RA Capital

Imbria Pharmaceuticals, founded in 2018 and headquartered in Boston, Massachusetts, is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies targeting cardiometabolic disorders. The company focuses on understanding the underlying mechanisms of these diseases, particularly energetic impairment at the cellular level, to inform its therapeutic candidates. Imbria's pipeline aims to improve the lives of patients suffering from conditions such as non-obstructive hypertrophic cardiomyopathy, stable angina, and heart failure with preserved ejection fraction by restoring or enhancing cellular energy production.

Vaxess Technologies is a biotechnology company focused on silk biomaterial platforms for vaccine and therapeutic delivery. It develops the MIMIX microneedle patch for transdermal, sustained-release delivery of vaccines and therapies, designed to improve stability and ease of use, including formulations that do not require cold chain storage. The MATRIX formulation and drying platform supports thermostable vaccine, therapeutic, and diagnostic products. The company aims to enable long-term immune responses and easier distribution, including in low-resource settings. Its technology supports oncology and infectious disease applications, including therapies that promote durable anti-tumor immunity and improved treatment outcomes. Founded in 2011 and based in Boston, the company positions its platforms to enable stable, targeted delivery and potentially reduce treatment burden for patients.

Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative treatments for severe, rare muscle disorders with significant unmet medical need. It employs a holistic drug discovery approach targeting the muscle as an organ, utilizing its proprietary platform to identify precision medicines regulating key proteins in muscle tissue.

Surrozen, Inc. is a clinical-stage biopharmaceutical company based in South San Francisco, California, focused on developing targeted regenerative antibodies to enhance tissue and organ repair in response to serious diseases. Founded in 2015, the company specializes in modulating the Wnt pathway, a crucial mediator of tissue regeneration, through its innovative antibody platforms that target specific organs and tissues. Notable programs include SZN-043, designed to stimulate liver regeneration in both acute and chronic liver diseases, and SZN-1326, aimed at repairing damaged epithelial tissue in patients with inflammatory bowel disease. Surrozen's approach extends to multiple organ systems, with potential applications in the intestine, liver, retina, cornea, lung, kidney, cochlea, skin, pancreas, and central nervous system.

Founded in 2014, Vivace Therapeutics is a biotechnology company based in the San Francisco Bay Area. It focuses on discovering and developing cancer therapeutics by targeting novel pathways, with a particular emphasis on the Hippo pathway which controls tissue regeneration and organ size.

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.

Abcuro develops immunotherapies targeting autoimmunity and cancer. Founded in 2015, the company uses bioinformatics and ex-vivo validation to uncover novel immune system targets.

Founded in 2020, Be Biopharma specializes in developing innovative therapeutics using engineered B-cells to treat diseases such as cancer, autoimmune disorders, and infectious diseases. Its approach aims to overcome limitations of current cell and gene therapies.

Vor Biopharma develops innovative cancer therapies focused on engineered hematopoietic stem cells (eHSCs). Its lead product candidate, VOR33, is designed to treat acute myeloid leukemia by generating healthy cells resistant to targeted immunotherapies. Incorporated in 2015, the company is based in Cambridge, Massachusetts.

Monopar Therapeutics Inc. is a clinical-stage biopharmaceutical company based in Wilmette, Illinois, focused on developing proprietary therapeutics to improve outcomes for cancer patients. The company is advancing several key products, including Validive, a clonidine mucobuccal tablet in Phase III trials aimed at preventing severe oral mucositis caused by chemoradiotherapy in oropharyngeal cancer patients. Another significant compound is camsirubicin, a targeted analog of doxorubicin designed to treat soft tissue sarcoma while minimizing cardiac toxicity. Additionally, Monopar is developing MNPR-101, a humanized monoclonal antibody targeting advanced solid tumors, and MNPR-202, an early-stage analog for various cancers. Monopar collaborates with organizations such as Grupo Español de Investigación en Sarcomas and NorthStar Medical Radioisotopes, focusing on treatments for both cancer and severe COVID-19. Founded in 2014, the company emphasizes the development of orphan oncology compounds to extend life and enhance the quality of life for patients.

Cidara Therapeutics is a biotechnology company dedicated to developing novel anti-infectives. Its primary focus is rezafungin acetate, an antifungal for treating serious invasive fungal infections. Additionally, it advances the Cloudbreak platform to develop antiviral therapies using Fc-conjugates.

Synapticure specializes in providing remote, disease-specific neurological care for patients with neurodegenerative diseases such as dementia, Parkinson's, and ALS across the United States. Founded by patients, they offer on-demand neurologists, speech-language therapists, round-the-clock care coordination, and support services tailored to each patient's needs.

