Perceptive Advisors

RAPT Therapeutics is a clinical-stage biopharmaceutical company specializing in immunology. It focuses on discovering, developing, and commercializing oral small-molecule therapies aimed at addressing significant unmet medical needs in oncology and inflammatory diseases. RAPT's proprietary discovery engine has led to the advancement of two drug candidates: FLX475, which selectively inhibits the migration of immunosuppressive regulatory T cells into tumors, and RPT193, which targets type 2 T helper cells in inflamed tissues. Both candidates have progressed to clinical trials within a short time frame since the company's inception. Additionally, RAPT is exploring further therapeutic targets, including general control nonderepressible 2 and hematopoietic progenitor kinase 1, which are currently in the discovery phase.

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.

Zevra Therapeutics is a development-stage biopharmaceutical company dedicated to the discovery and development of innovative therapies for pain, ADHD, and other central nervous system diseases. The company is particularly focused on creating safer, abuse-resistant opioid pain relievers. Utilizing its proprietary Ligand Activated Therapy (LAT) platform, Zevra aims to enhance existing drugs and streamline the development process while safeguarding its intellectual property. Additionally, Zevra addresses the needs of patients with rare diseases by employing unique, data-driven strategies to overcome challenges in drug development. The company's mission centers on delivering transformative therapies to individuals facing limited or no treatment options, ultimately striving to improve the quality of life for those affected by these conditions.

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.

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 is a developer of a mucosal immunotherapy platform designed to treat inflammatory bowel disease and diabetes. The company's mucosal immunotherapy platform has developed a flexible nucleotide delivery technology targeting mucosal tissues to treat numerous prevalent, chronic diseases via the induction or suppression of protein expression levels, enabling physicians and doctors to regenerate physiologic, meal-regulated insulin secretion from the gut of subjects with diabetes.

Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases, utilizing proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body’s immune system in the fight against cancer. The company designs targeted treatments aimed at addressing the underlying causes of diseases, thereby improving patient outcomes. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor that specifically targets the KIT D816V mutation and other mutations in KIT exon 17. This mutation is predominantly linked to Systemic Mastocytosis, a severe condition resulting from the uncontrolled growth of mast cells, and is also associated with advanced gastrointestinal stromal tumors. Cogent Biosciences is advancing its lead program, which is expected to enter Phase I clinical trials to evaluate safety and efficacy.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe 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. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Soleno Therapeutics, Inc. is a clinical-stage biopharmaceutical company dedicated to developing and commercializing innovative treatments 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). This product is currently undergoing evaluation in a Phase III clinical development program. Soleno Therapeutics was incorporated in 1999 and is based in Redwood City, California. The company was previously known as Capnia, Inc. before rebranding in May 2017.

Forte Biosciences is a biotechnology company specializing in dermatology and biopharmaceuticals. The company is developing innovative treatments for inflammatory skin diseases, with its lead product, FB-401, being a topically applied live biotherapeutic created in partnership with the National Institutes of Health and the National Institute of Allergy and Infectious Diseases. Additionally, Forte is advancing through preclinical trials for another product candidate, FB-102, which is a proprietary therapeutic molecule that may have applications in treating various autoimmune conditions, including graft-versus-host disease, vitiligo, and alopecia areata.

AsclepiX Therapeutics represents the next generation of biologics and drug development technology. They use bioinformatics and systems biology methods to design classes of short biomimetic anti-angiogenic (anti-neovascular), and anti-lymphangiogenic peptides. They also use the latest developments in biomaterials and drug delivery to design long-lasting biodegradable nano and microparticles to deliver these biomimetic therapeutic peptides throughout the body. AsclepiX Therapeutics utilizes its platform technology for indications in ophthalmology, oncology, and for other angiogenesis and lymphangiogenesis-dependent diseases.

