Perceptive Advisors

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.

Edgewise Therapeutics, founded in 2017 and based in Boulder, Colorado, is a clinical-stage biopharmaceutical company dedicated to developing precision medicine therapies for severe, rare muscle disorders. The company's core expertise lies in muscle biology and small molecule engineering, which it employs through its proprietary drug discovery platform. This platform uses custom-built systems to measure integrated muscle function, enabling the identification of small molecule therapies targeting key proteins in muscle tissue. Edgewise's pipeline focuses on addressing genetically defined muscle disorders such as Duchenne muscular dystrophy, Becker muscular dystrophy, and limb-girdle muscular dystrophies.

Ocuphire Pharma, Inc. is a clinical-stage biopharmaceutical company based in Farmington Hills, Michigan, dedicated to developing and commercializing therapies for various ophthalmic disorders. The company's primary focus is on innovative treatment options for front and back of the eye conditions, with its lead product candidate, Nyxol eye drops, currently in Phase 3 clinical development. Nyxol is a once-daily, preservative-free formulation aimed at addressing issues such as dim light or night vision disturbances, pharmacologically-induced mydriasis, and presbyopia. Additionally, Ocuphire is advancing another candidate, APX3330, a twice-daily oral tablet, which is entering Phase 2 clinical trials to target diabetic retinopathy and diabetic macular edema by inhibiting angiogenesis and inflammation. The company also seeks to explore opportunities for acquiring additional ophthalmic assets and forming strategic partnerships for global commercialization.

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.

Kardigan is a pioneering heart health company dedicated to transforming cardiovascular drug development. It focuses on identifying and matching critical disease drivers with responsive patients from clinical trials, aiming to deliver curative medicines that go beyond symptom management, ultimately providing functional cures for cardiovascular diseases.

RAPT Therapeutics is a clinical-stage biopharmaceutical company specializing in immunology. It is dedicated to discovering, developing, and commercializing oral small-molecule therapies aimed at addressing significant unmet medical needs in oncology and inflammatory diseases. The company employs a proprietary discovery and development engine to create highly selective small molecules that modulate immune responses related to these conditions. RAPT has advanced two notable drug candidates: FLX475, which targets the inhibition of immunosuppressive regulatory T cells in tumors, and RPT193, which selectively inhibits type 2 T helper cells in inflamed tissues. Both candidates have progressed to clinical trials within a short timeframe since the company's inception. Additionally, RAPT is exploring several other targets in the discovery phase, including general control nonderepressible 2 and hematopoietic progenitor kinase 1.

Inventiva Pharma is a clinical-stage biopharmaceutical company based in France, dedicated to developing oral small molecule therapies aimed at addressing significant unmet medical needs in fibrosis, lysosomal storage disorders, and oncology. The company has established a robust pipeline supported by a proprietary discovery engine and a wholly-owned research and development facility. It possesses an extensive library of molecules and a team with considerable expertise in developing compounds that target nuclear receptors, transcription factors, and epigenetic modulation. Inventiva is advancing clinical candidates, including lanifibranor, which is being developed for non-alcoholic steatohepatitis (NASH), a condition currently lacking approved treatments. Additionally, the company is engaged in a variety of pre-clinical therapy programs, further strengthening its portfolio in the fields of oncology, fibrosis, and rare diseases.

OnKure Therapeutics is a clinical-stage biotechnology company dedicated to the discovery and development of innovative cancer therapies. The company specializes in creating less toxic kinase inhibitor drugs and epigenetic therapies, including selective inhibitors of histone deacetylases. Its lead candidate, OKI-179, has demonstrated significant efficacy in preclinical models of hematological and solid tumor cancers, effectively reducing tumor growth both alone and in combination with other targeted therapies. Additionally, OnKure is advancing its HDAC3 selective inhibitor, OKI-422, into preclinical toxicology studies. The company's drug candidates are designed to directly target malignant cells, enhance the effects of small molecule therapies, and optimize the tumor microenvironment for improved checkpoint inhibition, addressing unmet medical needs in cancer treatment.

MeiraGTx Holdings is a clinical-stage gene therapy company dedicated to developing and commercializing innovative gene therapy products aimed at transforming the lives of patients with acquired and inherited disorders. The company focuses on addressing serious medical conditions, primarily targeting disorders related to the eye, salivary gland, and central nervous system. Its research and development efforts leverage a portfolio approach, which includes licensing, acquiring, and developing advanced technologies to support a diverse range of product candidates. Key pipeline products include AAV-CNGB3, AAV-CNGA3, and AAV-RPGR, among others. MeiraGTx operates in the United States, the United Kingdom, and the European Union, with plans to expand its focus to develop additional gene therapy treatments for various serious diseases in the future.

Cargo Therapeutics is a clinical-stage biotechnology company focused on developing innovative CAR T-cell therapies to improve outcomes for cancer patients. The company aims to address the challenges faced by existing autologous CAR therapies, which are only curative for less than half of cancer patients and hindered by issues such as manufacturing limitations, supply challenges, and reimbursement barriers. Cargo Therapeutics is advancing next-generation cell therapies designed to enhance effectiveness, safety, and supply reliability. One of its key products, CRG-022, is a novel CAR T-cell candidate targeting CD22, an antigen present in a majority of B-cell malignancies, specifically designed to overcome treatment resistance. By pursuing these novel solutions, Cargo Therapeutics seeks to facilitate broader access to curative therapies and significantly improve the treatment experience for cancer patients.

Bicycle Therapeutics plc is a clinical-stage biopharmaceutical company based in Cambridge, United Kingdom, specializing in the development of a novel class of medicines known as Bicycles. These are synthetic short peptides designed to form two loops that stabilize their structure. The company's lead product candidate, BT1718, is a Bicycle Toxin Conjugate (BTC) currently undergoing Phase I/IIa clinical trials targeting tumors that express Membrane Type 1 matrix metalloprotease. Other oncology candidates include BT5528, also a BTC in Phase I/IIa trials targeting EphA2, and BT8009, which is in preclinical studies targeting Nectin-4. Additionally, Bicycle is advancing THR-149, a plasma kallikrein inhibitor that has completed Phase I trials for diabetic macular edema. The company collaborates with various biopharmaceutical organizations to address therapeutic areas such as anti-infective, cardiovascular, ophthalmology, and respiratory indications. Bicycle Therapeutics was incorporated in 2009 and aims to address significant unmet medical needs in oncology and beyond.