Evommune, Inc. is a biotechnology company based in Los Altos, California, that develops and manufactures tissue-based medicines for inflammatory diseases. Incorporated in 2020, Evommune focuses on creating novel strategies to treat these disorders by addressing both symptoms and the underlying causes, thereby halting disease progression. The company employs a proprietary tissue-based platform that accelerates the discovery of innovative treatments, enhancing the potential for clinical success. With a commitment to improving the quality of life for patients, Evommune's pipeline includes initiatives aimed at tackling a range of prevalent inflammatory illnesses, positioning the company as a leader in advancing therapies in the fields of immunology and dermatology.

Eledon Pharmaceuticals is a clinical-stage biotechnology company focused on developing treatments for patients undergoing organ or cellular transplantation and those with autoimmune and neurodegenerative diseases. Its lead compound, tegoprubart, is an anti-CD40L antibody targeting the CD40L pathway.

Monopar Therapeutics Inc. is a clinical-stage biopharmaceutical company based in Wilmette, Illinois, focused on developing proprietary therapeutics to improve outcomes for cancer patients. The company is advancing several key products, including Validive, a clonidine mucobuccal tablet in Phase III trials aimed at preventing severe oral mucositis caused by chemoradiotherapy in oropharyngeal cancer patients. Another significant compound is camsirubicin, a targeted analog of doxorubicin designed to treat soft tissue sarcoma while minimizing cardiac toxicity. Additionally, Monopar is developing MNPR-101, a humanized monoclonal antibody targeting advanced solid tumors, and MNPR-202, an early-stage analog for various cancers. Monopar collaborates with organizations such as Grupo Español de Investigación en Sarcomas and NorthStar Medical Radioisotopes, focusing on treatments for both cancer and severe COVID-19. Founded in 2014, the company emphasizes the development of orphan oncology compounds to extend life and enhance the quality of life for patients.

Alpha9 Theranostics is a biopharma company that focuses on radiotherapeutics for the treatment of solid and hematologic malignancies. The company is creating a strong pipeline of radiopharmaceuticals to target solid and hematologic malignancies. Its systematic approach to radiotherapeutic design is fueled by a toolbox of technologies and chemistries, which offers broad potential for expansion into a multitude of oncology targets. Alpha9 Theranostics was founded in 2019 and is headquartered in Vancouver, Canada.

Founded in 2020, Be Biopharma specializes in developing innovative therapeutics using engineered B-cells to treat diseases such as cancer, autoimmune disorders, and infectious diseases. Its approach aims to overcome limitations of current cell and gene therapies.

Founded in Boston in 2016, Elektrofi is a preclinical-stage company focused on revolutionizing drug delivery. Its proprietary formulation system can efficiently process a wide range of therapeutic molecules, including large and complex proteins like monoclonal antibodies.

MBX Biosciences is a biotechnology company focused on developing therapies for endocrine and metabolic disorders, emphasizing rare endocrine diseases with limited treatment options. It pursues discovery, development, and commercialization of first-in-class peptide therapeutics that modulate glandular hormone signaling. MBX has developed a proprietary Precision Endocrine Peptide platform to engineer peptides with extended time-action profiles and consistent drug exposure, aiming to overcome limitations of traditional peptide therapies and improve disease management. Based in Carmel, Indiana, the company advances novel peptide programs through clinical development to address unmet medical needs in endocrine and metabolic disorders.

Vaxess Technologies is a biotechnology company focused on silk biomaterial platforms for vaccine and therapeutic delivery. It develops the MIMIX microneedle patch for transdermal, sustained-release delivery of vaccines and therapies, designed to improve stability and ease of use, including formulations that do not require cold chain storage. The MATRIX formulation and drying platform supports thermostable vaccine, therapeutic, and diagnostic products. The company aims to enable long-term immune responses and easier distribution, including in low-resource settings. Its technology supports oncology and infectious disease applications, including therapies that promote durable anti-tumor immunity and improved treatment outcomes. Founded in 2011 and based in Boston, the company positions its platforms to enable stable, targeted delivery and potentially reduce treatment burden for patients.

Ajax Therapeutics is a New York City-based biotechnology company that applies computational chemistry and structure-based technologies to develop novel small molecules for treating hematologic malignancies.

Medicenna Therapeutics is a clinical‑stage immuno‑oncology company headquartered in Toronto, Canada, founded in 2012. It develops and commercializes highly selective IL‑2, IL‑4, and IL‑13 Superkines and Empowered Cytokines (ECs) for cancer, inflammation, and immune‑mediated diseases. The pipeline includes MDNA55, an IL‑4 EC in Phase 2b trials for recurrent glioblastoma, as well as MDNA57 for solid tumors, MDNA109, an IL‑2 agonist for cancer immunotherapies, MDNA209, an IL‑2 antagonist for autoimmune conditions, MDNA413, a dual IL‑4/IL‑13 antagonist for solid tumors and fibrotic diseases, and MDNA132, an IL‑13 agonist targeting solid tumors and IL‑13Rα2‑expressing cells. The company also explores BiSKITs, a bifunctional SuperKine platform that delivers potent cell‑killing agents while modulating the tumor micro‑environment without harming healthy tissue.