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 lung cancer diagnostics and biopharmaceutical services. The company offers a range of blood-based tests designed to assist physicians in evaluating the risk of malignancy in patients with suspicious lung nodules, including the Nodify XL2 and Nodify CDT tests. Additionally, it provides GeneStrat tumor profiling and VeriStrat immune profiling tests that enable more informed treatment decisions. Biodesix utilizes a proprietary mass spectrometry-based platform for the development of multivariate protein diagnostics, with its first product, VeriStrat, aiding oncologists in choosing between specific therapies for advanced lung cancer patients. The tests are processed in a CLIA-certified laboratory, with results delivered within 72 hours. Besides its diagnostic offerings, Biodesix collaborates with biopharmaceutical companies to develop companion diagnostics, enhancing the therapeutic effectiveness of various agents. Incorporated in 2005, Biodesix is committed to improving patient outcomes through innovative diagnostic solutions.

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 severe 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. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Surf Bio is a preclinical biopharmaceutical company leveraging a breakthrough next-generation surfactant to develop enhanced therapeutic solutions for diabetes, oncology, infectious diseases, gene therapy, and other therapeutic areas.

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. The company utilizes its proprietary technology platform, SomaScan, which allows for the measurement of proteins with a precision comparable to DNA measurement. This technology enhances the understanding of the molecular bases of health and disease, surpassing traditional genomic methods. SomaLogic develops and offers SOMAmers, modified nucleic acid-based protein-binding reagents, and provides diagnostic tests and protein measurement solutions for various diseases, including those in oncology, neurology, and cardiovascular health. Their products serve multiple applications, including life sciences research, therapeutic drug development, and clinical diagnostics, making significant contributions to the fields of biological and medical sciences. Founded in 1999, SomaLogic continues to advance the capabilities of proteomics in both research and clinical settings.

Bridge to Life is a U.S.-based company headquartered in Northbrook, Illinois, dedicated to enhancing organ transplantation through innovative preservation solutions and technologies. The company specializes in the development and licensing of preservation products aimed at improving the quality of service provided to organ procurement organizations and transplant centers. By focusing on advancements in organ preservation and machine perfusion, Bridge to Life collaborates with surgeons and transplant professionals to explore new scientific developments in the field. As a leading supplier of preservation solutions worldwide, the company is committed to facilitating better storage and transportation of intra-abdominal organs to optimize transplant outcomes.

GeneDx is dedicated to providing personalized health insights that enhance diagnosis, treatment, and drug discovery. By integrating extensive genomic expertise with advanced data interpretation capabilities, the company aims to establish precision medicine as a standard in healthcare. GeneDx specializes in exome and genome testing and interpretation, leveraging one of the largest rare disease data sets available. Its operations are divided into two segments: GeneDx, which generates the majority of revenue, and Legacy Sema4 diagnostics. Through these efforts, GeneDx is positioned to significantly impact health outcomes by using genomic and large-scale clinical information.

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.

Born from the founders’ recognition that their diverse but complementary skills and experiences would enable them to successfully address challenges that others had not, the company set about solving a vexing problem: how to bring true proteomics to the world in a way that accelerates therapeutic development, dramatically improves medical diagnostics, and makes personalized and predictive medicine a reality. The extraordinary team at Nautilus represents a wide spectrum of disciplines and expertise, including protein chemists, chip designers, molecular biologists, data scientists, material scientists, biophysicists, optical engineers, microfluidics engineers, bioinformaticists, software engineers, and more. Nautilus is positioned to revolutionize proteomics, transform the way drugs are developed, and significantly improve the way human health is managed.

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.

Pyxis Oncology is a developer of antibody therapeutics intended to promote the body's immune response to cancer. The company has engaged in deep analyses of tumor antigen-specific tumor-infiltrating lymphocytes (TILs) within hot tumors and of tumor cell signaling pathways within cold tumors, providing laboratories with development of novel antibody-based immunotherapies. It was founded in 2019 and is based in Boston, Massachusetts.

Absci is a drug and target discovery company that utilizes deep learning AI and synthetic biology to enhance the therapeutic potential of proteins. By employing its Integrated Drug Creation Platform, Absci identifies novel drug targets and discovers optimal biotherapeutic candidates while simultaneously generating the necessary cell lines for manufacturing. This innovative approach allows biotech and pharmaceutical partners to develop next-generation protein-based drugs, including Bionic Proteins that incorporate nonstandard amino acids and other unique drug designs that may be unattainable through traditional methods. Absci's mission focuses on advancing 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 is a developer of regenerative therapies designed to promote functional recovery of neurological diseases. The company's therapies leverage the natural repair process in several different nervous system cell types to target diseases such as multiple sclerosis, providing patients with a treatment to repair damage to the nervous system.