Endeavor BioMedicines is a clinical-stage company based in Solana Beach, California, founded in 2018. The company is dedicated to developing innovative treatments that address the underlying causes of pulmonary fibrosis, particularly idiopathic pulmonary fibrosis (IPF). Endeavor BioMedicines is focused on creating a small-molecule inhibitor that has the potential to not only halt the progression of IPF but also to reverse its effects. By concentrating on these advancements, the company aims to enable healthcare professionals to provide more precise treatments for terminal conditions, ultimately improving patient outcomes and enhancing quality of life.

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.

TORL BioTherapeutics is a clinical-stage biopharmaceutical company dedicated to developing innovative, antibody-based therapies aimed at enhancing the lives of cancer patients. The company specializes in antibody-drug conjugates and other biologics, focusing on oncologic diseases with significant unmet medical needs. TORL BioTherapeutics employs a strategy that involves target identification and early discovery work, allowing them to selectively advance drug programs through novel licensing agreements. This approach enables their researchers to develop proprietary drugs with unique and optimized profiles, contributing to the advancement of cancer treatment options.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on developing precision oncology therapeutics. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond positively to specific treatments, thereby enhancing the effectiveness of its drug development efforts. Acrivon's pipeline features its lead program, ACR-368 (prexasertib), which is currently in Phase 2 trials, alongside several preclinical programs targeting key components of DNA damage response and cell cycle regulation, including WEE1 and PKMYT1. By concentrating on complex oncology drug targets that are often not addressed by traditional therapies, Acrivon aims to expedite the advancement of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials.

Spyre Therapeutics is a biotechnology company focused on developing innovative antibody therapies aimed at transforming the treatment landscape for inflammatory bowel disease (IBD). By leveraging advanced antibody engineering, rational therapeutic combinations, and precision medicine strategies for patient selection, Spyre Therapeutics seeks to create next-generation products that address the chronic inflammation associated with IBD, which includes ulcerative colitis and Crohn's disease. The company aims to enhance existing treatment options, which currently include anti-inflammatory drugs, immunosuppressants, and biologics, thereby improving outcomes for patients suffering from these debilitating gastrointestinal disorders.

Crinetics Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company headquartered in San Diego, California, dedicated to the discovery, development, and commercialization of innovative therapeutics for rare endocrine diseases and endocrine-related tumors. The company’s lead product candidate, paltusotine, is an oral selective nonpeptide somatostatin receptor type 2 biased agonist currently undergoing Phase I clinical trials aimed at treating acromegaly and neuroendocrine tumors. In addition to paltusotine, Crinetics is developing several other therapeutics, including CRN01941, an oral selective nonpeptide sst2 biased agonist for neuroendocrine tumors, and an oral somatostatin type 5 receptor agonist for congenital hyperinsulinism. The company is also working on an oral adrenocorticotrophic hormone antagonist for conditions such as Cushing’s syndrome and congenital adrenal hyperplasia. Founded in 2008, Crinetics Pharmaceuticals is focused on leveraging its internal discovery efforts to create effective treatments while utilizing hormonal biomarker endpoints to enhance the efficiency and cost-effectiveness of its clinical studies.

Applied Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative treatments for serious diseases that currently lack approved therapies. Established in 2016 and based in New York, the company is advancing a pipeline of novel product candidates that target high unmet medical needs, including cardiovascular diseases and metabolic disorders. Its lead candidate, AT-007, is in Phase I/II trials aimed at treating galactosemia. Additionally, AT-001 is undergoing Phase II trials for diabetic cardiomyopathy and Phase I trials for diabetic peripheral neuropathy, while AT-003 is in Phase I trials for diabetic retinopathy. The company also has preclinical candidates such as AT-104, intended for orphan hematological oncology conditions, demonstrating its commitment to addressing critical health challenges through targeted therapeutics.

Mainstay Medical is a global medical device company based in Dublin, Ireland, with additional operations in the United States and Australia. The company specializes in developing innovative therapies for individuals suffering from chronic low back pain, a condition that affects a significant underserved population. Mainstay Medical has created an FDA-approved restorative treatment designed to enhance muscle control and improve functionality in the lumbar spine. This therapy aims to help patients regain mobility and enhance their overall quality of life. By collaborating with scientists and clinical experts, Mainstay Medical is committed to advancing solutions for those affected by debilitating back pain.

Freenome, Inc. is an artificial intelligence genomics biotech company based in South San Francisco, California, founded in 2014. The company focuses on developing accurate, accessible, and non-invasive blood tests for the early detection of cancer and treatment selection. Utilizing a proprietary multi-omics platform, Freenome employs advanced algorithms to analyze cell-free genomic data, which aids in identifying early-stage cancers and other diseases. This innovative approach aims to transform disease management by enabling timely interventions and proactive treatment strategies. In addition to its diagnostic solutions, Freenome also offers clinical research services to further advance cancer detection and management.

Impulse Dynamics N.V. is a medical device company that specializes in developing innovative electrical therapies for chronic heart failure. Founded in 1996 and headquartered in Willemstad, Curacao, the company also maintains offices in New York and Hong Kong, with product development centers located in New Jersey and Israel. Its primary offering, the CCM® therapy, utilizes cardiac contractility modulation technology to deliver non-excitatory electrical impulses to the heart, enhancing its contraction and improving blood flow to the body. This minimally invasive treatment option addresses the needs of heart failure patients who have limited alternatives. Impulse Dynamics conducts clinical trials to assess the safety and efficacy of its therapies and collaborates with universities and hospitals for ongoing research and development.

enGene, Inc. is a biotechnology company based in Vancouver, Canada, specializing in mucosal immunotherapy to address inflammatory bowel disease and diabetes. The company has developed a non-integrating biopolymer-based nucleotide delivery technology that enables the localized delivery of immune-modulating proteins to mucosal tissues, targeting conditions affecting the gastrointestinal tract, lung, and bladder. This innovative platform allows for the systemic release of proteins from the gut, which can be beneficial for treating a variety of immune disorders, as well as other conditions such as diabetes, anemia, and hemophilia. Founded in 1999, enGene has formed a strategic alliance with Takeda Pharmaceutical Company to further advance its therapeutic offerings.

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).

PepGen Ltd. is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the next generation of nucleic acid therapeutics, particularly in the treatment of severe neuromuscular and neurologic diseases. Incorporated in 2018, PepGen has developed its Enhanced Delivery Oligonucleotide (EDO) platform, which is designed to improve the uptake and activity of conjugated oligonucleotide therapeutics. The company's innovative EDO peptides enhance tissue penetration, cellular uptake, and nuclear delivery, demonstrating their ability to transport oligonucleotides effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. PepGen aims to unlock the clinical potential of these transformative therapeutics, with a particular emphasis on the effective delivery of antisense oligonucleotides, and is advancing its novel conjugate therapeutics toward clinical application.