Asher Biotherapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2019. The company specializes in the development of immunotherapy drugs aimed at effectively treating cancer. Its innovative therapies are designed to enhance the immune system's ability to target and eliminate cancer cells while minimizing side effects. This focus allows healthcare providers to deliver effective treatments to patients in a shorter timeframe, aiming to improve overall outcomes in cancer care.

Eliem Therapeutics is a clinical-stage biotechnology company focused on developing treatments for nervous system disorders driven by neuronal hyperexcitability. It leverages neuroscience and translational medicine to advance analgesics and other therapies for conditions such as chronic pain, aiming to deliver best-in-class drug candidates that alleviate suffering and improve function. Founded in 2018 and based in Boston, Eliem concentrates on translating scientific insights into clinical solutions to address unmet medical needs in hyperexcitability disorders.

Protara Therapeutics is a clinical-stage company focused on identifying and advancing transformative therapies for cancer and rare diseases. Its lead program, TARA-002, is an investigational cell therapy being developed for non-muscle invasive bladder cancer and lymphatic malformations, while IV choline chloride is an investigational phospholipid substrate replacement therapy for intestinal failure-associated liver disease. The company is based in New York, New York.

Surrozen, Inc. is a clinical-stage biopharmaceutical company based in South San Francisco, California, focused on developing targeted regenerative antibodies to enhance tissue and organ repair in response to serious diseases. Founded in 2015, the company specializes in modulating the Wnt pathway, a crucial mediator of tissue regeneration, through its innovative antibody platforms that target specific organs and tissues. Notable programs include SZN-043, designed to stimulate liver regeneration in both acute and chronic liver diseases, and SZN-1326, aimed at repairing damaged epithelial tissue in patients with inflammatory bowel disease. Surrozen's approach extends to multiple organ systems, with potential applications in the intestine, liver, retina, cornea, lung, kidney, cochlea, skin, pancreas, and central nervous system.

SeLux Diagnostics, Inc. is a biotechnology company based in Charlestown, Massachusetts, focused on developing a next-generation phenotyping platform for antibiotic susceptibility testing and diagnostics of infectious diseases. Established in 2014, the company utilizes synthetic amplifier technology to enhance the speed and complexity of antibiotic susceptibility testing, thereby significantly improving patient care. By facilitating the rapid selection of personalized antimicrobial therapies, SeLux aims to transform the management of infectious diseases, ultimately saving lives, reducing hospital stays, and combating the growing issue of antibiotic resistance through more targeted treatments.

Geron Corporation is a late-stage clinical biopharmaceutical company based in Menlo Park, California, specializing in the development and commercialization of therapeutics for hematologic malignancies. Established in 1990, the company is primarily focused on its investigational drug, imetelstat, a first-in-class telomerase inhibitor currently undergoing Phase 2/3 clinical trials. Imetelstat targets the uncontrolled proliferation of malignant progenitor cells, aiming to improve blood cell production in patients with conditions such as myelodysplastic syndromes and myelofibrosis. Geron generates revenue through collaboration agreements, milestones, royalties, and licensing arrangements, positioning itself as a key player in oncology therapeutics.

Vesta Healthcare is a technology and clinical services organization based in New York, specializing in connecting caregivers to care teams through its innovative health care platform. The company offers 24/7 telehealth support aimed at caregivers and care recipients, particularly focusing on high-need, frail senior populations. Vesta partners with home care agencies, health plans, and healthcare providers to develop value-based population health programs that prioritize clinical quality and improved health outcomes. Founded in 2008 and originally known as Hometeam Technologies, Inc., Vesta Healthcare has established itself as a leader in enhancing caregiver insights and facilitating personalized engagement in the healthcare setting.

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.

Regulus Therapeutics Inc. is a clinical-stage biopharmaceutical company specializing in the discovery and development of microRNA-targeted therapies for various diseases. Founded in 2007 and headquartered in San Diego, California, the company focuses on innovative treatments for conditions such as kidney diseases and viral infections. Its lead product candidates include RG-012, an anti-miR targeting miR-21, currently in Phase II clinical trials for Alport syndrome, and RGLS4326, an anti-miR targeting miR-17, which is undergoing Phase Ib clinical trials for autosomal dominant polycystic kidney disease. Additionally, Regulus is advancing a pipeline of preclinical drug candidates, including RGLS5579 to inhibit miR-10b, as well as programs aimed at treating Hepatitis B virus and Non-Alcoholic Steatohepatitis. The company's work leverages the recent scientific advancements in microRNA research, which play a crucial role in regulating gene expression.