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.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe 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. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

LianBio’s mission is to catalyze the development and accelerate availability of paradigm-shifting medicines to patients in China and major Asian markets through partnerships that provide access to the best science-driven therapeutic discoveries. LianBio collaborates with world-class partners across a diverse array of therapeutic and geographic areas to build out a pipeline based on disease relevance and the ability to impact patients with transformative mechanisms and precision-based therapeutics.

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 developing innovative therapies for oncology, addressing unmet medical needs in the treatment of challenging cancers that have not responded to conventional therapies. Founded in 2018 and based in New York, with an additional office in San Francisco, the company has a portfolio of drug development candidates aimed at improving patient outcomes. One of its key clinical-stage candidates is NUV-868, an oral small molecule BET inhibitor that targets the BRD4 protein, which plays a crucial role in regulating tumor growth and differentiation. Additionally, Nuvation Bio is advancing its proprietary small molecule Drug-Drug Conjugate platform, further expanding its therapeutic arsenal in oncology.

Metabolon, Inc. is a health technology company specializing in metabolomics, which focuses on the analysis of metabolites to aid in disease detection and precision medicine. The company develops a range of clinical diagnostic tests, including QUANTOSE IR for early-stage insulin resistance and Meta IMD for identifying metabolites linked to inherited metabolic disorders. Metabolon also offers customizable research services and multiomics solutions, enabling academic and commercial researchers to explore various life sciences areas effectively. The company's technologies address a wide array of health conditions, including cardiovascular issues, diabetes, and cancer, and are utilized across sectors such as biotechnology, pharmaceuticals, and nutrition. Founded in 2000 and headquartered in Morrisville, North Carolina, Metabolon has a global presence, including offices in Munich, London, Madrid, and China, and maintains strategic partnerships with major firms in the pharmaceutical and consumer health industries.

Metabolon, Inc. is a health technology company specializing in metabolomics, which focuses on the analysis of metabolites to aid in disease detection and precision medicine. The company develops a range of clinical diagnostic tests, including QUANTOSE IR for early-stage insulin resistance and Meta IMD for identifying metabolites linked to inherited metabolic disorders. Metabolon also offers customizable research services and multiomics solutions, enabling academic and commercial researchers to explore various life sciences areas effectively. The company's technologies address a wide array of health conditions, including cardiovascular issues, diabetes, and cancer, and are utilized across sectors such as biotechnology, pharmaceuticals, and nutrition. Founded in 2000 and headquartered in Morrisville, North Carolina, Metabolon has a global presence, including offices in Munich, London, Madrid, and China, and maintains strategic partnerships with major firms in the pharmaceutical and consumer health industries.

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 is a developer of antiviral drugs designed to help patients and enlarge the antiviral market by developing novel first-in-class compounds. The company's antiviral drugs is a fusion inhibitor of RSV which is a highly potent, novel small molecule and is being developed to treat RSV in patients with severe disease including neonates, enabling patients to be treated who are infected with respiratory syncytial virus (RSV) by developing novel compounds.

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. Established in 2018 and based in Cambridge, Massachusetts, the company is advancing a diversified pipeline that includes several clinical-stage investigational therapies targeting conditions such as schizophrenia, epilepsy, Parkinson's disease, and substance use disorder. Notable products in development include CVL-231, a positive allosteric modulator currently in Phase Ib trials for schizophrenia, and CVL-865, also a positive allosteric modulator, which is in Phase II trials for drug-resistant focal onset seizures. Additionally, Tavapadon, a selective dopamine D1/D5 partial agonist, is in a Phase III program for Parkinson's disease, while CVL-936, a selective dopamine D3-preferring antagonist, is undergoing Phase I trials for substance use disorder. The company is also engaged in preclinical research aiming to address various neuroscience indications, leveraging expertise in neurocircuitry and receptor selectivity.

