RA Capital Management

Curevo is a clinical-stage biotechnology company developing next-generation, non-mRNA vaccines to address unmet medical needs. Its lead candidate, amezosvatein (also known as CRV-101), is an adjuvanted subunit vaccine designed to prevent shingles (herpes zoster) and chickenpox (varicella). The company focuses on improving vaccine efficacy, safety, and tolerability while advancing its pipeline through clinical trials.

Bia Energy is a pioneering company at the forefront of the energy industry. Bia, seamlessly merges smart metering infrastructure data with energy retailing, offering a customer-centric, data-driven platform that empowers users to take control of their energy consumption and costs. With Bia rapidly becoming the standard in Colombia's energy sector and beyond, transforming the region's energy service landscape.

Metsera is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for obesity and metabolic conditions. The company focuses on advancing a variety of oral and injectable treatment options, including incretin, non-incretin, and combination therapies. These treatments are designed to address multiple therapeutic targets, positioning Metsera to meet the evolving demands of the weight loss treatment landscape. Additionally, Metsera integrates proprietary health technology tools to provide personalized care, enhancing the effectiveness of its therapeutic offerings.

Halda Therapeutics is a research-stage drug discovery company focused on developing next-generation precision medicines. The company utilizes a novel therapeutic modality aimed at creating treatments that can address various disease states. By modifying disease-causing pathways, Halda Therapeutics seeks to enable healthcare providers to offer tailored therapies to patients, enhancing the effectiveness of treatment based on individual patient needs.

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.

Outpace Bio is a biotechnology company that creates smart cell therapies aimed at improving efficacy and safety. It offers a platform that combines custom-designed biological functions with cellular control modalities that program the right response at the right time and place. It creates a mechanism-driven solution tailor-made to improve efficacy and safety. The company collaborates with Lyell Immunopharma to develop and commercialize a potential immune cell therapy for the treatment of cancer, auto-immune diseases, infectious diseases, degenerative diseases, regenerative medicine, metabolic disorders, and genetic disorders, enabling healthcare providers to cure a wide range of diseases as per patient's need.

Gameto is a biotechnology company focused on improving female reproductive health through innovative therapies. Recognizing that women's health is an underserved area with limited treatment options, Gameto aims to develop advanced solutions that enhance patient outcomes. The company utilizes cellular engineering to create female reproductive cell lines, employing proprietary combinations of transcription factors to reprogram human induced pluripotent stem cells (hiPSCs) into engineered ovarian and endometrial cell lines that function similarly to natural cells. This platform supports a portfolio of cellular therapeutics targeting various female reproductive diseases. Gameto's flagship program, Fertilo, is designed to enhance the processes of in vitro fertilization (IVF) and egg freezing, making them shorter, safer, and more effective. Additional applications of Gameto's technology include Deovo, an organoid of the female reproductive system, and Ameno, a cell-based therapeutic aimed at alleviating the health issues associated with primary ovarian insufficiency and menopause.

AltruBio is a biopharmaceutical company focused on developing targeted antibody therapeutics for cancer and immune-related inflammatory diseases. Established in Delaware with its principal office in the San Francisco Bay Area, AltruBio integrates global resources to advance its research and development initiatives. The company has its roots in Taipei, Taiwan, where it has been working since June 2000 on discovering and developing novel therapeutics to address unmet medical needs. AltruBio employs a sophisticated discovery platform to identify and validate new antibody therapeutics. Currently, the company is developing several potential therapeutic antibodies that target a range of conditions, including autoimmune diseases and various types of cancer, such as pancreatic, stomach, colorectal, ovarian, and lung cancers, at different stages of development.

TORL BioTherapeutics is a clinical-stage biopharmaceutical company dedicated to developing innovative antibody-based medicines aimed at improving outcomes for cancer patients. The company specializes in creating antibody-drug conjugates (ADCs) and monoclonal antibodies (mAbs) to address oncologic diseases that exhibit significant unmet medical needs. TORL BioTherapeutics employs a strategic approach that includes target identification and early discovery work, allowing them to license promising drug programs and develop proprietary therapies with optimized characteristics. Through this pipeline, TORL BioTherapeutics is positioned to advance novel treatments that could significantly enhance patient care in oncology.

