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Maze Therapeutics is a biotechnology company focused on developing medicines that mimic the effects of rare, protective genetic variants to address unmet medical needs. Utilizing its Compass platform, the company identifies genetic variants associated with diseases and maps them to the biological pathways driving these conditions in specific patient populations.

35Pharma is a biopharmaceutical company that designs and develops innovative biologics to address diseases with high unmet medical need. The company applies protein engineering and a deep understanding of transforming growth factor-beta structure-function relationships to create potent ligand traps for cardio-pulmonary and metabolic diseases, including pulmonary hypertension and musculoskeletal disorders.

Founded in 2020 and headquartered in Boston, Massachusetts, Scorpion Therapeutics is a clinical-stage biotechnology company specializing in precision oncology. It develops targeted small-molecule drugs to treat cancer by focusing on three areas: therapies against known oncogenes, agents for currently undruggable targets, and drugs for new targets.

Founded in 2019, Enliven Therapeutics is a precision oncology company based in Boulder, Colorado. It focuses on developing small molecule therapies to extend and improve patient lives by targeting clinically validated biological targets with industry-leading chemistry.

Unicycive Therapeutics provides development programs that focus on treating kidney diseases with significant unmet medical needs. It aims to open the way for a broad range of disease-modifying therapies by modifying the intracellular and extracellular concentrations of these various electrolytes and ions in Chronic Kidney Disease and Gitelman’s Syndrome and by addressing the underlying mitochondrial pathology and inflammation associated with Acute Kidney Disease. Unicycive Therapeutics was founded in 2016 and is headquartered in Los Altos, California.

Cybin is a biopharmaceutical company dedicated to advancing research and development in psychedelic and medicinal mushrooms. It focuses on creating safe and effective therapeutics for mental health issues, with an emphasis on psilocybin-based products. The company is actively launching these products in jurisdictions where they are permitted and is engaged in clinical studies across North America and other regions. Through strategic partnerships with academic and institutional entities, Cybin seeks to develop novel compounds and delivery mechanisms, aiming to provide innovative treatments for various psychiatric and neurological conditions.

Silence Therapeutics is a London-based biotechnology company that develops RNA therapeutics using short interfering RNA to trigger RNA interference and degrade disease-causing messenger RNA. It focuses on discovery, development, and delivery of siRNA medicines for hematology, cardiovascular disease, and other rare and metabolic conditions. The company has a proprietary mRNAi GOLD platform to create internal candidates and collaborates on out-licensed programs, including a strategic partnership with AstraZeneca to discover, develop, and commercialize siRNA therapies across cardiovascular, renal, metabolic, and respiratory indications, as well as a collaboration with Genomics England. Its product candidates include SLN124 for iron overload, SLN360 for cardiovascular disease associated with high lipoprotein, and SLN500 for complement-mediated diseases. Silence Therapeutics also pursues delivery systems for targeting multiple organs and cell types, supporting a pipeline across its three focus areas.

Olema Oncology is a preclinical biotechnology company dedicated to developing innovative therapies for the treatment and prevention of estrogen receptor-positive breast cancer. The company specializes in discovering and commercializing targeted treatments specifically designed for women's cancers. By leveraging its extensive understanding of endocrine-driven cancers, nuclear receptors, and mechanisms of acquired resistance, Olema aims to create compounds that surpass existing therapies. The company's focus is on transforming the standard of care for both pre- and post-menopausal women with cancer, with an emphasis on developing more effective and convenient treatment options. Olema's pipeline includes drug candidates such as OP-1250 and OP-3136, which have progressed through discovery and preclinical studies.

Gracell Biotechnologies Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative cell therapies for cancer treatment. Based in Shanghai, China, Gracell's lead product candidates include GC012F, a dual-targeting autologous CAR-T therapy for multiple myeloma, and GC019F, which targets adult B cell acute lymphoblastic leukemia. Both candidates are currently undergoing Phase I clinical trials. Additionally, the company is advancing GC007F for B cell non-Hodgkin's lymphoma and GC027 for adult T cell acute lymphoblastic leukemia, among others. Gracell also has a range of earlier-stage product candidates addressing various cancer types, such as ovarian cancer and breast cancer. Founded in 2017, the company aims to provide effective cellular therapeutics to improve outcomes for patients with hematological malignancies and solid tumors.

