceutical company focused on creating new therapies for pain, ADHD, and other central nervous system (CNS) diseases, particularly in the realm of rare diseases. The company is dedicated to developing safer, abuse-resistant opioid pain relievers and employs a proprietary Ligand Activated Therapy (LAT) approach to drug discovery and development. This innovative platform allows Zevra to enhance existing medications and streamline the development process while preserving strong intellectual property rights. By integrating scientific research with patient needs and data-driven strategies, Zevra Therapeutics aims to address the challenges of drug development, delivering transformational therapies for conditions with limited or no treatment options.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on precision oncology, developing therapeutics that target challenging cancer types without straightforward single-gene mutations. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond to specific treatments. Acrivon's pipeline includes ACR-368, also known as prexasertib, which is currently in Phase 2 trials, alongside preclinical programs aimed at key components of DNA damage response and cell cycle regulation, such as the WEE1 and PKMYT1 proteins. Acrivon seeks to expedite the development of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials, aiming to enhance treatment success in oncology.

enGene, Inc. is a biotechnology company specializing in mucosal immunotherapy platforms aimed at treating inflammatory bowel disease and diabetes. The company has developed a non-integrating biopolymer-based nucleotide delivery technology that enables localized delivery of immune-modulating proteins to mucosal tissues, including the gastrointestinal tract, lung, and bladder. This innovative approach allows for the treatment of various immune disorders and supports systemic release of proteins from the gut to address conditions such as diabetes, anemia, and hemophilia. Founded in 1999 and based in Vancouver, Canada, enGene has formed a strategic alliance with Takeda Pharmaceutical Company Ltd. The company's platform facilitates the induction or suppression of protein expression levels, which can help regenerate physiologic, meal-regulated insulin secretion for diabetes patients.

Cogent Biosciences is a biotechnology company dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Soleno Therapeutics, Inc. is a clinical-stage biopharmaceutical company dedicated to the development and commercialization of innovative therapeutics for rare diseases. The company's primary focus is on its lead candidate, Diazoxide Choline Controlled Release (DCCR), a once-daily oral tablet designed for the treatment of Prader-Willi Syndrome (PWS). DCCR contains diazoxide choline, a choline salt of diazoxide, which is a benzothiadiazine. Currently, the product is being evaluated in a Phase III clinical development program. Established in 1999 and headquartered in Redwood City, California, Soleno Therapeutics was formerly known as Capnia, Inc. before rebranding in May 2017.

Forte Biosciences is a biotechnology company specializing in dermatology and biopharmaceuticals. The company's lead product, FB-401, is a topically applied live biotherapeutic designed to treat inflammatory skin diseases, developed in collaboration with the National Institutes of Health and the National Institute of Allergy and Infectious Diseases. Additionally, Forte Biosciences is advancing another product candidate, FB-102, which is a proprietary therapeutic molecule aimed at addressing a range of autoimmune conditions, including graft-versus-host disease, vitiligo, and alopecia areata. The company is currently in the preclinical trial stage for FB-102, reflecting its commitment to developing innovative treatments for dermatological and autoimmune disorders.

AsclepiX Therapeutics, Inc. is a biopharmaceutical company focused on the design and development of innovative treatments for diseases related to abnormal blood vessel growth, specifically through the use of short biomimetic anti-angiogenic and anti-lymphangiogenic peptides. The company's lead candidates, including AXT107, AXT201, AXT301, and AXT501, target conditions such as diabetic macular edema, wet age-related macular degeneration, and macular edema following retinal vein occlusion, which are among the leading causes of blindness. Additionally, AXT201, AXT301, and AXT501 are being explored for their potential in treating various solid tumors. AsclepiX leverages advanced computational biology, bioinformatics, and the latest developments in biomaterials to enhance drug delivery systems, aiming to create long-lasting biodegradable nanoparticles that can effectively deliver its therapeutic peptides. Incorporated in 2011 and based in Baltimore, Maryland, AsclepiX Therapeutics is committed to addressing unmet medical needs in ophthalmology, oncology, and other diseases driven by angiogenesis and lymphangiogenesis.

A-Alpha Bio, Inc. is a biotechnology company based in Seattle, Washington, established in 2017. The company specializes in synthetic biology, offering a platform known as AlphaSeq, which assists pharmaceutical companies in the development of drugs that interact with multiple targets. A-Alpha Bio provides cell-based tools for high-throughput and quantitative measurements of protein interactions, facilitating target discovery, library screening, and preclinical drug characterization. Its proprietary technology enables the mapping of protein-protein interactions at a large scale, allowing for the identification and quantification of interactions among thousands of protein pairs simultaneously. By delivering detailed maps of these interactions, A-Alpha Bio helps researchers identify potential drug targets and understand disease mechanisms, thereby streamlining the therapeutic discovery process and improving the efficiency of drug development. The company comprises a team of experts in synthetic biology, structural biology, and next-generation sequencing, focused on overcoming challenges in the drug development pipeline.

TORL BioTherapeutics is a clinical-stage biopharmaceutical company dedicated to developing innovative antibody-based medicines aimed at improving outcomes for cancer patients. The company specializes in creating antibody-drug conjugates (ADCs) and monoclonal antibodies (mAbs) to address oncologic diseases that exhibit significant unmet medical needs. TORL BioTherapeutics employs a strategic approach that includes target identification and early discovery work, allowing them to license promising drug programs and develop proprietary therapies with optimized characteristics. Through this pipeline, TORL BioTherapeutics is positioned to advance novel treatments that could significantly enhance patient care in oncology.

Biodesix, Inc. is a diagnostic solutions company based in Boulder, Colorado, specializing in blood-based tests for lung cancer and related services. The company offers a range of diagnostic tests, including the Nodify XL2 and Nodify CDT tests, which assist physicians in assessing the risk of malignancy in patients with suspicious lung nodules. Additionally, Biodesix provides tumor profiling and immune profiling tests, such as GeneStrat and VeriStrat, to support timely treatment decisions for oncologists. These tests are processed in a CLIA-certified laboratory, with results typically available within 72 hours. Beyond its diagnostic offerings, Biodesix also engages in clinical research, development, and testing services for biopharmaceutical companies, focusing on the development and commercialization of companion diagnostics to enhance therapeutic efficacy. Established in 2005, Biodesix continues to advance its proprietary mass spectrometry-based discovery platform to improve patient outcomes in lung disease.

immatics is a biopharmaceutical company focused on the discovery of true targets for cancer immunotherapies with the development of the right T cell receptors. The company's customized Adoptive Cell Therapies (ACTengine) and antibody-like TCR Bispecifics are the two main product classes in the pipeline (TCER). With a focus on particularly difficult-to-treat solid malignancies, each therapy modality has unique characteristics to create the desired therapeutic impact for patients at various disease stages and with various types of tumors. immatics biotechnologies was established in 2000 by Hans Rammensee, Harpreet Singh, and Steffen Walter in Tübingen, Baden-Wurttemberg.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Surf Bio is a preclinical biopharmaceutical company focused on developing innovative therapeutic solutions using a novel biotechnology platform. This platform centers around a next-generation surfactant designed to enhance drug effectiveness for a variety of medical conditions, including diabetes, oncology, infectious diseases, and gene therapy. By improving therapeutic applications, Surf Bio aims to provide healthcare organizations worldwide with access to new and more effective treatments for patients facing critical health issues.