Capstan Therapeutics is a biotechnology company focused on enhancing the therapeutic potential of in vivo RNA-based therapies. It develops proprietary targeted delivery systems, including targeted lipid nanoparticles, to improve the efficacy and control of dosage in engineered cell therapies. This innovative platform aims to address a variety of diseases, particularly in the fields of oncology, fibrosis, and inflammation, where there are significant unmet clinical needs. By advancing precision in vivo cell engineering, Capstan Therapeutics seeks to create transformative therapeutics that can offer new solutions for patients facing these challenging conditions.

ProfoundBio is a clinical-stage biotechnology company dedicated to creating innovative antibody-based therapeutics aimed at treating cancer. Utilizing proprietary technology platforms, the company has developed a diverse pipeline of antibody-drug conjugate (ADC) candidates targeting both solid tumors and hematological malignancies. These drug candidates are currently in various stages of discovery, preclinical, and clinical development. ProfoundBio's focus is on harnessing the immune system to provide patients with more effective treatments that have the potential for curative outcomes.

Nature’s Toolbox is a biotechnology company based in New Mexico that has developed a proprietary host-independent biological manufacturing and development platform. Founded in 2015, the company focuses on identifying, evaluating, and prioritizing naturally occurring drug discovery targets. Its platform provides high-throughput screening capabilities for the rapid prototyping of biological materials, allowing for real-time metabolic profiling of bacteria, fungi, and plants. This technology facilitates the discovery, characterization, and production of bioactive natural products, which can be applied to a range of applications, including protein-based therapies for cancer and autoimmune disorders. By enhancing reproducibility and reducing the resource footprint, Nature’s Toolbox aims to accelerate the drug development process for its clients.

AM Batteries manufactures Li-ion batteries using solvent-free electrode manufacturing technology. The company was founded in 2016 and is headquartered in Chelmsford, Massachusetts.

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.

Abcuro, Inc. is a biotechnology company focused on developing immunotherapies for the treatment of autoimmunity and cancer. Based in Newton, Massachusetts, Abcuro creates therapeutic antibodies targeting novel mechanisms related to these diseases. The company utilizes advanced bioinformatics to analyze transcription data from human diseases, which aids in the identification of new therapeutic targets. Through ex-vivo validation in diseased tissues, Abcuro seeks to uncover innovative methods to modulate key components of the immune system. Established in 2015, Abcuro aims to enhance treatment strategies in the healthcare industry by offering effective solutions for complex immunological conditions.

Septerna is a biotechnology company focused on discovering and advancing novel small-molecule medicines that target G protein-coupled receptors (GPCRs). The company aims to improve patient outcomes by integrating innovative drug discovery technologies with GPCR drug targets. Utilizing its Native Complex platform, Septerna facilitates industrial-scale drug discovery through advanced screening technologies and structure-based drug design. This approach enables medical researchers to efficiently identify treatments for a variety of diseases.

Hemab is a company that creates preventative treatments for bleeding and thrombosis problems. The medicines developed by the business aggregate, modify, balance, and at times recruit endogenous clotting components already present in the blood to the site of injury, allowing patients to produce hemostatic plugs or clots to avert life-threatening complications. Hemab Therapeutics was established in 2017 in Copenhagen, Hovedstaden by Johan H. Faber and Mads Behrndt.

Vaxess Technologies, Inc. is a biotechnology company based in Boston, Massachusetts, founded in 2011. The company focuses on developing and commercializing innovative silk biomaterial technologies. Its key product, the MIMIX microneedle platform, facilitates the transdermal delivery of vaccines and therapeutics. This sustained release patch technology allows for a controlled release of vaccines, mimicking the natural infection process to enhance immune response and effectiveness. Vaxess's patch is engineered for stability, eliminating the need for refrigeration, which enables shipping and application in low-resource settings. In addition to its vaccine delivery systems, Vaxess is also engaged in creating novel therapies aimed at oncology care and infectious diseases, ultimately striving to improve patient outcomes with reduced treatment burdens.