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 focused on developing precision therapies for genetically defined diseases, utilizing proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body’s immune system in the fight against cancer. The company designs targeted treatments aimed at addressing the underlying causes of diseases, thereby improving patient outcomes. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor that specifically targets the KIT D816V mutation and other mutations in KIT exon 17. This mutation is predominantly linked to Systemic Mastocytosis, a severe condition resulting from the uncontrolled growth of mast cells, and is also associated with advanced gastrointestinal stromal tumors. Cogent Biosciences is advancing its lead program, which is expected to enter Phase I clinical trials to evaluate safety and efficacy.

AsclepiX Therapeutics represents the next generation of biologics and drug development technology. They use bioinformatics and systems biology methods to design classes of short biomimetic anti-angiogenic (anti-neovascular), and anti-lymphangiogenic peptides. They also use the latest developments in biomaterials and drug delivery to design long-lasting biodegradable nano and microparticles to deliver these biomimetic therapeutic peptides throughout the body. AsclepiX Therapeutics utilizes its platform technology for indications in ophthalmology, oncology, and for other angiogenesis and lymphangiogenesis-dependent diseases.

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.

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 is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing therapies for patients with rare genetic diseases. The company is dedicated to addressing unmet medical needs, particularly in the realm of genetically defined conditions. Its lead product candidate, losmapimod, is a small molecule currently undergoing Phase 2b clinical trials for the treatment of facioscapulohumeral muscular dystrophy (FSHD), a progressive muscle-wasting disorder. Additionally, Fulcrum is developing FTX-6058, aimed at increasing fetal hemoglobin levels in patients with sickle cell disease and beta-thalassemia. The company also engages in research to identify drug targets for various conditions, including Duchenne muscular dystrophy and neurological diseases. Fulcrum Therapeutics has established collaborations with Acceleron Pharma Inc. and MyoKardia, Inc. to explore new therapeutic avenues in pulmonary disease and genetic cardiomyopathies, respectively. Founded in 2015, Fulcrum aims to enhance the quality of life for patients suffering from these challenging disorders.

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.

Born from the founders’ recognition that their diverse but complementary skills and experiences would enable them to successfully address challenges that others had not, the company set about solving a vexing problem: how to bring true proteomics to the world in a way that accelerates therapeutic development, dramatically improves medical diagnostics, and makes personalized and predictive medicine a reality. The extraordinary team at Nautilus represents a wide spectrum of disciplines and expertise, including protein chemists, chip designers, molecular biologists, data scientists, material scientists, biophysicists, optical engineers, microfluidics engineers, bioinformaticists, software engineers, and more. Nautilus is positioned to revolutionize proteomics, transform the way drugs are developed, and significantly improve the way human health is managed.

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 based in Boulder, Colorado, specializing in the development and commercialization of advanced molecular pathology products and services. The company focuses on next-generation sequencing technologies to enhance genetic mutation detection, offering a range of research products such as VariantPlex, FusionPlex, LiquidPlex, and Immunoverse. ArcherDX's proprietary Anchored Multiplexed PCR (AMP™) chemistry, combined with powerful bioinformatics software, allows for efficient identification of genetic mutations, including gene fusions and point mutations. The company also provides customization services for clinical and biopharmaceutical customers through its assay designer platform, enabling the development of personalized applications. Its products are primarily aimed at academic and reference laboratories, biopharmaceutical firms, and contract research organizations, supporting therapy optimization and cancer monitoring. Founded in 2013, ArcherDX operates as a subsidiary of Invitae Corporation.

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 severe 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. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

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 based in Boulder, Colorado, specializing in the development and commercialization of advanced molecular pathology products and services. The company focuses on next-generation sequencing technologies to enhance genetic mutation detection, offering a range of research products such as VariantPlex, FusionPlex, LiquidPlex, and Immunoverse. ArcherDX's proprietary Anchored Multiplexed PCR (AMP™) chemistry, combined with powerful bioinformatics software, allows for efficient identification of genetic mutations, including gene fusions and point mutations. The company also provides customization services for clinical and biopharmaceutical customers through its assay designer platform, enabling the development of personalized applications. Its products are primarily aimed at academic and reference laboratories, biopharmaceutical firms, and contract research organizations, supporting therapy optimization and cancer monitoring. Founded in 2013, ArcherDX operates as a subsidiary of Invitae Corporation.

