EcoR1 Capital

Attovia Therapeutics is a biotherapeutics pipeline developer focused on immune-mediated illness and oncology. The company uses the nanobody platform to create tiny format binders with low picomolar affinity, increased selectivity, quicker internalization, and rapid tissue penetration, thereby assisting cancer patients' treatment.

Tectonic Therapeutic is a biotechnology company dedicated to the discovery and development of innovative drugs that target G protein-coupled receptors (GPCRs). With a focus on therapeutic proteins and antibodies, Tectonic aims to address the complexities of GPCR-targeted drug discovery through its proprietary technology platform, GEODe. The company is committed to unlocking the therapeutic potential of challenging receptors, striving to create biologic medicines that can effectively modify disease progression. Founded by experts in biochemistry and molecular pharmacology, Tectonic combines a strong scientific foundation with a history of entrepreneurial success to advance its mission in the biopharmaceutical landscape.

Neurogene Inc., established in 2018 and headquartered in New York, specializes in developing genetic medicines for treating neurological disorders. The company focuses on utilizing adeno-associated virus (AAV) vectors to deliver therapeutic genes for conditions such as Charcot-Marie Tooth disease type 4J (CMT4J), Aspartylglucosaminuria (AGU), and an undisclosed Lysosomal Storage Disease (LSD). Neurogene aims to provide effective treatment options where none currently exist, aiming to improve the quality of life for patients and families affected by these rare neurological disorders.

Atara Biotherapeutics, Inc. is a biotechnology company focused on developing off-the-shelf T-cell immunotherapies for patients with cancer, autoimmune diseases, and viral infections. The company is advancing tabelecleucel, a T-cell immunotherapy currently in Phase III clinical trials for treating Epstein-Barr virus-associated post-transplant lymphoproliferative disorder, as well as other related hematologic and solid tumors, including nasopharyngeal carcinoma. Atara is also working on next-generation CAR T immunotherapies targeting various indications, including ATA2271 and ATA3271 for mesothelin, and ATA2431 and ATA3219 for B-cell lymphomas. Additionally, its pipeline includes ATA188 for multiple sclerosis and several other candidates for conditions such as acute myeloid leukemia and cytomegalovirus. Founded in 2012, Atara Biotherapeutics is headquartered in South San Francisco, California, and collaborates with leading academic institutions to enhance its research and development efforts.

AnaptysBio, Inc. is a clinical-stage biotechnology company based in San Diego, California, dedicated to developing therapeutic antibodies for inflammation and immuno-oncology. The company employs its proprietary somatic hypermutation platform to discover and optimize antibodies that target various diseases. Among its notable product candidates are Etokimab, an anti-IL-33 treatment aimed at conditions such as atopic dermatitis and eosinophilic asthma, and ANB019, an anti-IL-36 receptor for generalized pustular psoriasis. AnaptysBio is also advancing a range of immuno-oncology products, including checkpoint modulators and bispecific antibodies, to address cancer treatment needs. The company collaborates with notable partners like TESARO, Celgene Corporation, and GlaxoSmithKline to enhance its research and development efforts. Founded in 2005, AnaptysBio aims to fulfill unmet medical needs through innovative antibody therapies.

Tectonic Therapeutic is a biotechnology company dedicated to the discovery and development of innovative drugs that target G protein-coupled receptors (GPCRs). With a focus on therapeutic proteins and antibodies, Tectonic aims to address the complexities of GPCR-targeted drug discovery through its proprietary technology platform, GEODe. The company is committed to unlocking the therapeutic potential of challenging receptors, striving to create biologic medicines that can effectively modify disease progression. Founded by experts in biochemistry and molecular pharmacology, Tectonic combines a strong scientific foundation with a history of entrepreneurial success to advance its mission in the biopharmaceutical landscape.

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.

Ajax Therapeutics is a biotechnology company based in New York City that focuses on developing innovative small molecule therapies for hematologic malignancies. The company utilizes computational chemistry and structure-based technologies to design drugs that target critical cytokine signaling pathways involved in these cancers. By integrating insights from disease biology, genetics, and structural biology with an advanced computational drug discovery platform, Ajax Therapeutics aims to create precisely designed therapeutics that address significant unmet medical needs for patients suffering from hematologic malignancies.

Attovia Therapeutics is a biotherapeutics pipeline developer focused on immune-mediated illness and oncology. The company uses the nanobody platform to create tiny format binders with low picomolar affinity, increased selectivity, quicker internalization, and rapid tissue penetration, thereby assisting cancer patients' treatment.

BridgeBio Oncology Therapeutics is a biopharmaceutical company specializing in the development of precision oncology therapeutics. It focuses on targeting RAS-dependent cancers, leveraging a profound understanding of RAS signaling biology to create drugs that selectively inhibit RAS-driven PI3K activation and a wide spectrum of KRAS mutants. The company aims to provide patients with tailored treatments that address both active and inactive cancer-driving states.

Nkarta Therapeutics is a biopharmaceutical company focused on developing engineered natural killer (NK) cell therapies for treating various cancers and autoimmune diseases. Unlike T-cell therapies, NK cells have an inherent ability to recognize and destroy abnormal cells without genetic alteration, offering promise for diverse tumor types with potentially better tolerated side effects. Nkarta's approach involves leveraging its NK expansion platform technology alongside proprietary cell engineering techniques to generate a substantial supply of enhanced NK cells that can persistently target and eliminate cancer cells.

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.

CRISPR Therapeutics AG is a biotechnology company based in Zug, Switzerland, dedicated to developing transformative gene-based medicines for serious human diseases using CRISPR/Cas9 technology. This revolutionary gene-editing platform allows for precise changes to genomic DNA. The company's portfolio includes therapeutic programs targeting hemoglobinopathies, oncology, regenerative medicine, and rare diseases. Their lead product candidate is CTX001, a gene-edited therapy for transfusion-dependent beta thalassemia or severe sickle cell disease, which has been approved as Casgevy in collaboration with Vertex Pharmaceuticals. Additionally, CRISPR Therapeutics is developing CTX110, an allogeneic CAR-T therapy targeting CD19-positive malignancies, and other programs like CTX120 for multiple myeloma and CTX130 for solid tumors and hematologic malignancies. The company also engages in regenerative medicine programs for diabetes and genetic disease treatments such as glycogen storage disease Ia, Duchenne muscular dystrophy, and cystic fibrosis. CRISPR Therapeutics has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and ViaCyte, Inc.

Kura Oncology is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing targeted therapies for cancer treatment. The company’s primary product candidate, Tipifarnib, is an orally available farnesyl transferase inhibitor currently undergoing Phase II clinical trials for solid tumors, peripheral T-cell lymphomas, and various hematologic malignancies. In addition to Tipifarnib, Kura is advancing other small molecule candidates, including KO-947, which targets dysregulated extracellular signal-related kinases, and KO-539, aimed at inhibiting the menin-mixed lineage leukemia protein interaction. Kura Oncology emphasizes precision medicine, leveraging insights from cancer genomics to create targeted treatments for patients with specific genetic profiles, addressing significant unmet medical needs across various cancer types, including lung, colorectal, thyroid, pancreatic cancers, and blood cancers such as lymphoma and leukemia. The company was founded in 2014 and operates primarily within the United States.

Accent Therapeutics, Inc. is a biopharmaceutical company focused on developing small molecule therapies for oncology, utilizing the emerging field of epitranscriptomics. Founded in 2017 and headquartered in Lexington, Massachusetts, the company explores the post-transcriptional chemical modifications of RNA, which play a crucial role in regulating proteins essential for cellular growth and differentiation. By precisely targeting cancer-associated RNA-modifying proteins, Accent Therapeutics aims to innovate and deliver transformative therapies that can significantly improve patient outcomes in cancer treatment.

