RA Capital

Boundless Bio, Inc., biopharma company, develops therapies and medicines for the treatment of cancers. It offers therapeutics based on extrachromosomal DNA (ecDNA) one of the drivers of the aggressive cancers, such as cancers characterized by high copy number amplification of oncogenes. The company was formerly known as Pretzel Therapeutics, Inc. Boundless Bio, Inc. was founded in 2018 and is based in LA Jolla, California.

Capstan Therapeutics is advancing precision in vivo cell engineering to develop therapeutics for a broad range of disease categories with unmet or underserved clinical need.

Nimbus Apollo is a program under Nimbus Therapeutics that focuses on the development of innovative treatments for serious diseases, particularly targeting non-alcoholic steatohepatitis (NASH) and hepatocellular carcinoma (HCC). The program's lead candidate, NDI-010976, is an allosteric inhibitor of Acetyl-CoA Carboxylase (ACC), which has received Fast Track designation from the U.S. Food and Drug Administration. Nimbus Apollo is engaged in advancing this candidate through various stages of clinical development, with Phase 1 data expected to be presented at notable medical conferences. Nimbus Therapeutics, founded in 2009 and based in Cambridge, Massachusetts, leverages computational technology to design selective and potent small molecule therapeutics aimed at addressing metabolic diseases, cancer, and immune-inflammatory disorders. The company has established strategic partnerships to enhance its research and development efforts.

MMI S.r.l manufactures and develops micro instruments to support surgical interventions at a dimensional scale. The company develops wristed micro instruments and their fabrication process. The company also develops and offers robotic platform. The company's proprietary robotic platform and wristed micro instruments simplifies reconstruction procedures after traumatic injuries and after tumor removal in breast, head, and bones. The company was founded in 2015 and is headquartered in Calci, Italy.

We are the genome editing company specialized for mitochondrial engineering. We address unmet needs of future food supplies by creating hybrid crop plants to boost their yields to an unprecedented level. The company’s technology creates robust male sterile lines that are ideal over conventional lines because of their stability and elite backgrounds. This allows the production of non-GM hybrid seeds in crop plants that are currently mostly non-hybrid such as wheat. Our technology solution is expected to fulfill the highest level of public acceptance and aspiration.

Cerebral Therapeutics, Inc. is a pharmaceutical company that develops implanted drug-device combination therapies for the treatment of neurologic diseases. The company uses implanted, refillable catheter and pump system to deliver a continuous intracerebroventricular (ICV) dose for the formulation of anti-epileptic drug valproic acid, called CT-010, to treat refractory epilepsy. Cerebral Therapeutics, Inc. was formerly known as Cerebral Therapeutics LLC. The company was incorporated in 2010 and is based in Aurora, Colorado.

DBV Technologies S.A. is a clinical-stage biopharmaceutical company focused on the research and development of epicutaneous immunotherapy products aimed at treating food allergies. The company’s lead candidate, Viaskin Peanut, has completed Phase III clinical trials for peanut allergies in children, adolescents, and adults. Additionally, DBV is developing Viaskin Milk, which is in Phase I/II trials for cow's milk protein allergy and related conditions, and Viaskin Egg, which is in pre-clinical stages for hen’s egg allergy. The company is also working on a booster vaccine for Bordetella pertussis, along with earlier-stage research programs targeting respiratory syncytial virus, Crohn’s disease, celiac disease, and type I diabetes. DBV Technologies collaborates with Nestlé Health Science to create MAG1C, a diagnostic patch test for non-IgE mediated cow's milk protein allergy in infants and toddlers. Founded in 2002, the company is headquartered in Montrouge, France.

Mineralys Therapeutics, Inc., a private, clinical-stage biopharmaceutical company founded by Catalys Pacific committed to developing best-in-class, novel therapy for the treatment of hypertension. The Company is driven to bring a targeted approach to the management of hypertension via the development of MLS-101. MLS-101, licensed from Mitsubishi Tanabe Pharma Corporation, is a highly selective and potent aldosterone synthase inhibitor that is being investigated for the treatment of hypertension.

Founded in April 2006, Concert Pharmaceuticals is a clinical stage biotechnology company dedicated to creating new medicines through a novel scientific approach utilizing the naturally-occurring element deuterium. Concert applies its innovative precision deuterium chemistry platform to modify specific properties of validated drug molecules, yielding a rich pipeline of new chemical entities (NCEs). Concert leverages decades of pharmaceutical experience to create novel drug candidates with potential for best-in-class efficacy and safety, while greatly reducing R&D risk, time and expense.

Celcuity Inc. is a clinical-stage biotechnology company focused on identifying cancer sub-types and therapeutic options for patients in the United States. Utilizing its CELsignia diagnostic platform, the company analyzes a patient’s living tumor cells to determine the specific abnormal cellular processes driving their cancer and the corresponding targeted therapies. Celcuity is developing the CELsignia HSF test to diagnose two new sub-types of HER2-negative breast cancer and is also working on the CELsignia MP test, which aims to identify 11 cancer sub-types across various cancers, including breast, lung, colon, ovarian, kidney, and bladder cancers. Founded in 2011 and headquartered in Minneapolis, Minnesota, Celcuity seeks to enhance treatment options and improve patient care in oncology.

Be Biopharma, Inc. is focused on developing innovative therapeutics derived from engineered B-cells within the immune system. The company aims to address significant limitations associated with existing cell and gene therapies, such as the challenges of administering multiple doses, unpredictable patient reactions, and the necessity of potentially harmful chemotherapy regimens prior to treatment. Be Biopharma's approach allows for the creation of durable therapies that can be re-administered without toxic conditioning, making these treatments suitable for a range of diseases, including cancer, autoimmune disorders, and infectious diseases. Founded in 2020, Be Biopharma is headquartered in Cambridge, Massachusetts.