Astria Therapeutics is a biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative therapies for patients affected by rare allergic and immunological diseases. The company focuses on creating life-changing treatments, with its lead program, STAR-0215, being a monoclonal antibody designed to inhibit plasma kallikrein, currently in preclinical development for hereditary angioedema. Additionally, Astria is advancing STAR-0310, another monoclonal antibody, which targets the OX40 receptor and is also in preclinical development for atopic dermatitis, a condition characterized by impaired skin barrier function and significant itching. Through its targeted approach, Astria Therapeutics aims to address unmet medical needs in the field of immunology.

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.

Spyre Therapeutics is a biotechnology company focused on developing innovative antibody therapies aimed at transforming the treatment landscape for inflammatory bowel disease (IBD). By leveraging advanced antibody engineering, rational therapeutic combinations, and precision medicine strategies for patient selection, Spyre Therapeutics seeks to create next-generation products that address the chronic inflammation associated with IBD, which includes ulcerative colitis and Crohn's disease. The company aims to enhance existing treatment options, which currently include anti-inflammatory drugs, immunosuppressants, and biologics, thereby improving outcomes for patients suffering from these debilitating gastrointestinal disorders.

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.

Avalyn Pharma, Inc. is a biopharmaceutical company focused on developing innovative therapies for idiopathic pulmonary fibrosis (IPF) and other serious respiratory conditions. Headquartered in Seattle, Washington, with an additional office in San Diego, the company specializes in an inhaled formulation of pirfenidone, known as AP01. This formulation aims to enhance the amount of medication that reaches the lungs while minimizing side effects associated with oral treatments. Established in 2011, Avalyn Pharma is dedicated to improving care and outcomes for patients with severe respiratory diseases through its advanced therapeutic pipeline and novel inhaled therapeutics. Initially named Genoa Pharmaceuticals, the company rebranded to its current name in July 2017.

Inhibrx, Inc. is a clinical-stage biotechnology company headquartered in La Jolla, California, that specializes in developing a diverse pipeline of novel biologic therapeutic candidates. The company's focus areas include oncology, orphan diseases, and infectious diseases, employing advanced protein engineering techniques, including its proprietary sdAb platform. Among its leading candidates is INBRX-109, a multivalent agonist of death receptor 5, currently in Phase 1 trials for treating solid tumors, including sarcomas. Another candidate, INBRX-105, serves as an antagonist of programmed death ligand 1 (PD-L1) and a conditional agonist of 4-1BB, also in Phase 1 trials for PD-L1 expressing tumors. Additional candidates include INBRX-101, aimed at alpha-1 antitrypsin deficiency, and INBRX-103, targeting cluster of differentiation 47. The company's preclinical programs encompass innovative therapies such as INBRX-106, a hexavalent agonist of OX40, and INBRX-111, a multifunctional antibody targeting Pseudomonas aeruginosa. Founded in 2010, Inhibrx has established collaborations with prominent organizations and received funding from various granting agencies.

Rapport Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing precision medicines for neurological disorders. The company focuses on creating transformational small-molecule therapies aimed at patients with central nervous system conditions. Leveraging foundational research in neuronal receptor biology, Rapport Therapeutics maps and targets specific neuronal receptor complexes, which are complex assemblies of proteins that include principal receptor subunits and receptor-associated proteins. These receptor-associated proteins are essential for regulating receptor expression and functionality, allowing the company to innovate in the treatment of neurological diseases.

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 developing innovative treatments for retinal diseases and certain cancers. The company specializes in designing short biomimetic peptides that target angiogenesis and lymphangiogenesis, utilizing advanced bioinformatics and systems biology approaches. Its lead product candidates, including AXT107, AXT201, AXT301, and AXT501, are aimed at addressing conditions such as diabetic macular edema, age-related macular degeneration, and macular edema from retinal vein occlusion, all of which are significant causes of blindness in the western world. Additionally, AXT201, AXT301, and AXT501 are in early development stages for treating various solid tumors. AsclepiX Therapeutics employs cutting-edge biomaterials and drug delivery systems, including biodegradable nano and microparticles, to enhance the therapeutic efficacy of its peptide-based treatments. Incorporated in 2011 and headquartered in Baltimore, Maryland, the company is leveraging its platform technology to advance solutions in both ophthalmology and oncology.

Lyra Therapeutics, Inc. is a clinical-stage therapeutics company dedicated to developing and commercializing innovative drug and delivery solutions for the localized treatment of ear, nose, and throat diseases. The company utilizes its proprietary XTreo technology platform, which allows for the precise and sustained delivery of medications directly to affected tissues through a single administration. Its lead product candidates, LYR-210 and LYR-220, are bioresorbable polymeric matrices designed for non-invasive in-office procedures, providing up to six months of continuous drug therapy for chronic rhinosinusitis. The active ingredient in these products is mometasone furoate, known for its established efficacy and safety profile in various FDA-approved medications. Founded in 2005 and headquartered in Watertown, Massachusetts, Lyra Therapeutics aims to improve treatment outcomes for patients suffering from ENT diseases.

OnKure Therapeutics is a clinical-stage biotechnology company dedicated to the discovery and development of innovative cancer therapies. The company specializes in creating less toxic kinase inhibitor drugs and epigenetic therapies, including selective inhibitors of histone deacetylases. Its lead candidate, OKI-179, has demonstrated significant efficacy in preclinical models of hematological and solid tumor cancers, effectively reducing tumor growth both alone and in combination with other targeted therapies. Additionally, OnKure is advancing its HDAC3 selective inhibitor, OKI-422, into preclinical toxicology studies. The company's drug candidates are designed to directly target malignant cells, enhance the effects of small molecule therapies, and optimize the tumor microenvironment for improved checkpoint inhibition, addressing unmet medical needs in cancer treatment.

TORL BioTherapeutics is a clinical-stage biopharmaceutical company dedicated to developing innovative, antibody-based therapies aimed at enhancing the lives of cancer patients. The company specializes in antibody-drug conjugates and other biologics, focusing on oncologic diseases with significant unmet medical needs. TORL BioTherapeutics employs a strategy that involves target identification and early discovery work, allowing them to selectively advance drug programs through novel licensing agreements. This approach enables their researchers to develop proprietary drugs with unique and optimized profiles, contributing to the advancement of cancer treatment options.

Cargo Therapeutics is a clinical-stage biotechnology company focused on developing innovative CAR T-cell therapies to improve outcomes for cancer patients. The company aims to address the challenges faced by existing autologous CAR therapies, which are only curative for less than half of cancer patients and hindered by issues such as manufacturing limitations, supply challenges, and reimbursement barriers. Cargo Therapeutics is advancing next-generation cell therapies designed to enhance effectiveness, safety, and supply reliability. One of its key products, CRG-022, is a novel CAR T-cell candidate targeting CD22, an antigen present in a majority of B-cell malignancies, specifically designed to overcome treatment resistance. By pursuing these novel solutions, Cargo Therapeutics seeks to facilitate broader access to curative therapies and significantly improve the treatment experience for cancer patients.