MBX Biosciences, Inc. is a biotechnology company based in Carmel, Indiana, specializing in the development of therapeutics for rare endocrine disorders. Established in 2018, the company focuses on creating peptides aimed at treating genetic diseases that affect glandular hormones, which play a crucial role in the body’s chemical messaging system. By targeting conditions with inadequate treatment options, MBX Biosciences is dedicated to discovering, developing, and commercializing innovative endocrine therapeutics.

Rivus Pharmaceuticals operates as a biopharmaceutical company dedicated to improving cardio-metabolic health. Rivus Pharmaceuticals is dedicated to improving cardio-metabolic health by developing controlled metabolic accelerators (CMAs) that address the underlying metabolic risk factors for cardiovascular mortality and morbidity.

Empatica Inc. is a company that specializes in developing wearable medical sensor devices aimed at health monitoring, treatment, and research. Founded in 2013 and based in Cambridge, Massachusetts, Empatica's offerings include the E3 wristband, which monitors physiological signals in real-time, along with a mobile application and a web dashboard designed for stress and sleep monitoring. The company employs a combination of biosensors and artificial intelligence to analyze human physiology, providing valuable insights for patients, clinicians, and researchers. Its client base includes notable organizations such as Microsoft Research, Sony, University of Trento, and Intel.

We believe that all patients deserve the most innovative, open surgical treatment options so they can live their fullest lives. We’re on a mission to advance robotic technology that pushes the limits of soft tissue open surgery and opens new opportunities for surgeons to restore quality of life for more patients with complex conditions. The Symani® Surgical System is a first-of-its kind robotic technology that uniquely addresses the scale and complexities of microsurgery and supermicrosurgery to restore quality of life for more patients.

Mineralys Therapeutics is a biopharmaceutical company that provides hypertension treatment. They also bring a targeted approach to the management of hypertension.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

Be Biopharma, Inc. is focused on developing innovative therapeutics derived from engineered B-cells to address various diseases, including cancer, autoimmune disorders, and infectious diseases. Founded in 2020 and based in Cambridge, Massachusetts, the company aims to overcome significant challenges associated with traditional cell and gene therapies, such as the limitations of dosing, unpredictable reactions in patients, and the necessity of pre-treatment chemotherapy. By leveraging precision genome editing, Be Biopharma creates durable and effective cell therapies that can be administered without toxic conditioning, thereby improving treatment options for conditions like Hemophilia B and enhancing overall patient outcomes.

Incyclix Bio develops small molecule inhibitors of CDKs for the treatment of advanced and resistant cancers. Founded by Patrick Roberts, Jay Strum, John Bisi, and Fred Eshelman in 2020 and is headquartered in Durham, North Carolina.

Plexium, Inc. is a biotechnology company based in San Diego, California, that specializes in developing targeted protein degradation therapies aimed at treating cancer and neurodegenerative diseases. Founded in 2017, Plexium utilizes a platform that identifies small molecules capable of modulating E3 ligases, which play a crucial role in protein degradation. This platform includes the DELPhe screening technology, designed for high-content readouts from cell-based assays. By harnessing E3 ligases, Plexium's approach enables selective targeting of various cancers, including small cell lung and breast tumors. The company's innovative research extends beyond existing therapies, exploring new drug-like protein degraders and molecular glues to enhance treatment options for cancer and inflammatory diseases.

Metagenomi, Inc. is a biotechnology company focused on developing gene editing systems aimed at treating genetic diseases. Founded in 2016 and based in Emeryville, California, Metagenomi utilizes metagenomics and machine learning to uncover novel genome editing technologies derived from previously uncharacterized organisms. The company's innovative discovery platform enables the creation of highly efficient and specific gene editing tools, including programmable nucleases, base editors, and advanced integration systems such as prime editing and CRISPR-associated transposases. By harnessing these naturally evolved systems, Metagenomi seeks to address a wide range of genetic mutations that traditional genome engineering approaches have struggled to target, with the ultimate goal of providing transformative therapies for patients globally.

Third Harmonic Bio is a clinical-stage company developing a first-in-class, highly selective, oral KIT inhibitor for treatment of severe allergy and inflammation. KIT is a cell surface receptor that acts as the master survival and functional regulator of mast cells, which are immune cells concentrated at the body’s interfaces with the external environment and that act as the key mediator of the inflammation associated with allergic diseases.