Synthego Corporation is a genome engineering company focused on advancing life science research and therapeutic development to improve human health. Founded in 2012 and headquartered in Redwood City, California, Synthego offers a comprehensive platform that integrates proprietary hardware, software, bioinformatics, chemistries, and molecular biology. The company's product portfolio includes engineered cells, CRISPR kits, and bioinformatics tools designed for various applications such as pathway analysis, target validation, disease modeling, and diagnostics. By leveraging machine learning and automation, Synthego enables researchers in biotechnology and academia to conduct efficient and precise gene editing, facilitating rapid advancements in drug discovery and development. The company serves a global customer base, distributing its products through various channels, including online sales and partnerships in multiple countries.

Endeavor BioMedicines is a clinical-stage company based in Solana Beach, California, that focuses on developing innovative treatments for pulmonary fibrosis, particularly idiopathic pulmonary fibrosis (IPF). Founded in 2018, the company is working on a small-molecule inhibitor designed to target the underlying causes of this progressive lung disease. The treatment aims to not only halt the progression of IPF but also has the potential to reverse its effects, offering healthcare professionals a new avenue for precision treatments in managing terminal conditions. Endeavor BioMedicines strives to improve patient outcomes and enhance quality of life for those affected by this challenging illness.

Ceptur Therapeutics is focused on developing innovative genetic medicines aimed at addressing diseases that currently lack effective treatments. The company utilizes its proprietary U1 Adaptor technology, which enables long-lasting and non-genotoxic silencing of disease-causing genes, particularly in challenging tissues beyond the liver and central nervous system. This technology addresses the limitations of existing gene silencing methods and holds promise for cancer treatment, allowing healthcare providers to offer new therapeutic options for patients with various types of cancers.

AvengeBio is a biotechnology company based in Cambridge, Massachusetts, focused on developing cell-generated immunotherapies aimed at eradicating solid tumors. Founded in 2019, the company is advancing a drug delivery platform designed to enable the precision delivery of cytokines and other potent immunomodulatory molecules. This innovative approach allows for controlled release over time, fostering robust local and systemic anti-tumor immune responses while minimizing the toxicities typically associated with conventional immunotherapies. Through its research and development efforts, AvengeBio seeks to address the challenges posed by intractable solid tumors and improve treatment outcomes for cancer patients.

Sermonix Pharmaceuticals is a biotechnology company based in Columbus, Ohio, founded in 2014. The company specializes in the development and commercialization of female-specific oncology products, focusing on late-stage women’s health treatments. Its primary product, lasofoxifene, is a selective estrogen receptor modulator designed for the treatment of vulvovaginal atrophy and the treatment and prevention of post-menopausal osteoporosis. Additionally, Sermonix is involved in developing pharmaceutical drugs aimed at treating ESR1-mutated metastatic breast and gynecological cancers, thereby addressing critical needs in women's health and oncology.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on precision oncology, developing therapeutics that target challenging cancer types without straightforward single-gene mutations. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond to specific treatments. Acrivon's pipeline includes ACR-368, also known as prexasertib, which is currently in Phase 2 trials, alongside preclinical programs aimed at key components of DNA damage response and cell cycle regulation, such as the WEE1 and PKMYT1 proteins. Acrivon seeks to expedite the development of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials, aiming to enhance treatment success in oncology.

A-Alpha Bio, Inc. is a biotechnology company based in Seattle, Washington, established in 2017. The company specializes in synthetic biology, offering a platform known as AlphaSeq, which assists pharmaceutical companies in the development of drugs that interact with multiple targets. A-Alpha Bio provides cell-based tools for high-throughput and quantitative measurements of protein interactions, facilitating target discovery, library screening, and preclinical drug characterization. Its proprietary technology enables the mapping of protein-protein interactions at a large scale, allowing for the identification and quantification of interactions among thousands of protein pairs simultaneously. By delivering detailed maps of these interactions, A-Alpha Bio helps researchers identify potential drug targets and understand disease mechanisms, thereby streamlining the therapeutic discovery process and improving the efficiency of drug development. The company comprises a team of experts in synthetic biology, structural biology, and next-generation sequencing, focused on overcoming challenges in the drug development pipeline.

Emulate, Inc. specializes in organ-on-chip technology that simulates human biology to enhance the understanding of how diseases, medications, chemicals, and food impact human health. The company develops organ-chips for various organs, including the lung, intestine, liver, and skin, which replicate normal functions and disease conditions. Emulate's innovative technology offers researchers a more accurate alternative to traditional experimental methods, such as cell cultures and animal testing, by providing detailed insights into human biological responses. Additionally, the company supplies drug development and instrumentation software to facilitate research and development workflows for its clients. Emulate serves a diverse range of sectors, including biotechnology, pharmaceuticals, consumer health, cosmetics, chemicals, food, agrochemicals, government agencies, and academic institutions. Founded in 2013 and headquartered in Boston, Massachusetts, Emulate was previously known as Emulate Living MicroDevices, Inc. until its name change in 2014.

SomaLogic, Inc. is a commercial-stage company based in Boulder, Colorado, specializing in protein biomarker discovery and clinical diagnostics. Founded in 1999, the company has developed advanced technologies such as SOMAmers, slow off-rate modified aptamers that specifically bind to target proteins, and the SOMAscan platform, which facilitates comprehensive protein measurement. This innovative approach allows for the analysis of protein biomarker signatures, significantly enhancing the understanding of health and disease beyond traditional genomic methods. SomaLogic's products are utilized in a variety of applications, including diagnostics for conditions in oncology, neurology, and cardiovascular and metabolic diseases, as well as in drug discovery and development. The company also offers diagnostic tests and protein measurement solutions for life science and biopharma researchers, contributing to advancements in medical science.

Bridge to Life is a U.S.-based company headquartered in Northbrook, Illinois, dedicated to enhancing organ transplantation through the development and licensing of innovative preservation solutions and technologies. As a leading supplier of preservation products worldwide, the company focuses on advancing organ preservation and machine perfusion techniques, which are crucial for the effective storage of intra-abdominal organs. By collaborating closely with surgeons and transplant professionals, Bridge to Life continually explores emerging scientific advancements and preservation technologies to improve the quality of service provided to organ procurement organizations and transplant centers.

Bridge to Life is a U.S.-based company headquartered in Northbrook, Illinois, dedicated to enhancing organ transplantation through the development and licensing of innovative preservation solutions and technologies. As a leading supplier of preservation products worldwide, the company focuses on advancing organ preservation and machine perfusion techniques, which are crucial for the effective storage of intra-abdominal organs. By collaborating closely with surgeons and transplant professionals, Bridge to Life continually explores emerging scientific advancements and preservation technologies to improve the quality of service provided to organ procurement organizations and transplant centers.