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 immuno-oncology company. It is mainly engaged in the development and commercialization of genetically engineered allogeneic T cell therapies for the treatment of cancer. The company is involved in developing a pipeline of multiple allogeneic T cell product candidates utilizing validated gene editing and advanced proprietary cell manufacturing technologies. Its pipeline includes UCART19 which is developed for the treatment of relapsed or refractory acute lymphoblastic leukemia (ALL), as well as several preclinical allogeneic CAR T therapies.

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 the United Kingdom that specializes in developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which modifies a patient's own hematopoietic stem cells to create a targeted treatment administered in a single dose. Orchard's portfolio includes Strimvelis, the first autologous gene therapy approved by the European Medicines Agency for the treatment of adenosine deaminase-severe combined immunodeficiency (ADA-SCID). Additionally, it is advancing clinical programs for various conditions, including metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has an extensive preclinical pipeline targeting mucopolysaccharidosis types I, IIIA, and IIIB. Orchard Therapeutics collaborates with leading institutions in gene therapy research, enhancing its efforts to transform the lives of patients with rare diseases in the UK, European Union, and the United States. Founded in 2015 and headquartered in London, it aims to provide sustainable clinical benefits through its innovative therapies.

ReViral is a developer of antiviral drugs designed to help patients and enlarge the antiviral market by developing novel first-in-class compounds. The company's antiviral drugs is a fusion inhibitor of RSV which is a highly potent, novel small molecule and is being developed to treat RSV in patients with severe disease including neonates, enabling patients to be treated who are infected with respiratory syncytial virus (RSV) by developing novel compounds.

Alector, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapies for neurodegenerative diseases, including Alzheimer's disease and frontotemporal dementia. The company’s lead products, AL001, AL101, AL002, and AL003, are currently undergoing various stages of clinical trials, with AL001 in a phase II trial for frontotemporal dementia and AL101 in phase I trials targeting multiple neurodegenerative conditions. Alector employs advanced antibody technology combined with insights from neuroimmunology and human genetics to create therapeutics that address underlying disease mechanisms. The company has established a collaboration with Adimab, which specializes in the discovery and optimization of antibody therapeutics, to enhance its research and development efforts. Alector also has several additional programs in the research and development stage, reflecting its commitment to advancing novel treatments for complex neurological disorders. Founded in 2013 and headquartered in South San Francisco, California, Alector aims to restore healthy immune function in the brain through its unique therapeutic approaches.

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, based in Durham, North Carolina, is a clinical-stage pharmaceutical company dedicated to acquiring, developing, and commercializing drug candidates for rare diseases with significant unmet medical needs. Its primary focus is on thrombocytopenia, a condition characterized by a low platelet count. The company's lead product, DOPTELET, is approved for treating thrombocytopenia in adult patients with chronic liver disease who are scheduled for procedures. Additionally, Dova is exploring DOPTELET's potential for treating chronic immune thrombocytopenia and chemotherapy-induced thrombocytopenia. Founded in 2016, Dova Pharmaceuticals 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 severe 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. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

VBI Vaccines Inc. is a biopharmaceutical company focused on developing and commercializing vaccines for infectious diseases and immuno-oncology, operating in Israel, the United States, and internationally. The company’s flagship product, Sci-B-Vac, is a commercial-stage hepatitis B vaccine that effectively mimics all three viral surface antigens, providing rapid protection and high antibody levels with lower dosing requirements compared to traditional vaccines. VBI's innovative enveloped virus-like particle (eVLP) platform technology facilitates the creation of vaccines that closely resemble target viruses, enhancing immune responses. The company is advancing several clinical programs, including VBI-1901, an immunotherapeutic candidate for glioblastoma, and VBI-1501, a prophylactic vaccine for cytomegalovirus. VBI collaborates with various organizations, including Brii Biosciences and GlaxoSmithKline, and partners with the National Research Council of Canada to develop a pan-coronavirus vaccine. Headquartered in Cambridge, Massachusetts, VBI Vaccines Inc. aims to address unmet medical needs through its innovative vaccine solutions.