Zymeworks Inc. is a clinical-stage biopharmaceutical company based in Vancouver, Canada, focused on the discovery, development, and commercialization of innovative biotherapeutics for cancer treatment. The company's lead candidates include ZW25, a bispecific antibody undergoing Phase I and II clinical trials for various tumors, including biliary tract and gastroesophageal adenocarcinomas, and ZW49, a bispecific antibody-drug conjugate in Phase I trials for advanced HER2-expressing cancers. Zymeworks employs a computational biotechnology approach, utilizing proprietary molecular modeling and simulation technologies to optimize therapeutic antibodies and other protein-based therapeutics. The company has established strategic partnerships with notable organizations such as Merck, Eli Lilly, and Bristol-Myers Squibb, as well as collaborations aimed at advancing antibody discovery and development. Founded in 2003, Zymeworks is dedicated to addressing unmet medical needs through its advanced therapeutic platforms and integrated drug development capabilities.

Arvinas, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, that specializes in the discovery, development, and commercialization of innovative therapies aimed at degrading disease-causing proteins. The company's lead product candidates include ARV-110, a proteolysis targeting chimera (PROTAC) currently in phase I clinical trials for metastatic castration-resistant prostate cancer, and ARV-471, which targets the estrogen receptor for patients with metastatic ER-positive/HER2-negative breast cancer. In addition to these, Arvinas is developing various other PROTACs aimed at degrading clinically relevant androgen receptor mutations and treatments for neurodegenerative diseases, including tauopathies. The company has established collaborations with prominent pharmaceutical firms such as Pfizer Inc., Genentech, and Bayer AG, enhancing its research capabilities and reach within the biopharmaceutical landscape. Founded in 2015, Arvinas aims to improve patient outcomes by eliminating harmful proteins from the body through its proprietary technology.

Terremoto Biosciences is a biotechnology company focused on the development and discovery of small-molecule medicines through innovative drug delivery systems. By utilizing lysine-based covalency, the company aims to optimize clinical profiles and enhance therapeutic benefits for various diseases. Terremoto's platform is designed to improve existing medications while also facilitating the creation of new therapies aimed at addressing critical healthcare challenges. Through its research and technology, Terremoto Biosciences strives to provide effective solutions for the treatment of devastating diseases, contributing to advancements in the healthcare sector.

Aligos Therapeutics, Inc. is a clinical-stage biopharmaceutical company founded in 2018 and based in South San Francisco, California. The company is dedicated to developing innovative therapeutics to meet unmet medical needs in viral and liver diseases. Its primary focus is on chronic hepatitis B (CHB), with a robust pipeline that includes several drug candidates: ALG-010133, a synthetic oligonucleotide currently in Phase I clinical trials; ALG-000184, a capsid assembly modulator; ALG-020572, another oligonucleotide; and ALG-125097, an siRNA drug candidate. Additionally, Aligos is advancing ALG-055009, a small molecule THR-ß agonist aimed at treating non-alcoholic steatohepatitis (NASH). Aligos aims to leverage its expertise in liver diseases and viral infections to develop targeted antiviral therapies and establish itself as a leader in this therapeutic area.

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Actio Biosciences specializes in developing precision medicines tailored to homogeneous rare patient populations. Its pipeline focuses on creating new drugs designed to enhance clinical success rates and ultimately deliver treatments to all patients in need, including those with challenging-to-cure illnesses.

Immunome Inc., founded in 2006 and headquartered in Exton, Pennsylvania, is a biotechnology company focused on discovering, developing, and commercializing highly targeted and native human cancer antibodies against universal cancer antigens. The company utilizes its proprietary RealMAb technology to identify novel antigens and the corresponding native human antibodies that target them. Additionally, Immunome employs ScreenMab multiplex functional screening technology to pinpoint monoclonal antibodies with high specificity for tumor neoantigens.

ORIC Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing innovative therapies to combat cancer by overcoming resistance mechanisms. The company's lead product candidate, ORIC-101, is a small molecule antagonist of the glucocorticoid receptor, which plays a significant role in resistance to various cancer treatments across multiple solid tumors. Another key candidate, ORIC-533, is an orally bioavailable inhibitor of CD73, targeting a critical pathway in the adenosine metabolism that contributes to resistance against chemotherapy and immunotherapy. Additionally, ORIC is working on a pipeline of precision medicines aimed at addressing other cancer resistance mechanisms. Founded in 2014 and headquartered in South San Francisco, California, ORIC Pharmaceuticals leverages the expertise of its scientific founders, who have extensive backgrounds in cancer research and target discovery.

Paratus Sciences is a biotechnology company focused on enhancing human health and health security through the study of bat biology. By investigating the unique characteristics of the bat genome, the company aims to identify and develop therapeutics for various diseases. Leveraging insights gained from bat biology, Paratus Sciences seeks to enable patients to combat and potentially cure the diseases they face. Through its innovative approach, the company aspires to contribute significantly to advancements in medical treatments and public health.

BioAtla, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-based therapeutics for cancer treatment. The company utilizes its proprietary platforms, Conditionally Active Biologics (CAB) and Comprehensive Integrated Antibody Optimization (CIAO!), to create highly selective and effective drugs. Its lead product candidate, BA3011, is a conditionally active biologic antibody-drug conjugate targeting soft tissue and bone sarcomas, non-small cell lung cancer, and other tumor types. Additionally, BioAtla is developing BA3021, aimed at non-small cell lung cancer and melanoma, as well as BA3071, which targets multiple cancers, including renal cell carcinoma and hepatocellular carcinoma. The company holds over 150 issued patents and applications, emphasizing its commitment to improving drug selectivity and safety while expanding the range of treatable cancers.

Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing gene and cell therapies for life-threatening rare genetic diseases. Its lead programs include EB-101, an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa; ABO-102, an adeno-associated virus-based gene therapy for Sanfilippo syndrome type A; and ABO-101, an AAV-based gene therapy for Sanfilippo syndrome type B. The company also develops therapies for Batten disease, cystic fibrosis, and genetic eye disorders. Additionally, Abeona Therapeutics is advancing the AIM Vector Platform, which includes over 100 next-generation AAV capsids for delivering gene therapies to various organs. The company operates a manufacturing facility producing therapies and vectors for preclinical and clinical studies.

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.

Ventyx Biosciences, Inc. is a clinical-stage biopharmaceutical company based in Encinitas, California, that specializes in developing innovative therapies for autoimmune diseases and inflammatory disorders. Incorporated in 2018, Ventyx focuses on creating selective inhibitors targeting TYK2, a key mediator in various inflammatory conditions. The company's clinical pipeline includes VTX958, an oral allosteric TYK2 inhibitor currently in Phase 1 trials, which aims to provide a safer alternative by avoiding the toxicities linked to broader Janus kinase inhibition. Additionally, Ventyx is advancing VTX002, a Phase 2-ready S1P1 receptor modulator intended for ulcerative colitis treatment, and VTX2735, a Phase 1 peripheral inhibitor of the NLRP3 inflammasome, which plays a significant role in multiple inflammatory diseases.

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.