GeneDx is a company dedicated to providing personalized and actionable health insights that enhance diagnosis and treatment, ultimately improving health outcomes. Positioned at the intersection of diagnostics and data science, GeneDx combines decades of genomic expertise with the capability to interpret clinical data at scale. The company focuses on accelerating the application of genomic information and large-scale clinical data to establish precision medicine as a standard practice in healthcare. GeneDx leads the transformation of healthcare through its comprehensive exome and genome testing services, leveraging one of the largest rare disease data sets in the world. The business operates through two segments: GeneDx, which generates the majority of its revenue, and Legacy Sema4 diagnostics.

Amylyx Pharmaceuticals develops innovative therapies for Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases. The company's primary product, AMX0035, is an investigational therapy that combines two active compounds, sodium phenylbutyrate and Taurursodiol. This fixed-dose formulation aims to address mitochondrial energy crises and the accumulation of toxic proteins in the endoplasmic reticulum, which contribute to neurological decline. In addition to ALS, Amylyx's research pipeline includes potential treatments for Wolfram syndrome and Alzheimer's disease. Founded in 2013 and based in Cambridge, Massachusetts, Amylyx Pharmaceuticals is dedicated to advancing solutions for serious brain disorders.

RayzeBio, Inc. is a biotechnology company based in San Diego, California, founded in 2020. It specializes in developing tumor-targeted small molecule medicines that utilize the therapeutic potential of radioisotopes, particularly Actinium-225, an alpha-emitting radioisotope aimed at treating solid tumors. RayzeBio is focused on enhancing cancer treatment outcomes by creating a robust pipeline of radiopharmaceutical candidates targeting validated oncology drug targets. The company's portfolio includes a range of drug candidates at various stages of development, from discovery to late-stage clinical programs, addressing significant market opportunities in the field of cancer therapeutics.

Nautilus Biotechnology is a development-stage life sciences company focused on advancing proteomics, the study of proteins and their functions, to enhance therapeutic development and medical diagnostics. The company has created a platform technology that aims to quantify and unravel the complexities of the human proteome. Its flagship product, the Proteomic Analysis System, is a prototype single-molecule instrument designed to facilitate these efforts. The team at Nautilus comprises experts from various disciplines, including protein chemistry, molecular biology, and data science, enabling a multifaceted approach to overcoming challenges in the field. By harnessing this diverse expertise, Nautilus aims to revolutionize drug development and improve healthcare management through personalized and predictive medicine.

Affinia Therapeutics Inc. is focused on developing gene therapies for various diseases, particularly those affecting the muscles and central nervous system. The company specializes in creating adeno-associated virus (AAV) vectors, leveraging advanced techniques in synthetic and systems biology, as well as high-throughput screening and tissue and single-cell resolution. Founded in 2019 and based in Waltham, Massachusetts, Affinia aims to address significant unmet medical needs by providing rationally designed gene therapies with improved properties. The company's innovative approach seeks to offer effective treatments for devastating conditions that currently lack adequate therapeutic options.

Icosavax, Inc. is a biotechnology company based in Seattle, Washington, founded in 2017. The company specializes in developing vaccines using its innovative virus-like particle (VLP) technology platform, which allows for the multivalent display of complex viral antigens. This technology aims to provide broad and durable protection against various infectious diseases, with an initial focus on life-threatening respiratory illnesses. Icosavax's pipeline includes vaccine candidates targeting respiratory syncytial virus (RSV), human metapneumovirus (hMPV), and SARS-CoV-2. The company is dedicated to advancing its VLP technology to discover, develop, and ultimately commercialize effective vaccines against these infectious diseases.

Scribe Therapeutics Inc. is a biotechnology company focused on developing innovative therapeutics based on CRISPR technology to address genetic disorders. Founded in 2017 and headquartered in Berkeley, California, the company utilizes its proprietary X-Editing (XE) platform, which features highly engineered CRISPR enzymes designed to improve the efficacy, specificity, and deliverability of genome editing. Scribe Therapeutics aims to overcome existing limitations in genetic medicine by creating custom-engineered enzymes and advanced delivery systems. Their approach seeks to establish CRISPR-based therapies as a new standard in clinical care, potentially transforming the treatment landscape for various conditions, including immuno-oncology, degenerative disorders, and enzyme replacement therapy. By prioritizing therapeutic precision and allele-specific targeting, Scribe Therapeutics is committed to expanding access to groundbreaking treatments that can significantly impact patient lives.

Pyxis Oncology, established in 2019 in Boston, Massachusetts, is a biotechnology company specializing in the development of antibody therapeutics for cancer treatment. The company focuses on promoting the body's immune response to cancer by analyzing tumor antigen-specific tumor-infiltrating lymphocytes (TILs) within hot tumors and tumor cell signaling pathways within cold tumors. Pyxis aims to create novel antibody-based immunotherapies to directly kill tumor cells and address underlying pathologies that enable cancer proliferation and immune evasion, with the goal of improving patient outcomes for difficult-to-treat cancers.

Absci is a drug and target discovery company that leverages deep learning AI and synthetic biology to enhance the therapeutic potential of proteins. Its Integrated Drug Creation Platform streamlines the process of identifying novel drug targets, discovering optimal biotherapeutic candidates, and generating the necessary cell lines for manufacturing. By combining biologic drug discovery and cell line development into a single, efficient workflow, Absci collaborates with biotech and pharmaceutical innovators to develop next-generation protein-based drugs. These innovations include Bionic Proteins, which feature nonstandard amino acids, and other unique drug designs that may be unattainable through conventional methods. Absci's mission is to facilitate the development of improved medicines by effectively translating innovative ideas into viable drugs.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that specializes in targeted DNA integration to develop innovative therapies for severe diseases. The company utilizes a precision gene editing approach to precisely correct genetic mutations, replace malfunctioning genes, and insert therapeutic genetic sequences at specific locations within the genome. Its technology aims to transform treatment paradigms by enabling the targeted integration of genetic payloads, which could potentially cure a range of serious and life-threatening conditions. Graphite Bio was formerly known as Integral Medicines, Inc. before rebranding in August 2020, and it was incorporated in 2019.

Pipeline Therapeutics Inc. is a biotechnology company focused on developing and commercializing small molecules aimed at neuroregeneration, which includes promoting synaptogenesis, remyelination, and axonal repair. Based in San Diego, California, the company’s lead product candidate, PIPE-505, is a gamma secretase inhibitor designed to treat mild-to-moderate sensorineural hearing loss associated with cochlear synaptopathy. Additionally, Pipeline Therapeutics is advancing earlier-stage programs, such as PIPE-307, which target remyelination and axonal repair to address various neurological disorders, including multiple sclerosis. Founded in 2017, the company aims to harness the body's natural repair mechanisms to facilitate recovery from neurological damage.