Ansa Biotechnologies, Inc. is a biotechnology company based in Berkeley, California, focused on developing innovative DNA synthesis technology utilizing enzymes. Established in 2018, the company aims to improve upon traditional chemical methods of DNA synthesis that have remained largely unchanged for decades. Ansa's enzymatic approach offers significant advantages, including increased speed, accuracy, and cleanliness in the production of DNA fragments such as oligonucleotides. This technology enhances the capabilities of biological research, therapeutics, diagnostics, and biomanufacturing by enabling the synthesis of gene-length oligonucleotides. Through its advancements, Ansa Biotechnologies seeks to facilitate innovation in the fields of biological research and engineering, ultimately providing valuable services to medical researchers and contributing to the broader biotechnology landscape.

Incyclix Bio is a biotechnology company based in Durham, North Carolina, focused on the development of small molecule inhibitors targeting cyclin-dependent kinases (CDKs) for the treatment of advanced and resistant cancers. Founded in 2020 by a team including Patrick Roberts, Jay Strum, John Bisi, and Fred Eshelman, the company aims to provide potent and selective inhibition of challenging CDK targets. This approach enables pharmaceutical companies and medical practitioners to access innovative therapeutic options, thereby enhancing treatment opportunities for cancer patients facing difficult cases. Through its research and development efforts, Incyclix Bio seeks to contribute to the advancement of cancer treatment solutions.

Ocelot Bio brings new treatment options to patients with severe liver disease. The company was founded in 2020 and is headquartered in San Diego, California.

Plexium, Inc. is a biotechnology company based in San Diego, California, established in 2017. The company specializes in drug development, focusing on the identification of small molecules that modulate E3 ligases for therapeutic applications in cancer and neurodegenerative diseases. Plexium employs a DNA-encoded library platform to conduct high-throughput screening, utilizing cell-based assays to discover molecular glues that can alter E3 ligase substrate recognition, thereby inducing, inhibiting, or modulating protein degradation. This innovative approach allows Plexium to selectively target various cancers, including small cell lung and breast tumors, while also exploring mechanisms to enhance immune responses through E3 ligase modulation.

Somatus is a healthcare company that partners with health plans, health systems, and nephrology, and primary care groups. It provides integrated care for patients with or at risk of developing kidney disease. Somatus' is vertically integrated clinical services and technology delay or prevent disease progression, improve quality and care coordination, and increase the use of home dialysis modalities and rates of kidney transplantation. The company is headquartered in McLean, Virginia, and founded by a team of world-class healthcare operators, successful entrepreneurs, and leading clinicians treating kidney disease.

Synthego is a genome engineering company that enables the acceleration of life science research and development in the pursuit of improved human health. The company leverages machine learning, automation, and gene editing to build platforms for science at scale. With its foundations in engineering disciplines, the company’s full-stack platform vertically integrates proprietary hardware, software, bioinformatics, chemistries, and molecular biology to advance both basic research and therapeutic development programs. By providing both commercial and academic researchers and therapeutic developers with unprecedented access to cutting-edge genome engineering, Synthego is at the forefront of innovation, accelerating the development of truly engineered biology. The company was founded in 2012 and is based in Redwood City, California.

Electra Therapeutics is a clinical-stage biotechnology company focused on developing therapies that target signal regulatory proteins (SIRP) for treating immunological diseases and cancer. The company has one program in clinical development and two additional preclinical programs. Its lead product candidate, ELA026, is a monoclonal antibody designed to target SIRP on the surface of myeloid and T cells, effectively depleting pathological immune cells. ELA026 is currently being investigated for secondary hemophagocytic lymphohistiocytosis (sHLH), a severe hyperinflammatory condition for which no approved treatment exists.

Indapta Therapeutics is a privately held biotechnology company focused on developing a natural killer (NK) cell therapy platform aimed at treating both blood and solid tumor cancers. The company specializes in a proprietary off-the-shelf, non-engineered, and allogeneic G-NK cell designed to enhance the immune response and significantly improve the effectiveness of antibody therapies. Through its innovative approach, Indapta Therapeutics seeks to provide healthcare providers with advanced treatment options for patients suffering from cancer, leveraging the potent antibody-dependent cellular cytotoxicity (ADCC) activity of its NK cell therapy.

Ventus Therapeutics U.S., Inc. is a biopharmaceutical company based in Natick, Massachusetts, with an additional location in Montreal, Canada. Founded in 2019, it focuses on discovering and developing novel small-molecule medicines that target the innate immune system to address autoimmune diseases, inflammatory diseases, and cancer. The company employs a structural immunology platform that integrates protein engineering to elucidate molecular structures and mechanisms of action. This platform facilitates the development of precise binding and functional screening assays, as well as structure-based modeling. Ventus Therapeutics is advancing an emerging pipeline of drug programs aimed at key targets within the innate immune system, leveraging its proprietary capabilities in rational and structure-based drug design.

Metagenomi, Inc. is a genetic medicines company based in Emeryville, California, focused on developing innovative gene editing systems for the treatment of genetic diseases. Since its incorporation in 2016, the company has utilized a proprietary metagenomics-derived genome editing toolbox that enables the creation of curative therapeutics. This toolbox encompasses a range of advanced technologies, including programmable nucleases, base editors, and various RNA and DNA-mediated integration systems, such as prime editing systems and CRISPR-associated transposases (CASTs). Metagenomi aims to address the challenges posed by diverse mutations that have traditionally been difficult to target with existing genome engineering approaches.