Kymera Therapeutics, Inc. is a biopharmaceutical company based in Watertown, Massachusetts, specializing in targeted protein degradation. Founded in 2015, the company employs a proprietary platform that utilizes the body's natural protein degradation system to selectively degrade disease-causing proteins. Kymera is advancing several therapeutic programs, including its IRAK4 program aimed at treating immunology-inflammation diseases such as hidradenitis suppurativa, atopic dermatitis, and rheumatoid arthritis. Additionally, it is developing the IRAKIMiD program for MYD88-mutated diffuse large B cell lymphoma and the STAT3 program targeting hematologic malignancies, solid tumors, and autoimmune diseases. By focusing on previously untreatable conditions and using innovative small molecule modalities, Kymera Therapeutics aims to revolutionize drug discovery and create effective treatments for challenging diseases.

AlloVir, Inc. is a clinical-stage cell therapy company based in Cambridge, Massachusetts, specializing in the research and development of allogeneic, off-the-shelf multi-virus specific T cell (VST) therapies aimed at preventing and treating serious viral-associated diseases. The company's lead product, Viralym-M, targets multiple viruses including BK virus, cytomegalovirus, adenovirus, Epstein-Barr virus, and human herpesvirus 6. Additionally, AlloVir is advancing several product candidates through preclinical and clinical stages, such as ALVR106 for respiratory syncytial virus and influenza, ALVR109 for SARS-CoV-2, ALVR107 for hepatitis B, and ALVR108 for human herpesvirus-8. The company, originally founded in 2013 as ViraCyte, Inc., rebranded to AlloVir in May 2019 and is committed to addressing the urgent medical needs of patients with limited treatment options for viral diseases.

Gossamer Bio is a clinical-stage biopharmaceutical company based in San Diego that specializes in the discovery, development, and commercialization of innovative therapeutic products targeting significant unmet medical needs. Founded by former executives from Receptos, the company focuses on areas such as immunology, inflammation, fibrosis, and oncology. Its pipeline includes several promising candidates: Seralutinib, aimed at addressing pulmonary arterial hypertension; GB004, for inflammatory bowel disease; GB1275, targeting various oncology indications; and GB001, designed for moderate-to-severe eosinophilic asthma. Gossamer Bio's strategy involves leveraging a rich in-licensing environment to enhance its therapeutic offerings, supported by a team recognized for their successful track record in drug development.

Tempest Therapeutics Inc. is a biotechnology company based in South San Francisco, California, focused on developing small molecule therapeutics for cancer treatment. Established in 2011, the company aims to enhance anti-tumor immunity through its innovative drug pipeline, which includes clinical-stage candidates such as TPST-8844, TPST-1120, and TPST-1495. TPST-8844 targets an enzyme that helps tumor cells evade immune responses, while TPST-1120 blocks the PPAR-alpha pathway to stimulate immune effector cells essential for fighting tumors. Additionally, the company's therapeutics address pathways that directly kill tumor cells and activate tumor-specific immunity, positioning Tempest Therapeutics at the forefront of cancer treatment innovation.

22seventy bio is an immuno-oncology cell therapy company that discovers and develops cancer therapies. The company combines a deep knowledge of cancer cell biology and genetics with an understanding of the immune response to cancer to advance its pipeline. It was established in Cambridge, Massachusetts in 2021.

Dewpoint Therapeutics, Inc. is a Boston-based biotech company focused on the research and development of biomolecular condensates, aiming to create innovative treatments for a range of diseases, including cancer, neurodegenerative disorders, immunological conditions, cardiovascular issues, women's health, and viral infections. Founded in 2018, the company utilizes a proprietary platform that combines experimental techniques with machine-learning-based image analysis to visualize and study biomolecular condensates. This technology includes a collection of genetically engineered cell lines designed to track condensates and mitigate harmful protein sequestration. By leveraging its advanced capabilities, Dewpoint Therapeutics seeks to address unmet medical needs and enhance drug discovery across multiple therapeutic areas, collaborating with leading academic and pharmaceutical partners to further its mission.

Pardes Biosciences is a clinical-stage biopharmaceutical company focused on addressing significant health challenges, particularly those posed by viral infections such as COVID-19. Utilizing modern reversible-covalent chemistry, the company is dedicated to discovering and developing novel oral drug candidates. Its lead product, PBI-0451, is an oral antiviral designed to treat and prevent infections caused by SARS-CoV-2, the virus responsible for COVID-19. Pardes Biosciences aims to enhance patient access to effective treatments, catering to both adult and pediatric populations, while striving to provide convenient and accessible options for those affected by life-threatening viral infections. The company is committed to contributing to global health solutions and improving patient outcomes.

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Cyclerion Therapeutics is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on discovering, developing, and commercializing treatments for serious and orphan diseases. The company leverages soluble guanylate cyclase (sGC) pharmacology to create next-generation sGC stimulators. Its pipeline includes Olinciguat, an orally administered vascular sGC stimulator in Phase II studies for sickle cell disease; Praliciguat, another oral sGC stimulator in Phase II trials for diabetic nephropathy and heart failure with preserved ejection fraction; and IW-6463, a CNS-penetrant sGC stimulator in Phase I trials for neurodegenerative diseases. Additionally, Cyclerion is exploring liver-targeted and lung-targeted sGC stimulators. The company was incorporated in 2018.

Ajax Therapeutics is a biotechnology company based in New York City that focuses on developing innovative small molecule therapies for hematologic malignancies. The company utilizes computational chemistry and structure-based technologies to design drugs that target critical cytokine signaling pathways involved in these cancers. By integrating insights from disease biology, genetics, and structural biology with an advanced computational drug discovery platform, Ajax Therapeutics aims to create precisely designed therapeutics that address significant unmet medical needs for patients suffering from hematologic malignancies.

Corvus Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Burlingame, California, specializing in the development of immuno-oncology therapies. The company focuses on creating first-in-class agents that modulate the immune system to treat cancer and other diseases. Its lead product candidate, Ciforadenant (CPI-444), is an oral small molecule antagonist of the A2A receptor currently undergoing Phase Ib/2 clinical trials. Additionally, Corvus is developing CPI-006, an anti-CD73 monoclonal antibody in Phase I/Ib trials aimed at inhibiting adenosine production and stimulating immune cells, as well as a Phase I trial for COVID-19. Another candidate, CPI-818, is a small molecule inhibitor targeting interleukin-2-inducible T-cell kinase, also in Phase I/Ib trials. The company has established a strategic collaboration with Angel Pharmaceuticals to further advance its pipeline of investigational medicines. Founded in 2014, Corvus Pharmaceuticals aims to contribute significantly to the field of immunotherapy and improve patient outcomes in various diseases.

Janux Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative immunotherapies aimed at treating cancer. Utilizing its proprietary tumor-activated T Cell Engager platform technology, Janux aims to generate targeted immune responses that effectively combat tumors while minimizing effects on healthy tissue. The company's approach seeks to enhance all three stages of the anti-tumor immune response, making it a promising player in the field of cancer treatment.

Tectonic Therapeutic is a biotechnology company dedicated to the discovery and development of innovative drugs that target G protein-coupled receptors (GPCRs). With a focus on therapeutic proteins and antibodies, Tectonic aims to address the complexities of GPCR-targeted drug discovery through its proprietary technology platform, GEODe. The company is committed to unlocking the therapeutic potential of challenging receptors, striving to create biologic medicines that can effectively modify disease progression. Founded by experts in biochemistry and molecular pharmacology, Tectonic combines a strong scientific foundation with a history of entrepreneurial success to advance its mission in the biopharmaceutical landscape.

Aktis Oncology is a biotechnology company focused on the discovery and development of a new class of targeted radiopharmaceuticals aimed at treating various solid tumor cancers. The company has developed proprietary platforms to create tumor-targeting agents optimized for alpha radiotherapy. These agents are engineered for high tumor penetration and prolonged residence time, allowing for effective tumor elimination while reducing the side effects typically associated with cancer treatments. Additionally, Aktis Oncology's innovative approach enables clinicians to visualize and confirm target engagement before administering therapeutic radioisotopes, enhancing treatment precision and efficacy.