Affinivax, Inc. is a biotechnology company based in Lexington, Massachusetts, focused on developing innovative vaccines using its proprietary Multiple Antigen Presenting System (MAPS) technology. This approach allows for the combination of protective polysaccharides and proteins into a single vaccine, thereby enhancing immune responses against a variety of infectious diseases affecting both children and adults. Affinivax targets several significant pathogens, including Streptococcus pneumoniae, bacterial nosocomial infections, Salmonella typhi, and Staphylococcus aureus. The company aims to create vaccines that not only provide broader disease coverage compared to existing options but also address infectious diseases lacking effective immunization strategies. Founded in 2014, Affinivax benefits from the expertise of leading professionals in infectious diseases and vaccines, and it has received backing from the Bill & Melinda Gates Foundation, along with an exclusive license from Boston Children’s Hospital for its MAPS technology.

IsoPlexis is a life science technology company based in Branford, Connecticut, founded in 2013. It focuses on developing a single-cell detection system that identifies patient immune responses at the single-cell level. This innovative platform enables the assessment of therapeutic safety and efficacy, as well as the monitoring of disease progression. IsoPlexis specializes in cell-based proteomic systems and biomarkers, providing insights into functional protein biology and cellular signaling networks with high resolution. By offering these capabilities, the company aids in predicting patient responses to novel immunotherapies and cellular therapies, ultimately enhancing therapeutic development and informing clinical decisions.

IsoPlexis is a life science technology company based in Branford, Connecticut, founded in 2013. It focuses on developing a single-cell detection system that identifies patient immune responses at the single-cell level. This innovative platform enables the assessment of therapeutic safety and efficacy, as well as the monitoring of disease progression. IsoPlexis specializes in cell-based proteomic systems and biomarkers, providing insights into functional protein biology and cellular signaling networks with high resolution. By offering these capabilities, the company aids in predicting patient responses to novel immunotherapies and cellular therapies, ultimately enhancing therapeutic development and informing clinical decisions.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

LianBio is a biopharmaceutical company based in Shanghai, China, with an additional office in Princeton, New Jersey. Founded in 2019, the company focuses on the discovery and development of therapeutic drugs aimed at treating oncology and cardiorenal diseases, as well as other medical conditions. LianBio's mission is to enhance access to innovative medicines by partnering with leading organizations in the field to leverage advanced scientific discoveries. The company is dedicated to addressing unmet medical needs in Greater China and other Asian markets by in-licensing assets and building a diverse pipeline of clinically validated product candidates. Its portfolio aims to establish new standards of care across various therapeutic areas, including cardiovascular, oncology, ophthalmology, inflammatory diseases, and respiratory conditions.

AavantiBio, Inc. is a biopharmaceutical company based in Gainesville, Florida, focused on developing gene transfer and gene editing therapies for rare genetic diseases. Established in 2019, the company primarily targets Friedreich’s Ataxia, a severe inherited condition that leads to significant cardiac and central nervous system dysfunction. In addition to addressing this specific disorder, AavantiBio is advancing a pipeline of innovative gene therapies aimed at treating various autoimmune diseases and other conditions with unmet medical needs. Through its commitment to research and development, the company seeks to improve the lives of patients affected by these debilitating genetic disorders.

Nuvation Bio Inc. is a biopharmaceutical company dedicated to addressing unmet needs in oncology through the development of novel therapies for challenging cancers. Founded in 2018 and headquartered in New York, with an additional office in San Francisco, Nuvation Bio focuses on treating patients whose conditions have not responded to conventional treatments. The company's leading clinical-stage candidate, NUV-868, is a BD2-selective oral small molecule BET inhibitor that targets the BRD4 protein, which plays a crucial role in regulating tumor growth and differentiation. In addition to NUV-868, Nuvation Bio is advancing a proprietary small molecule Drug-Drug Conjugate platform aimed at enhancing therapeutic efficacy. The company’s comprehensive portfolio includes multiple drug development candidates designed to meet the specific needs of cancer patients.

Metabolon, Inc. is a health technology company specializing in metabolomics, focusing on the development of diagnostic tests and analytical methods for early disease detection and precision medicine. The company offers a range of clinical diagnostics, including QUANTOSE IR for measuring insulin resistance, QUANTOSE IGT for assessing glucose tolerance, and Meta IMD for identifying metabolites linked to inherited metabolic disorders. In addition to its diagnostic capabilities, Metabolon provides customizable research services and multiomics solutions to support academic and commercial investigations across various life science sectors. Its technologies are applicable in areas such as cardiovascular health, diabetes, oncology, and respiratory disorders. Founded in 2000 and headquartered in Morrisville, North Carolina, with additional offices in Europe and sales locations in the UK, Spain, and China, Metabolon collaborates with numerous strategic partners in the pharmaceutical and biotechnology industries to enhance research and development outcomes.

I-Mab Biopharma (Shanghai) Co., Ltd. is a clinical-stage biopharmaceutical company based in Shanghai, China, with additional offices in Beijing and Rockville, Maryland. Founded in 2016, the company focuses on the discovery, development, and commercialization of innovative biologics aimed at addressing significant unmet medical needs, particularly in the fields of immuno-oncology and immuno-inflammation. I-Mab's diverse drug pipeline targets various therapeutic areas, including multiple myeloma, autoimmune diseases, pediatric growth hormone deficiency, and several cancer indications such as head and neck cancer. Key candidates in its portfolio include Uliledlimab, an antibody for solid tumors, along with Ragistomig and Givastomig, highlighting the company's commitment to developing transformative therapies for patients.

Metabolon, Inc. is a health technology company specializing in metabolomics, focusing on the development of diagnostic tests and analytical methods for early disease detection and precision medicine. The company offers a range of clinical diagnostics, including QUANTOSE IR for measuring insulin resistance, QUANTOSE IGT for assessing glucose tolerance, and Meta IMD for identifying metabolites linked to inherited metabolic disorders. In addition to its diagnostic capabilities, Metabolon provides customizable research services and multiomics solutions to support academic and commercial investigations across various life science sectors. Its technologies are applicable in areas such as cardiovascular health, diabetes, oncology, and respiratory disorders. Founded in 2000 and headquartered in Morrisville, North Carolina, with additional offices in Europe and sales locations in the UK, Spain, and China, Metabolon collaborates with numerous strategic partners in the pharmaceutical and biotechnology industries to enhance research and development outcomes.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering and developing innovative cancer therapeutics. Founded in 2017 and headquartered in San Mateo, California, the company focuses on targeting dysregulated transcription factors and oncogenic signaling pathways. Its lead candidate, entospletinib, is a selective inhibitor of spleen tyrosine kinase aimed at treating acute myeloid leukemia. Additionally, Kronos Bio is developing KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9, intended for MYC-amplified solid tumors. By leveraging advanced technologies such as high-throughput small-molecule microarrays and targeted protein degradation, Kronos Bio aims to identify effective compounds that can modulate historically undruggable cancer targets, ultimately striving to improve patient outcomes through precision medicine.