AN2 Therapeutics, Inc. is a biopharmaceutical company based in Menlo Park, California, founded in 2017. The company is dedicated to the research, development, and commercialization of innovative medicines aimed at addressing infectious diseases. AN2 Therapeutics is focused on developing epetraborole, a once-daily oral treatment for patients suffering from chronic non-tuberculous mycobacterial lung disease, which represents a significant unmet medical need. Additionally, the company has established a strategic partnership with Brii Biosciences to enhance its development efforts.

Tasso, Inc. is a Seattle-based company founded in 2012 that specializes in manufacturing blood sample collection devices designed for patient convenience. The company's innovative devices enable individuals to collect their own blood samples safely and easily at home, eliminating the need for clinic visits. These disposable, compact devices feature interchangeable cartridges that ensure proper blood preparation immediately upon sample collection. By allowing patients to manage their blood sampling process, Tasso aims to streamline blood-based diagnostics and enhance the overall experience of medical testing.

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.

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 focused on creating small molecule medicines for RNA-mediated diseases. The company specializes in developing novel RNA-targeted drug candidates that have the potential to treat a wide range of severe health conditions. By leveraging its innovative technology, Expansion Therapeutics aims to provide effective treatment options for patients suffering from these challenging 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.

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, that focuses on drug discovery through its innovative platform targeting solute carrier (SLC) transporters. Established in 2016, the company aims to improve patient outcomes by developing small molecule therapies for various diseases, including those related to immunometabolism, lysosomal function, and mucosal defense. By utilizing advanced chemoproteomics techniques, Jnana accelerates the identification of therapeutic targets, addressing significant unmet medical needs in areas such as immuno-oncology, inflammatory disorders, and neurological diseases. The company's approach seeks to enhance drug discovery efficiency by comprehensively targeting the vast family of membrane proteins associated with SLC transporters.

Sydnexis is focused on developing a proprietary treatment for pediatric progressive myopia, addressing a significant global medical need. The company manufactures a topical eyedrop formulation specifically designed to treat the progression of myopia in children. This innovative approach enables healthcare professionals to effectively manage and reduce the incidence of eye disorders in pediatric patients.

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.

Frontier Medicines Corporation is a pre-clinical stage biopharmaceutical company based in South San Francisco, California, founded in 2018. The company focuses on the discovery and development of medicines targeting cancer-causing proteins through its innovative chemoproteomics platform. This platform utilizes advanced computational methods and machine learning to identify and pharmacologically target new binding pockets on proteins, facilitating the development of small-molecule drugs. By enhancing the accessibility of these proteins, Frontier Medicines aims to provide effective treatments that can redefine the management of debilitating diseases.

Bright Peak Therapeutics is a biotechnology company focused on developing immunotherapies for cancer and autoimmune diseases. The company specializes in cytokine therapeutics that enable tissue and cell-specific targeting of cytokine payloads. This approach aims to enhance the efficacy of treatments, ultimately improving patient outcomes and quality of life. By leveraging innovative immuno-cytokines, Bright Peak seeks to provide more effective therapeutic options for complex medical conditions.

Eliem Therapeutics, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, dedicated to developing innovative therapeutic drugs for hyperexcitability disorders affecting the nervous system. Founded in 2018, the company focuses on creating treatments for conditions such as chronic pain, depression, epilepsy, and anxiety, which significantly impact the quality of life for millions. Eliem employs its expertise in neuroscience and translational medicine to advance a promising pipeline of drug candidates, aiming to address unmet medical needs in these areas. Notably, its leading drug candidate, ETX-155, targets major depressive disorder and focal onset seizures. Through its efforts, Eliem Therapeutics seeks to improve the lives of patients suffering from these debilitating disorders.

Forge Biologics is a gene therapy development engine, focused on enabling access to life-changing gene therapies and helping bring them from an idea into reality. The company partners with innovators in the gene therapy community: scientists, physicians, biotech/pharma companies, and patient groups. It was founded in 2020 and headquartered in Grove City, Ohio.