VBI Vaccines Inc. is a biopharmaceutical company focused on developing and commercializing vaccines for infectious diseases and immuno-oncology, operating in Israel, the United States, and internationally. The company’s flagship product, Sci-B-Vac, is a commercial-stage hepatitis B vaccine that effectively mimics all three viral surface antigens, providing rapid protection and high antibody levels with lower dosing requirements compared to traditional vaccines. VBI's innovative enveloped virus-like particle (eVLP) platform technology facilitates the creation of vaccines that closely resemble target viruses, enhancing immune responses. The company is advancing several clinical programs, including VBI-1901, an immunotherapeutic candidate for glioblastoma, and VBI-1501, a prophylactic vaccine for cytomegalovirus. VBI collaborates with various organizations, including Brii Biosciences and GlaxoSmithKline, and partners with the National Research Council of Canada to develop a pan-coronavirus vaccine. Headquartered in Cambridge, Massachusetts, VBI Vaccines Inc. aims to address unmet medical needs through its innovative vaccine solutions.

VBI Vaccines Inc. is a biopharmaceutical company focused on developing and commercializing vaccines for infectious diseases and immuno-oncology, operating in Israel, the United States, and internationally. The company’s flagship product, Sci-B-Vac, is a commercial-stage hepatitis B vaccine that effectively mimics all three viral surface antigens, providing rapid protection and high antibody levels with lower dosing requirements compared to traditional vaccines. VBI's innovative enveloped virus-like particle (eVLP) platform technology facilitates the creation of vaccines that closely resemble target viruses, enhancing immune responses. The company is advancing several clinical programs, including VBI-1901, an immunotherapeutic candidate for glioblastoma, and VBI-1501, a prophylactic vaccine for cytomegalovirus. VBI collaborates with various organizations, including Brii Biosciences and GlaxoSmithKline, and partners with the National Research Council of Canada to develop a pan-coronavirus vaccine. Headquartered in Cambridge, Massachusetts, VBI Vaccines Inc. aims to address unmet medical needs through its innovative vaccine solutions.

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 is a biopharmaceutical company focused on developing and delivering treatments for serious eye diseases, aiming to restore and preserve vision. Founded in 2011 and headquartered in Alpharetta, Georgia, the company employs a proprietary ocular microinjection platform that allows for targeted delivery of therapeutic agents to specific compartments of the eye, such as the retina and choroid. Among its products, XIPERE is a triamcinolone acetonide injectable suspension designed for treating macular edema linked to conditions like uveitis and diabetic macular edema. Additionally, the company's CLS-AX is being developed as an axitinib formulation for suprachoroidal injection. Clearside's innovative approach provides a non-surgical and repeatable method for administering therapies, which could significantly benefit patients with sight-threatening eye diseases.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe 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. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

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.

REGENXBIO Inc. is a clinical-stage biotechnology company specializing in gene therapy aimed at addressing genetic defects and enabling cells to produce therapeutic proteins and antibodies. The company utilizes its proprietary NAV Technology Platform, which involves recombinant adeno-associated virus vectors for gene delivery. REGENXBIO’s lead candidate, RGX-314, is currently in Phase I/IIa clinical trials for the treatment of wet age-related macular degeneration. Additional product candidates under development include RGX-121 for mucopolysaccharidosis type II, RGX-111 for mucopolysaccharidosis type I, RGX-181 for late infantile neuronal ceroid lipofuscinosis type II, and RGX-501 for homozygous familial hypercholesterolemia. Beyond its own product candidates, REGENXBIO also licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies and has a collaboration agreement with Neurimmune AG to develop vectorized antibodies for neurodegenerative diseases. Founded in 2008 and headquartered in Rockville, Maryland, the company was previously known as ReGenX Biosciences, LLC before rebranding in 2014.

