RA Capital Management

Vivace Therapeutics, Inc. is a drug discovery and development company focused on creating cancer therapies that target the Hippo pathway, which plays a crucial role in tissue regeneration and organ development. Established in 2014 and located in the San Francisco Bay Area, Vivace Therapeutics employs an innovative approach to develop small molecule treatments aimed at improving patient outcomes in oncology. The company collaborates with leading academic institutions to advance research and develop therapies that can be used alone or in combination with existing treatments. With a commitment to enhancing cancer care, Vivace Therapeutics is dedicated to delivering novel solutions that can help patients combat cancer more effectively.

IDRx is a clinical-stage biopharmaceutical company focused on developing precision therapies for oncology, specifically targeting gastrointestinal stromal tumors. The company specializes in designing combination therapies that utilize engineered drug combinations to address key tumor escape mechanisms, aiming to enhance treatment efficacy and prolong patient responses. By creating these targeted therapies, IDRx seeks to revolutionize cancer treatment and support healthcare professionals in delivering more effective medicines to patients.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on precision oncology, developing therapeutics that target challenging cancer types without straightforward single-gene mutations. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond to specific treatments. Acrivon's pipeline includes ACR-368, also known as prexasertib, which is currently in Phase 2 trials, alongside preclinical programs aimed at key components of DNA damage response and cell cycle regulation, such as the WEE1 and PKMYT1 proteins. Acrivon seeks to expedite the development of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials, aiming to enhance treatment success in oncology.

Nkarta Therapeutics is a biopharmaceutical company focused on developing engineered natural killer (NK) cells for the treatment of cancer and autoimmune diseases. The company aims to enhance the efficacy of cell therapy by leveraging the inherent ability of NK cells to identify and eliminate abnormal cells without the need for genetic alteration. This approach allows for a broader application across various hematologic and solid tumor malignancies while potentially minimizing side effects associated with traditional T-cell therapies. Nkarta combines its NK expansion platform technology with proprietary cell engineering methods to produce a substantial supply of NK cells, enhancing their ability to recognize therapeutic targets and improving their persistence in the body for sustained activity. Through this innovative strategy, Nkarta seeks to provide more potent, well-tolerated, and rapidly available therapies for patients facing diverse cancer types.

LENZ Therapeutics is a late-stage biopharmaceutical company dedicated to the development and commercialization of innovative ophthalmic therapies aimed at improving vision. The company's primary focus is on addressing presbyopia, a common condition that leads to the gradual loss of near vision, affecting nearly all individuals over the age of 45. LENZ Therapeutics is currently advancing an aceclidine-based eye drop specifically designed to restore near vision, addressing a significant unmet need in the market. Through its efforts, the company aims to enhance the quality of life for individuals experiencing vision changes related to aging.

BioAge Labs, Inc. is a biotechnology company focused on developing therapies to address aging and age-related diseases. The company utilizes a systems biology and artificial intelligence platform to identify key drug targets that influence aging and analyze extensive datasets to uncover the molecular drivers of age-related conditions. BioAge Labs is advancing several drug candidates, including BGE-117, a hypoxia-inducible factor prolyl hydroxylase inhibitor designed to activate genes involved in critical biological processes such as tissue regeneration and vascular remodeling. Additionally, BGE-175, an orally administered inhibitor of the prostaglandin D2 DP1 signaling pathway, aims to mitigate risks associated with immune aging and respiratory infections. Founded in 2015 and based in Richmond, California, BioAge Labs is committed to discovering and developing innovative treatments that harness the biology of human aging.

Tenaya Therapeutics is a developer of novel therapies designed to offer treatment for heart disease. The company's therapies address heart failure through multipronged efforts that target the fundamental cellular pathologies present in diseased cardiac muscle and that leverage cutting-edge research in cardiac development and regeneration, enabling physicians to regenerate heart tissue, and additional programs focused on cardiomyopathies.

Vaxart, Inc. is a clinical-stage biotechnology company based in South San Francisco, California, that specializes in the discovery and development of oral recombinant protein vaccines using its proprietary oral vaccine platform. The company’s pipeline includes several oral tablet vaccines targeting norovirus, seasonal influenza, respiratory syncytial virus (RSV), and a therapeutic vaccine for human papillomavirus (HPV). Vaxart's vaccines are designed to elicit broad and durable immune responses, with the advantage of being administered as room temperature-stable tablets, which may enhance distribution and increase vaccination rates. Additionally, Vaxart is developing antiviral treatments, including teslexivir, aimed at specific viral infections. The company also collaborates with Janssen Vaccines & Prevention B.V. to explore the application of its oral vaccine platform for a universal influenza vaccine program.