Boundless Bio is a clinical-stage biopharmaceutical company focused on developing innovative therapies for the treatment of aggressive cancers linked to extrachromosomal DNA (ecDNA). Founded in 2018 and based in La Jolla, California, the company aims to address the significant unmet medical needs of patients with oncogene amplified tumors, which affect over 14% of cancer patients. By targeting ecDNA, a key factor in oncogene amplification, Boundless Bio seeks to provide transformative treatments for previously difficult-to-treat cancers. One of its leading candidates, BBI-355, is an oral, selective inhibitor of checkpoint kinase 1 (CHK1), designed to manage the replication and transcription of ecDNA in cancer cells.

Janux Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative immunotherapies aimed at generating immune responses to combat tumors while preserving healthy tissue. Utilizing its proprietary tumor-activated T Cell Engager platform technology, Janux targets all three stages of the anti-tumor immune response. The company's approach is designed to enhance the efficacy of cancer treatments by specifically engaging the immune system to identify and eliminate cancer cells.

GH Research PLC is a clinical-stage biopharmaceutical company that aims to transform the treatment of psychiatric and neurological disorders. The company is primarily focused on developing novel and proprietary therapies based on 5-MeO-DMT, a compound known as Mebufotenin, to address the needs of patients suffering from Treatment-Resistant Depression (TRD). Through its innovative approach, GH Research seeks to provide new therapeutic options for individuals who have not responded to conventional treatments.

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.

Ventus Therapeutics is a biopharmaceutical company focused on discovering and developing novel small molecule medicines aimed at treating autoimmune diseases, inflammatory diseases, and cancer by targeting the innate immune system. The company employs a structural immunology platform that provides deep insights into the mechanisms and molecular structures involved in immune responses. This platform integrates proprietary protein engineering capabilities with advanced rational and structure-based drug design tools, allowing for precise targeting of the innate immune system. Ventus is actively building a pipeline of drug programs that address critical targets within this system, particularly proteins involved in inflammasome and nucleic acid-sensing signaling pathways, thereby enabling the treatment of both acute and chronic inflammatory and immune-related conditions.

Icosavax, Inc. is a biotechnology company based in Seattle, Washington, founded in 2017. The company specializes in developing vaccines using its innovative virus-like particle (VLP) technology platform, which allows for the multivalent display of complex viral antigens. This technology aims to provide broad and durable protection against various infectious diseases, with an initial focus on life-threatening respiratory illnesses. Icosavax's pipeline includes vaccine candidates targeting respiratory syncytial virus (RSV), human metapneumovirus (hMPV), and SARS-CoV-2. The company is dedicated to advancing its VLP technology to discover, develop, and ultimately commercialize effective vaccines against these infectious diseases.

Tyra Biosciences is a clinical-stage biotechnology company based in Carlsbad, California, that specializes in developing small molecule therapies for cancer treatment. Founded in 2018, the company aims to address drug resistance in cancer therapies through its proprietary SNÅP platform, which facilitates rapid and precise drug design by generating iterative molecular snapshots. Tyra is particularly focused on creating selective inhibitors of the Fibroblast Growth Factor Receptor (FGFR) family, which are implicated in approximately 7% of all cancers. The company's lead product candidate, TYRA-300, specifically targets FGFR3 and is initially aimed at patients with metastatic urothelial carcinoma of the bladder and urinary tract. Through its innovative approach, Tyra Biosciences seeks to improve treatment outcomes for cancer patients by providing alternatives to existing therapies.

Pyxis Oncology is a developer of antibody therapeutics intended to promote the body's immune response to cancer. The company has engaged in deep analyses of tumor antigen-specific tumor-infiltrating lymphocytes (TILs) within hot tumors and of tumor cell signaling pathways within cold tumors, providing laboratories with development of novel antibody-based immunotherapies. It was founded in 2019 and is based in Boston, Massachusetts.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that specializes in targeted DNA integration to develop innovative therapies for severe diseases. The company utilizes a precision gene editing approach to precisely correct genetic mutations, replace malfunctioning genes, and insert therapeutic genetic sequences at specific locations within the genome. Its technology aims to transform treatment paradigms by enabling the targeted integration of genetic payloads, which could potentially cure a range of serious and life-threatening conditions. Graphite Bio was formerly known as Integral Medicines, Inc. before rebranding in August 2020, and it was incorporated in 2019.