Cidara Therapeutics is a biotechnology company based in San Diego, California, established in 2012. The company specializes in the discovery and development of innovative anti-infective therapies for serious diseases, particularly those that affect individuals with compromised immune systems. Its lead candidate, rezafungin acetate, is an antifungal agent designed to treat and prevent severe invasive fungal infections, such as candidemia and invasive candidiasis, which carry significant mortality risks. In addition to its antifungal efforts, Cidara is advancing its proprietary Cloudbreak platform, which focuses on developing antiviral Fc-conjugates aimed at preventing and treating various viral infections, including influenza, RSV, HIV, and coronavirus. Cidara's mission is to transform treatment and prevention strategies for life-threatening illnesses.

Blueprint Medicines is a biopharmaceutical company dedicated to developing targeted therapies for cancers and rare diseases driven by abnormal kinase activation. Founded in 2008 and based in Cambridge, Massachusetts, the company utilizes its proprietary Insights-to-Validation Platform and chemical library to create small molecule kinase inhibitors that address specific genomic drivers. Key drug candidates include avapritinib for systemic mastocytosis and gastrointestinal stromal tumors, BLU-263 for indolent systemic mastocytosis, and pralsetinib for RET-altered cancers such as non-small cell lung cancer and medullary thyroid carcinoma. Additionally, fisogatinib is under investigation for advanced hepatocellular carcinoma, while BLU-782 targets fibrodysplasia ossificans progressiva. Blueprint Medicines collaborates with several pharmaceutical companies to enhance its research and development efforts.

Invitae is a genetic information company focused on integrating genetic insights into everyday medical practice to enhance healthcare quality for a large population. The company specializes in genetic diagnostics for hereditary disorders, offering a comprehensive service that consolidates multiple genetic tests into a single platform. Invitae's offerings include assessments for genes linked to hereditary cancer, neurological disorders, cardiovascular conditions, pediatric issues, metabolic disorders, and other hereditary ailments. By providing high-quality testing with quicker turnaround times and competitive pricing, Invitae aims to make genetic testing more accessible and effective for patients and healthcare providers alike.

Bellicum Pharmaceuticals is a clinical-stage biopharmaceutical company based in Houston, Texas, focused on developing innovative cellular immunotherapies for the treatment of hematological cancers and solid tumors. The company is advancing its product candidates, including BPX-601, an autologous GoCAR-T therapy aimed at solid tumors expressing prostate stem cell antigen, and BPX-603, a dual-switch GoCAR-T therapy targeting solid tumors that express human epidermal growth factor receptor 2. Bellicum utilizes its proprietary Chemical Induction of Dimerization technology platform, which allows for real-time control of immune system components to enhance therapeutic efficacy. The company collaborates with various institutions, including Adaptimmune Therapeutics and Baylor College of Medicine, to further its research and development efforts. Founded in 2004, Bellicum Pharmaceuticals continues to make strides in cancer treatment through its novel immunotherapeutic approaches.

Virobay Inc. is a clinical-stage biopharmaceutical company based in Menlo Park, California, focused on developing and commercializing novel therapies for various medical conditions, including neuropathic pain, autoimmune diseases, liver fibrosis, cancer, and Alzheimer's disease. The company's pipeline includes VBY-129, a cathepsin S inhibitor aimed at treating neuropathic pain and Crohn's disease, and VBY-376, an orally-administered cathepsin B inhibitor for liver fibrosis associated with nonalcoholic steatohepatitis. Additionally, VBY-825 is designed to inhibit multiple cathepsins to mitigate autoimmune attacks on bile ducts and reverse fibrosis. Virobay has also licensed VBY-891 to LEO Pharma A/S for specific dermatological uses, such as psoriasis. Founded in 2006, Virobay is dedicated to addressing significant unmet medical needs through its cysteine cathepsin platform, which focuses on developing safer and more effective treatments by regulating essential cellular processes.

Sevion is a biopharmaceutical company which discovers, develops and acquires innovative product candidates for the treatment of cancer and immunological diseases. The Company is advancing a pipeline of proprietary and partnered product candidates that leverages its unique antibody technology platforms. The Company’s antibody pipeline is being developed from technology that allows for the discovery of innovative biologic therapies to previously inaccessible targets, such as multispanning membrane proteins and ion channels that play an important role in multiple diseases. The Company also developed the first protein nanocage system allowing delivery of nucleic acids and other payloads to target cells.