Fusion Pharmaceuticals Inc. is a clinical-stage biopharmaceutical company based in Hamilton, Canada, specializing in the development of next-generation radiopharmaceuticals as precision medicines for challenging cancers. The company leverages its Targeted Alpha Therapies platform and proprietary Fast-Clear linker technology to connect alpha particle-emitting isotopes with antibodies and other targeting molecules, enabling selective delivery of cytotoxic payloads to tumors. Its lead product candidate, FPI-1434, is currently undergoing Phase 1 clinical trials as a monotherapy for solid tumors expressing insulin-like growth factor 1 receptor. Additionally, Fusion is conducting preclinical studies to explore the combination of FPI-1434 with approved checkpoint inhibitors and DNA damage response inhibitors to enhance anti-tumor activity. The company is also advancing FPI-1966, aimed at treating head and neck and bladder cancers that express fibroblast growth factor receptor. Founded in 2014, Fusion Pharmaceuticals aims to innovate within the field of radiopharmaceuticals, transforming internalizing antibodies into effective cytotoxic drugs for improved cancer treatment outcomes.

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.

Zenas BioPharma is a clinical-stage biopharmaceutical company headquartered in Florida, USA, that specializes in developing immune-based therapies for patients. The company’s lead product candidate, obexelimab, is a bifunctional monoclonal antibody designed to target both CD19 and FcγRIIb, which are found in B cell lineages. This innovative approach aims to inhibit the activity of cells involved in various autoimmune diseases while preserving their presence, thus avoiding depletion. Through its focus on these advanced therapies, Zenas BioPharma seeks to address unmet medical needs in the treatment of autoimmune conditions.

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.

SpringWorks Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for patients with rare diseases and cancer. Founded in 2017 and headquartered in Stamford, Connecticut, the company employs a precision medicine approach to create targeted oncology solutions. Its lead product candidate, nirogacestat, is an oral small molecule gamma secretase inhibitor currently in Phase III clinical trials for desmoid tumors. Additionally, SpringWorks is advancing mirdametinib, an oral small molecule MEK inhibitor, which is undergoing Phase IIb trials for neurofibromatosis type 1-associated plexiform neurofibromas. The company is also exploring various combination therapies involving nirogacestat and mirdametinib for multiple myeloma and solid tumors. SpringWorks has established collaborations with notable organizations to enhance its research and development efforts, aiming to unlock new treatment options for underserved patient populations.

MeiraGTx Holdings is a clinical-stage gene therapy company dedicated to developing and commercializing innovative gene therapy products aimed at transforming the lives of patients with acquired and inherited disorders. The company focuses on addressing serious medical conditions, primarily targeting disorders related to the eye, salivary gland, and central nervous system. Its research and development efforts leverage a portfolio approach, which includes licensing, acquiring, and developing advanced technologies to support a diverse range of product candidates. Key pipeline products include AAV-CNGB3, AAV-CNGA3, and AAV-RPGR, among others. MeiraGTx operates in the United States, the United Kingdom, and the European Union, with plans to expand its focus to develop additional gene therapy treatments for various serious diseases in the future.

Cerebral Therapeutics, Inc. is a clinical-stage pharmaceutical company based in Aurora, Colorado, focused on developing innovative drug-device combination therapies for neurologic diseases, particularly refractory epilepsy. The company employs a unique implanted, refillable catheter and pump system to deliver a continuous intracerebroventricular (ICV) dose of valproic acid, an anti-epileptic drug formulated as CT-010. By utilizing reformulated off-patent drugs delivered directly to the brain's ventricles, Cerebral Therapeutics aims to bypass the blood-brain barrier, thereby enhancing treatment outcomes for patients suffering from chronic neurological conditions. Established in 2010, the company is committed to improving the quality of life for those with uncontrolled neurological disorders.

Lyra Therapeutics, Inc. is a clinical-stage therapeutics company dedicated to developing and commercializing innovative drug and delivery solutions for the localized treatment of ear, nose, and throat diseases. The company utilizes its proprietary XTreo technology platform, which allows for the precise and sustained delivery of medications directly to affected tissues through a single administration. Its lead product candidates, LYR-210 and LYR-220, are bioresorbable polymeric matrices designed for non-invasive in-office procedures, providing up to six months of continuous drug therapy for chronic rhinosinusitis. The active ingredient in these products is mometasone furoate, known for its established efficacy and safety profile in various FDA-approved medications. Founded in 2005 and headquartered in Watertown, Massachusetts, Lyra Therapeutics aims to improve treatment outcomes for patients suffering from ENT diseases.

VivoSense, also known as Vivonoetics, is a data analysis and software company that focuses on wearable sensor physiological monitoring solutions for research and clinical trials. The company develops innovative digital clinical measures aimed at enhancing patient clinical research and care. Its platform specializes in providing digital biomarkers derived from wearable sensor data, facilitating the collection of real-world data in regulated clinical trials. By enabling researchers to analyze this data accurately, VivoSense supports informed decision-making and risk evaluation associated with various medical treatments.

DNAnexus, Inc. is a cloud-based provider of genome informatics and data management tools, primarily serving enterprises and organizations involved in genomic medicine. Founded in 2009 and headquartered in Mountain View, California, the company offers a platform that facilitates the analysis of raw sequencing data and the integration of genomic data with clinical information. Its suite of products includes MOSAIC Microbiome for collaborative research on the human microbiome, DNAnexus Apollo for clinico-genomic data exploration, and DNAnexus Titan for data analysis. Additionally, the DNAnexus Portals provide secure online workspaces for cross-disciplinary collaboration, while the Clinico-Genomic Data Solution addresses the need for disease-specific datasets through partnerships with healthcare providers. The company has established itself as a key player in the biopharmaceutical, diagnostic, and academic research sectors, enabling clients to tackle complex challenges in human health with enhanced security and scalability. DNAnexus also maintains a strategic alliance with WuXi NextCODE Genomics and has locations in San Francisco, London, and Prague.

Endeavor BioMedicines is a clinical-stage company based in Solana Beach, California, founded in 2018. The company is dedicated to developing innovative treatments that address the underlying causes of pulmonary fibrosis, particularly idiopathic pulmonary fibrosis (IPF). Endeavor BioMedicines is focused on creating a small-molecule inhibitor that has the potential to not only halt the progression of IPF but also to reverse its effects. By concentrating on these advancements, the company aims to enable healthcare professionals to provide more precise treatments for terminal conditions, ultimately improving patient outcomes and enhancing quality of life.