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.

Frontier Medicines Corporation, established in 2018 and headquartered in South San Francisco, California, is a pre-clinical stage biopharmaceutical company specializing in cancer research. The company employs chemoproteomics to discover and develop novel medicines targeting cancer-causing proteins. Frontier Medicines' innovative platform integrates advanced computational methods and machine learning algorithms to identify and pharmacologically target new binding pockets on proteins, thereby enabling small-molecule drug discovery and development for previously inaccessible targets.

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

BioAge Labs develops medicines to treat aging and age-related diseases using its proprietary BioAge platform, which combines systems biology and AI. Its pipeline includes BGE-117 for multiple aging-related conditions, BGE-175 for immune aging and respiratory infections, and azelaprag for obesity in the elderly.

Tenaya Therapeutics is a preclinical-stage biotechnology company focused on developing therapies for heart disease and heart failure. It operates three product platforms: cellular regeneration, which aims to regenerate heart tissue by reprogramming cardiac fibroblasts into cardiomyocytes via delivery of proprietary transcription factors; gene therapy, which targets cardiac fibroblasts to deliver therapeutic payloads; and precision medicine, which enables personalized approaches to treat heart disease. Founded in 2016 and headquartered in South San Francisco, California, Tenaya seeks to address the underlying cellular pathologies of diseased myocardium with curative therapies that advance from discovery through development.

Tyra Biosciences is a biotechnology company focused on developing precision therapies for cancer. It uses its proprietary SNAP platform to create next-generation drug candidates that target acquired drug resistance, with an initial focus on the Fibroblast Growth Factor Receptor (FGFR) family.

Astria Therapeutics is a biopharmaceutical company focused on developing therapies for rare and niche allergic and immunological diseases. It is advancing monoclonal antibody programs such as STAR-0215, a plasma kallikrein inhibitor being developed for hereditary angioedema, and STAR-0310, an OX40 antagonist for atopic dermatitis, both in preclinical development to address immune-mediated inflammatory conditions.

Vaxart is a clinical-stage biotechnology company developing oral recombinant protein vaccines using its proprietary platform. Its pipeline includes vaccines for norovirus, influenza, RSV, and HPV, with products administered via convenient room-temperature stable tablets.

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.

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.

NTx is a biotechnology company specializing in a proprietary host-independent biological manufacturing platform. This technology extends beyond vaccines, potentially revolutionizing the production of protein-based therapies for cancer and autoimmune disorders.

Founded in 2016, AM Batteries specializes in the manufacture of Li-ion batteries using solvent-free electrode technology. This innovative approach aims to enhance energy efficiency, enable faster charging, and reduce production costs.

Commons Clinic is a multispecialty healthcare provider based in Austin, Texas, founded in 2021. It delivers comprehensive specialty and surgical care for pain, orthopedics, women's health, and men's health. The company also develops a healthcare platform that combines preventive diagnostics with specialist-driven interventions, uniting advanced diagnostics, personalized care pathways, and interventional procedures within an accountable system across musculoskeletal care, women's health, and preventive oncology to support informed clinical decisions and improved patient outcomes.

SAB Biotherapeutics is a clinical-stage biopharmaceutical company established in 2014, headquartered in Sioux Falls, South Dakota with additional offices in Cambridge, Massachusetts. The company specializes in developing immunotherapies using its proprietary DiversitAb platform, which employs transchromosomic cattle to produce large quantities of targeted human polyclonal antibodies without relying on human plasma or serum. SAB's pipeline includes therapies for conditions such as Type 1 diabetes (SAB-142), seasonal influenza (SAB-176), and Middle East Respiratory Syndrome Coronavirus (MERS-CoV, SAB-301).

Vaxess Technologies is a biotechnology company focused on silk biomaterial platforms for vaccine and therapeutic delivery. It develops the MIMIX microneedle patch for transdermal, sustained-release delivery of vaccines and therapies, designed to improve stability and ease of use, including formulations that do not require cold chain storage. The MATRIX formulation and drying platform supports thermostable vaccine, therapeutic, and diagnostic products. The company aims to enable long-term immune responses and easier distribution, including in low-resource settings. Its technology supports oncology and infectious disease applications, including therapies that promote durable anti-tumor immunity and improved treatment outcomes. Founded in 2011 and based in Boston, the company positions its platforms to enable stable, targeted delivery and potentially reduce treatment burden for patients.

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.

Sortera Alloys manufactures low-cost, high-quality metal alloys for domestic manufacturing using its artificial intelligence technology. Sortera Alloys is a company based in Fort Wayne, Indiana, and was established in 2020.