Third Harmonic Bio is a clinical-stage biopharmaceutical company dedicated to developing innovative treatments for severe allergies and inflammation. The company is focused on creating first-in-class oral small-molecule inhibitors targeting the KIT receptor, which plays a crucial role in the survival and function of mast cells—key immune cells involved in allergic responses. Its lead product candidate, THB001, is designed to provide selective inhibition of KIT, while another candidate, THB335, aims to address multiple mast cell-driven inflammatory diseases affecting the skin, respiratory system, and gastrointestinal tract. Through its research and development efforts, Third Harmonic Bio seeks to advance the management of inflammatory diseases, enhancing patient outcomes and quality of life.

AN2 Therapeutics, Inc., a biopharmaceutical company, engages in the research, development, and commercialization of novel medicines targeting infectious diseases. The company focuses on developing a clinical-stage antibacterial compound. It has a strategic partnership with Brii Biosciences. The company was incorporated in 2017 and is based in Menlo Park, California.

AmbAgon Therapeutics is a developer of cancer molecule therapeutics. The company offers research of a small molecule that augments a tumor-suppressing protein. AmbAgon Therapeutics was founded by Christian Ottmann, Lucas Brunsveld, and Michelle Arkin.

Pardes Biosciences is a biotechnology company that develops and commercializes direct-acting antivirals for the treatment of coronavirus. The Carlsbad, California-based company was established by Uri Lopatin in 2020.

Nodexus is a Venture- and SBIR-backed biotech startup commercializing the NX One platform to address widespread unmet needs for accessible live single cell isolation in industrial/biopharma, clinical research, and academic sectors. The NX One platform consists of a low-infrastructure, low-cost hardware system and single-use disposable microfluidic cartridges tailored toward live single-cell workflows involving gene editing (e.g. CRISPR engineering), cell line/antibody development, and tumor heterogeneity studies.

Emergence Therapeutics AG develops novel antibody drug conjugate (ADC) immuno-therapeutics to treat high need cancers. The company was incorporated in 2019 and is based in Duisburg, Germany.

Freenome, Inc., is an artificial intelligence (AI) genomics biotech company. The company develops and provides a medical arti?cial intelligence platform for early detection of cancer. It develops blood tests for early-cancer screening and treatment selection. The company also provides clinical research services. Freenome, Inc. was founded in 2014 and is based in South San Francisco, California.

NanoMosaic specializes in innovative solutions for early illness detection, prognostic monitoring, and biomarker identification. The company has developed a unique platform that utilizes nanoneedles for protein detection and quantification. This technology facilitates the analysis of multiple biomarkers on a single chip, making it possible for the medical and healthcare sectors to enhance the speed and accuracy of biomarker discovery and validation. By enabling more efficient proteomic analysis, NanoMosaic aims to improve early disease detection and monitoring, ultimately contributing to better health outcomes.

Curie Therapeutics is a fully integrated, next-generation radiopharmaceuticals company focused on pioneering a new class of targeted radiopharmaceuticals that are both safe and effective. Curie's radiopharmaceuticals are composed of rationally designed conjugates against carefully selected biological targets, to deliver alpha and beta-emitting radionuclide payloads.

Avilar Therapeutics is a biopharmaceutical company focusing on the discovery and development of extracellular protein degraders, a new frontier in targeted protein degradation.

Antios Therapeutics, Inc. is a biopharmaceutical company based in Atlanta, Georgia, founded in 2018. The company focuses on developing innovative therapies for viral diseases, particularly through its oral drug candidate, ATI-2173, aimed at treating patients infected with Hepatitis B and Hepatitis D viruses. With a leadership team that possesses extensive experience in the field, Antios Therapeutics is dedicated to advancing its research and development efforts toward potentially curative treatments for Hepatitis B virus infections.

Cedilla is a developer of therapeutics technologies intended to broaden the reach of small molecule therapeutics. The company's technology is focused on destabilizing oncoprotein targets that are also applicable to therapies outside of oncology, enabling doctors to treat diseases caused by protein dysregulation. The company was founded in 2018 and based in Cambridge, Massachusetts.

Expansion Therapeutics is a drug discovery and development company pursuing the vast potential of small molecule medicines for RNA-mediated diseases.

Obsidian Therapeutics is a developer of a next-generation cell and gene therapies created to extend adoptive immunotherapy to every patient with cancer.The company's next-generation cell and gene therapies with pharmacologic operating systems provide exquisite control of protein activity in cells and create adoptive cell therapies with new functions that are under control of the treating physician using simple, safe, orally-active, marketed drugs, enabling patients to improve on the current generation of cell therapies.

Qvella Corporation is a Toronto-based molecular diagnostics company established in 2009 by a team of scientists and engineers. The company focuses on advancing sample preparation and detection technologies in clinical microbiology, aiming to significantly reduce the time required to obtain results. Qvella's innovative Field Activated Sample Treatment (FAST™) technology employs a unique electrical lysing method known as e-lysis™, allowing for the direct and fully automated rapid detection of infectious agents from unenriched biological samples. By providing actionable results in clinically relevant time frames, Qvella seeks to improve patient outcomes and transform medical practices in the field of microbiology.

Asher Biotherapeutics, Inc. is a biotechnology company focused on developing immunotherapy drugs for cancer treatment. Founded in 2019 and headquartered in South San Francisco, California, the company aims to create innovative therapeutic solutions to improve patient outcomes in oncology. Asher Bio leverages advanced technologies and research to enhance the effectiveness of immune-based therapies, positioning itself as a key player in the evolving field of cancer treatment.