DTx Pharma, LLC is a biotechnology company focused on developing and commercializing fatty acid-conjugated peptide therapeutics for patients with rare and chronic diseases. The company specializes in RNA-based therapeutics, utilizing a delivery technology platform that enhances the distribution of nucleic acid drugs to various tissues and organ systems beyond the liver. This innovative approach addresses limitations associated with previous-generation RNA delivery methods, such as poor pharmacokinetics and inadequate cellular uptake. DTx Pharma's products target conditions including retinitis pigmentosa, Duchenne muscular dystrophy, and central nervous system disorders. Founded in 2017 and based in San Diego, California, DTx Pharma aims to provide personalized treatment options across multiple therapeutic areas.

Artiva Biotherapeutics is a biotech company focused on developing and manufacturing cellular immunotherapies for cancer patients. Founded in 2019 and based in San Diego, California, Artiva specializes in off-the-shelf, allogeneic natural killer (NK) cell therapies aimed at treating various hematologic cancers and solid tumors. The company's product pipeline targets specific antigens, including CD20 and CD19 for B-cell lymphomas, as well as HER2 for certain solid tumors. Artiva's approach emphasizes the therapeutic potential of NK cells and aims to overcome challenges in scaling and manufacturing these therapies. The company is committed to delivering safe, effective, and readily accessible cell therapies to benefit cancer patients. Artiva operates as a subsidiary of Green Cross Holdings Corporation.

Design Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing innovative therapies for degenerative disorders linked to nucleotide repeat expansions. Founded in 2017 and headquartered in Solana Beach, California, the company is advancing its lead program aimed at treating Friedreich’s ataxia. Additionally, Design Therapeutics is exploring treatments for other conditions such as Fragile X syndrome and myotonic dystrophy, utilizing its proprietary GeneTACTM technology, which involves small-molecule gene-targeted therapeutics designed to modify the underlying causes of these inherited diseases. The company is committed to addressing serious monogenic disorders driven by nucleotide repeat mutations through its ongoing research and development efforts.

Verve Therapeutics, Inc. is a genetic medicines company focused on innovative treatments for cardiovascular disease through gene editing. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company aims to shift the management of cardiovascular conditions from chronic treatments to single-course gene editing therapies. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are known to significantly impact blood lipid levels, a major factor in cardiovascular disease. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance its gene editing delivery technologies. The company is staffed by a team of experts in cardiovascular medicine, human genetics, and drug development, positioning itself to address the challenges of cardiovascular health effectively.

IO Biotech is a clinical-stage biopharmaceutical company focused on developing innovative immune therapies for cancer treatment. Utilizing its proprietary T-win technology platform, the company creates immune-modulating cancer vaccines that activate T cells specifically targeting immune-suppressive molecules. This approach aims to induce the immune system to disrupt multiple pathways responsible for tumor-induced immunosuppression. IO Biotech has a solid track record in advancing both preclinical and clinical compounds, with its two lead candidates targeting IDO and PD-L1 currently in clinical development, while several additional therapies remain in the preclinical stage.

Werewolf Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative therapeutics that enhance the body’s immune response to cancer. Founded in 2017, the company utilizes its proprietary PREDATOR platform to engineer conditionally activated molecules, known as INDUKINE molecules. These therapeutics are designed to remain inactive in peripheral tissues but activate selectively within the tumor microenvironment, addressing the limitations of traditional proinflammatory immune therapies. By stimulating both adaptive and innate immunity, Werewolf Therapeutics aims to improve cancer treatment outcomes.

Kira Pharmaceuticals is a biotechnology company founded in 2017 and based in Suzhou, China. It specializes in developing complement-targeted therapies aimed at treating immune-mediated diseases. The company focuses on pioneering drug discovery related to the complement system, with the goal of providing transformative therapies for individuals suffering from complement-mediated conditions.

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.