ReViral Ltd is a biotechnology company based in Stevenage, United Kingdom, that specializes in the discovery and development of antiviral drugs targeting the respiratory syncytial virus (RSV). Founded in 2011, ReViral focuses on creating novel first-in-class compounds, including a highly potent fusion inhibitor designed to treat severe RSV infections, particularly in vulnerable populations such as neonates. The company's innovative approach aims to expand the antiviral market by providing effective treatment options for patients affected by RSV, a virus known for causing significant respiratory illness. Through its research and development efforts, ReViral seeks to address unmet medical needs in the realm of viral infections.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, established in 2019, focused on developing adeno-associated virus (AAV)-based gene therapies for monogenic diseases affecting the central nervous system (CNS). The company’s product pipeline includes several therapies currently in development, such as TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha Gene Therapies aims to translate innovative treatments from research to clinical practice efficiently, leveraging a strategic partnership with The University of Texas Southwestern Medical Center to enhance its development and commercialization capabilities. The company's mission is to create curative medicines that target both rare and prevalent CNS conditions, thereby addressing severe and life-threatening diseases.

Cerevel Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapies for neuroscience diseases. Founded in 2018 and headquartered in Cambridge, Massachusetts, the company is advancing a diverse pipeline that includes five clinical-stage investigational therapies and several preclinical compounds targeting conditions such as schizophrenia, epilepsy, Parkinson's disease, and substance use disorder. Key products under development include CVL-231, a positive allosteric modulator for schizophrenia; CVL-865, which is being tested for drug-resistant focal onset epilepsy and acute anxiety; and Tavapadon, a selective dopamine D1/D5 partial agonist for Parkinson's disease. Cerevel Therapeutics employs a unique approach that leverages expertise in neurocircuitry and receptor selectivity to address unmet medical needs in the field of neuroscience.

Frequency Therapeutics is a clinical-stage biotechnology company based in Woburn, Massachusetts, that aims to repair or reverse damage from various degenerative diseases by utilizing the body’s own biological mechanisms. The company employs a Progenitor Cell Activation approach, which involves the use of small molecules to activate progenitor cells and promote the regeneration of functional tissue. Its leading product candidate, FX-322, is currently undergoing phase 2a clinical trials to address the underlying causes of sensorineural hearing loss. In addition to hearing loss, Frequency Therapeutics is also exploring treatments for a range of other conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. The company has established a collaboration with Astellas Pharma for the development and commercialization of FX-322. Founded in 2014, Frequency Therapeutics focuses on disease modification without the complexities associated with genetic engineering.

Cogent Biosciences is a biotechnology company dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

AsclepiX Therapeutics, Inc. is a biopharmaceutical company focused on the design and development of innovative treatments for diseases related to abnormal blood vessel growth, specifically through the use of short biomimetic anti-angiogenic and anti-lymphangiogenic peptides. The company's lead candidates, including AXT107, AXT201, AXT301, and AXT501, target conditions such as diabetic macular edema, wet age-related macular degeneration, and macular edema following retinal vein occlusion, which are among the leading causes of blindness. Additionally, AXT201, AXT301, and AXT501 are being explored for their potential in treating various solid tumors. AsclepiX leverages advanced computational biology, bioinformatics, and the latest developments in biomaterials to enhance drug delivery systems, aiming to create long-lasting biodegradable nanoparticles that can effectively deliver its therapeutic peptides. Incorporated in 2011 and based in Baltimore, Maryland, AsclepiX Therapeutics is committed to addressing unmet medical needs in ophthalmology, oncology, and other diseases driven by angiogenesis and lymphangiogenesis.

C4 Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutic candidates that target and eliminate disease-causing proteins, primarily for the treatment of cancer and neurodegenerative disorders. The company's lead product candidate, CFT7455, is an orally bioavailable degrader designed to target IKZF1/3 for multiple myeloma and other lymphomas. Additionally, C4 is advancing CFT8634, which targets BRD9 for specific solid tumors, alongside programs aimed at genetically defined resistant solid tumors. C4 Therapeutics utilizes its proprietary Degronimid platform, which allows for the selective degradation of proteins through the natural ubiquitin/proteasome system, potentially addressing previously undruggable targets while minimizing drug resistance. The company has formed strategic collaborations with major industry players, and it aims to make a significant impact on the treatment landscape through its advanced protein degradation science and multiple development programs. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics continues to innovate in the field of targeted therapy.

4D Molecular Therapeutics is a clinical-stage gene therapy company based in Emeryville, California, that specializes in developing innovative gene therapy products to address unmet medical needs. The company utilizes targeted and evolved adeno-associated virus vectors to create a diverse portfolio of product candidates focused on ophthalmology, cardiology, and pulmonology. Among its key projects are 4D-125, currently in a Phase I/II clinical trial for X-linked retinitis pigmentosa; 4D-110, in a Phase I clinical trial for choroideremia; and 4D-310, also in a Phase I/II trial for Fabry disease. Additionally, 4D Molecular Therapeutics is advancing investigational new drug candidates such as 4D-150 for wet age-related macular degeneration and 4D-710 for cystic fibrosis lung disease. Founded in 2013, the company is dedicated to transforming the treatment landscape in these therapeutic areas.

C4 Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutic candidates that target and eliminate disease-causing proteins, primarily for the treatment of cancer and neurodegenerative disorders. The company's lead product candidate, CFT7455, is an orally bioavailable degrader designed to target IKZF1/3 for multiple myeloma and other lymphomas. Additionally, C4 is advancing CFT8634, which targets BRD9 for specific solid tumors, alongside programs aimed at genetically defined resistant solid tumors. C4 Therapeutics utilizes its proprietary Degronimid platform, which allows for the selective degradation of proteins through the natural ubiquitin/proteasome system, potentially addressing previously undruggable targets while minimizing drug resistance. The company has formed strategic collaborations with major industry players, and it aims to make a significant impact on the treatment landscape through its advanced protein degradation science and multiple development programs. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics continues to innovate in the field of targeted therapy.

Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing novel therapies for patients with genetically defined diseases that have significant unmet medical needs. The company's lead product candidate, losmapimod, is a small molecule currently undergoing Phase 2b clinical trials for the treatment of facioscapulohumeral muscular dystrophy, a rare condition characterized by progressive muscle wasting and disability. Another key candidate, FTX-6058, aims to upregulate fetal hemoglobin to treat sickle cell disease and beta-thalassemia. Fulcrum is also actively researching drug targets for other genetic disorders, including Duchenne muscular dystrophy and Friedreich ataxia, as well as neurological and pulmonary diseases. The company has established collaborations with Acceleron Pharma to explore biological targets in pulmonary disease and with MyoKardia to develop therapies for genetic cardiomyopathies. Founded in 2015, Fulcrum Therapeutics continues to focus on improving patient outcomes in rare disease contexts.