ARS Pharmaceuticals is a stealth pharmaceutical company that developed a novel intranasal epinephrine spray with a unique absorption technology that could be easy-to-use, convenient, and more reliable for patients and loved ones at risk of severe allergic reactions to food, medications and insect bites that could lead to life-threatening anaphylaxis.

DiCE Therapeutics, founded in 2013 and based in Menlo Park, California, specializes in developing a platform for the discovery of novel small molecules aimed at targets that have previously been difficult to address in the pharmaceutical sector. The company utilizes innovative technology that extends directed evolution, allowing for the direct translation of DNA-encoded information into organic compounds, thereby streamlining the traditionally labor-intensive processes of medicinal chemistry. This platform not only serves the pharmaceutical industry but also has applications in agriculture and materials sciences. The founding team includes notable figures such as Stanford Professor Pehr Harbury, who developed the core technology, and other experienced professionals from various sectors, contributing to the company's mission of transforming drug discovery and development.

Jnana Therapeutics Inc. is a Boston-based company founded in 2016 that operates a drug discovery platform centered on solute carrier (SLC) transporters. The company focuses on key disease pathways related to immunometabolism, lysosomal function, and mucosal defense, targeting areas such as immuno-oncology, inflammatory disorders, and neurological diseases. By leveraging its innovative approach, Jnana Therapeutics aims to discover and develop transformative medicines that address significant medical needs and improve patient outcomes.

GentiBio, Inc. is a biotherapeutics company focused on developing engineered regulatory T cells (EngTregs) to treat a range of conditions, including autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and based in Boston, Massachusetts, with additional locations in Seattle and Israel, GentiBio utilizes a proprietary platform that combines autologous and allogeneic EngTregs. This innovative approach aims to restore immune tolerance and address the limitations found in current regulatory T-cell therapies. The company was co-founded by experts in Treg biology and synthetic immunology, emphasizing its commitment to addressing the underlying causes of diseases linked to immune system dysfunction.

Tango Therapeutics, Inc., a biotechnology company, develops cancer drugs based on synthetic lethality. The company uses the genetic principle of synthetic lethality to discover and develop therapies that take aim at driver genes in cancer. It focuses counteracting tumor suppressor gene loss; reversing the ability of cancer cells to evade the immune system; and identifying novel combinations that are more effective than single agent therapy. Tango Therapeutics, Inc. was formerly known as Cancer Combos NewCo, Inc. The company was incorporated in 2014 and is based in Cambridge, Massachusetts.

Sydnexis is engaged in the development of a proprietary treatment for pediatric progressive myopia, a major global unmet medical need. Sydnexis manufactures pharmaceutical product for ophthalmic disorder or condition.

PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the delivery of nucleic acid therapeutics, particularly antisense oligonucleotides. Established in 2018, PepGen aims to enhance the clinical efficacy of these therapeutics through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative technology employs engineered peptides designed for optimized tissue penetration, cellular uptake, and nuclear delivery, enabling effective transport of oligonucleotides to various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. By leveraging its drug delivery capabilities, PepGen seeks to transform the treatment landscape for severe neuromuscular and neurological diseases.

T-knife is a developer of T cell receptors intended for T cell therapy of cancer.The company uses a humanized TCR mice platform to carry human TCRαβ gene loci and recombine a broad repertoire of human TCRs, enabling the efficient generation and virtual selection of cell receptors for any human tumor antigen.

Exo is a medical device startup handheld ultrasound platform and AI for imaging and therapeutic applications. The company is committed to delivering affordable and easy-to-use medical imaging to healthcare professionals around the world. By delivering easy-to-use, high-quality medical imaging. Exo empowers healthcare professionals to make critical, real-time decisions that improve patient outcomes. The Exo ultrasound platform combines advances in nano-materials, novel sensor technologies, advanced signal processing, and computation with the economies of scale of semiconductor manufacturing to dramatically reduce the cost of imaging. It also represents a giant leap in imaging and therapeutics technology one that will drive the development of high-performance products that are accessible, easy-to-use, and available at a price point never before possible.

Frontier Medicines Corporation is a pre-clinical stage biopharmaceutical company based in South San Francisco, California, founded in 2018. The company specializes in discovering and developing medicines that target cancer-causing proteins through its innovative chemoproteomics platform. This platform utilizes advanced computational techniques and machine learning to identify new binding pockets on proteins, thereby facilitating the discovery and development of small-molecule drugs. By enhancing the drug development process, Frontier Medicines aims to deliver breakthrough therapies that can significantly change the treatment landscape for debilitating diseases.

Nimbus Apollo is a program under Nimbus Therapeutics that focuses on the development of innovative treatments for serious diseases, particularly targeting non-alcoholic steatohepatitis (NASH) and hepatocellular carcinoma (HCC). The program's lead candidate, NDI-010976, is an allosteric inhibitor of Acetyl-CoA Carboxylase (ACC), which has received Fast Track designation from the U.S. Food and Drug Administration. Nimbus Apollo is engaged in advancing this candidate through various stages of clinical development, with Phase 1 data expected to be presented at notable medical conferences. Nimbus Therapeutics, founded in 2009 and based in Cambridge, Massachusetts, leverages computational technology to design selective and potent small molecule therapeutics aimed at addressing metabolic diseases, cancer, and immune-inflammatory disorders. The company has established strategic partnerships to enhance its research and development efforts.

POINT Biopharma Global Inc. is a globally focused radiopharmaceutical company building a platform for the clinical development and commercialization of radioligands that fight cancer. POINT Biopharma Global Inc. is combining a portfolio of best-in-class radiopharmaceutical assets, a seasoned management team, expertise in radioisotopes such as Actinium-225 (Ac-225) and Lutetium-177 (Lu-177), manufacturing technology and novel direct-to-patient targeting to revolutionize theragnostic drug development and radioligand commercialization.