Notch Therapeutics Inc. is an immune cell therapy company focused on developing gene-edited allogenic T cell therapies for cancer treatment. Founded in 2018 and headquartered in Toronto, Canada, the company specializes in induced pluripotent stem cell (iPSC) AlloCAR therapy products targeting non-Hodgkin lymphoma, leukemia, and multiple myeloma. Notch Therapeutics has created a proprietary T-cell production platform that allows for precise control of Notch signaling, a critical factor in T-cell development. This innovative approach addresses key limitations in cell therapy development, enabling the design and manufacture of a consistent and limitless supply of therapeutic T cells. The technology empowers healthcare professionals to engineer stem cells tailored to the complex biology of various diseases, thereby enhancing the efficacy of cell therapies.

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.

Gritstone bio is a biotechnology company focused on developing immunotherapies for cancer and infectious diseases. The company initially concentrated on tumor-specific neoantigens but has expanded its programs to include viral antigens found on the surface of infected cells. Gritstone bio's approach leverages the body's natural immune system recognition of targets on abnormal cells, which is applicable to both anti-tumor and anti-viral immunity. Their pipeline includes several product candidates for treating solid tumors, such as GRANITE, SLATE, and CORAL. The company operates in a field that has seen significant advancements with the development and commercialization of immunotherapy drugs like checkpoint inhibitors, transforming cancer treatment.

Neurogene Inc., established in 2018 and headquartered in New York, specializes in developing genetic medicines for treating neurological disorders. The company focuses on utilizing adeno-associated virus (AAV) vectors to deliver therapeutic genes for conditions such as Charcot-Marie Tooth disease type 4J (CMT4J), Aspartylglucosaminuria (AGU), and an undisclosed Lysosomal Storage Disease (LSD). Neurogene aims to provide effective treatment options where none currently exist, aiming to improve the quality of life for patients and families affected by these rare neurological disorders.

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.

Syros Pharmaceuticals, Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, that specializes in developing innovative treatments for cancer and monogenic diseases through gene control medicines. The company is advancing its lead product candidates, including SY-1425, a selective retinoic acid receptor alpha agonist currently in Phase II clinical trials for specific patient populations with acute myeloid leukemia and myelodysplastic syndrome, and SY-5609, a cyclin-dependent kinase 7 inhibitor under investigation in a Phase I clinical trial for advanced solid tumors. Syros is also engaged in collaborative research, including a partnership with Incyte Corporation aimed at identifying therapeutic targets for myeloproliferative neoplasms and a licensing agreement with TMRC Co. Ltd. for the development of tamibarotene. With a focus on mapping gene regulatory circuits, Syros leverages its proprietary platform and expertise to address significant unmet medical needs in oncology. Founded in 2011, the company aims to establish new standards of care for patients with hematologic malignancies.

Cellares Corporation, founded in 2019 and based in South San Francisco, California, specializes in the development of cell therapies for cancer treatment. The company addresses significant manufacturing challenges associated with these therapies, which are often produced on a per-patient basis, leading to high costs and limited availability. Cellares is pioneering the Cell Shuttle, an automated, closed end-to-end manufacturing solution designed to enhance the scalability and efficiency of cell therapy production. This innovative system allows for the simultaneous production of ten times more patient doses compared to traditional manual methods, while also reducing process failure rates by three-fold and lowering manufacturing costs by up to 70 percent. By providing flexible and scalable manufacturing options, Cellares aims to accelerate the delivery of life-saving therapies to patients in need and support the broader biopharmaceutical industry in meeting global demand for these treatments.

Dewpoint Therapeutics, Inc. is a Boston-based biotech company focused on the research and development of biomolecular condensates, aiming to create innovative treatments for a range of diseases, including cancer, neurodegenerative disorders, immunological conditions, cardiovascular issues, women's health, and viral infections. Founded in 2018, the company utilizes a proprietary platform that combines experimental techniques with machine-learning-based image analysis to visualize and study biomolecular condensates. This technology includes a collection of genetically engineered cell lines designed to track condensates and mitigate harmful protein sequestration. By leveraging its advanced capabilities, Dewpoint Therapeutics seeks to address unmet medical needs and enhance drug discovery across multiple therapeutic areas, collaborating with leading academic and pharmaceutical partners to further its mission.

Silverback Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Seattle, Washington, specializing in the development of tissue-targeted therapeutics aimed at treating cancer, chronic viral infections, and other serious diseases. The company’s lead product candidate, SBT6050, is currently in a Phase I/Ib clinical trial and is designed as a TLR8 agonist linker-payload conjugated to a HER2-directed monoclonal antibody, targeting tumors such as breast and non-small cell lung cancers. Silverback is also advancing SBT6290, a preclinical candidate targeting Nectin4 in various cancers, and SBT8230, which aims to treat chronic hepatitis B virus infection. The company employs its proprietary ImmunoTAC technology platform to create therapies that can be systemically administered while acting specifically at disease sites, thereby potentially modulating fundamental disease pathways previously considered inaccessible. Founded in 2016, Silverback Therapeutics is committed to innovative approaches in biopharmaceutical development.

Eledon Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in Irvine, California, specializing in the development of innovative treatments for patients with disorders of the ear, nose, and throat, as well as for those undergoing organ or cellular transplantation and individuals with autoimmune and neurodegenerative diseases. The company’s lead product, OP0201, is a surfactant-based nasal aerosol designed for patients at risk for or suffering from otitis media. Additionally, Eledon has developed a foam-based drug delivery technology, OP0101 and OP0102, aimed at delivering medications to the ear, nose, and sinus cavities. The company also focuses on the CD40L pathway, with its main compound in development, tegoprubart, being an IgG1 anti-CD40L antibody that shows promise for treating autoimmune diseases and supporting organ transplants. Eledon Pharmaceuticals was formerly known as Novus Therapeutics, Inc. before its rebranding in January 2021.

Neogene Therapeutics, Inc., founded in 2018 and based in New York, develops innovative T cell therapies aimed at treating cancer. The company specializes in creating personalized engineered T cells that target mutated proteins, known as neo-antigens, found in cancer cells as a result of DNA mutations. By leveraging advanced technologies, Neogene isolates neo-antigen specific T cell receptor genes from tumor biopsies, which are routinely collected during cancer treatment. These tumor-infiltrating lymphocytes often express T cell receptors that can recognize and attack cancer cells displaying neo-antigens. Utilizing state-of-the-art DNA sequencing, synthesis, and genetic screening, Neogene's proprietary platform enables the identification of these specific T cell receptors with high sensitivity and at scale, ultimately enhancing the efficacy of cancer treatments.

Chinook Therapeutics, Inc. is a clinical-stage biotechnology company based in Seattle, Washington, that specializes in the discovery, development, and commercialization of precision medicines for kidney diseases. The company's lead program is atrasentan, an investigational endothelin receptor antagonist currently prepared for Phase III trials targeting IgA nephropathy and other primary glomerular diseases. In addition to atrasentan, Chinook is developing BION-1301, an investigational anti-APRIL monoclonal antibody evaluated in a Phase Ib trial for IgA nephropathy, and CHK-336, a preclinical candidate aimed at treating an undisclosed ultra-orphan kidney disease. Chinook Therapeutics is also engaged in research programs addressing other rare and severe chronic kidney conditions, including polycystic kidney disease.