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

Abcuro develops immunotherapies targeting autoimmunity and cancer. Founded in 2015, the company uses bioinformatics and ex-vivo validation to uncover novel immune system targets.

COMPASS Pathways is a UK-based mental health care company dedicated to accelerating patient access to innovative treatments for mental health challenges. The company focuses on developing psilocybin therapy, administered in conjunction with psychological support, for patients with treatment-resistant depression who have not responded to conventional treatments.

AN2 Therapeutics is a biopharmaceutical company dedicated to researching, developing, and commercializing innovative medicines targeting infectious diseases. Established in 2017, the company focuses on advancing a clinical-stage antibacterial compound and has a strategic partnership with Brii Biosciences.

Taysha Gene Therapies develops adeno-associated virus-based gene therapies to treat monogenic diseases of the central nervous system. Its pipeline includes treatments for GM2 gangliosidosis, CLN1 disease, Rett syndrome, SLC6A1 haploinsufficiency disorder, and Surfeit locus 1 deficiency. The company collaborates with The University of Texas Southwestern Medical Center to develop and commercialize transformative gene therapy treatments.

Carmot Therapeutics, Inc. is a clinical-stage biotechnology company based in Berkeley, California, with an additional office in San Francisco. Founded in 2008, it focuses on discovering and developing innovative therapies for patients with metabolic diseases, including obesity and diabetes. Utilizing its proprietary Chemotype Evolution technology, Carmot Therapeutics aims to address the underlying causes of these conditions and accelerate the discovery of effective treatments. The company’s portfolio includes several clinical-stage drug candidates, such as CT-388 and CT-996, which are designed as GLP-1 receptor agonists for the treatment of obesity and type 2 diabetes, and CT-868, intended for type 1 diabetes patients who are overweight or obese. Through these efforts, Carmot Therapeutics seeks to create life-changing therapeutics that improve the health outcomes of individuals affected by metabolic 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.

Cortica Inc. is a company dedicated to providing neurological therapies for children facing autism and other neurodevelopmental challenges. Founded in 2014 and based in San Diego, California, Cortica offers tailored treatment programs that are informed by a comprehensive understanding of each child's neurobiology and developmental profile. The company operates multiple centers across California, including locations in Carlsbad, Irvine, San Rafael, Torrance, and Westlake Village. Cortica's services encompass a wide range of assessments and therapies, including diagnoses for autism, ADHD, and developmental delays. Treatment options available include gene sequencing, metabolic testing, electroencephalography, nutrition evaluation, medication management, and various therapeutic interventions such as ABA therapy, parent coaching, speech-language therapy, occupational therapy, physical therapy, and music therapy. The company provides these services through in-home, in-clinic, and telehealth modalities.

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.

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

Vaxess Technologies is a biotechnology company focused on silk biomaterial platforms for vaccine and therapeutic delivery. It develops the MIMIX microneedle patch for transdermal, sustained-release delivery of vaccines and therapies, designed to improve stability and ease of use, including formulations that do not require cold chain storage. The MATRIX formulation and drying platform supports thermostable vaccine, therapeutic, and diagnostic products. The company aims to enable long-term immune responses and easier distribution, including in low-resource settings. Its technology supports oncology and infectious disease applications, including therapies that promote durable anti-tumor immunity and improved treatment outcomes. Founded in 2011 and based in Boston, the company positions its platforms to enable stable, targeted delivery and potentially reduce treatment burden for patients.

Apogee Therapeutics is a biotechnology company focused on developing therapies for immunological and inflammatory disorders. Based in San Francisco and founded in 2022, it aims to advance novel biologics to address patients’ unmet needs. The company pursues differentiated antibody programs targeting well-established mechanisms implicated in atopic dermatitis, chronic obstructive pulmonary disease, and related inflammatory and immunology indications, supported by antibody engineering to optimize half-life and other properties.

Vor Biopharma develops innovative cancer therapies focused on engineered hematopoietic stem cells (eHSCs). Its lead product candidate, VOR33, is designed to treat acute myeloid leukemia by generating healthy cells resistant to targeted immunotherapies. Incorporated in 2015, the company is based in Cambridge, Massachusetts.

Jnana Therapeutics Inc. is a biotechnology company based in Boston, Massachusetts, founded in 2016. It specializes in drug discovery, particularly focusing on solute carrier (SLC) transporters, which are vital for understanding various disease pathways. The company’s efforts are directed towards immunometabolism, lysosomal function, and mucosal defense, with applications in immuno-oncology, inflammatory disorders, and neurological diseases. Utilizing its proprietary RAPID platform, Jnana accelerates the identification of therapeutic targets and the development of small molecule therapies. By leveraging advanced chemoproteomics techniques, the company aims to enhance the efficiency of drug discovery and address significant unmet medical needs in patient care.