Septerna is a biotechnology company focused on discovering and developing oral small-molecule medicines that target G protein-coupled receptors (GPCRs). It operates a proprietary Native Complex Platform to accelerate GPCR drug discovery and aims to deliver therapies across endocrinology, immunology and inflammation, and metabolic diseases. The company is advancing a GPCR-focused pipeline with candidates such as SEP-786 (PTH1R) and SEP-631 (MRGPRX2), along with programs targeting TSHR, GLP-1R, GIPR, and GCGR, with the goal of translating GPCR biology into orally available medicines that address unmet patient needs.

Icosavax develops virus-like particle (VLP) vaccines using its proprietary computational design platform. Its primary focus is creating vaccines for life-threatening respiratory diseases, with current candidates targeting respiratory syncytial virus (RSV), human metapneumovirus (hMPV), and severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). Founded in 2017, the company aims to discover, develop, and commercialize vaccines against infectious diseases.

Boundless Bio develops innovative cancer therapeutics focused on understanding and treating previously untreatable cancers. It specializes in targeting extrachromosomal DNA (ecDNA) biology, a key driver of aggressive cancers.

Unicycive Therapeutics provides development programs that focus on treating kidney diseases with significant unmet medical needs. It aims to open the way for a broad range of disease-modifying therapies by modifying the intracellular and extracellular concentrations of these various electrolytes and ions in Chronic Kidney Disease and Gitelman’s Syndrome and by addressing the underlying mitochondrial pathology and inflammation associated with Acute Kidney Disease. Unicycive Therapeutics was founded in 2016 and is headquartered in Los Altos, California.

Entact Bio is a biotechnology company that is in the preclinical stages of developing medications to improve protein function. It was founded by a team with deep roots in deubiquitinase (DUB) biochemistry, chemical biology, disease biology, and small-molecule drug development. The company's proprietary EncompassTM platform was designed to create enhancement-targeting chimeric (ENTACTM) medicines. ENTACs take advantage of DUBs' ability to regulate proteins.

Triana Biomedicines develops a molecular glue discovery platform targeting challenging disease targets. Its drug discovery engine combines high-resolution structural insights, advanced computational tools, and proprietary chemical libraries.

Synthego is a biotechnology company specializing in genome engineering. It offers integrated hardware, software, bioinformatics, chemistries, and molecular biology platforms to advance basic research and therapeutic development. Its products include engineered cells, CRISPR kits, and bioinformatics tools for gene knockout, target validation, and disease modeling. Synthego serves scientists globally, with sales through distributors in multiple countries and online.

Parse Biosciences is a biotechnology company specializing in single-cell analysis and sequencing solutions. Founded in 2018 and based in Seattle, Washington, the company focuses on developing innovative technologies that simplify the sequencing process for researchers. By employing combinatorial cDNA barcoding directly within cells, Parse Biosciences eliminates the necessity for complex and costly microfluidic instruments. This technology allows for the fixing, freezing, and storage of samples, enabling researchers to conduct experiments with samples collected on different dates in a single run. This capability enhances sample handling and minimizes errors, ultimately improving the accuracy of results. The company aims to increase gene detection in individual cells while addressing common challenges associated with single-cell sequencing, thereby advancing research in various scientific fields.

Septerna is a biotechnology company focused on discovering and developing oral small-molecule medicines that target G protein-coupled receptors (GPCRs). It operates a proprietary Native Complex Platform to accelerate GPCR drug discovery and aims to deliver therapies across endocrinology, immunology and inflammation, and metabolic diseases. The company is advancing a GPCR-focused pipeline with candidates such as SEP-786 (PTH1R) and SEP-631 (MRGPRX2), along with programs targeting TSHR, GLP-1R, GIPR, and GCGR, with the goal of translating GPCR biology into orally available medicines that address unmet patient needs.