Tourmaline Bio is a late-stage clinical biotechnology company focused on developing transformative medicines aimed at improving the lives of patients with immune diseases. Its primary product candidate, TOUR006, is a fully human monoclonal antibody that targets interleukin-6 (IL-6), a significant proinflammatory cytokine implicated in various autoimmune and inflammatory disorders. The company leverages over twenty years of clinical and commercial experience associated with the anti-IL-6 and anti-IL-6 receptor antibody class, which has effectively treated over a million patients suffering from a range of autoimmune conditions. Tourmaline Bio is committed to addressing the needs of individuals with life-altering immune diseases through its innovative therapeutic approaches.

Ventricle Health offers evidence-based therapy, remote patient monitoring, patient education, and advocacy services for cardiac care.

Mariana Oncology is a biotechnology company focused on developing radiopharmaceuticals designed to target cancer cells using radioactive drugs. The company leverages expertise in ligand discovery, radiochemistry, and radiobiology, along with oncology translational and clinical research, to create a diverse pipeline aimed at treating various solid tumor types. By advancing the use of radiomedicines, Mariana Oncology seeks to enhance treatment options for medical professionals in their fight against cancer.

Convergent Therapeutics is a clinical-stage biotechnology company focused on developing innovative radiopharmaceutical therapies specifically for prostate cancer. The company utilizes a proprietary dual-targeted radionuclide therapy technology, initially developed by Dr. Neil Bander from Weill Cornell Medicine, which is licensed from Cornell University. Its lead product, CONV 01-α, is a monoclonal antibody conjugated with the radioactive alpha particle emitter 225Ac. This therapy is designed to bind to the prostate-specific membrane antigen (PSMA) and, upon binding, internalize to deliver a potent radioactive payload directly into prostate cancer cells. CONV 01-α is protected by multiple U.S. and foreign patents, and if approved by the FDA, it would be the first antibody to deliver a radioisotope specifically for prostate cancer treatment, marking a significant advancement in cancer therapy. Convergent Therapeutics aims to explore dual-targeted combination strategies to enhance cancer treatment efficacy, leveraging various targeting agents and therapeutic doses.

Orchard Therapeutics is a biopharmaceutical company based in the United Kingdom that specializes in developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which modifies a patient's own hematopoietic stem cells to create a targeted treatment administered in a single dose. Orchard's portfolio includes Strimvelis, the first autologous gene therapy approved by the European Medicines Agency for the treatment of adenosine deaminase-severe combined immunodeficiency (ADA-SCID). Additionally, it is advancing clinical programs for various conditions, including metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has an extensive preclinical pipeline targeting mucopolysaccharidosis types I, IIIA, and IIIB. Orchard Therapeutics collaborates with leading institutions in gene therapy research, enhancing its efforts to transform the lives of patients with rare diseases in the UK, European Union, and the United States. Founded in 2015 and headquartered in London, it aims to provide sustainable clinical benefits through its innovative therapies.

Avilar Therapeutics is a biopharmaceutical company dedicated to the discovery and development of extracellular protein degraders, which represent an innovative approach to targeted protein degradation. The company has created an integrated discovery platform that extends the concept of protein degradation beyond intracellular proteins. Avilar’s novel degraders are designed to bind to disease-causing proteins in circulation and shuttle them to the hepatocyte endolysosome for degradation. This mechanism aims to facilitate the treatment of serious diseases by removing unwanted proteins from the body, thereby offering potential therapeutic benefits in various medical conditions.

Nucleix Ltd. is a company that develops, manufactures, and markets non-invasive molecular cancer diagnostic tests. Founded in 2008 and headquartered in Rehovot, Israel, with an additional office in San Diego, California, Nucleix focuses on identifying subtle changes in DNA methylation patterns to enhance cancer detection. The company's key products include Bladder EpiCheck, a urine test designed for monitoring bladder cancer, and Lung EpiCheck, a blood test aimed at the early detection of lung cancer. By leveraging proprietary biomarker assays, Nucleix enables healthcare providers to improve treatment outcomes through more accurate cancer screening and monitoring. The company has established distribution partnerships in several countries, including Israel, the Netherlands, Turkey, Russia, Italy, and Denmark.