MBX Biosciences is a biotechnology company focused on developing therapies for endocrine and metabolic disorders, emphasizing rare endocrine diseases with limited treatment options. It pursues discovery, development, and commercialization of first-in-class peptide therapeutics that modulate glandular hormone signaling. MBX has developed a proprietary Precision Endocrine Peptide platform to engineer peptides with extended time-action profiles and consistent drug exposure, aiming to overcome limitations of traditional peptide therapies and improve disease management. Based in Carmel, Indiana, the company advances novel peptide programs through clinical development to address unmet medical needs in endocrine and metabolic disorders.

Vaxess Technologies is a biotechnology company focused on silk biomaterial platforms for vaccine and therapeutic delivery. It develops the MIMIX microneedle patch for transdermal, sustained-release delivery of vaccines and therapies, designed to improve stability and ease of use, including formulations that do not require cold chain storage. The MATRIX formulation and drying platform supports thermostable vaccine, therapeutic, and diagnostic products. The company aims to enable long-term immune responses and easier distribution, including in low-resource settings. Its technology supports oncology and infectious disease applications, including therapies that promote durable anti-tumor immunity and improved treatment outcomes. Founded in 2011 and based in Boston, the company positions its platforms to enable stable, targeted delivery and potentially reduce treatment burden for patients.

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.

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.

Empatica Inc. is a Cambridge, Massachusetts-based company that specializes in the development of wearable medical sensor devices aimed at health monitoring, forecasting, treatment, and research. Founded in 2013, Empatica creates AI-enabled wearables, including the E3 wristband, which monitors physiological signals in real time, along with accompanying software applications and web dashboards for stress and sleep analysis. The company's technology employs a combination of biosensors to capture and analyze unique aspects of human physiology, offering valuable insights to patients, clinicians, and researchers. Notable clients include Microsoft Research, Sony, University of Trento, and Intel, highlighting the company's impact in the field of health technology.

Carmot Therapeutics, Inc. is a clinical-stage biotechnology company based in Berkeley, California, with an additional office in San Francisco. Founded in 2008, it focuses on discovering and developing innovative therapies for patients with metabolic diseases, including obesity and diabetes. Utilizing its proprietary Chemotype Evolution technology, Carmot Therapeutics aims to address the underlying causes of these conditions and accelerate the discovery of effective treatments. The company’s portfolio includes several clinical-stage drug candidates, such as CT-388 and CT-996, which are designed as GLP-1 receptor agonists for the treatment of obesity and type 2 diabetes, and CT-868, intended for type 1 diabetes patients who are overweight or obese. Through these efforts, Carmot Therapeutics seeks to create life-changing therapeutics that improve the health outcomes of individuals affected by metabolic diseases.

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

Concert Pharmaceuticals is a clinical‑stage biopharmaceutical company headquartered in Lexington, Massachusetts, founded in 2006. The company develops small‑molecule therapies for autoimmune and central nervous system disorders using a precision deuterium chemistry platform that modifies drug molecules to improve efficacy, safety, and reduce development risk. Its pipeline includes CTP‑543, a Phase III candidate for alopecia areata; CTP‑692, a Phase II candidate for schizophrenia; and AVP‑786, a Phase III combination therapy for neurologic and psychiatric conditions such as agitation in Alzheimer’s disease. Concert collaborates with Avanir, Cipla Technologies, Processa, and Jazz Pharmaceuticals to advance its candidates. The firm focuses on orally administered agents and repurposed drugs for conditions including cystic fibrosis, inflammation, narcolepsy, residual schizophrenia, major depressive disorder, and Alzheimer’s agitation.

Celcuity is a clinical-stage biotechnology company that discovers new cancer sub-types and therapeutic options using its proprietary CELsignia diagnostic platform. This platform analyzes living tumor cells to identify specific abnormal cellular processes driving cancer, enabling the development of targeted therapies for patients.

Founded in 2020, Be Biopharma specializes in developing innovative therapeutics using engineered B-cells to treat diseases such as cancer, autoimmune disorders, and infectious diseases. Its approach aims to overcome limitations of current cell and gene therapies.

Incyclix Bio is a biotechnology company based in Durham, North Carolina, focused on developing small molecule inhibitors of cyclin-dependent kinases (CDKs) for the treatment of advanced and resistant cancers. Founded in 2020 by Patrick Roberts, Jay Strum, John Bisi, and Fred Eshelman, the company aims to provide potent and selective inhibition of challenging CDK targets. Incyclix Bio's innovative approach seeks to equip pharmaceutical companies and medical practitioners with novel therapeutic options, enhancing the treatment landscape for cancer patients.