Element Biosciences, Inc. develops genetic analysis tools for the research and diagnostic markets. The company provides a disruptive DNA sequencing technology and a sequencing platform, such as surface chemistry, sequencing chemistry, detection, and data analysis. The company was incorporated in 2017 and is based in San Diego, California.

Adela is focused on the detection of cancer and other high-morbidity, high-mortality conditions through a routine blood test.

Presbyopia Therapies LLC moves forward with development of its Liquid Vision drops, a temporary presbyopia-correcting therapeutic designed to last five hours or longer.

Synthekine Inc. is a biotechnology company focused on developing innovative cytokine therapies and immunotherapies for cancer and autoimmune disorders. Founded in 2018 and based in Menlo Park, California, the company leverages advanced platform technologies, including engineered partial agonists and orthogonal cell therapies, to create targeted treatments. Synthekine's notable innovations include the Synthekine Platform, which utilizes surrogate agonists instead of traditional mutant cytokines. The company's product pipeline features STK-009, an orthogonal ligand paired with SYNCAR-001, an engineered CAR-T therapy targeting CD-19, as well as STK-012, a partial agonist of IL-2. Synthekine's approach emphasizes safety and efficacy, utilizing immunological insights to guide the development of therapeutics that selectively modulate immune system pathways. Preclinical studies have indicated promising efficacy and tolerability for its lead programs, positioning Synthekine as a key player in the biotechnology landscape.

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

NiKang Therapeutics, Inc. is a biotechnology company established in 2017 and located in Wilmington, Delaware. The company is dedicated to discovering and developing innovative small molecule oncology medicines aimed at addressing unmet medical needs in cancer treatment. By employing a discovery approach that leverages target structure biology and structure-based drug design, NiKang Therapeutics aims to facilitate the rapid and efficient development of proprietary drug candidates with optimal pharmacological properties. The company seeks to enhance the treatment options available to healthcare providers, ultimately improving patient outcomes in the fight against cancer.

Eliem Therapeutics, Inc. develops therapeutic drugs for the treatment of hyperexcitability disorders. The company focuses on the clinical development of analgesics for the treatment of hyperexcitability disorders, such as chronic pain using neuroscience and translational medicine. The company was incorporated in 2018 and is based in Boston, Massachusetts.

Larimar Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. The company’s lead compound, CTI-1601, is currently being evaluated in a Phase 1 clinical program as a potential treatment for Friedreich’s ataxia, a rare and progressive genetic disease. Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds.

Interius BioTherapeutics is a biopharmaceutical company that targets cancer. The company spun out the University of Pennsylvania and is targeting cancer.

Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for various diseases, particularly those affecting muscle and the central nervous system (CNS). Founded in 2019 and based in Waltham, Massachusetts, the company specializes in creating rationally designed adeno-associated virus (AAV) vectors. By integrating synthetic and systems biology with high-throughput screening and advanced resolution techniques, Affinia Therapeutics aims to address significant unmet medical needs. The company’s innovative approach allows for the development of novel gene therapies with enhanced properties, targeting the treatment of devastating diseases and improving the lives of affected individuals.

Forge Biologics is a gene therapy development engine founded in 2020 and headquartered in Grove City, Ohio. The company specializes in the development and manufacturing of gene therapies, focusing on transforming innovative concepts into accessible treatments for rare genetic diseases. With end-to-end capabilities, Forge Biologics helps accelerate gene therapy programs from preclinical stages through to clinical and commercial-scale production. The company collaborates with various stakeholders in the gene therapy community, including scientists, healthcare professionals, biotech and pharmaceutical companies, and patient advocacy groups. Among its notable initiatives is a unique approach combining adeno-associated virus (AAV) vectors and umbilical cord transplants to treat infantile Krabbe disease, a severe neurodegenerative disorder. Through its comprehensive services, Forge Biologics aims to provide transformative medicines that improve patient outcomes.

CeriBell, Inc. designs and manufactures medical devices for acquisition and interpreting electroencephalography (EEG) data in patients with various neurological conditions. It offers Ceribell EEG system that includes Ceribell EEG Headband that allows healthcare providers to accommodate various hair types and head sizes; and Ceribell EEG Recorder, a pocket-sized, battery operated recorder that provides clinical quality EEG and on-device EEG display. It serves medical staff in hospital ICUs, inpatient units, and emergency departments. CeriBell, Inc. was formerly known as Brain Stethoscope, Inc. The company was incorporated in 2014 and is based in Mountain View, California.

Boundless Bio, Inc., biopharma company, develops therapies and medicines for the treatment of cancers. It offers therapeutics based on extrachromosomal DNA (ecDNA) one of the drivers of the aggressive cancers, such as cancers characterized by high copy number amplification of oncogenes. The company was formerly known as Pretzel Therapeutics, Inc. Boundless Bio, Inc. was founded in 2018 and is based in LA Jolla, California.

ViaCyte, Inc. is a regenerative medicine company based in San Diego, California, focused on developing innovative cell replacement therapies for diabetes. The company is working on several product candidates, including VC-01, designed for type 1 diabetes, and VC-02, which facilitates blood vessel interaction with implanted pancreatic precursor cells. Their VCTX210 product utilizes a proprietary human stem cell line that helps prevent immune system rejection, eliminating the need for immunosuppressants. ViaCyte's therapies differentiate stem cells into pancreatic beta-cell precursors that are implanted in an encapsulation device, enabling the production of insulin in response to blood glucose levels. The company's aim is to provide long-term solutions for both type 1 and type 2 diabetes patients, reducing their dependence on insulin and minimizing associated health complications. Founded in 1999 and formerly known as Novocell, Inc., ViaCyte also has operations in Athens, Georgia.