Simcha Therapeutics is a biopharmaceutical company focused on developing engineered cytokine immunotherapy for cancer treatment. Founded in 2018 and headquartered in New Haven, Connecticut, the company utilizes directed evolution to create novel cytokines that enhance the immune system's ability to combat diseases. Its lead program, ST-067, is a designer IL-18 cytokine that has demonstrated significant antitumor effects in animal models, both as a standalone treatment and in combination with anti-PD-1 checkpoint inhibitors. This innovative approach aims to provide patients with more effective immunotherapies, thereby improving their ability to fight cancer.

Founded in 2019, Enliven Therapeutics is a precision oncology company based in Boulder, Colorado. It focuses on developing small molecule therapies to extend and improve patient lives by targeting clinically validated biological targets with industry-leading chemistry.

Quanta Therapeutics is a biotechnology company focused on developing novel cancer therapeutic candidates. It specializes in allosteric modulation to directly inhibit RAS-driven cancer types by restoring conformational control of active RAS signaling at the cell membrane.

Cedilla Therapeutics, Inc. is a biotechnology company focused on developing targeted small-molecule therapeutics for cancer and diseases linked to protein dysregulation. The company employs innovative strategies to destabilize oncoprotein targets, utilizing methods such as direct ligand-induced degradation, disruption of stabilizing protein-protein interactions, and the identification of upstream regulators that affect protein stability. Cedilla's comprehensive approach also includes large-scale proteomic analyses to map protein susceptibility, enhancing the potential for effective therapies. By concentrating on the transitions between stable and susceptible protein states, Cedilla aims to create novel treatments that broaden the applicability of small molecule therapeutics beyond oncology. Founded in 2017 and headquartered in Cambridge, Massachusetts, the company was formerly known as Protein Stability Newco, Inc. before rebranding in 2018.

RareCyte, Inc. is a life sciences company based in Seattle, Washington, founded in 2009. The company specializes in developing advanced platforms for liquid and tissue biopsy analysis, focusing on the detection and characterization of rare cells, particularly circulating tumor cells (CTCs) and circulating endothelial cells (CECs) in blood samples. RareCyte's AccuCyte system provides an open platform that facilitates the unbiased discovery and isolation of these rare cells, supporting various applications in cancer research, cardiovascular disease studies, and diagnostic development. The company offers a range of products, including sample preparation systems, staining kits, automated fluorescence microscopy instruments, and slide imaging systems. By enabling precision medicine approaches, RareCyte aims to provide new tools for medical professionals and researchers to address complex questions in the fields of cancer and other therapeutic areas.

Founded in 2015 and based in Cambridge, Massachusetts, Obsidian Therapeutics develops next-generation controllable cell and gene therapies designed to extend adoptive immunotherapy to every cancer patient. Its innovative approach uses pharmacologic operating systems to precisely control protein activity in cells, enabling physicians to improve upon current cell therapies using simple, safe, orally-active marketed drugs.

DICE Therapeutics is a biopharmaceutical company dedicated to developing novel oral therapeutic candidates aimed at treating chronic diseases, particularly in immunology. Utilizing its proprietary DELSCAPE technology platform, DICE focuses on creating selective oral small molecules that can effectively modulate protein-protein interactions, similar to the efficacy of existing systemic biologics. The company's lead candidate, S011806, targets interleukin-17 (IL-17), a key pro-inflammatory molecule associated with various immunological conditions. Additionally, DICE is advancing oral therapies aimed at α4ß7 integrin and αVß1/αVß6 integrin, which are intended for the treatment of inflammatory bowel disease and idiopathic pulmonary fibrosis, respectively. Through its innovative approach, DICE Therapeutics aims to bring effective oral treatments to patients suffering from chronic inflammatory diseases.

Tango Therapeutics is a biotechnology company focused on developing cancer therapies based on the genetic principle of synthetic lethality. It aims to counteract tumor suppressor gene loss, reverse immune evasion in cancer cells, and identify effective drug combinations.

Nimbus Therapeutics, LLC is a biotechnology company based in Cambridge, Massachusetts, focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. Founded in 2009, the company utilizes a computational technology-driven approach to advance a pipeline of novel small-molecule therapeutics. Nimbus Therapeutics specializes in designing allosteric inhibitors, notably targeting Acetyl-CoA Carboxylase for conditions such as non-alcoholic steatohepatitis and hepatocellular carcinoma. The company has established strategic partnerships with organizations like Charles River Laboratories and Celgene Corporation to enhance its drug development efforts. In March 2015, Nimbus Discovery rebranded itself as Nimbus Therapeutics to better reflect its transition into a clinical-stage enterprise.