SeLux Diagnostics, Inc. is a biotechnology company based in Charlestown, Massachusetts, focused on developing a next-generation phenotyping platform for antibiotic susceptibility testing and diagnostics of infectious diseases. Established in 2014, the company utilizes synthetic amplifier technology to enhance the speed and complexity of antibiotic susceptibility testing, thereby significantly improving patient care. By facilitating the rapid selection of personalized antimicrobial therapies, SeLux aims to transform the management of infectious diseases, ultimately saving lives, reducing hospital stays, and combating the growing issue of antibiotic resistance through more targeted treatments.

Plexium, Inc. is a biotechnology company based in San Diego, California, specializing in the development of targeted protein degradation therapeutics aimed at treating cancer and neurodegenerative diseases. Founded in 2017, Plexium focuses on identifying E3 ligase modulating small molecules to create innovative therapies. The company has developed a screening platform known as DELPhe, which utilizes advanced techniques to miniaturize cell-based assays for high-content readouts. This platform enables the modulation of E3 ligases to selectively target various cancers, including small cell lung and breast tumors, while also inducing immune responses. By exploring next-generation drug candidates that extend beyond traditional approaches, Plexium is poised to uncover new therapeutic possibilities across a wide range of medical applications.

Somatus, Inc. is a healthcare company focused on providing specialized kidney care services aimed at delaying or preventing the progression of chronic kidney disease. Founded in 2016 and headquartered in McLean, Virginia, Somatus collaborates with health plans, health systems, nephrology, and primary care groups to deliver integrated care for patients with or at risk of kidney disease. Its comprehensive services include home hemodialysis, peritoneal dialysis, in-center hemodialysis, kidney transplant, and conservative care. By utilizing vertically integrated clinical services and technology, Somatus seeks to improve patient outcomes, enhance care coordination, and promote the use of home dialysis modalities while increasing the rates of kidney transplantation. The company's mission is to ensure that patients can access the care they need to manage kidney disease effectively.

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.

Metagenomi is a genetic medicines company headquartered in Emeryville, California. It leverages metagenomics and machine learning to discover novel genome editing systems from diverse organisms, then convert these natural systems into compact, highly efficient, and specific tools aimed at reducing immune responses. The company maintains a proprietary, metagenomics-derived genome editing toolbox that includes programmable nucleases, base editors, small RNA-mediated integration systems (RIGS), large template RIGS, and CRISPR-associated transposases (CASTs). Its development focus is in vivo gene editing with a wholly owned pipeline, while exploring technology out-licensing for ex vivo cell therapies to pursue curative therapeutics for genetic diseases.

AN2 Therapeutics is a biopharmaceutical company dedicated to researching, developing, and commercializing innovative medicines targeting infectious diseases. Established in 2017, the company focuses on advancing a clinical-stage antibacterial compound and has a strategic partnership with Brii Biosciences.

Founded in 2008, Nucleix Ltd. specializes in developing non-invasive molecular diagnostic tests for early cancer detection. Its proprietary tests, Bladder EpiCheck and Lung EpiCheck, identify subtle methylation changes in urine and blood samples respectively.

Tasso, Inc., established in 2012 and based in Seattle, Washington, specializes in manufacturing clinical-grade, at-home blood sample collection devices. Their innovative product allows patients to conveniently and painlessly collect their own blood at home using a simple, push-button mechanism, and then mail the samples to a qualified lab for analysis. This approach aims to enhance accessibility and comfort in blood-based diagnostics, reducing the need for clinic visits.

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

Antios Therapeutics, Inc. is a biopharmaceutical company based in Atlanta, Georgia, focused on developing innovative therapies for viral diseases. Founded in 2018 by Abel De La Rosa, Douglas Mayers, and Idean Marvasty, the company is dedicated to addressing the needs of patients infected with Hepatitis B and Hepatitis D viruses. Its lead product, ATI-2173, is an oral drug candidate aimed at providing a potentially curative treatment for Hepatitis B virus infections. With a leadership team that brings a wealth of experience, Antios Therapeutics is committed to advancing its therapeutic solutions in the field of viral diseases.

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.

Delix Therapeutics is a neuroscience company focused on harnessing the power of psychoplastogens—novel neuroplasticity-promoting therapeutics—to better treat mental health disorders at scale. The company was founded in 2019 and is headquartered in Boston, Massachusetts.

Qvella Corporation is a Toronto-based molecular diagnostics company founded in 2009 by a group of scientists and engineers. The company focuses on transforming clinical microbiology by significantly reducing the time required to obtain results for bloodstream infections. Qvella's innovative Field Activated Sample Treatment (FAST™) technology employs a novel electrical lysing technique, known as e-lysis™, which facilitates the rapid and fully automated detection of infectious agents directly from unenriched biological samples. This capability allows clinicians to identify microorganisms in infected whole blood samples without the lengthy sample processing typically required, ultimately enhancing patient outcomes and saving lives.