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.

Mythic Therapeutics is a biotechnology company dedicated to advancing cancer treatments through innovative therapies. The company specializes in the development of antibody-drug conjugates (ADCs), utilizing a unique protein engineering approach that enhances the effectiveness of these targeted therapies. Mythic Therapeutics' technology significantly improves the uptake of therapeutic agents in cancerous cells while minimizing their release in healthy cells, thereby increasing efficacy across various cancer types. This method not only boosts the potency of the treatments but also ensures a favorable safety profile, enabling healthcare providers to harness the full potential of ADCs. As such, Mythic Therapeutics aims to make a meaningful impact on cancer care by addressing a broad spectrum of tumor targets.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on developing precision oncology therapeutics. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond positively to specific treatments, thereby enhancing the effectiveness of its drug development efforts. Acrivon's pipeline features its lead program, ACR-368 (prexasertib), which is currently in Phase 2 trials, alongside several preclinical programs targeting key components of DNA damage response and cell cycle regulation, including WEE1 and PKMYT1. By concentrating on complex oncology drug targets that are often not addressed by traditional therapies, Acrivon aims to expedite the advancement of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials.

CinCor Pharma, Inc. is a clinical-stage biopharmaceutical company based in Cincinnati, Ohio, founded in 2018. The company specializes in the development of innovative treatments for cardiovascular, metabolic, and kidney diseases. Its primary focus is on CIN-107, an aldosterone synthase inhibitor aimed at addressing resistant hypertension and primary aldosteronism. By advancing its clinical candidates toward marketing approval, CinCor seeks to provide effective solutions for patients suffering from these conditions.

Science 37, Inc. is a technology-enabled clinical trial company that specializes in developing patient-centric models for clinical research, aimed at accelerating biomedical discovery. The company utilizes its innovative platform, NORA (Network Oriented Research Assistant), a cloud-based mobile research tool that facilitates communication between researchers, patients, and mobile nurses through videos, surveys, and photographs. By providing decentralized clinical trial services, Science 37 enables access to diverse patient populations that traditional site-based research may overlook. The company has established itself as a leader in conducting virtual trials, leveraging an extensive network of telemedicine investigators and home-health nurses to enhance patient enrollment and retention. Serving a wide range of clients, including pharmaceutical sponsors, biotech firms, and academic institutions, Science 37 aims to transform clinical research through its comprehensive, fully integrated trial platform. Founded in 2014 and headquartered in Los Angeles, California, Science 37 continues to innovate in the field of clinical research.

HilleVax is a clinical-stage biopharmaceutical company headquartered in Boston, Massachusetts, founded in 2021. The company specializes in the development and commercialization of innovative vaccines. Its primary focus is on HIL-214, a virus-like particle (VLP) based vaccine candidate aimed at preventing moderate-to-severe acute gastroenteritis (AGE) caused by norovirus infection. HilleVax is dedicated to addressing significant public health challenges through its vaccine initiatives.

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.

Genome Medical, Inc. is a genomic medicine delivery company based in South San Francisco, California, established in 2016. It operates a technology platform known as Genome Care Delivery, which connects individuals and healthcare providers with genetic specialists to facilitate virtual genetic consultations. The company focuses on various areas of clinical care, including cancer, cardiovascular health, reproductive health, pediatrics, and pharmacogenomics. By providing expert genetic assessments and personalized clinical action plans, Genome Medical helps patients understand their genetic risks and make informed treatment decisions. Additionally, the company supports healthcare providers in navigating the growing field of genetics, enhancing disease diagnosis, and improving patient care while aiming to reduce overall healthcare costs.

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, Inc. is a biotechnology company focused on developing innovative therapies for neurodegenerative diseases, particularly Amyotrophic Lateral Sclerosis (ALS), Alzheimer’s disease, and Wolfram syndrome. Founded in 2013 and based in Cambridge, Massachusetts, the company is best known for its investigational therapy AMX0035, a fixed-dose co-formulation of sodium phenylbutyrate and Taurursodiol. This treatment aims to address the energy crisis within mitochondria and the accumulation of toxic, unfolded proteins in the endoplasmic reticulum, both of which contribute to the progression of neurodegenerative conditions. Through its research and development efforts, Amylyx seeks to provide effective solutions to mitigate the suffering caused by these debilitating diseases.

Point Biopharma Global Inc., established in 2019 and headquartered in Toronto, Canada, specializes in the development and commercialization of radioligand therapies for cancer treatment. The company's portfolio comprises several assets, including PNT2002, PNT2004, PNT2003, and PNT2001. Point Biopharma leverages its expertise in radioisotopes like Actinium-225 and Lutetium-177, along with advanced manufacturing technology and a patient-centric approach, to revolutionize theragnostic drug development and radioligand commercialization on a global scale.

RayzeBio, Inc. is a biotechnology company based in San Diego, California, founded in 2020. The company specializes in developing tumor-targeted small molecule medicines that leverage radioisotopes to improve cancer treatment outcomes. RayzeBio focuses on creating innovative radiopharmaceuticals, particularly utilizing alpha-emitting radioisotopes like Actinium-225, to target solid tumors effectively. With a robust pipeline of drug candidates, RayzeBio is committed to addressing significant market opportunities in oncology through late-stage clinical programs, development initiatives, and discovery efforts. The company's mission is to provide effective therapeutic solutions aimed at defeating cancer.

Juno Diagnostics, Inc. is a health technology company based in San Diego, California, founded in 2017. The company specializes in developing cell-free DNA-based noninvasive prenatal testing, providing women and their healthcare providers with crucial genetic information. Juno Diagnostics aims to democratize access to high-value genetic data by creating simple and cost-effective testing devices that facilitate point-of-need genetic testing. Their innovative technology enhances sample collection and delivers high-quality test results without the need for traditional phlebotomy procedures, thus reducing costs and lead times associated with genetic testing. Additionally, Juno is involved in the development of genetic testing tools for applications in epigenetics, inherited diseases, and cancer diagnostics.

Forge Biologics, Inc. is a contract development and manufacturing organization (CDMO) specializing in gene therapies and genetic medicines. Founded in 2019 and based in Columbus, Ohio, the company focuses on developing and manufacturing therapeutics, particularly for viral vector gene therapies aimed at treating genetic diseases. Notably, it offers a novel approach combining adeno-associated virus (AAV) and umbilical cord transplant to address infantile Krabbe disease, a severe neurodegenerative disorder. Additionally, Forge Biologics collaborates with scientists, physicians, biotech and pharmaceutical companies, and patient groups to facilitate the progression of gene therapy programs from preclinical development through to clinical and commercial-scale manufacturing. The company is committed to enabling access to transformative medicines for patients suffering from rare genetic diseases.