Pardes Biosciences is a clinical-stage biopharmaceutical company focused on addressing significant health challenges, particularly those posed by viral infections. It utilizes modern reversible-covalent chemistry to discover and develop novel oral drug candidates. The company's lead product candidate, PBI-0451, is an oral antiviral designed to treat and prevent infections from SARS-CoV-2, the virus responsible for COVID-19. By reimagining the patient journey, Pardes aims to provide accessible and convenient treatment options for both adult and pediatric patients, ultimately striving to improve health outcomes and respond effectively to global health crises.

Emergence Therapeutics AG is a biopharmaceutical company based in Duisburg, Germany, focused on developing novel antibody-drug conjugate (ADC) immuno-therapeutics for high-need cancers. Founded in 2019, the company is advancing its lead program, which combines a highly specific antibody with optimized linker and payload technology to target Nectin-4, a clinically validated target for various cancers. This approach is inspired by the success of enfortumab vedotin, an approved treatment for urothelial cancers. Emergence Therapeutics is also exploring opportunities to create additional first- or best-in-class ADCs, aiming to address significant therapeutic needs in oncology.

Mariana Oncology is a biotechnology company focused on developing radiopharmaceuticals designed to target cancer cells using radioactive drugs. The company leverages expertise in ligand discovery, radiochemistry, and radiobiology, along with oncology translational and clinical research, to create a diverse pipeline aimed at treating various solid tumor types. By advancing the use of radiomedicines, Mariana Oncology seeks to enhance treatment options for medical professionals in their fight against cancer.

Avilar Therapeutics is a biopharmaceutical company dedicated to the discovery and development of extracellular protein degraders, which represent an innovative approach to targeted protein degradation. The company has created an integrated discovery platform that extends the concept of protein degradation beyond intracellular proteins. Avilar’s novel degraders are designed to bind to disease-causing proteins in circulation and shuttle them to the hepatocyte endolysosome for degradation. This mechanism aims to facilitate the treatment of serious diseases by removing unwanted proteins from the body, thereby offering potential therapeutic benefits in various medical conditions.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on precision oncology, developing therapeutics that target challenging cancer types without straightforward single-gene mutations. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond to specific treatments. Acrivon's pipeline includes ACR-368, also known as prexasertib, which is currently in Phase 2 trials, alongside preclinical programs aimed at key components of DNA damage response and cell cycle regulation, such as the WEE1 and PKMYT1 proteins. Acrivon seeks to expedite the development of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials, aiming to enhance treatment success in oncology.

Antios Therapeutics is a biopharmaceutical company devoted to developing innovative therapies for viral diseases. With an experienced and proven leadership team, the company is focused on the development of its oral drug candidate for potentially curative treatment of HBV infections. Antios Therapeutics was founded by Abel De La Rosa, Douglas Mayers, and Idean Marvasty.

GentiBio, Inc. is a biotherapeutics company focused on developing engineered regulatory T cells (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, GentiBio utilizes a proprietary platform that combines autologous and allogeneic EngTregs technologies. This innovative approach is designed to restore immune tolerance and address limitations present in current regulatory T-cell therapies. By targeting the underlying causes of diseases related to immune system dysfunction, GentiBio aims to provide effective therapeutic solutions for conditions that arise from immune system overshooting or malfunctioning. The company was co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of this emerging field.

LENZ Therapeutics is a late-stage biopharmaceutical company dedicated to the development and commercialization of innovative ophthalmic therapies aimed at improving vision. The company's primary focus is on addressing presbyopia, a common condition that leads to the gradual loss of near vision, affecting nearly all individuals over the age of 45. LENZ Therapeutics is currently advancing an aceclidine-based eye drop specifically designed to restore near vision, addressing a significant unmet need in the market. Through its efforts, the company aims to enhance the quality of life for individuals experiencing vision changes related to aging.

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.

Antios Therapeutics is a biopharmaceutical company devoted to developing innovative therapies for viral diseases. With an experienced and proven leadership team, the company is focused on the development of its oral drug candidate for potentially curative treatment of HBV infections. Antios Therapeutics was founded by Abel De La Rosa, Douglas Mayers, and Idean Marvasty.

Nucleix Ltd. is a company that develops, manufactures, and markets non-invasive molecular cancer diagnostic tests. Founded in 2008 and headquartered in Rehovot, Israel, with an additional office in San Diego, California, Nucleix focuses on identifying subtle changes in DNA methylation patterns to enhance cancer detection. The company's key products include Bladder EpiCheck, a urine test designed for monitoring bladder cancer, and Lung EpiCheck, a blood test aimed at the early detection of lung cancer. By leveraging proprietary biomarker assays, Nucleix enables healthcare providers to improve treatment outcomes through more accurate cancer screening and monitoring. The company has established distribution partnerships in several countries, including Israel, the Netherlands, Turkey, Russia, Italy, and Denmark.