Alpine Immune Sciences, Inc. is a clinical-stage biopharmaceutical company based in Seattle, Washington, that specializes in the discovery and development of protein-based immunotherapies aimed at treating cancer, autoimmune, and inflammatory disorders. The company leverages a proprietary scientific platform to transform native immune system proteins into innovative therapeutic candidates. Its leading programs include ALPN-101, a dual antagonist targeting the inducible T cell costimulator and CD28 pathways, designed for autoimmune and inflammatory conditions, and ALPN-303, a dual antagonist of the B cell activating factor and proliferation-inducing ligand, which is vital for B cell activation and survival. Additionally, Alpine Immune Sciences has established a collaboration with Kite Pharma to explore immunotherapies targeting the immune synapse for cancer treatment.

Shattuck Labs, Inc. is a clinical-stage biotechnology company based in Austin, Texas, focused on developing innovative therapeutics for cancer and autoimmune diseases. Founded in 2016, the company is advancing its proprietary Agonist Redirected Checkpoint (ARC) platform, which utilizes dual-function fusion proteins to enhance immune responses against tumors. Its lead product candidate, SL-172154, is currently undergoing Phase 1 clinical trials for ovarian cancer, aiming to inhibit the CD47/SIRPa checkpoint interaction while activating the CD40 receptor to restore and amplify anti-tumor immune responses. Additionally, Shattuck Labs is collaborating with Takeda Pharmaceuticals on SL-279252, which targets the PD-1/PD-L1 interaction and activates the OX40 receptor, and is also in Phase 1 trials for patients with advanced solid tumors and lymphoma. Through its innovative approach to immuno-oncology, Shattuck Labs seeks to address various cancer types and improve treatment options for patients.

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

Surface Oncology, Inc. is a clinical-stage immuno-oncology company based in Cambridge, Massachusetts, focused on developing innovative cancer therapies. The company specializes in creating human immunoglobulin isotype G4 monoclonal antibodies, including SRF231, which inhibits CD47; NZV930, which targets CD73; SRF617, which inhibits CD39; SRF388, aimed at interleukin 27; and SRF813, which targets CD112R. Additionally, Surface Oncology is advancing several preclinical programs that address other critical elements of the tumor microenvironment, such as regulatory T cells and natural killer cells. The company has established strategic collaborations with Novartis Institutes for Biomedical Research and Merck Sharp & Dohme Corp. to advance the development of its therapies, including evaluating the combination of SRF617 with Merck’s KEYTRUDA. Founded in 2014, Surface Oncology aims to leverage novel immunotherapy targets and insights into cancer immuno-biology to enhance anti-tumor immune responses.

Accent Therapeutics, Inc. is a biopharmaceutical company focused on developing small molecule therapies for oncology, utilizing the emerging field of epitranscriptomics. Founded in 2017 and headquartered in Lexington, Massachusetts, the company explores the post-transcriptional chemical modifications of RNA, which play a crucial role in regulating proteins essential for cellular growth and differentiation. By precisely targeting cancer-associated RNA-modifying proteins, Accent Therapeutics aims to innovate and deliver transformative therapies that can significantly improve patient outcomes in cancer treatment.

ElevateBio, LLC is a holding company based in Cambridge, Massachusetts, founded in 2017. It focuses on developing cell and gene therapies for the treatment of cancer and viral infections. The company operates a portfolio of subsidiaries that aim to manufacture and commercialize innovative medicines. ElevateBio collaborates with leading scientists and inventors to create a robust portfolio of therapeutic solutions. Additionally, it has established a centralized facility designed to streamline the translation of cell and gene therapy research into viable medical products, ensuring that patients have access to treatments for severe diseases in a timely manner.

Nurix Therapeutics is a biopharmaceutical company dedicated to the discovery, development, and commercialization of small molecule therapies aimed at modulating cellular protein levels for the treatment of cancer and immune disorders. The company has developed an integrated discovery platform known as DELigase, which leverages its expertise in E3 ligases—enzymes capable of modulating proteins within cells. Nurix's pipeline includes promising candidates such as NX-2127, an orally available Bruton’s tyrosine kinase degrader targeting relapsed or refractory B-cell malignancies, and NX-1607, an orally available inhibitor of Casitas B-lineage lymphoma proto-oncogene B, intended for immuno-oncology applications. Founded in 2009 and headquartered in San Francisco, California, Nurix Therapeutics was formerly known as Nurix Inc. and rebranded in October 2018. The company has established strategic collaborations with major industry players, enhancing its research and development capabilities.

Pieris Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on the development of anticalin-based therapeutics for treating various medical conditions, including cancer and severe asthma. Anticalins are engineered proteins derived from lipocalins, naturally occurring human proteins that can bind and transport a variety of molecules. The company's lead programs include PRS-060, a drug candidate in Phase I clinical trials targeting IL-4Ra for asthma and inflammatory diseases, and PRS-343, a bispecific protein in Phase I trials for oncology. Pieris is also advancing PRS-344, a bispecific anticalin-antibody fusion protein in preclinical development, and PRS-080, currently in Phase IIa trials for targeting hepcidin in patients with functional iron deficiency. Pieris collaborates with strategic partners and research institutions to enhance its development efforts, showcasing a robust pipeline aimed at addressing significant unmet medical needs. The company was founded in 2000 and is recognized for its innovative approach in biotherapeutics.

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.

AlloVir, Inc. is a clinical-stage cell therapy company based in Cambridge, Massachusetts, specializing in the research and development of allogeneic, off-the-shelf multi-virus specific T cell (VST) therapies aimed at preventing and treating serious viral-associated diseases. The company's lead product, Viralym-M, targets multiple viruses including BK virus, cytomegalovirus, adenovirus, Epstein-Barr virus, and human herpesvirus 6. Additionally, AlloVir is advancing several product candidates through preclinical and clinical stages, such as ALVR106 for respiratory syncytial virus and influenza, ALVR109 for SARS-CoV-2, ALVR107 for hepatitis B, and ALVR108 for human herpesvirus-8. The company, originally founded in 2013 as ViraCyte, Inc., rebranded to AlloVir in May 2019 and is committed to addressing the urgent medical needs of patients with limited treatment options for viral diseases.

ElevateBio, LLC is a holding company based in Cambridge, Massachusetts, founded in 2017. It focuses on developing cell and gene therapies for the treatment of cancer and viral infections. The company operates a portfolio of subsidiaries that aim to manufacture and commercialize innovative medicines. ElevateBio collaborates with leading scientists and inventors to create a robust portfolio of therapeutic solutions. Additionally, it has established a centralized facility designed to streamline the translation of cell and gene therapy research into viable medical products, ensuring that patients have access to treatments for severe diseases in a timely manner.

Prevail Therapeutics Inc. is a biotechnology company headquartered in New York, specializing in the development and commercialization of gene therapies targeting neurodegenerative diseases. Founded in 2017, the company focuses on adeno-associated virus (AAV)-based therapies, with its lead candidate, PR001, currently undergoing Phase I/II clinical trials for Parkinson's disease associated with GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is advancing PR006 for frontotemporal dementia linked to GRN mutations and PR004 for various synucleinopathies. The company's mission is to provide disease-modifying treatments for patients suffering from these challenging conditions.

Ashvattha Therapeutics, Inc. is a pharmaceutical company focused on developing innovative therapeutics that address unmet medical needs in neurology, oncology, ophthalmology, and inflammation. Founded in 2015 and headquartered in Redwood City, California, with research facilities in Baltimore, Maryland, the company specializes in hydroxyl dendrimer therapeutics. These products are designed to selectively target and treat diseases by engaging actively endocytosing cells such as reactive microglia and macrophages, which play a critical role in chronic inflammation and disease progression. By utilizing this precision targeting approach, Ashvattha Therapeutics aims to minimize off-target toxicity, systemic side effects, and immunogenicity often associated with conventional treatments, thereby enhancing treatment efficacy and patient outcomes. The company has a robust pipeline containing nine therapeutics across its areas of focus.