Janux Therapeutics develops immunotherapies that generate immune responses to prevent tumors and not affecting a patient’s healthy tissue. Janux technology targets all three stages of the anti-tumor immune response. Janux Therapeutics, Inc. was founded in 2017 by David Campbell and is based in La Jolla, California.

Invivyd is a biotechnology company focused on developing advanced antibodies to neutralize SARS-CoV-2, SARS-CoV-1, and other potentially emerging coronaviruses. Founded in 2020 and headquartered in Waltham, Massachusetts, the company aims to create best-in-class therapeutic and prophylactic treatments that surpass the efficacy of existing antibody programs. Its innovative portfolio features multiple non-competing antibodies, each targeting distinct viral epitopes, which is designed to minimize the risk of viral escape. By leveraging cutting-edge antibody discovery technologies, Invivyd seeks to enhance the potency and durability of its antibody candidates, addressing a critical need in the ongoing fight against coronaviruses.

Antios Therapeutics, Inc. is a biopharmaceutical company based in Atlanta, Georgia, founded in 2018. The company focuses on developing innovative therapies for viral diseases, particularly through its oral drug candidate, ATI-2173, aimed at treating patients infected with Hepatitis B and Hepatitis D viruses. With a leadership team that possesses extensive experience in the field, Antios Therapeutics is dedicated to advancing its research and development efforts toward potentially curative treatments for Hepatitis B virus infections.

Ventus Therapeutics U.S., Inc. is a biopharmaceutical company based in Natick, Massachusetts, with an additional location in Montreal, Canada. Founded in 2019, it focuses on discovering and developing novel small-molecule medicines that target the innate immune system to address autoimmune diseases, inflammatory diseases, and cancer. The company employs a structural immunology platform that integrates protein engineering to elucidate molecular structures and mechanisms of action. This platform facilitates the development of precise binding and functional screening assays, as well as structure-based modeling. Ventus Therapeutics is advancing an emerging pipeline of drug programs aimed at key targets within the innate immune system, leveraging its proprietary capabilities in rational and structure-based drug design.

Icosavax is focused on developing safe and effective vaccines against infectious diseases that address important unmet medical needs and reduce healthcare costs. tHEY were founded on breakthrough computationally-designed virus-like particle technology, exclusively licensed for a variety of infectious disease indications from the Institute for Protein Design at the University of Washington.

Tyra Biosciences is a clinical-stage biotechnology company based in Carlsbad, California, focused on developing small molecule therapies for cancer treatment. Founded in 2018, the company aims to address drug resistance in oncology by targeting specific pathways associated with acquired resistance. Tyra Biosciences employs its proprietary precision medicine platform, SNÅP, to facilitate rapid drug design through molecular snapshots, enabling the prediction of genetic alterations that may lead to resistance against existing therapies. The company's lead product candidate, TYRA 300, is an FGFR3 selective inhibitor, primarily aimed at treating patients with metastatic urothelial carcinoma of the bladder and urinary tract. Through innovative approaches, Tyra Biosciences seeks to create next-generation precision medicines that significantly improve treatment outcomes for cancer patients.

New Equilibrium Biosciences provides a platform that reveals opportunities for structure-based design by learning biophysics with AI.

Scribe Therapeutics Inc. engages in the engineering, delivery, and development of in vivo therapies and CRISPR molecules to rewrite and repair the underlying cause of genetic disorders. Its technology includes X-Editing (XE), an engineered molecule for therapeutic use and in vivo genetic modification. The company was incorporated in 2017 and is based in Berkeley, California.

Pyxis Oncology, Inc. is a biotechnology company focused on developing antibody therapeutics that enhance the immune response against cancer. Founded in 2018 and based in Boston, Massachusetts, the company specializes in analyzing tumor antigen-specific tumor-infiltrating lymphocytes within hot tumors and tumor cell signaling pathways in cold tumors. This research informs the creation of novel antibody-based immunotherapies aimed at targeting difficult-to-treat cancers. Pyxis Oncology's goal is to develop next-generation therapeutics that not only kill tumor cells but also address the underlying issues that facilitate cancer's proliferation and immune evasion, ultimately improving the quality of life for patients.

Eliem Therapeutics, Inc. develops therapeutic drugs for the treatment of hyperexcitability disorders. The company focuses on the clinical development of analgesics for the treatment of hyperexcitability disorders, such as chronic pain using neuroscience and translational medicine. The company was incorporated in 2018 and is based in Boston, Massachusetts.

At Graphite Bio, they are rapidly building a next-generation gene editing company that is driven to apply their breakthrough technology with urgency to advance gene replacement therapies and cures for people living with serious diseases. They stand apart through our ability to harness natural and highly precise cellular DNA repair machinery to achieve high efficiency, targeted gene integration to correct the underlying causes of diseases.

Century Therapeutics, Inc. is a biotechnology company focused on developing induced pluripotent stem cell (iPSC)-based therapies for cancer treatment. Founded in 2018 and headquartered in Philadelphia, Pennsylvania, the company aims to create allogeneic living drugs that specifically target both hematologic and solid tumors. By utilizing iPSCs, Century Therapeutics can engineer immune effector cells through multiple rounds of cellular modification, producing master cell banks that can be expanded and differentiated for therapeutic use. Their innovative approach incorporates advanced technologies such as CRISPR-mediated gene editing and proprietary chimeric antigen receptors, along with Allo-Evasion technology, which helps prevent rejection by the host immune system. These advancements position Century Therapeutics to enhance the effectiveness and accessibility of cancer treatments.