Element Biosciences develops innovative genetic analysis tools for research and diagnostic markets. Founded in 2017, the company specializes in a disruptive DNA sequencing technology that offers high-quality data, workflow flexibility, and affordable access to next-generation sequencing.

Gennao Bio is a privately held company focused on developing innovative targeted nucleic acid therapeutics, primarily aimed at treating oncology and rare monogenic skeletal muscle diseases. The company utilizes its proprietary gene monoclonal antibody (GMAB) platform technology, which features a novel, cell-penetrating antibody that facilitates the non-covalent binding and delivery of various nucleic acid payloads to specific cells. This non-viral delivery system stands out from traditional gene therapy methods by allowing for multiple types of nucleic acids, enabling repeat dosing, and employing established manufacturing processes. Gennao Bio's approach aims to address significant unmet medical needs and improve patient outcomes in the relevant therapeutic areas.

Boundless Bio develops innovative cancer therapeutics focused on understanding and treating previously untreatable cancers. It specializes in targeting extrachromosomal DNA (ecDNA) biology, a key driver of aggressive cancers.

Graphite Bio is a clinical-stage gene editing company focused on targeted DNA integration to treat serious diseases. Its platform aims to precisely insert, repair, or replace genetic sequences by integrating therapeutic cargoes at predefined genomic locations, enabling correction of mutations or replacement of disease-causing genes while preserving normal regulatory control. Based in South San Francisco, the company pursues high-efficiency precision gene editing to address underlying causes of disease.

Century Therapeutics is a biotechnology company developing allogeneic cell therapies derived from induced pluripotent stem cells to treat cancer and related diseases. Its platform enables engineering of iPSCs into NK and T cells with master cell banks of modified cells that can be expanded and differentiated into standardized, off-the-shelf therapies. The company combines CRISPR-mediated gene editing, proprietary chimeric antigen receptors, Allo-Evasion technology, and manufacturing capabilities to enable scalable production of homogeneous cell products for hematologic and solid tumors. While focused on oncology, its platform also targets autoimmune and inflammatory diseases with the goal of improving cell therapy performance and reducing host immune rejection.

DTx Pharma, LLC is a biotechnology company headquartered in San Diego, California, specializing in the development and commercialization of fatty acid-conjugated peptide therapeutics. Founded in 2017, the company focuses on delivering RNA-based medicines to treat patients with rare and chronic diseases, including retinitis pigmentosa, Duchenne muscular dystrophy, and various central nervous system disorders. DTx Pharma's innovative delivery technology addresses challenges faced by previous-generation platforms, such as poor pharmacokinetics and limited cellular uptake, thereby enhancing the efficiency of nucleic acid drug delivery across diverse tissues and cell types. The company aims to establish RNA therapeutics as a leading option for personalized treatment in multiple therapeutic areas.

Artiva Biotherapeutics, Inc. is a San Diego-based biotechnology company focused on developing and manufacturing cellular immunotherapies for cancer treatment. Founded in 2019, Artiva specializes in off-the-shelf, allogeneic natural killer (NK) cell therapies aimed at treating both hematologic cancers and solid tumors. The company's product pipeline includes therapies that target CD20 and CD19 in B-cell lymphomas, as well as HER2 in various solid tumors. Artiva is committed to harnessing the therapeutic potential of NK cells while addressing challenges in their scalability and manufacturing. Its mission centers on delivering effective and safe cell therapies that are readily accessible to cancer patients in need.

Viracta Therapeutics, Inc. is a clinical-stage drug development company based in Cardiff, California, that specializes in precision oncology focused on virus-associated malignancies. The company utilizes a viral activation therapy platform to develop treatments for cancers linked to the Epstein-Barr Virus (EBV) and other virus-related diseases. Its lead investigational drug, nanatinostat, is being evaluated in combination with the antiviral valganciclovir as a potential oral therapy for EBV-positive lymphoma, including post-transplant lymphoproliferative disorder, plasmablastic lymphoma, and angioimmunoblastic T cell lymphoma. Viracta is also exploring the application of this approach to other EBV-associated conditions, such as nasopharyngeal carcinoma and gastric carcinoma. Founded in 2007 and rebranded from HemaQuest Pharmaceuticals in 2015, Viracta Therapeutics is actively conducting multiple clinical trials to advance its therapeutic candidates.