Renovia Inc., founded in 2016 and based in Boston, Massachusetts, was a healthcare company focused on developing prescription digital therapeutics for pelvic floor disorders. The company's innovative device provided a clinically validated treatment for conditions such as stress, mixed, and urgent urinary incontinence, including overactive bladder. This technology allowed for real-time visualization of pelvic movement during muscle training sessions, facilitating a non-surgical and drug-free approach to treatment. However, Renovia ceased operations in late 2022 following a strategic review by its investors, and in January 2023, the majority of its assets, including intellectual property, were acquired by Axena Health, Inc.

Icosavax, Inc. is a biotechnology company based in Seattle, Washington, founded in 2017. The company specializes in developing vaccines utilizing its proprietary virus-like particle (VLP) platform technology. This innovative approach allows for the multivalent, particle-based display of complex viral antigens, which is aimed at generating broad and durable immune responses. Icosavax focuses primarily on life-threatening respiratory diseases, with its lead vaccine candidate, IVX-121, targeting respiratory syncytial virus (RSV) in older adults. The company's pipeline also includes candidates for human metapneumovirus (hMPV) and SARS-CoV-2, reflecting its commitment to advancing vaccine solutions for infectious diseases.

Scribe Therapeutics Inc. is a biotechnology company focused on developing innovative therapies utilizing engineered CRISPR technologies to address genetic disorders. Founded in 2018 and based in Berkeley, California, Scribe Therapeutics is known for its proprietary X-Editing (XE) molecules, which are advanced CRISPR enzymes designed to enhance efficacy, specificity, and deliverability compared to existing genome editing methods. The company's platform aims to overcome the limitations of current technologies by creating custom-engineered enzymes and delivery systems that facilitate precise genetic modifications. Scribe Therapeutics is committed to establishing CRISPR-based therapies as a new standard of clinical care, with the potential to significantly impact the treatment landscape for a variety of diseases, including those related to immuno-oncology, degenerative disorders, and enzyme replacement therapy.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that focuses on targeted DNA integration to address severe genetic diseases. The firm employs advanced technology to enable the precise insertion of genetic sequences, effectively correcting mutations, replacing malfunctioning genes, and introducing therapeutic genetic materials into specific regions of the genome. By harnessing high-efficiency targeted gene integration, Graphite Bio aims to develop novel therapies that could potentially cure a wide array of serious and life-threatening conditions. Formerly known as Integral Medicines, Inc., the company rebranded in August 2020 and has operated since its incorporation in 2019. Graphite Bio is committed to the pioneering of a precision gene editing approach designed to fulfill the medical challenge of accurately "finding and replacing" genes.

OnKure Therapeutics is a clinical-stage biotechnology company dedicated to the discovery and development of innovative cancer therapies. The company specializes in creating less toxic kinase inhibitor drugs and epigenetic therapies, including selective inhibitors of histone deacetylases. Its lead candidate, OKI-179, has demonstrated significant efficacy in preclinical models of hematological and solid tumor cancers, effectively reducing tumor growth both alone and in combination with other targeted therapies. Additionally, OnKure is advancing its HDAC3 selective inhibitor, OKI-422, into preclinical toxicology studies. The company's drug candidates are designed to directly target malignant cells, enhance the effects of small molecule therapies, and optimize the tumor microenvironment for improved checkpoint inhibition, addressing unmet medical needs in cancer treatment.

Pipeline Therapeutics Inc. is a biotechnology company based in San Diego, California, specializing in the development and commercialization of small molecules aimed at neuroregeneration. Founded in 2017, the company focuses on promoting functional recovery for various neurological disorders. Its lead product candidate, PIPE-505, is a small molecule gamma secretase inhibitor designed to treat mild-to-moderate sensorineural hearing loss associated with cochlear synaptopathy. Additionally, Pipeline Therapeutics has a portfolio of earlier-stage programs, including PIPE-307, which targets remyelination and axonal repair to address conditions like multiple sclerosis. By harnessing the natural repair processes of different nervous system cell types, the company seeks to provide innovative treatments that repair damage within the nervous system.

Day One Biopharmaceuticals is a clinical-stage biotechnology company dedicated to developing targeted cancer therapies for patients of all ages, with a particular emphasis on pediatric patients who have historically been underserved in cancer treatment advancements. The company is committed to addressing the unique needs of children and adults facing cancer by creating therapies that are informed by the biology of childhood cancers. Day One Biopharmaceuticals aims to bring promising treatments rapidly to market, focusing on those that can benefit both younger and older patients. The company's lead product candidate, DAY101, is an oral, brain-penetrant pan-RAF kinase inhibitor designed to target genetically defined cancers. By licensing and acquiring innovative products from research institutions and other biopharmaceutical companies, Day One Biopharmaceuticals seeks to provide effective treatment options for families confronted with the challenges of a cancer diagnosis.

Affinivax, Inc. is a biotechnology company based in Lexington, Massachusetts, specializing in the development of vaccines using its innovative Multiple Antigen Presenting System (MAPS) technology. Founded in 2014, the company aims to enhance the protection against a range of infectious diseases that affect both children and adults globally. MAPS technology allows for the effective binding of protective polysaccharides and proteins within a single vaccine, inducing robust immune responses. Affinivax is advancing vaccine programs targeting significant pathogens, including Streptococcus pneumoniae, which contributes to considerable morbidity and mortality worldwide. The company leverages insights from leading experts in infectious diseases and has secured exclusive intellectual property rights related to its MAPS platform from Boston Children’s Hospital, with notable backing from the Bill & Melinda Gates Foundation.

IsoPlexis is a life science technology company based in Branford, Connecticut, established in 2013. It specializes in developing a single-cell detection system that identifies a wide array of patient immune responses at the single-cell level. The company's platform focuses on cell-based proteomic systems and biomarkers, allowing for the assessment of therapeutic safety and efficacy, monitoring of disease progression, and exploration of functional protein biology and cellular signaling networks. IsoPlexis's technology enables clients to predict cancer patient responses to various novel immunotherapies and cellular therapies, thereby enhancing therapeutic development and informing clinical decision-making.

IsoPlexis is a life science technology company based in Branford, Connecticut, established in 2013. It specializes in developing a single-cell detection system that identifies a wide array of patient immune responses at the single-cell level. The company's platform focuses on cell-based proteomic systems and biomarkers, allowing for the assessment of therapeutic safety and efficacy, monitoring of disease progression, and exploration of functional protein biology and cellular signaling networks. IsoPlexis's technology enables clients to predict cancer patient responses to various novel immunotherapies and cellular therapies, thereby enhancing therapeutic development and informing clinical decision-making.