Janux Therapeutics develops immunotherapies that generate immune responses to prevent tumors and not affecting a patient’s healthy tissue. Janux technology targets all three stages of the anti-tumor immune response. Janux Therapeutics, Inc. was founded in 2017 by David Campbell and is based in La Jolla, California.

DTx Pharma, LLC is a biotechnology company focused on developing and commercializing fatty acid-conjugated peptide therapeutics aimed at treating patients with rare and chronic diseases globally. The company utilizes innovative technology to enhance the delivery of RNA medicines to various tissues and organ systems, effectively addressing limitations faced by earlier platforms, such as poor pharmacokinetics and inadequate cellular uptake. DTx Pharma's therapies target conditions including retinitis pigmentosa, Duchenne muscular dystrophy, and central nervous system disorders. Founded in 2017, the company is headquartered in San Diego, California.

Tenaya Therapeutics, Inc. develops therapeutics for heart failure. It offers cellular regeneration platform, that enables in vivo reprogramming of cardiac fibroblasts into cardio myocytes by delivering proprietary transcription factors; gene therapy platform, that enables the targeted delivery of a payload to cardiac fibroblasts ; and precision medicine platform, which provides personalized medicine approach to treat heart disease. The company was founded in 2016 and is headquartered in South San Francisco, California.

Satsuma Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative treatments for migraines. The company's primary product candidate, STS101, is a drug-device combination featuring a proprietary dry-powder formulation of dihydroergotamine mesylate. This medication is designed for self-administration using a pre-filled, single-use nasal delivery device. Currently, STS101 is undergoing Phase III clinical trials, marking a significant step in its development process. Founded in 2016 and based in South San Francisco, California, Satsuma Pharmaceuticals aims to provide effective solutions for individuals suffering from acute migraine attacks.

Artiva Biotherapeutics, Inc., a biotech company, develops and manufactures cellular immunotherapies for cancer patients. The company offers a pipeline of off-the-shelf, allogeneic natural killer (NK) cell therapies for the treatment of hematologic cancers or solid tumors. The company’s products target CD20 and CD19 in B-cell lymphomas and HER2 in various solid tumors. The company was founded in 2019 and is headquartered in San Diego, California. Artiva Biotherapeutics, Inc. operates as a subsidiary of Green Cross Holdings Corporation.

Vividion Therapeutics, Inc., a biotechnology company, develops therapeutics to treat major unmet medical needs using the platform for proteome-wide small molecule drug discovery. The company combines a proteome-wide small molecule screening approach and synthetic chemistry techniques. It provides small molecule selectivity and creates a path to drugging any protein. The company was incorporated in 2013 and is based in San Diego, California.

Xilio Therapeutics is a biotechnology company advancing next-generation cancer immunotherapies designed to improve patient outcomes by unleashing the power of the immune system selectively at the site of the tumor. The company’s tumor-selective immunotherapies are based on its proprietary technology, which maximizes the potency of proven immuno-oncology therapies and restricts their activity to the tumor to minimize peripheral side effects. The broad applicability of these therapies across cancer types means that all patients could benefit from these potentially curative medicines. These tumor-selective therapies are designed to overcome the significant toxicities associated with validated IO therapies, such as IL-2 and aCTLA4, which have historically limited the number of patients that can be treated and prevented patients from completing full courses of treatment Xilio Therapeutics proprietary technology enables the explosion of therapeutic activity specifically within the tumor while reducing toxicity throughout the body a nod to the company name, Xilio, derived from the Latin term, Ex Nihilo, meaning creation or big-bang. The company applying its technology to build a broad pipeline of engineered antibodies, cytokines and chemokines as potential new options for people living with cancer. Xilio Therapeutics was founded in 2016 and is headquartered in Waltham, Massachusetts, United States.

Cyteir is developing the next generation of synthetic lethal therapies to treat cancer.Cyteir Therapeutics is creating breakthrough solutions to the major challenges facing cancer researchers today. Currently in development, is the answer to the oncology triumvirate: reducing side effects, reducing therapy resistance, inducing diseased cell self destruction.

Day One Biopharmaceuticals is a biotechnology company focused on developing new cancer therapies for patients of all ages. The company understands that advances in cancer research now enable drug developers to identify children and adults who may benefit from the same medicine. They develop new therapies that meet the critical needs of people of all ages living with cancer starting from the biology of childhood cancer. Day One Biopharmaceuticals also focuses its efforts to bring the best medicines to families receiving the life-altering news of a new cancer diagnosis and the need for a treatment plan. It prioritizes programs that can be rapidly brought to market for both younger and older patients. The company's goal is to license and acquire the most promising products from research institutions as well as for biotechnology and pharmaceutical companies. Day One Biopharmaceuticals was founded in 2018 and is headquartered in South San Francisco, CA, USA.

Orchard Therapeutics plc, a biopharmaceutical company, develops gene therapies for serious and life-threatening rare diseases in the United Kingdom, European Union, and the United States. The company’s gene therapy approach seeks to transform a patient’s hematopoietic stem cells into a gene-modified drug product to treat the patient’s disease through a single administration. It provides Strimvelis, a gammaretroviral-based product for the treatment of adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company’s clinical development products comprise OTL-101 for the treatment of ADA-SCID; OTL-200 to treat metachromatic leukodystrophy; OTL-103 for the treatment of Wiskott-Aldrich syndrome; OTL-102 for X-linked chronic granulomatous disease; and OTL-300 for transfusion-dependent beta-thalassemia. Its preclinical programs include OTL-203 for mucopolysaccharidosis type I, OTL-201 for mucopolysaccharidosis type MPS-IIIA, and OTL-202 for mucopolysaccharidosis type IIIB. The company was formerly known as Orchard Rx Limited. Orchard Therapeutics plc was founded in 2015 and is headquartered in London, the United Kingdom.