Peloton Therapeutics, Inc. is a clinical stage biopharmaceutical company based in Dallas, Texas, dedicated to developing innovative medicines for patients with cancer and other serious conditions. The company focuses on targeting HIF-2a, a transcription factor previously considered difficult to address with small molecules. Its lead drug candidate, PT2977, is an oral HIF-2a inhibitor currently undergoing Phase 2 clinical trials for advanced or metastatic clear cell renal cell carcinoma, and it is being evaluated in various settings, including in patients with von Hippel-Lindau disease-associated renal cell carcinoma and in combination with cabozantinib. Additionally, Peloton is advancing PT2567, another oral HIF-2a inhibitor in preclinical development for non-oncology indications such as pulmonary arterial hypertension. Founded in 2010 and previously known as Damascus Pharmaceutics, Peloton Therapeutics aims to leverage its scientific expertise and collaborations to become a leading biotech company in the region.

Neurogene Inc., established in 2018 and headquartered in New York, specializes in developing genetic medicines for treating neurological disorders. The company focuses on utilizing adeno-associated virus (AAV) vectors to deliver therapeutic genes for conditions such as Charcot-Marie Tooth disease type 4J (CMT4J), Aspartylglucosaminuria (AGU), and an undisclosed Lysosomal Storage Disease (LSD). Neurogene aims to provide effective treatment options where none currently exist, aiming to improve the quality of life for patients and families affected by these rare neurological disorders.

Dewpoint Therapeutics, Inc. is a Boston-based biotech company focused on the research and development of biomolecular condensates, aiming to create innovative treatments for a range of diseases, including cancer, neurodegenerative disorders, immunological conditions, cardiovascular issues, women's health, and viral infections. Founded in 2018, the company utilizes a proprietary platform that combines experimental techniques with machine-learning-based image analysis to visualize and study biomolecular condensates. This technology includes a collection of genetically engineered cell lines designed to track condensates and mitigate harmful protein sequestration. By leveraging its advanced capabilities, Dewpoint Therapeutics seeks to address unmet medical needs and enhance drug discovery across multiple therapeutic areas, collaborating with leading academic and pharmaceutical partners to further its mission.

Relay Therapeutics, Inc. is a clinical-stage precision medicine company based in Cambridge, Massachusetts, focused on transforming the drug discovery process. The company employs a novel allosteric drug-discovery platform, known as Dynamo, which integrates advanced computational techniques with experimental methods to study protein motion and interactions. This innovative approach enables the identification and development of therapies targeting complex diseases, particularly in oncology. Relay Therapeutics is advancing a pipeline of product candidates, including RLY-1971, a small molecule inhibitor targeting SHP2, RLY-4008, a selective inhibitor of FGFR2 for advanced solid tumors, and RLY-PI3K1047, aimed at mutant variants of phosphoinositide 3-kinase alpha. Founded in 2015, the company aims to deliver life-changing therapies to patients by addressing previously intractable targets in precision oncology and genetic diseases.

Avidity Biosciences, Inc. is a biopharmaceutical company based in La Jolla, California, specializing in the development of oligonucleotide-based therapies, particularly through its innovative Antibody Oligonucleotide Conjugates (AOC) platform. This technology aims to address the limitations of traditional oligonucleotide therapies by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide treatments. Avidity's lead product candidate, AOC 1001, targets myotonic dystrophy type 1, a rare genetic muscle disease. In addition to this, the company is advancing multiple programs focused on muscle-related conditions, including muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Furthermore, Avidity is expanding its research to include immune and other cell types, positioning itself at the forefront of biopharmaceutical innovation for serious diseases.

Morphic Therapeutic is a biotechnology company focused on developing oral integrin therapies to address serious chronic diseases such as autoimmune disorders, cardiovascular issues, metabolic diseases, fibrosis, and cancer. The company leverages its expertise in integrins to create a pipeline of small-molecule inhibitors targeting various integrin pathways. This innovative approach aims to provide effective treatments for patients suffering from immunological, fibrotic, neoplastic, and vascular conditions, contributing to advancements in biopharmaceuticals.

Atreca, Inc. is a biopharmaceutical company focused on the discovery and development of antibody-based immunotherapeutics for various solid tumor types. Founded in 2010 and headquartered in South San Francisco, California, Atreca utilizes a unique platform to identify antibodies produced during immune responses, facilitating the development of novel therapies without prior knowledge of antigens. Its lead product candidate, ATRC-101, is a monoclonal antibody currently in clinical development, targeting a range of cancers including ovarian, non-small cell lung, colorectal, and breast cancer. The company has established a strategic research collaboration with Merck Sharp & Dohme Corp. to explore antigenic targets for select antibodies with potential applications in oncology.

Accent Therapeutics, Inc. is a biopharmaceutical company focused on developing small molecule therapies for oncology, utilizing the emerging field of epitranscriptomics. Founded in 2017 and headquartered in Lexington, Massachusetts, the company explores the post-transcriptional chemical modifications of RNA, which play a crucial role in regulating proteins essential for cellular growth and differentiation. By precisely targeting cancer-associated RNA-modifying proteins, Accent Therapeutics aims to innovate and deliver transformative therapies that can significantly improve patient outcomes in cancer treatment.

Magenta Therapeutics, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on advancing stem cell transplantation and related therapies. Founded in 2015, the company aims to enhance the patient experience in transplant medicine by developing innovative treatments that make stem cell transplants safer and more accessible. Its pipeline includes several key candidates, such as C100, C200, and C300 targeted antibody-drug conjugates designed for transplant conditioning; MGTA-145, a stem cell mobilization product; and MGTA-456, an allogeneic stem cell therapy. Additionally, Magenta is exploring E478, a small molecule aimed at expanding gene-modified stem cells, and G100, an antibody-drug conjugate to mitigate acute graft-versus-host disease. The company has established research collaborations with other biotechnology firms to further investigate the potential of its therapies in treating blood cancers, genetic disorders, and autoimmune diseases, ultimately striving to transform stem cell transplantation from a last-resort option into a more viable and effective treatment for patients.

Omniox is a biopharmaceutical company focused on developing innovative treatments for diseases associated with hypoxia, a condition characterized by inadequate oxygen levels in tissues. This lack of oxygen is linked to various serious health issues, including certain cancers and cardiovascular diseases. The company utilizes its proprietary H-NOX platform technology, which is designed to deliver oxygen and nitric oxide specifically to hypoxic tissues. This targeted approach aims to restore normal tissue function and improve patient outcomes by addressing the underlying causes of hypoxia-related diseases. Through its advancements in biologics, Omniox seeks to provide meaningful therapeutic benefits and enable healthcare professionals to develop effective oxygen-based therapies that enhance patient health and recovery.

Unity Biotechnology, Inc. is a biotechnology company dedicated to the research and development of therapeutics aimed at extending human health span by addressing age-related diseases. The company specializes in clearing senescent cells through the creation of senolytic medicines, which specifically target vulnerabilities unique to these cells while sparing healthy ones. Its lead drug candidates include UBX0101, currently in Phase II clinical trials for musculoskeletal conditions like osteoarthritis, and UBX1325, which is in Phase I trials for diabetic macular edema. Additionally, Unity is developing treatments for various conditions related to aging, including pulmonary and neurological disorders, as well as kidney and liver diseases. Founded in 2009 and headquartered in South San Francisco, California, Unity Biotechnology was previously known as Forge, Inc. before changing its name in 2015.