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

Asher Biotherapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2019. The company specializes in the development of immunotherapy drugs aimed at effectively treating cancer. Its innovative therapies are designed to enhance the immune system's ability to target and eliminate cancer cells while minimizing side effects. This focus allows healthcare providers to deliver effective treatments to patients in a shorter timeframe, aiming to improve overall outcomes in cancer care.

Jnana Therapeutics Inc. is a biotechnology company based in Boston, Massachusetts, founded in 2016. It specializes in drug discovery, particularly focusing on solute carrier (SLC) transporters, which are vital for understanding various disease pathways. The company’s efforts are directed towards immunometabolism, lysosomal function, and mucosal defense, with applications in immuno-oncology, inflammatory disorders, and neurological diseases. Utilizing its proprietary RAPID platform, Jnana accelerates the identification of therapeutic targets and the development of small molecule therapies. By leveraging advanced chemoproteomics techniques, the company aims to enhance the efficiency of drug discovery and address significant unmet medical needs in patient care.

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.

GentiBio, Inc. is a biotherapeutics company that specializes in developing engineered regulatory T cells, known as EngTregs, aimed at treating autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and headquartered in Boston, Massachusetts, with additional locations in Israel and Seattle, Washington, GentiBio employs a proprietary platform that combines autologous and allogeneic technologies. This platform enhances the ability to restore immune tolerance and addresses significant limitations found in existing regulatory T-cell therapeutics. By focusing on the underlying mechanisms of immune system disorders, GentiBio aims to provide innovative therapeutic solutions that tackle the root causes of diseases caused by dysregulated immune responses. The company is co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of advanced immunotherapy development.

Frontier Medicines Corporation, established in 2018 and headquartered in South San Francisco, California, is a pre-clinical stage biopharmaceutical company specializing in cancer research. The company employs chemoproteomics to discover and develop novel medicines targeting cancer-causing proteins. Frontier Medicines' innovative platform integrates advanced computational methods and machine learning algorithms to identify and pharmacologically target new binding pockets on proteins, thereby enabling small-molecule drug discovery and development for previously inaccessible targets.

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.

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

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.

Synthekine Inc. is a biotechnology company based in Menlo Park, California, specializing in the development of engineered cytokine therapies and immunotherapies aimed at treating cancer and autoimmune disorders. Founded in 2018, Synthekine utilizes advanced platform technologies, including engineered partial agonists and orthogonal cell therapies, to enhance the efficacy of cytokines while minimizing their toxic side effects. The company's innovative Synthekine Platform employs surrogate agonists instead of traditional mutant cytokines to create selective immunotherapies. Its product pipeline features candidates such as STK-009, an orthogonal ligand paired with SYNCAR-001, an orthogonal receptor-modified CAR-T therapy targeting CD-19. Additionally, Synthekine is developing STK-012, partial agonists of IL-2, and other novel immunotherapies that integrate cytokine receptors to initiate new signaling pathways without relying on wild-type cytokines.

NiKang Therapeutics, Inc. is a biotechnology company established in 2017 and headquartered in Wilmington, Delaware. The company specializes in the discovery and development of small molecule oncology medicines aimed at addressing unmet medical needs in cancer treatment. Utilizing a discovery approach informed by target structure biology, NiKang employs structure-based drug design to facilitate the rapid and efficient development of proprietary drug candidates. This methodology enables the company to create innovative therapies with desirable pharmacological properties, ultimately aimed at improving the lives of patients through enhanced treatment options.

Eliem Therapeutics is a clinical-stage biotechnology company focused on developing treatments for nervous system disorders driven by neuronal hyperexcitability. It leverages neuroscience and translational medicine to advance analgesics and other therapies for conditions such as chronic pain, aiming to deliver best-in-class drug candidates that alleviate suffering and improve function. Founded in 2018 and based in Boston, Eliem concentrates on translating scientific insights into clinical solutions to address unmet medical needs in hyperexcitability disorders.

Larimar Therapeutics is a clinical-stage biotechnology company dedicated to developing treatments for complex rare diseases. Its lead compound, CTI-1601, is currently in Phase 1 clinical trials as a potential treatment for Friedreich's ataxia, a rare and progressive genetic disease.

Affinia Therapeutics develops adeno-associated virus (AAV) gene therapies for muscular and central nervous system diseases. The company uses an AAV vector design platform built on synthetic and systems biology, high-throughput screening, and tissue- and single-cell-resolution analyses to create vectors with improved properties. It leverages sequencing, artificial intelligence, and structural modeling to enable rational design of therapies. Headquartered in Waltham, Massachusetts, Affinia Therapeutics was founded in 2019 and targets diseases with significant unmet needs in muscle and CNS.