Tenaya Therapeutics is a developer of novel therapies designed to offer treatment for heart disease. The company's therapies address heart failure through multipronged efforts that target the fundamental cellular pathologies present in diseased cardiac muscle and that leverage cutting-edge research in cardiac development and regeneration, enabling physicians to regenerate heart tissue, and additional programs focused on cardiomyopathies.

Orchard Therapeutics is a biopharmaceutical company based in the United Kingdom that specializes in developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which modifies a patient's own hematopoietic stem cells to create a targeted treatment administered in a single dose. Orchard's portfolio includes Strimvelis, the first autologous gene therapy approved by the European Medicines Agency for the treatment of adenosine deaminase-severe combined immunodeficiency (ADA-SCID). Additionally, it is advancing clinical programs for various conditions, including metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has an extensive preclinical pipeline targeting mucopolysaccharidosis types I, IIIA, and IIIB. Orchard Therapeutics collaborates with leading institutions in gene therapy research, enhancing its efforts to transform the lives of patients with rare diseases in the UK, European Union, and the United States. Founded in 2015 and headquartered in London, it aims to provide sustainable clinical benefits through its innovative therapies.

Vivace Therapeutics, Inc. is a drug discovery and development company focused on creating cancer therapies that target the Hippo pathway, which plays a crucial role in tissue regeneration and organ development. Established in 2014 and located in the San Francisco Bay Area, Vivace Therapeutics employs an innovative approach to develop small molecule treatments aimed at improving patient outcomes in oncology. The company collaborates with leading academic institutions to advance research and develop therapies that can be used alone or in combination with existing treatments. With a commitment to enhancing cancer care, Vivace Therapeutics is dedicated to delivering novel solutions that can help patients combat cancer more effectively.

Locanabio, Inc. is a biotechnology company based in San Diego, California, founded in 2016. It specializes in developing RNA-targeted gene therapies aimed at treating a range of underserved diseases, particularly in the areas of neuromuscular, neurodegenerative, and retinal conditions. The company's innovative platform focuses on modifying disease-causing RNA, allowing for the identification and correction of specific RNA sequences associated with rare genetic disorders. This approach distinguishes Locanabio's therapies from traditional DNA-targeted methods, offering a unique solution for patients with severe and challenging health issues. The company, which rebranded from Locana, Inc. in July 2020, is dedicated to advancing therapeutic candidates that address significant medical needs.

Adagio Medical is engaged in the research and development of treatments for cardiovascular diseases. Adagio Medical is committed to providing electrophysiologists innovative cryoablation catheters that create safe, continuous, transmural lesions to treat cardiac arrhythmias anywhere in the heart, including paroxysmal and persistent atrial fibrillation, atrial flutter, and ventricular tachycardia. The company was founded in 2011 and is based in Laguna Hills, California.

PMV Pharmaceuticals, Inc. is a precision oncology company focused on discovering and developing small molecule therapies targeting p53 mutations in cancer. The company's lead candidate, PC14586, aims to correct and restore the function of p53, a critical tumor suppressor protein. In addition to this product, PMV Pharmaceuticals is also working on therapies for specific p53 hotspot mutations, including the R273H mutation. Founded in 2013 and headquartered in Cranbury, New Jersey, the company leverages over three decades of expertise in p53 biology, aiming to develop first-in-class modulators that could improve treatment outcomes for cancer patients.

FORMA Therapeutics is a clinical-stage biopharmaceutical company based in Watertown, Massachusetts, specializing in the development and commercialization of innovative therapeutics for rare hematologic diseases and various cancers. The company's notable product candidates include FT-4202, currently in Phase 1 trials for sickle cell disease and other hemoglobinopathies, and FT-7051, which targets metastatic castration-resistant prostate cancer. Additionally, FORMA is advancing FT-2102, an oral investigational agent designed to inhibit mutated IDH1 enzymes, now being evaluated in Phase 2 trials for relapsed/refractory acute myeloid leukemia and exploratory Phase 1 trials for glioma. The company also develops FT-4101 and FT-8225, selective inhibitors of fatty acid synthase. By integrating advanced drug discovery technologies and oncology expertise, FORMA aims to target challenging biological pathways that contribute to cancer, thereby enhancing the development of small molecule drug candidates with unique mechanisms of action.