Prevail Therapeutics Inc. is a biotechnology company headquartered in New York, specializing in the development and commercialization of gene therapies targeting neurodegenerative diseases. Founded in 2017, the company focuses on adeno-associated virus (AAV)-based therapies, with its lead candidate, PR001, currently undergoing Phase I/II clinical trials for Parkinson's disease associated with GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is advancing PR006 for frontotemporal dementia linked to GRN mutations and PR004 for various synucleinopathies. The company's mission is to provide disease-modifying treatments for patients suffering from these challenging conditions.

Scholar Rock, Inc. is a biotechnology company based in Cambridge, Massachusetts, specializing in the discovery and development of biologic therapies that selectively target dysregulated growth factors within disease microenvironments. The company's innovative approach focuses on modulating supracellular activation to address serious diseases, including fibrosis, musculoskeletal disorders, immuno-oncology, and autoimmune diseases. Scholar Rock's lead product candidate, SRK-015, is a first-in-class inhibitor designed to target the activation of the growth factor myostatin in skeletal muscle, specifically for the treatment of spinal muscular atrophy. The company is advancing this candidate through clinical development while also leveraging its proprietary platform to create a pipeline of additional product candidates aimed at transforming treatment for various serious conditions, including other neuromuscular disorders, cancer, and anemia.

Relay Therapeutics, Inc. is a clinical-stage precision medicine company based in Cambridge, Massachusetts, focused on transforming the drug discovery process. The company employs a novel allosteric drug-discovery platform, known as Dynamo, which integrates advanced computational techniques with experimental methods to study protein motion and interactions. This innovative approach enables the identification and development of therapies targeting complex diseases, particularly in oncology. Relay Therapeutics is advancing a pipeline of product candidates, including RLY-1971, a small molecule inhibitor targeting SHP2, RLY-4008, a selective inhibitor of FGFR2 for advanced solid tumors, and RLY-PI3K1047, aimed at mutant variants of phosphoinositide 3-kinase alpha. Founded in 2015, the company aims to deliver life-changing therapies to patients by addressing previously intractable targets in precision oncology and genetic diseases.

Codiak BioSciences is a clinical-stage biopharmaceutical company dedicated to developing exosome-based therapeutics, a novel class of medicines with the potential to address various diseases with significant unmet medical needs. The company employs its proprietary engEx Platform, which allows for the engineering and manufacturing of exosomes, leveraging their natural role as intercellular messengers. Codiak's pipeline includes product candidates such as exoSTING and exoIL-12, targeting diverse therapeutic areas including oncology, neuro-oncology, neurology, neuromuscular diseases, and infectious diseases. Additionally, the company collaborates with the Ragon Institute to explore the potential of its exoVACC platform for vaccines against SARS-CoV-2 and HIV. Founded in 2015 and headquartered in Cambridge, Massachusetts, Codiak aims to transform therapeutic approaches by harnessing the unique properties of exosomes.

Arcus Biosciences, Inc. is a clinical-stage biopharmaceutical company based in Hayward, California, focused on developing innovative immunotherapies for cancer treatment. Founded in 2015, the company leverages insights from immunology to create novel therapies targeting the ATP-adenosine pathway, a critical driver of immunosuppression in the tumor microenvironment. Arcus has a diverse product pipeline featuring several candidates, including Etrumadenant (AB928), a dual A2a/A2b adenosine receptor antagonist in Phase 1b/2 trials; Zimberelimab, an anti-PD-1 monoclonal antibody in Phase Ib trials; and AB154, an anti-TIGIT monoclonal antibody in Phase 2 development for first-line metastatic non-small cell lung cancer. Additionally, AB680, a small-molecule CD73 inhibitor, is undergoing Phase 1/1b studies for the treatment of first-line metastatic pancreatic cancer. The company collaborates with other organizations, including AstraZeneca and Strata Oncology, to advance its clinical programs.

aTyr Pharma, Inc., established in 2005 and headquartered in San Diego, California, is a biotherapeutics company dedicated to discovering and developing innovative medicines based on novel immunological pathways. The company's primary focus is efzofitimod (formerly ATYR1923), a fusion protein currently in clinical trials for treating interstitial lung diseases by modulating immune engagement through the neuropilin-2 receptor. aTyr Pharma has collaborations with academic institutions and industry partners, including the University of Nebraska Medical Center, CSL Behring, Boston Children's Hospital, Kyorin Pharmaceutical Co., Ltd., and the Medical University of South Carolina, to advance its pipeline of immunomodulatory therapeutics derived from tRNA synthetases. The company is backed by prominent life sciences investors such as Alta Partners, Cardinal Partners, Domain Associates, and Polaris Ventures.

Kezar Life Sciences, Inc. is a clinical-stage biotechnology company based in South San Francisco, California, focused on the discovery and development of small molecule therapeutics aimed at addressing unmet medical needs in autoimmunity and cancer. The company's lead product candidate, KZR-616, is a selective immunoproteasome inhibitor currently undergoing Phase 2 clinical trials for five autoimmune conditions, including lupus nephritis and autoimmune hemolytic anemia, as well as Phase 1b/2 trials in systemic lupus erythematosus. In addition to KZR-616, Kezar is developing KZR-261, which targets oncology and autoimmune indications. Founded in 2015, the company leverages innovative research in protein homeostasis and has established a pipeline of potential therapies to advance treatment options for patients.

Dicerna Pharmaceuticals is a biopharmaceutical company that specializes in the discovery and development of RNA interference (RNAi)-based therapeutics. The company focuses on innovative treatments for rare inherited liver diseases, viral infections, chronic liver diseases, and cardiometabolic conditions. Utilizing its proprietary GalXC RNAi technology platform, Dicerna is advancing several key product candidates, including nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B, and programs targeting genetic liver diseases and cardiovascular conditions. Founded in 2007 and headquartered in Lexington, Massachusetts, Dicerna has established strategic collaborations with major pharmaceutical firms to enhance its research and development efforts.

Ardelyx, Inc. is a biopharmaceutical company based in Fremont, California, specializing in the development and commercialization of innovative oral therapeutics for cardiorenal diseases. Founded in 2007, Ardelyx focuses on addressing significant unmet medical needs with its proprietary drug discovery platform. The company's lead product candidate, tenapanor, has completed Phase 3 clinical trials for the treatment of irritable bowel syndrome with constipation and is also being evaluated for hyperphosphatemia in end-stage renal disease patients on dialysis. In addition, Ardelyx is advancing RDX013, a potassium secretagogue aimed at treating hyperkalemia, and other candidates including RDX002, a phosphate transport inhibitor for chronic kidney disease, and RDX009, an agonist for type 2 diabetes, which are currently in preclinical development. Ardelyx's approach emphasizes the creation of minimally absorbed medications that target specific receptors and transporters, thereby reducing the risk of systemic side effects.

ORIC Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing innovative therapies to combat cancer by overcoming resistance mechanisms. The company's lead product candidate, ORIC-101, is a small molecule antagonist of the glucocorticoid receptor, which plays a significant role in resistance to various cancer treatments across multiple solid tumors. Another key candidate, ORIC-533, is an orally bioavailable inhibitor of CD73, targeting a critical pathway in the adenosine metabolism that contributes to resistance against chemotherapy and immunotherapy. Additionally, ORIC is working on a pipeline of precision medicines aimed at addressing other cancer resistance mechanisms. Founded in 2014 and headquartered in South San Francisco, California, ORIC Pharmaceuticals leverages the expertise of its scientific founders, who have extensive backgrounds in cancer research and target discovery.