Shoreline Biosciences, Inc. is a biotechnology company based in La Jolla, California, founded in 2020. The company specializes in developing cell-based immunotherapies aimed at treating seriously ill patients. Shoreline's proprietary technology platform leverages advanced methods in induced pluripotent stem cell (iPSC) differentiation and genetic programming, specifically targeting the IL-15/CISH pathway. This approach enhances the metabolic fitness and persistence of engineered natural killer (NK) cells, improving their anti-cancer activity. Shoreline's focus includes creating clinically effective immunotherapies using iPSC-derived NK cells and macrophages, providing innovative and cost-effective treatment options for oncology and other serious health conditions.

Verve Therapeutics is a biotechnology company developing genetic medicines for cardiovascular disease, aiming to transform treatment from chronic management to single-course gene editing therapies. It leverages human genetics analysis and gene-editing technology, with expertise in cardiovascular medicine, human genetics, gene editing, delivery technologies, drug development, and commercialization.

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.

Visen Pharmaceuticals is an innovative biopharmaceutical company focused on developing and providing advanced treatments for endocrine diseases in China. With a patient-centric approach, Visen aims to deliver first-in-class or best-in-class products, covering both common and rare endocrine conditions in adults and children. The company leverages cutting-edge technologies and global resources, with established offices in Shanghai, Beijing, Hong Kong, and Taipei, along with a Greater China R&D and manufacturing site in Suzhou.

Founded in 2020 and headquartered in Boston, Massachusetts, Scorpion Therapeutics is a clinical-stage biotechnology company specializing in precision oncology. It develops targeted small-molecule drugs to treat cancer by focusing on three areas: therapies against known oncogenes, agents for currently undruggable targets, and drugs for new targets.

Biomea Fusion is a precision medicine company focused on accelerating cancer drug development by targeting specific gene alterations that drive tumor growth. Its lead program aims to disrupt the protein-to-protein interaction between menin and the MLL complex for treating various tumors.

IconOVir Bio is a preclinical-stage biotechnology company focused on developing oncolytic virus therapies for cancer treatment. Its proprietary platform aims to overcome limitations of earlier generations, enabling personalized cancer care.

Founded in 1999, SomaLogic specializes in the discovery, development, and commercialization of life science research tools and clinical diagnostic products centered around its proprietary SOMAmer/SOMAscan technology. This platform enables protein measurement at scale, facilitating biomarker discovery for various diseases, drug development, and clinical diagnostics.

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.

Talaris Therapeutics, Inc. is a biotechnology company focused on developing innovative cell therapies aimed at eliminating chronic immunosuppression for organ transplant recipients. The company's leading product, FCR001, is designed to enable living donor kidney transplant recipients to discontinue all chronic immunosuppression therapy within twelve months post-transplant. In addition to its applications in organ transplantation, Talaris’s technology facilitates the safe use of hematopoietic stem cells from unmatched and unrelated donors for bone marrow transplants, reducing the associated toxicity and risks of traditional methods. Founded in 1988 and originally named Regenerex, Inc., Talaris Therapeutics rebranded in March 2019. The company is headquartered in Louisville, Kentucky, with an additional office in Wellesley, Massachusetts.

Olema Oncology is a preclinical biotechnology company dedicated to developing innovative therapies for the treatment and prevention of estrogen receptor-positive breast cancer. The company specializes in discovering and commercializing targeted treatments specifically designed for women's cancers. By leveraging its extensive understanding of endocrine-driven cancers, nuclear receptors, and mechanisms of acquired resistance, Olema aims to create compounds that surpass existing therapies. The company's focus is on transforming the standard of care for both pre- and post-menopausal women with cancer, with an emphasis on developing more effective and convenient treatment options. Olema's pipeline includes drug candidates such as OP-1250 and OP-3136, which have progressed through discovery and preclinical studies.