Athira Pharma, Inc. is a clinical-stage biopharmaceutical company based in Seattle, Washington, focused on developing small molecules aimed at restoring neuronal health and addressing neurodegenerative diseases. The company's lead candidate, ATH-1017, is a small hepatocyte growth factor/MET activator designed to penetrate the blood-brain barrier and is currently undergoing various clinical trials for the treatment of Alzheimer's and Parkinson's diseases. Additionally, Athira is advancing other candidates, including ATH-1019, which is in preclinical development for depression, and ATH-1018, aimed at treating peripheral neuropathy. Founded in 2011 and originally named M3 Biotechnology, Athira Pharma changed its name in April 2019 to reflect its commitment to innovative therapies that positively impact individuals affected by brain disorders.

Nautilus Biotechnology is a development-stage life sciences company focused on advancing proteomics, the study of proteins and their functions, to enhance therapeutic development and medical diagnostics. The company has created a platform technology that aims to quantify and unravel the complexities of the human proteome. Its flagship product, the Proteomic Analysis System, is a prototype single-molecule instrument designed to facilitate these efforts. The team at Nautilus comprises experts from various disciplines, including protein chemistry, molecular biology, and data science, enabling a multifaceted approach to overcoming challenges in the field. By harnessing this diverse expertise, Nautilus aims to revolutionize drug development and improve healthcare management through personalized and predictive medicine.

Dynacure is a clinical-stage drug development company dedicated to enhancing the lives of patients with rare and orphan diseases. The company is advancing its lead product, DYN101, an investigational antisense medicine aimed at modulating the expression of dynamin 2 to treat Centronuclear Myopathies, in collaboration with Ionis Pharmaceuticals. In addition to DYN101, Dynacure is developing a complementary research portfolio that targets various other orphan disorders, including a program focused on hereditary spastic paraplegias linked to specific genetic mutations. Through its innovative approach and expertise in rare disease drug development, Dynacure seeks to address significant unmet medical needs in this underserved patient population.

immatics is a biopharmaceutical company focused on the discovery of true targets for cancer immunotherapies with the development of the right T cell receptors. The company's customized Adoptive Cell Therapies (ACTengine) and antibody-like TCR Bispecifics are the two main product classes in the pipeline (TCER). With a focus on particularly difficult-to-treat solid malignancies, each therapy modality has unique characteristics to create the desired therapeutic impact for patients at various disease stages and with various types of tumors. immatics biotechnologies was established in 2000 by Hans Rammensee, Harpreet Singh, and Steffen Walter in Tübingen, Baden-Wurttemberg.

ArcherDX, Inc. is a genomics company headquartered in Boulder, Colorado, specializing in the development and commercialization of advanced genetic analysis products. Established in 2013, the company focuses on next-generation sequencing technologies to enhance genetic mutation detection, particularly in oncology. ArcherDX offers a range of research-use-only products, including DNA-based VariantPlex, RNA-based FusionPlex, and ctDNA-based LiquidPlex assays. These products enable laboratories to conduct genomic analyses for therapy optimization and cancer monitoring. Additionally, ArcherDX provides customized assay design services for clinical and biopharmaceutical clients, allowing them to target specific biomarkers and develop new applications. The company aims to deliver actionable genomic information for the treatment of solid tumors, blood cancers, and sarcomas, catering to academic laboratories, biopharmaceutical firms, and contract research organizations. ArcherDX also seeks regulatory approval for various companion diagnostic assays to further support precision medicine initiatives.

Quellis Biosciences, Inc. is a biopharmaceutical company headquartered in Waltham, Massachusetts, established in 2017. The company is dedicated to developing new monoclonal antibody therapies aimed at treating rare diseases, particularly for patients who lack effective treatment options. By focusing on next-generation antibody therapies, Quellis seeks to enhance patient outcomes and deliver innovative solutions that address unmet medical needs. Through its research and development efforts, the company aims to optimize therapeutic leads and identify promising antibodies, ultimately improving the quality of life for individuals affected by serious rare conditions.

Zentalis Pharmaceuticals is a clinical-stage biopharmaceutical company based in New York, dedicated to developing small molecule therapeutics aimed at treating various cancers. Founded in 2014, the company focuses on discovering and advancing drug candidates that target critical biological pathways in cancer. Its lead product candidate, ZN-c5, is an oral selective estrogen receptor degrader currently undergoing Phase I/II clinical trials for advanced or metastatic breast cancer. In addition, Zentalis is developing ZN-c3, an inhibitor of the WEE1 protein tyrosine kinase, for advanced solid tumors; ZN-d5, a selective B-cell lymphoma 2 inhibitor for hematological malignancies; and ZN-e4, an irreversible inhibitor of mutant epidermal growth factor receptor for advanced non-small cell lung cancer, all of which are also in Phase I/II clinical trials. The company utilizes an integrated discovery engine to identify new chemical entities with the potential for differentiated therapeutic profiles.

Avidity Biosciences, Inc. is a biotechnology company focused on developing oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These innovative therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. This approach allows Avidity to target previously undruggable tissue and cell types, thereby addressing the underlying genetic drivers of various diseases. The company's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, a rare muscle disease. Additionally, Avidity is advancing multiple programs aimed at treating muscle-related conditions such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Founded in 2012 and based in La Jolla, California, Avidity is also exploring therapeutic applications in immune and other cell types.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Frequency Therapeutics is a clinical-stage biotechnology company based in Woburn, Massachusetts, that aims to repair or reverse damage from various degenerative diseases by utilizing the body’s own biological mechanisms. The company employs a Progenitor Cell Activation approach, which involves the use of small molecules to activate progenitor cells and promote the regeneration of functional tissue. Its leading product candidate, FX-322, is currently undergoing phase 2a clinical trials to address the underlying causes of sensorineural hearing loss. In addition to hearing loss, Frequency Therapeutics is also exploring treatments for a range of other conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. The company has established a collaboration with Astellas Pharma for the development and commercialization of FX-322. Founded in 2014, Frequency Therapeutics focuses on disease modification without the complexities associated with genetic engineering.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering and developing innovative cancer therapeutics. Founded in 2017 and headquartered in San Mateo, California, the company focuses on targeting dysregulated transcription factors and oncogenic signaling pathways. Its lead candidate, entospletinib, is a selective inhibitor of spleen tyrosine kinase aimed at treating acute myeloid leukemia. Additionally, Kronos Bio is developing KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9, intended for MYC-amplified solid tumors. By leveraging advanced technologies such as high-throughput small-molecule microarrays and targeted protein degradation, Kronos Bio aims to identify effective compounds that can modulate historically undruggable cancer targets, ultimately striving to improve patient outcomes through precision medicine.

ArcherDX, Inc. is a genomics company headquartered in Boulder, Colorado, specializing in the development and commercialization of advanced genetic analysis products. Established in 2013, the company focuses on next-generation sequencing technologies to enhance genetic mutation detection, particularly in oncology. ArcherDX offers a range of research-use-only products, including DNA-based VariantPlex, RNA-based FusionPlex, and ctDNA-based LiquidPlex assays. These products enable laboratories to conduct genomic analyses for therapy optimization and cancer monitoring. Additionally, ArcherDX provides customized assay design services for clinical and biopharmaceutical clients, allowing them to target specific biomarkers and develop new applications. The company aims to deliver actionable genomic information for the treatment of solid tumors, blood cancers, and sarcomas, catering to academic laboratories, biopharmaceutical firms, and contract research organizations. ArcherDX also seeks regulatory approval for various companion diagnostic assays to further support precision medicine initiatives.