Acumen Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on discovering, developing, and commercializing therapies for Alzheimer's disease and related neurodegenerative conditions. The company is advancing a targeted immunotherapy drug candidate, ACU193, which is designed to selectively target amyloid-beta oligomers, believed to be a key factor in the progression of Alzheimer's disease. Acumen employs a sensitivity assay to measure soluble amyloid-beta oligomer levels in cerebrospinal fluid, which aids in the enrollment of patients for clinical trials. The Phase 1 clinical trial for ACU193 began in 2021, aiming to assess its safety, tolerability, and pharmacokinetics in patients with mild cognitive impairment and mild dementia resulting from Alzheimer's disease. Founded in 1996 and based in Livermore, California, Acumen Pharmaceuticals is committed to addressing the challenges posed by Alzheimer’s disease through innovative therapeutic approaches.

Olema Oncology is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for estrogen receptor (ER) positive breast cancer. The company specializes in the discovery and commercialization of targeted treatments aimed at improving outcomes for women with cancer. Olema utilizes its extensive knowledge of endocrine-driven cancers and the molecular mechanisms of the ER to create drug candidates that are designed to outperform existing therapies. Currently, the company's product pipeline includes OP-1250 and OP-3136, both of which have completed discovery and preclinical studies. Olema's mission is to transform the standard of care for both pre- and post-menopausal women by providing more effective and convenient treatment options.

Vor Biopharma, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing engineered hematopoietic stem cell (eHSC) therapies for cancer treatment. The company's lead product candidate, VOR33, is designed specifically for the treatment of acute myeloid leukemia (AML) and is currently in preclinical development. VOR33 eHSCs are engineered to lack CD33, a protein commonly expressed by AML cells, allowing for selective targeting of the cancer without harming normal cells. This innovative approach seeks to minimize the toxicities associated with traditional immunotherapies, which often affect both cancerous and healthy tissues. By protecting healthy stem cells from depletion while generating functional immune cells, Vor Biopharma aims to enhance therapeutic outcomes for patients with hematological malignancies.

Cybin is a clinical-stage biopharmaceutical company on a mission to create safe and effective psychedelic-based therapeutics to address the large unmet need for new and innovative treatment options for people who suffer from mental health conditions. Cybin’s goal of revolutionizing mental healthcare is supported by a network of world-class partners and internationally recognized scientists aimed at progressing proprietary drug discovery platforms, innovative drug delivery systems, and novel formulation approaches and treatment regimens. The Company is currently developing CYB003, a proprietary deuterated psilocybin analog for the treatment of major depressive disorder, and CYB004, a proprietary deuterated DMT molecule for generalized anxiety disorder, and has a research pipeline of investigational psychedelic-based compounds. Headquartered in Canada and founded in 2019, Cybin is operational in Canada, the United States, the United Kingdom, the Netherlands, and Ireland. For company updates and to learn more about Cybin, visit www.cybin.com or follow the team on Twitter, LinkedIn, YouTube, and Instagram.

C4 Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutic candidates that target and eliminate disease-causing proteins, primarily for the treatment of cancer and neurodegenerative disorders. The company's lead product candidate, CFT7455, is an orally bioavailable degrader designed to target IKZF1/3 for multiple myeloma and other lymphomas. Additionally, C4 is advancing CFT8634, which targets BRD9 for specific solid tumors, alongside programs aimed at genetically defined resistant solid tumors. C4 Therapeutics utilizes its proprietary Degronimid platform, which allows for the selective degradation of proteins through the natural ubiquitin/proteasome system, potentially addressing previously undruggable targets while minimizing drug resistance. The company has formed strategic collaborations with major industry players, and it aims to make a significant impact on the treatment landscape through its advanced protein degradation science and multiple development programs. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics continues to innovate in the field of targeted therapy.

Pandion Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapeutics for patients with autoimmune diseases and inflammatory conditions. The company's lead product candidate, PT101, is an engineered variant of interleukin-2 currently undergoing Phase 1a clinical trials for moderate-to-severe ulcerative colitis and systemic lupus erythematosus. In addition to PT101, Pandion's pipeline includes PT627, a systemic PD-1 agonist in preclinical studies, and PT001 and PT002, which are bifunctional molecules designed for tissue-selective immunomodulation in the gastrointestinal tract. These molecules utilize a unique approach to achieve localized treatment, potentially enhancing efficacy and safety compared to traditional therapies. Pandion also collaborates with Astellas Pharma to develop locally acting immunomodulators targeting autoimmune diseases of the pancreas. The company is headquartered in Watertown, Massachusetts.