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.

Achilles Therapeutics Ltd. is a biopharmaceutical company based in London, United Kingdom, specializing in the development of patient-specific immunotherapies designed to combat cancer. Founded in 2016, the company focuses on personalized vaccines and T cell therapies that utilize the body's immune system to target and eliminate cancer cells. Achilles Therapeutics aims to identify and target truncal tumor neoantigens and other markers present on cancer cells, allowing for precise attacks on tumors while sparing healthy tissue. The company is currently conducting clinical trials, including the CHIRON trial for non-small-cell lung cancer and the THETIS trial for recurrent or metastatic melanoma, to evaluate the efficacy and safety of its innovative therapies.

Adagio Medical, Inc. is a medical device company specializing in the design and manufacture of innovative solutions for treating cardiac arrhythmias. Founded in 2011 and based in Laguna Hills, California, Adagio Medical develops advanced cryoablation technologies, including its continuous lesion ablation system and a range of specialized catheters. The company's products are aimed at electrophysiologists and are designed to create safe, continuous, transmural lesions to effectively address various heart conditions such as paroxysmal and persistent atrial fibrillation, atrial flutter, and ventricular tachycardia. Adagio Medical is committed to advancing the treatment of cardiovascular diseases through its research and development efforts.

LianBio is a biopharmaceutical company founded in 2019, headquartered in Shanghai, China, with an additional office in Princeton, New Jersey. The company is dedicated to the discovery and development of therapeutic drugs aimed at treating oncology and cardiorenal diseases, as well as addressing other unmet medical needs in Greater China and major Asian markets. LianBio focuses on in-licensing assets and collaborates with leading partners to enhance its pipeline, which includes clinically validated product candidates across various therapeutic areas such as cardiovascular, oncology, ophthalmology, inflammatory diseases, and respiratory indications. The company aims to accelerate the availability of innovative medicines to improve patient outcomes through a science-driven approach.

Impulse Dynamics N.V. is a medical device company that specializes in developing innovative electrical therapies for chronic heart failure. Founded in 1996 and headquartered in Willemstad, Curacao, the company also maintains offices in New York and Hong Kong, with product development centers located in New Jersey and Israel. Its primary offering, the CCM® therapy, utilizes cardiac contractility modulation technology to deliver non-excitatory electrical impulses to the heart, enhancing its contraction and improving blood flow to the body. This minimally invasive treatment option addresses the needs of heart failure patients who have limited alternatives. Impulse Dynamics conducts clinical trials to assess the safety and efficacy of its therapies and collaborates with universities and hospitals for ongoing research and development.

AavantiBio, Inc. is a biopharmaceutical company focused on developing gene transfer and gene editing therapies aimed at treating rare genetic diseases. Incorporated in 2019 and based in Gainesville, Florida, the company primarily concentrates on Friedreich’s Ataxia, a rare inherited disorder that leads to significant cardiac and central nervous system dysfunction. AavantiBio is committed to transforming the lives of patients suffering from this condition by advancing innovative therapies that address unmet medical needs in the field of genetic disorders. The company’s efforts are directed toward both treatment development and research, with the goal of providing effective solutions for patients facing the challenges of rare diseases.

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.

Escient Pharmaceuticals, Inc. is a biotechnology company based in San Diego, California, specializing in the development and manufacture of G protein-coupled receptor (GPCR)-targeted drugs aimed at treating neuro-immuno-inflammatory and autoreactive diseases. Founded in 2017, Escient focuses on harnessing the therapeutic potential of specific orphan GPCRs, particularly the Mas-Related G-Protein Receptors (Mrgprs). The company aims to provide innovative therapies for a variety of conditions, including mast cell-mediated disorders and cholestatic pruritus, thereby expanding treatment options for healthcare providers facing these complex diseases.

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.

Rain Oncology Inc. is a late-stage precision oncology company focused on developing targeted therapies aimed at oncogenic drivers. The company employs a genetic selection strategy to identify patients who are most likely to benefit from its treatments, utilizing a tumor-agnostic approach that prioritizes the underlying genetics of tumors over traditional histological classifications. Its lead product candidate, milademetan, is a small molecule oral inhibitor of MDM2, which plays a role in various cancers. Additionally, Rain Oncology is advancing a preclinical program aimed at inducing synthetic lethality in cancer cells by inhibiting RAD52, further diversifying its therapeutic pipeline.

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.

Freenome, Inc. is an artificial intelligence genomics biotech company based in South San Francisco, California, founded in 2014. The company focuses on developing accurate, accessible, and non-invasive blood tests for the early detection of cancer and treatment selection. Utilizing a proprietary multi-omics platform, Freenome employs advanced algorithms to analyze cell-free genomic data, which aids in identifying early-stage cancers and other diseases. This innovative approach aims to transform disease management by enabling timely interventions and proactive treatment strategies. In addition to its diagnostic solutions, Freenome also offers clinical research services to further advance cancer detection and management.

ReViral Ltd is a biotechnology company based in Stevenage, United Kingdom, specializing in the discovery and development of antiviral drugs targeting respiratory syncytial virus (RSV). Founded in 2011, ReViral is focused on creating innovative first-in-class compounds, including a highly potent fusion inhibitor designed specifically for treating severe RSV infections, particularly in vulnerable populations such as neonates. By advancing these novel antiviral treatments, ReViral aims to address significant unmet medical needs and expand the antiviral market, ultimately improving patient outcomes for those affected by RSV.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company based in San Mateo, California, focused on the discovery and development of innovative cancer therapeutics. Established in 2017, the company aims to address historically challenging cancer targets through precision medicine. Its primary product candidates include entospletinib, a selective inhibitor of spleen tyrosine kinase intended for acute myeloid leukemia patients, and KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9 for treating MYC-amplified solid tumors. Kronos Bio employs advanced technologies, such as high-throughput small-molecule microarrays and targeted protein degradation, to identify potent and selective compounds that target dysregulated transcription factors and other key drivers of oncogenic signaling.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, focused on developing adeno-associated virus (AAV)-based therapies for monogenic diseases of the central nervous system (CNS). Founded in 2019, the company aims to create curative treatments for severe and life-threatening conditions. Its pipeline includes several product candidates, 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 collaborates with The University of Texas Southwestern Medical Center to enhance its development and commercialization efforts, leveraging expertise in gene therapy to advance its mission of eradicating monogenic CNS 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.