Athenex, Inc. is a biopharmaceutical company focused on the discovery, development, and commercialization of innovative therapies for cancer and related conditions. Founded in 2003 and headquartered in Buffalo, New York, Athenex operates across North America and Asia, with a clinical pipeline that includes several promising product candidates. The company’s Orascovery platform features oral formulations of well-known chemotherapeutic agents such as Paclitaxel, Irinotecan, Docetaxel, Topotecan, and Eribulin, which are in various stages of clinical trials targeting multiple cancer types. Additionally, Athenex is developing Src Kinase inhibitors for skin cancers and psoriasis, as well as advanced immunotherapies and arginine deprivation therapies. Through its commitment to innovative research and collaborations, Athenex aims to become a leader in delivering effective cancer treatments and improving patient health outcomes globally.

Poseida Therapeutics is a clinical-stage biopharmaceutical company based in San Diego, California, dedicated to developing innovative therapeutics for patients with significant unmet medical needs. The company utilizes non-viral gene engineering technologies to create a pipeline of targeted therapies, including both autologous and allogeneic chimeric antigen receptor T cell (CAR-T) product candidates aimed at treating hematological malignancies and solid tumors. Additionally, Poseida is advancing gene therapies for rare and life-threatening conditions, such as Ornithine transcarbamylase deficiency, methylmalonic acidemia, and various genetic liver diseases. Founded in 2014, Poseida is committed to addressing critical health challenges through its focused research and development efforts.

Saama Technologies, Inc. is a data science solutions and services company that specializes in advanced analytics and data management primarily for the life sciences sector. Established in 1997 and headquartered in Campbell, California, Saama offers a suite of solutions, including the Life Science Analytics Cloud (LSAC), which supports biotech companies in conducting efficient clinical development and regulatory programs. The LSAC platform is utilized by over 50 biotech firms and major pharmaceutical companies to enhance the oversight of clinical research data, facilitating quicker New Drug Applications (NDAs) and faster market entry for new drugs. Additionally, Saama provides custom analytical solutions, operations analytics, and social governance risk compliance analytics. With a global presence, including locations in the United States, India, the United Kingdom, and Switzerland, the company serves a diverse range of industries, including healthcare, insurance, and technology. Saama has established strategic partnerships with leading organizations to enhance its analytics capabilities and remains committed to advancing data-driven decision-making in the life sciences.

BridgeBio is a biotechnology company that focuses on genetic disease therapies and develops those assets with the right approach in supporting R&D. BridgeBio creates a bridge from advancements in genetic science to patients with unmet needs via the entrepreneurial engine required to make life-changing medicines as rapidly as possible. BridgeBio's mission is to discover, create, test, and deliver transformative medicines to treat patients with genetic diseases.

Allogene Therapeutics is a clinical-stage biotechnology company focused on developing off-the-shelf, genetically engineered allogeneic T cell therapies for cancer treatment. The company's pipeline includes UCART19, in development for relapsed/refractory acute lymphoblastic leukemia, and several preclinical allogeneic CAR T therapies targeting various cancers. Allogene's approach uses gene editing and advanced manufacturing technologies to create a scalable, broader patient eligibility option compared to autologous therapies. Revenue is primarily generated through collaborations and licensing agreements.

4D Molecular Therapeutics is a clinical-stage gene therapy company based in Emeryville, California, that specializes in developing innovative gene therapy products to address unmet medical needs. The company utilizes targeted and evolved adeno-associated virus vectors to create a diverse portfolio of product candidates focused on ophthalmology, cardiology, and pulmonology. Among its key projects are 4D-125, currently in a Phase I/II clinical trial for X-linked retinitis pigmentosa; 4D-110, in a Phase I clinical trial for choroideremia; and 4D-310, also in a Phase I/II trial for Fabry disease. Additionally, 4D Molecular Therapeutics is advancing investigational new drug candidates such as 4D-150 for wet age-related macular degeneration and 4D-710 for cystic fibrosis lung disease. Founded in 2013, the company is dedicated to transforming the treatment landscape in these therapeutic areas.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

ReViral Ltd is a biotechnology company based in Stevenage, United Kingdom, that specializes in the discovery and development of antiviral drugs targeting the respiratory syncytial virus (RSV). Founded in 2011, ReViral focuses on creating novel first-in-class compounds, including a highly potent fusion inhibitor designed to treat severe RSV infections, particularly in vulnerable populations such as neonates. The company's innovative approach aims to expand the antiviral market by providing effective treatment options for patients affected by RSV, a virus known for causing significant respiratory illness. Through its research and development efforts, ReViral seeks to address unmet medical needs in the realm of viral infections.

Alector, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapies for neurodegenerative diseases, including Alzheimer’s disease and frontotemporal dementia. Founded in 2013 and headquartered in South San Francisco, California, the company utilizes advanced antibody technology and insights from neuroimmunology and human genetics to create innovative treatments. Alector’s pipeline includes several candidates, such as AL001, which is undergoing phase II clinical trials for frontotemporal dementia, and AL101, currently in phase I trials for Alzheimer's and Parkinson's diseases. Additionally, AL002 and AL003 are in phase 1b trials for Alzheimer’s disease. The company is actively engaged in research and development, with a total of ten programs at various stages. Alector has formed a strategic collaboration with Adimab, LLC to enhance its antibody research and development capabilities, aiming to efficiently generate and validate antibody drugs that target key disease-related mechanisms.

Athenex, Inc. is a biopharmaceutical company focused on the discovery, development, and commercialization of innovative therapies for cancer and related conditions. Founded in 2003 and headquartered in Buffalo, New York, Athenex operates across North America and Asia, with a clinical pipeline that includes several promising product candidates. The company’s Orascovery platform features oral formulations of well-known chemotherapeutic agents such as Paclitaxel, Irinotecan, Docetaxel, Topotecan, and Eribulin, which are in various stages of clinical trials targeting multiple cancer types. Additionally, Athenex is developing Src Kinase inhibitors for skin cancers and psoriasis, as well as advanced immunotherapies and arginine deprivation therapies. Through its commitment to innovative research and collaborations, Athenex aims to become a leader in delivering effective cancer treatments and improving patient health outcomes globally.

BridgeBio is a biotechnology company that focuses on genetic disease therapies and develops those assets with the right approach in supporting R&D. BridgeBio creates a bridge from advancements in genetic science to patients with unmet needs via the entrepreneurial engine required to make life-changing medicines as rapidly as possible. BridgeBio's mission is to discover, create, test, and deliver transformative medicines to treat patients with genetic diseases.