Intellia Therapeutics, Inc. is a genome editing company headquartered in Cambridge, Massachusetts, specializing in the development of therapeutics using the CRISPR/Cas9 technology. This innovative platform allows for precise alterations of genomic DNA, enabling the company to pursue treatments for a range of severe and life-threatening diseases. Intellia's pipeline includes both in vivo programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as ex vivo programs focused on engineered cell therapies for oncological and autoimmune disorders. The company is also exploring treatments for conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Intellia has established collaborations with several organizations, enhancing its research and development capabilities. Founded in 2014, Intellia holds a significant intellectual property portfolio related to the therapeutic applications of CRISPR/Cas9, aiming to address high unmet medical needs through its advanced gene-editing approaches.

Syndax Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Waltham, Massachusetts, specializing in the development of therapies for cancer treatment. The company's lead candidate, entinostat, is a class I HDAC inhibitor currently in Phase III trials for advanced hormone receptor-positive, HER2-negative breast cancer. Additionally, Syndax is advancing SNDX-5613, a selective inhibitor targeting the Menin-MLL binding interaction, in Phase I/II trials for acute myeloid leukemia with specific mutations. The company also explores entinostat in combination with immunotherapies such as Keytruda and Tecentriq for various cancers, including non-small cell lung cancer and metastatic breast cancer. Furthermore, Syndax is developing axatilimab, a monoclonal antibody targeting the CSF-1 receptor, currently in trials for chronic graft-versus-host disease. The company maintains collaborative relationships with several major pharmaceutical entities and research organizations to enhance its drug development efforts. Founded in 2005, Syndax Pharmaceuticals is committed to advancing innovative treatments for patients with challenging malignancies.

Metacrine, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, established in 2014. It specializes in the discovery and development of innovative therapies aimed at treating liver and gastrointestinal diseases. The company is advancing a pipeline of drug candidates, with a primary focus on the farnesoid X receptor (FXR), a key target in managing various liver and GI disorders. Its lead candidates, MET409 and MET642, are currently in clinical trials to evaluate their efficacy in treating non-alcoholic steatohepatitis. Additionally, Metacrine collaborates with Novo Nordisk A/S to explore further research opportunities related to fibroblast growth factor 1. The company is committed to developing best-in-class therapies to improve patient outcomes in these challenging medical areas.

Clementia Pharmaceuticals Inc. is a clinical-stage biopharmaceutical company based in Montreal, Canada, focused on developing innovative treatments for patients with ultra-rare bone disorders and other diseases. Founded in 2010, the company specializes in creating disease-modifying therapies to address high unmet medical needs. Its lead product candidate, palovarotene, is an oral small molecule that has demonstrated significant efficacy in preventing abnormal bone formation and fibrosis in various tissues. Currently, palovarotene is undergoing evaluation in the Phase III MOVE trial for conditions such as fibrodysplasia ossificans progressive and multiple osteochondromas, as well as dry eye disease. Clementia Pharmaceuticals aims to improve the lives of patients suffering from debilitating conditions through its advanced therapeutic solutions.

Kezar Life Sciences, Inc. is a clinical-stage biotechnology company based in South San Francisco, California, focused on the discovery and development of small molecule therapeutics aimed at addressing unmet medical needs in autoimmunity and cancer. The company's lead product candidate, KZR-616, is a selective immunoproteasome inhibitor currently undergoing Phase 2 clinical trials for five autoimmune conditions, including lupus nephritis and autoimmune hemolytic anemia, as well as Phase 1b/2 trials in systemic lupus erythematosus. In addition to KZR-616, Kezar is developing KZR-261, which targets oncology and autoimmune indications. Founded in 2015, the company leverages innovative research in protein homeostasis and has established a pipeline of potential therapies to advance treatment options for patients.

Nabriva Therapeutics plc is a biopharmaceutical company focused on developing and commercializing innovative anti-infective agents to address serious infections. The company's lead product candidate, XENLETA, is the first systemic pleuromutilin antibiotic approved by the U.S. Food and Drug Administration for the treatment of community-acquired bacterial pneumonia. In addition to XENLETA, Nabriva is developing CONTEPO, a potential first-in-class epoxide antibiotic aimed at treating complicated urinary tract infections, including acute pyelonephritis. The company is also involved in clinical trials for other indications, including pediatric infections and sexually transmitted infections. Founded in 2005 and headquartered in Dublin, Ireland, Nabriva was previously known as Nabriva Therapeutics Forschungs GmbH before rebranding in 2007.

aTyr Pharma, Inc., established in 2005 and headquartered in San Diego, California, is a biotherapeutics company dedicated to discovering and developing innovative medicines based on novel immunological pathways. The company's primary focus is efzofitimod (formerly ATYR1923), a fusion protein currently in clinical trials for treating interstitial lung diseases by modulating immune engagement through the neuropilin-2 receptor. aTyr Pharma has collaborations with academic institutions and industry partners, including the University of Nebraska Medical Center, CSL Behring, Boston Children's Hospital, Kyorin Pharmaceutical Co., Ltd., and the Medical University of South Carolina, to advance its pipeline of immunomodulatory therapeutics derived from tRNA synthetases. The company is backed by prominent life sciences investors such as Alta Partners, Cardinal Partners, Domain Associates, and Polaris Ventures.

Kura Oncology is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing targeted therapies for cancer treatment. The company’s primary product candidate, Tipifarnib, is an orally available farnesyl transferase inhibitor currently undergoing Phase II clinical trials for solid tumors, peripheral T-cell lymphomas, and various hematologic malignancies. In addition to Tipifarnib, Kura is advancing other small molecule candidates, including KO-947, which targets dysregulated extracellular signal-related kinases, and KO-539, aimed at inhibiting the menin-mixed lineage leukemia protein interaction. Kura Oncology emphasizes precision medicine, leveraging insights from cancer genomics to create targeted treatments for patients with specific genetic profiles, addressing significant unmet medical needs across various cancer types, including lung, colorectal, thyroid, pancreatic cancers, and blood cancers such as lymphoma and leukemia. The company was founded in 2014 and operates primarily within the United States.

Collegium Pharmaceutical, Inc. is a specialty pharmaceutical company dedicated to developing and commercializing products aimed at addressing the challenges associated with pain management and the abuse of prescription drugs. The company utilizes its proprietary DETERx platform technology to create abuse-deterrent formulations that maintain the extended-release and safety profiles of medications, making them less susceptible to misuse through methods such as chewing or injecting. Collegium's primary products include Xtampza ER, an abuse-deterrent extended-release formulation of oxycodone, and Nucynta, which encompasses both extended-release and immediate-release formulations of tapentadol for managing severe pain. Founded in 2002 and headquartered in Stoughton, Massachusetts, Collegium focuses on delivering innovative solutions that enhance patient safety while effectively treating chronic pain conditions.

Naurex Inc. is a clinical-stage company based in Evanston, Illinois, focused on developing innovative therapies for depression and other central nervous system disorders. The company is pioneering a new mechanism of action for modulating the N-methyl-D-aspartic acid receptor (NMDAR), resulting in the creation of a novel class of compounds known as glycine-site functional partial agonists (GFPAs). These compounds offer a different approach to receptor modulation compared to existing NMDAR agents. Naurex's drug development pipeline includes the first-generation molecule GLYX-13, as well as the second-generation NRX-1050 small molecule series and additional compounds derived from its platform of novel NMDAR modulators. The company's mission is to address unmet needs in psychiatry and neurology by providing safe and effective treatment options.