Flame Biosciences is a clinical-stage biotechnology company based in New York, founded in 2017. The company specializes in developing innovative therapies for cancer and other inflammatory conditions. Its portfolio includes FL-101 therapy, designed to treat lung cancers driven by inflammation, and FL-201, aimed at reducing chronic inflammation in patients. Flame Biosciences is dedicated to improving the lives of individuals suffering from debilitating and life-threatening diseases through its transformative therapeutic approaches.

Kinnate Biopharma Inc. is a biopharmaceutical company dedicated to the discovery and development of small molecule kinase inhibitors aimed at treating genomically defined cancers. Established in 2018 and headquartered in San Diego, California, the company focuses on precision oncology therapeutics, leveraging its expertise in structure-based drug discovery and translational research. Kinnate's lead product candidate, KIN002787, is a rapidly accelerated fibrosarcoma inhibitor currently in preclinical development for lung cancer, melanoma, and other solid tumors. Additionally, the company is advancing other preclinical candidates, including KIN003, which targets cancer-associated alterations in FGFR2 and FGFR3 genes, and a Cyclin-Dependent Kinase 12 inhibitor in the KIN004 program. Kinnate Biopharma is committed to developing targeted therapeutics for underserved patient populations, aiming to expand the reach of precision medicine in cancer treatment.

Olema Oncology is a preclinical biotechnology company dedicated to developing innovative therapies for the treatment and prevention of estrogen receptor-positive breast cancer. The company specializes in discovering and commercializing targeted treatments specifically designed for women's cancers. By leveraging its extensive understanding of endocrine-driven cancers, nuclear receptors, and mechanisms of acquired resistance, Olema aims to create compounds that surpass existing therapies. The company's focus is on transforming the standard of care for both pre- and post-menopausal women with cancer, with an emphasis on developing more effective and convenient treatment options. Olema's pipeline includes drug candidates such as OP-1250 and OP-3136, which have progressed through discovery and preclinical studies.

C4 Therapeutics is a biopharmaceutical company developing targeted protein degradation therapies using its proprietary Degronimid platform. This platform enables the selective destruction of disease-causing proteins, expanding the scope of treatable conditions and potentially reducing drug resistance. The company focuses on oncology indications and has strategic collaborations with industry leaders.

Pliant Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in South San Francisco, California, dedicated to discovering and developing innovative therapies for fibrotic diseases. Its lead product candidate, PLN-74809, is an oral small-molecule dual selective inhibitor of avß6 and avß1 integrins, targeting idiopathic pulmonary fibrosis and primary sclerosing cholangitis. PLN-74809 has received Orphan Drug Designation from the U.S. Food and Drug Administration for both indications and is currently undergoing Phase 2a clinical trials. The company is also developing PLN-1474, a small-molecule selective inhibitor of avß1, for the treatment of liver fibrosis associated with nonalcoholic steatohepatitis, which is in Phase 1 trials. Founded in 2015, Pliant Therapeutics is also advancing preclinical programs aimed at addressing oncology and muscular dystrophies.

Avidity Biosciences is a biopharmaceutical company developing antibody oligonucleotide conjugates to enable tissue-selective delivery and precision RNA therapies for serious diseases. Its AOC platform combines monoclonal antibody tissue targeting with oligonucleotide therapies to access previously undruggable tissues and genetic drivers. The lead candidate, AOC 1001, targets myotonic dystrophy type 1, and its muscle programs address muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. The pipeline also includes immune and other cell-type programs, including Del-zota for DMD, Del-desiran for DM1, and Del-brax for FSHD. Avidity is based in La Jolla, California.

Repare Therapeutics is a precision oncology company that discovers and develops novel therapeutics using its synthetic lethality approach. It employs its proprietary SNIPRx platform to systematically identify and target genomic instability, particularly DNA damage repair pathways in cancer cells. The company's lead product candidate, RP-3500, is an oral small molecule inhibitor for treating solid tumors with specific DNA damage repair-related alterations.

Passage Bio is a gene therapy company dedicated to developing transformative treatments for rare, monogenic central nervous system disorders. It has an integrated pipeline of five AAV-delivered therapeutics and collaborates with the University of Pennsylvania's Gene Therapy Program for preclinical work.