Dova Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company based in Durham, North Carolina, specializing in the acquisition, development, and commercialization of drug candidates for rare diseases with significant unmet medical needs. The company primarily targets thrombocytopenia, a condition marked by a low blood platelet count, and its lead product candidate, DOPTELET, is indicated for adult patients with chronic liver disease who are scheduled for procedures. Additionally, Dova Pharmaceuticals is developing DOPTELET for chronic immune thrombocytopenia and chemotherapy-induced thrombocytopenia. Founded in 2016, the company aims to provide innovative treatments for patients suffering from orphan diseases and operates as a subsidiary of Swedish Orphan Biovitrum AB.

Xontogeny LLC is a life sciences accelerator based in Boston, Massachusetts, founded in 2016. The company specializes in the development of pharmaceuticals and related technologies, aiming to enhance the success rate of early-stage drug and technology ventures. Xontogeny provides entrepreneurs with essential leadership, strategic guidance, and operational support, fostering collaboration with founding scientists to realize their visions. By adopting a differentiated approach, Xontogeny supports efficient development models that benefit both company founders and early equity holders, thereby advancing innovation in the healthcare and life science sectors.

Singulex, Inc. is an immunodiagnostics and biotechnology company that specializes in single molecule counting (SMC) technology for clinical diagnostics and scientific research. The company has developed the Sgx Clarity System, an in vitro diagnostics platform capable of detecting low abundance biomarkers at levels down to femtograms/mL. This system assists clinicians in managing cardiovascular health, while also providing heart disease monitoring services and personalized wellness programs for patients. Singulex's offerings include ultra-sensitive immunoassay solutions and laboratory testing services that facilitate both reactive and proactive health management, as well as support the discovery of new therapeutics. The company serves a diverse clientele, including pharmaceutical research laboratories, clinical research organizations, and academic institutions globally. Additionally, Singulex is involved in pilot studies to validate its Erenna BioAssay System, which enhances molecular detection capabilities. Founded in 1997 and based in Alameda, California, Singulex has an additional office in Basel, Switzerland, and was previously known as BioProfile Corporation until its name change in 2003.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

VBI Vaccines Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, with research facilities in Ottawa, Canada. It develops and sells vaccines for infectious diseases and immuno-oncology, with a focus on expanding protection in large, underserved markets. The company's enveloped virus-like particle (eVLP) platform enables the creation of vaccines that closely mimic target viruses, eliciting potent immune responses. VBI's commercial product, Sci-B-Vac, is a hepatitis B vaccine approved in Israel and 14 other countries, offering rapid onset of protection and high antibody levels. The company is also developing VBI-2601 for a functional cure of chronic hepatitis B, VBI-1901 for glioblastoma, and VBI-1501 for cytomegalovirus, among other candidates. VBI collaborates with pharmaceutical companies and research institutions, and its products are primarily distributed through direct sales to physicians and pharmacists.

VBI Vaccines Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, with research facilities in Ottawa, Canada. It develops and sells vaccines for infectious diseases and immuno-oncology, with a focus on expanding protection in large, underserved markets. The company's enveloped virus-like particle (eVLP) platform enables the creation of vaccines that closely mimic target viruses, eliciting potent immune responses. VBI's commercial product, Sci-B-Vac, is a hepatitis B vaccine approved in Israel and 14 other countries, offering rapid onset of protection and high antibody levels. The company is also developing VBI-2601 for a functional cure of chronic hepatitis B, VBI-1901 for glioblastoma, and VBI-1501 for cytomegalovirus, among other candidates. VBI collaborates with pharmaceutical companies and research institutions, and its products are primarily distributed through direct sales to physicians and pharmacists.

VBI Vaccines Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, with research facilities in Ottawa, Canada. It develops and sells vaccines for infectious diseases and immuno-oncology, with a focus on expanding protection in large, underserved markets. The company's enveloped virus-like particle (eVLP) platform enables the creation of vaccines that closely mimic target viruses, eliciting potent immune responses. VBI's commercial product, Sci-B-Vac, is a hepatitis B vaccine approved in Israel and 14 other countries, offering rapid onset of protection and high antibody levels. The company is also developing VBI-2601 for a functional cure of chronic hepatitis B, VBI-1901 for glioblastoma, and VBI-1501 for cytomegalovirus, among other candidates. VBI collaborates with pharmaceutical companies and research institutions, and its products are primarily distributed through direct sales to physicians and pharmacists.

Corbus Pharmaceuticals is a clinical-stage pharmaceutical company based in Norwood, Massachusetts, that focuses on developing and commercializing therapeutics for inflammatory and fibrotic diseases. Founded in 2009, the company’s lead product candidate, lenabasum, is a synthetic oral CB2 agonist currently undergoing Phase III clinical trials for systemic sclerosis and dermatomyositis, and Phase IIb trials for systemic lupus erythematosus and cystic fibrosis. In addition to lenabasum, Corbus is developing CRB-4001, a peripherally-restricted CB1 inverse agonist aimed at treating nonalcoholic steatohepatitis and other fibrotic conditions. The company is leveraging a diverse pipeline of drug candidates sourced from a library of over 600 novel compounds targeting the endocannabinoid system. Furthermore, Corbus has established collaborations for the development and commercialization of its products, including a partnership with Kaken Pharmaceutical Co., Ltd. for lenabasum in Japan.

Clearside Biomedical, Inc. is a biopharmaceutical company focused on developing treatments for serious eye diseases with the aim of restoring and preserving vision. Founded in 2011 and headquartered in Alpharetta, Georgia, the company specializes in a proprietary ocular microinjection platform that allows for targeted delivery of therapeutic products to specific compartments of the eye. This innovative approach includes the use of the SCS Microinjector for administering therapies directly into the suprachoroidal space, enabling a non-surgical, repeatable procedure. Among its product pipeline, Clearside is developing XIPERE, a triamcinolone acetonide injectable suspension for macular edema, and CLS-AX, an axitinib for suprachoroidal injection. The company is dedicated to advancing pharmacological therapies for sight-threatening conditions by ensuring precise and effective treatment delivery.

Celladon Corporation is a biotechnology company focused on developing molecular therapies for the treatment of heart failure. Founded in 2000 and headquartered in La Jolla, California, the company is known for its product candidates that address key enzyme deficiencies associated with advanced heart failure. One of its primary products, SERCA2a, is an enzyme that plays a crucial role in calcium cycling and contractility in heart muscle cells. Additionally, Celladon is developing MYDICAR, an enzyme replacement therapy aimed at improving heart function in patients suffering from heart failure.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on developing gene therapy treatments for retinal degenerative diseases and neurodegenerative conditions affecting the central nervous system. Utilizing its core technology platforms—Mitochondrial Targeting Sequence and Optogenetics—the company aims to preserve and restore vision in patients with severe retinal disorders. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III trials for treating Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. In addition to these, GenSight is advancing several products in preclinical stages targeting various ophthalmic and neurodegenerative diseases. Founded in 2012, the company is dedicated to innovative approaches that aim to prevent vision loss and improve the quality of life for affected patients.