Forge Biologics, Inc. is a contract development and manufacturing organization (CDMO) specializing in gene therapies and genetic medicines. Founded in 2019 and based in Columbus, Ohio, the company focuses on developing and manufacturing therapeutics, particularly for viral vector gene therapies aimed at treating genetic diseases. Notably, it offers a novel approach combining adeno-associated virus (AAV) and umbilical cord transplant to address infantile Krabbe disease, a severe neurodegenerative disorder. Additionally, Forge Biologics collaborates with scientists, physicians, biotech and pharmaceutical companies, and patient groups to facilitate the progression of gene therapy programs from preclinical development through to clinical and commercial-scale manufacturing. The company is committed to enabling access to transformative medicines for patients suffering from rare genetic diseases.

Verona Pharma plc is a clinical-stage biopharmaceutical company based in London, dedicated to developing and commercializing innovative therapies for respiratory diseases with significant unmet medical needs. Founded in 2005, the company primarily focuses on its lead product candidate, ensifentrine, an inhaled dual inhibitor of phosphodiesterase enzymes 3 and 4, which functions as both a bronchodilator and an anti-inflammatory agent. Ensifentrine is currently in Phase 2b clinical development as a nebulized formulation for the maintenance treatment of chronic obstructive pulmonary disease (COPD), with additional formulations including a dry powder inhaler and a pressurized metered-dose inhaler also under development. In addition to COPD, Verona Pharma is exploring the use of ensifentrine for other respiratory conditions, including cystic fibrosis and asthma, thereby aiming to enhance the health and quality of life for patients suffering from these chronic disorders.

Frequency Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to repairing or reversing damage caused by degenerative diseases through its innovative Progenitor Cell Activation approach. This method utilizes a combination of small molecules to activate progenitor cells in the body, which facilitates the creation of functional tissue without the complexities of genetic engineering. The company’s lead product candidate, FX-322, is currently undergoing phase 2a clinical trials aimed at treating the underlying causes of sensorineural hearing loss. In addition to FX-322, Frequency Therapeutics is exploring treatments for various degenerative conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. Established in 2014 and based in Woburn, Massachusetts, Frequency Therapeutics has established a collaboration agreement with Astellas Pharma Inc. for the development and commercialization of FX-322.

Pear Therapeutics, Inc. is a Boston-based commercial-stage healthcare company specializing in software-based digital therapeutics, also known as Prescription Digital Therapeutics (PDTs). Founded in 2013, the company develops clinically validated software solutions designed to treat a range of behavioral health disorders, including addiction, anxiety, depression, and chronic insomnia. Its suite of products includes mobile digital therapies that reward patients for engagement, and specific tools like reSET and reSET-O, aimed at treating substance use disorders. The company also offers Somryst, which provides neurobehavioral interventions for insomnia, and operates PearConnect, a patient services center that connects patients with advocates and resources, ensuring support throughout their treatment journey. Pear Therapeutics aims to enhance treatment outcomes by integrating digital therapies with pharmaceuticals and providing comprehensive support for patients, clinicians, and payors.

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).

Genome Medical, Inc. is a genomic medicine delivery company based in South San Francisco, California, established in 2016. It operates a technology platform known as Genome Care Delivery, which connects individuals and healthcare providers with genetic specialists to facilitate virtual genetic consultations. The company focuses on various areas of clinical care, including cancer, cardiovascular health, reproductive health, pediatrics, and pharmacogenomics. By providing expert genetic assessments and personalized clinical action plans, Genome Medical helps patients understand their genetic risks and make informed treatment decisions. Additionally, the company supports healthcare providers in navigating the growing field of genetics, enhancing disease diagnosis, and improving patient care while aiming to reduce overall healthcare costs.

AsclepiX Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative treatments for retinal diseases and certain cancers. The company specializes in designing short biomimetic peptides that target angiogenesis and lymphangiogenesis, utilizing advanced bioinformatics and systems biology approaches. Its lead product candidates, including AXT107, AXT201, AXT301, and AXT501, are aimed at addressing conditions such as diabetic macular edema, age-related macular degeneration, and macular edema from retinal vein occlusion, all of which are significant causes of blindness in the western world. Additionally, AXT201, AXT301, and AXT501 are in early development stages for treating various solid tumors. AsclepiX Therapeutics employs cutting-edge biomaterials and drug delivery systems, including biodegradable nano and microparticles, to enhance the therapeutic efficacy of its peptide-based treatments. Incorporated in 2011 and headquartered in Baltimore, Maryland, the company is leveraging its platform technology to advance solutions in both ophthalmology and oncology.

C4 Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutic candidates aimed at targeting and eliminating disease-causing proteins for the treatment of cancer, neurodegenerative diseases, and other conditions. The company's lead product candidate, CFT7455, is an orally bioavailable degrader targeting IKZF1/3, designed to treat multiple myeloma and various lymphomas. Additionally, C4 Therapeutics is advancing CFT8634, which targets BRD9 for synovial sarcoma and SMARCB1-deleted solid tumors, as well as programs targeting BRAF V600E and RET for genetically defined resistant solid tumors. The company employs its proprietary Degronimid platform, which utilizes small molecule binders to flag harmful proteins for degradation by the cellular proteasome system, allowing for the potential treatment of previously undruggable targets. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics engages in strategic collaborations to enhance its research and development efforts.

4D Molecular Therapeutics, Inc. is a clinical-stage gene therapy company based in Emeryville, California, focused on developing innovative gene therapies for unmet medical needs. The company utilizes targeted and evolved adeno-associated virus (AAV) vectors to create its product candidates, which are concentrated in the therapeutic areas of ophthalmology, cardiology, and pulmonology. Key candidates in its portfolio include 4D-125, currently in a Phase I/II trial for X-linked retinitis pigmentosa, and 4D-110, undergoing Phase I trials for choroideremia. Additionally, 4D-310 is in Phase I/II trials for Fabry disease, while investigational drugs 4D-150 and 4D-710 are being developed for wet age-related macular degeneration and cystic fibrosis lung disease, respectively. Founded in 2013, 4D Molecular Therapeutics aims to advance its gene therapies to address significant health challenges.

C4 Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutic candidates aimed at targeting and eliminating disease-causing proteins for the treatment of cancer, neurodegenerative diseases, and other conditions. The company's lead product candidate, CFT7455, is an orally bioavailable degrader targeting IKZF1/3, designed to treat multiple myeloma and various lymphomas. Additionally, C4 Therapeutics is advancing CFT8634, which targets BRD9 for synovial sarcoma and SMARCB1-deleted solid tumors, as well as programs targeting BRAF V600E and RET for genetically defined resistant solid tumors. The company employs its proprietary Degronimid platform, which utilizes small molecule binders to flag harmful proteins for degradation by the cellular proteasome system, allowing for the potential treatment of previously undruggable targets. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics engages in strategic collaborations to enhance its research and development efforts.