Design Therapeutics, Inc. develops therapies for the treatment of degenerative disorders caused by nucleotide repeat expansions. The company engages in the development of a program for the treatment of Friedreich’s ataxia and degenerative diseases such as Fragile X syndrome and myotonic dystrophy. The company was incorporated in 2017 and is based in Solana Beach, California.

TScan Therapeutics is a biopharmaceutical company dedicated to developing innovative T cell therapies to treat various cancers, including liquid tumors and solid tumors. The company focuses on engineering T cell receptor (TCR) therapies, with specific candidates aimed at addressing hematologic malignancies and preventing relapse after hematopoietic stem cell transplantation. TScan utilizes a specialized platform that identifies shared T cell antigens and minimizes off-target effects, enhancing the safety and effectiveness of its treatments. The company’s pipeline includes both liquid tumor therapies and multiplexed TCR-T candidates for solid tumors. Founded in 2018, TScan Therapeutics is headquartered in Waltham, Massachusetts, and comprises a team of experts committed to expanding treatment options for patients with limited therapeutic alternatives.

Verve Therapeutics, Inc. is a biotechnology company focused on developing innovative therapies aimed at editing the adult human genome to reduce the risk of coronary artery disease, a leading cause of death globally. Founded in 2018 and headquartered in Cambridge, Massachusetts, with a research facility in Philadelphia, the company leverages human genetic analysis and gene-editing technologies to create treatments that offer lifelong protection from this condition. Verve has established strategic alliances, including collaborations with Beam Therapeutics to advance delivery technologies targeting cardiovascular issues and with Verily for developing gene editing delivery vehicles. Formerly known as Endcadia, Verve Therapeutics rebranded in September 2019 to reflect its commitment to addressing heart disease through advanced genetic solutions.

DiCE Therapeutics, founded in 2013 and based in Menlo Park, California, specializes in developing a platform for the discovery of novel small molecules aimed at targets that have previously been difficult to address in the pharmaceutical sector. The company utilizes innovative technology that extends directed evolution, allowing for the direct translation of DNA-encoded information into organic compounds, thereby streamlining the traditionally labor-intensive processes of medicinal chemistry. This platform not only serves the pharmaceutical industry but also has applications in agriculture and materials sciences. The founding team includes notable figures such as Stanford Professor Pehr Harbury, who developed the core technology, and other experienced professionals from various sectors, contributing to the company's mission of transforming drug discovery and development.

Werewolf Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative receptor agonists for cancer immunotherapy. Established in 2017, the company is advancing a pipeline of transformative biotherapeutics aimed at treating cancer. Werewolf Therapeutics creates medicines intended for systemic delivery that remain inactive within the body until they arrive at the tumor microenvironment. At this point, the treatments activate to stimulate a robust immune response, thereby targeting and attacking cancer cells effectively.

Abcuro’s mission is to develop a new class of immune-modulatory bio-therapeutics for treating both autoimmunity and cancer. They leverage the systematic target validation and clinical insights gained by its founders to identify new targets. Their intensive use of bioinformatics to interrogate the transcriptome of human disease together with ex-vivo validation in diseased tissue has uncovered new approaches to target key compartments of the immune system. The company was incorporated in 2015 and is based in Newton, Massachusetts.

Vivace Therapeutics is a venture-backed start-up dedicated to discovering and developing cancer therapeutics by targeting a novel pathway. At Vivace Therapeutics, they pursue their work with passion, driven by a conviction to help people fight cancer and live life to the fullest. Based in the San Francisco Bay Area, Vivace Therapeutics is an oncology-focused portfolio-based drug discovery and development company adopting a capital-efficient and modality-indifferent approach to bring novel therapies to patients in need. Their experienced management team and world-class scientists work to advance promising drugs and therapeutic technologies that they believe can help conquer cancer.

Locanabio, Inc., is an RNA-targeting gene therapy company focused on developing therapies for patients with severe neurodegenerative, neuromuscular, and retinal diseases.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic disorder primarily affecting boys. The company's lead candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in muscle tissues. In addition to SGT-001, Solid Biosciences is advancing Anti-LTBP4, a monoclonal antibody designed to mitigate fibrosis and inflammation by targeting the LTBP4 protein. The company also works on developing biomarkers and wearable assistive devices under the Solid Suit program, which aims to provide therapeutic benefits to patients. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to further develop and commercialize gene therapies for DMD. The company’s initiatives address a critical need, as there are currently no effective cures or satisfactory treatments for this progressive and life-threatening condition.

PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the delivery of nucleic acid therapeutics, particularly antisense oligonucleotides. Established in 2018, PepGen aims to enhance the clinical efficacy of these therapeutics through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative technology employs engineered peptides designed for optimized tissue penetration, cellular uptake, and nuclear delivery, enabling effective transport of oligonucleotides to various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. By leveraging its drug delivery capabilities, PepGen seeks to transform the treatment landscape for severe neuromuscular and neurological diseases.

Edgewise Therapeutics is a clinical-stage biopharmaceutical company based in Boulder, Colorado, specializing in the development of small molecule therapies for severe musculoskeletal diseases. Founded in 2017, the company leverages its expertise in skeletal muscle physiology to create innovative treatments for genetically defined muscle disorders, including Duchenne, Becker, and limb girdle muscular dystrophies. Edgewise Therapeutics employs a precision medicine platform to identify and target key muscle proteins and modulators, aiming to address the underlying causes of these conditions. Additionally, the company has established a state-of-the-art research facility to enhance its capabilities in understanding muscle adaptation and injury related to disease.