Orchard Therapeutics is a biopharmaceutical company based in the United Kingdom that specializes in developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which modifies a patient's own hematopoietic stem cells to create a targeted treatment administered in a single dose. Orchard's portfolio includes Strimvelis, the first autologous gene therapy approved by the European Medicines Agency for the treatment of adenosine deaminase-severe combined immunodeficiency (ADA-SCID). Additionally, it is advancing clinical programs for various conditions, including metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has an extensive preclinical pipeline targeting mucopolysaccharidosis types I, IIIA, and IIIB. Orchard Therapeutics collaborates with leading institutions in gene therapy research, enhancing its efforts to transform the lives of patients with rare diseases in the UK, European Union, and the United States. Founded in 2015 and headquartered in London, it aims to provide sustainable clinical benefits through its innovative therapies.

Farcast Biosciences is a clinical diagnostic company focused on improving cancer treatment through personalized medicine. Founded in 2019 and based in Madison, Wisconsin, with a laboratory in Bangalore, India, Farcast has developed a human tumor microenvironment platform. This platform allows oncologists and drug developers to evaluate individual tumors by culturing fresh, unmodified tumor fragments, preserving their natural state. By generating detailed response data, Farcast's technology aids in identifying optimal treatment options for patients before they begin therapy. This approach not only enhances the understanding of tumor behavior but also supports researchers in drug development by providing insights into treatment responses using real human tumors and blood samples.

Natera, Inc. is a diagnostic company that specializes in preconception and prenatal genetic testing services. Its primary offerings include the Panorama test, which is a non-invasive prenatal test that screens for chromosomal abnormalities in a fetus through a blood sample from the mother, and the Vistara test, designed to identify single-gene disorders. Additionally, Natera provides Horizon carrier screening to determine genetic disease carrier status and Spectrum, which analyzes chromosomal anomalies during in vitro fertilization. The company also offers Anora, a test for analyzing fetal chromosomes to investigate miscarriage causes, and non-invasive paternity testing. Its Signatera product utilizes circulating tumor DNA technology to monitor cancer recurrence. Natera markets its tests through a direct sales force and a network of approximately 100 laboratory and distribution partners both domestically and internationally. The company, founded in 2003 and headquartered in San Carlos, California, maintains partnerships with BGI Genomics and Foundation Medicine for the development of genetic testing assays.

Lumena Pharmaceuticals is focused on developing oral therapeutics for rare liver diseases, aiming to enhance liver function and alleviate disease symptoms. The company's clinical-stage product candidates are designed to selectively target intestinal transporters, minimizing systemic toxicity and absorption in the body. Their lead candidate, LUM001, has undergone extensive evaluation through 12 clinical studies involving over 1,400 subjects, positioning it for rapid advancement in treating cholestatic liver disease in both pediatric and adult patients. In addition to its primary goal of addressing rare liver diseases, Lumena's innovative therapeutic approach shows promise for treating metabolic liver diseases, a significant and rising health concern affecting both children and adults.

OvaScience, Inc. was a fertility company focused on developing innovative treatments for women facing infertility challenges. The company was founded in 2011 and was headquartered in Waltham, Massachusetts. OvaScience specialized in proprietary technology derived from the discovery of egg precursor (EggPC) cells, which allowed for the enhancement of egg quality and improved outcomes in in vitro fertilization (IVF). Their treatment options included OvaPrime, which aimed to restore a woman's egg production using her own EggPC cells, OvaTure, designed to mature these cells into fertilizable eggs outside the body, and AUGMENT, which utilized mitochondria from EggPC cells to enhance fertilization and pregnancy rates. The company also explored OvaXon for preventing inherited diseases. OvaScience was co-founded by a team of experts from Harvard Medical School and was backed by prominent life science investors. In December 2018, OvaScience was acquired by Millendo Therapeutics in a reverse merger.

Allegiance Hospice Group specializes in providing hospice care to patients with terminal illnesses in the Merrimack Valley and Southeastern Massachusetts. The organization employs an interdisciplinary team that includes physicians, nurses, home healthcare aides, social workers, counselors, and volunteers to deliver comprehensive palliative care. Their services encompass inpatient nursing and rehabilitation treatments, ensuring that patients receive the necessary support and comfort during this critical time. Allegiance Hospice Group is dedicated to enhancing the quality of life for patients and their families through compassionate care tailored to individual needs.