Akouos, Inc. is a biotechnology company based in Boston, Massachusetts, dedicated to developing gene therapies aimed at restoring, improving, and preserving hearing for individuals affected by various inner ear disorders. Founded in 2016, the company utilizes an innovative platform that includes a proprietary adeno-associated viral vector library and a unique delivery method. Its leading product candidate, AK-OTOF, targets hearing loss resulting from mutations in the OTOF gene. Akouos focuses on genetically-defined patient populations, addressing conditions ranging from single genetic mutations to hearing loss caused by ototoxic drugs and aging. The company has established strategic partnerships with key institutions, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing precision genetic therapies for sensorineural hearing loss.

Cerebral Therapeutics, Inc. is a clinical-stage pharmaceutical company based in Aurora, Colorado, focused on developing implanted drug-device combination therapies for neurologic diseases, particularly refractory epilepsy. The company utilizes a refillable catheter and pump system to deliver a continuous intracerebroventricular (ICV) dose of its formulation of the anti-epileptic drug valproic acid, known as CT-010. This innovative approach aims to bypass the blood-brain barrier, enhancing treatment outcomes for patients with chronic neurological conditions. Founded in 2010, Cerebral Therapeutics is dedicated to improving the lives of individuals affected by uncontrolled neurological diseases.

Achilles Therapeutics is a developer of immunotherapies intended to offer next-generation, patient-specific therapies to treat cancer. The company's immunotherapies harness the immune system to destroy cancer cells that target truncal tumor neo-antigens and flags to the immune system present on the surface of every cancer cell, enabling scientists to target and destroy tumors without harming healthy tissues.

Freenome, Inc. is a biotechnology company based in South San Francisco, California, founded in 2014. It specializes in developing artificial intelligence-driven genomic solutions for the early detection of cancer. The company focuses on creating simple, non-invasive blood tests that can identify early-stage cancer and assist in treatment selection, thus enabling proactive disease management. Freenome’s multi-omics platform employs proprietary algorithms to enhance disease screening processes, aiming to facilitate timely interventions and improve treatment effectiveness. In addition to its diagnostic products, Freenome also offers clinical research services to support its mission of transforming cancer management through systematic early detection.

Satsuma Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing innovative treatments for migraine. The company's lead product candidate, STS101, is a drug-device combination that features a proprietary dry-powder formulation of dihydroergotamine mesylate. This product is designed for self-administration through a pre-filled, single-use nasal delivery device and is currently undergoing Phase III clinical trials. Founded in 2016 and based in South San Francisco, California, Satsuma aims to provide an effective therapeutic option for individuals suffering from acute migraine episodes.

Prevail Therapeutics is a biotechnology company focused on developing novel gene therapies for patients with Parkinson’s disease and other neurodegenerative diseases. Prevail was launched in 2017 by The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, and is headquartered in New York, NY.

Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on developing and commercializing therapeutics for rare genetic disorders of hemoglobin, including sickle cell disease and beta-thalassemia. Founded in 2016, the company is advancing its lead product candidate, IMR-687, which is a highly selective small molecule inhibitor of PDE9. IMR-687 is designed as an oral, once-a-day treatment that aims to modify disease through a multimodal mechanism of action affecting various cell types, including red and white blood cells. Imara is dedicated to addressing the unmet medical needs of patients suffering from these serious hemoglobinopathies.

Attune Pharmaceuticals is a private biotechnology company focused on discovering and developing novel orally-administered small-molecule therapeutics for the treatment of rare diseases.

Black Diamond Therapeutics is a precision oncology medicine company focused on discovering and developing small molecule therapies that are tumor-agnostic. The company’s lead product candidate, BDTX-189, targets non-canonical and oncogenic mutations in ErbB kinases, specifically the epidermal growth factor receptor and tyrosine-protein kinase. In addition to BDTX-189, Black Diamond is developing allosteric-EGFR mutation inhibitors and other early-stage pipeline programs that address allosteric mutations in kinases associated with cancer and rare genetic diseases. The company's approach is centered on its Mutation-Allostery-Pharmacology platform, which aims to create targeted treatments for genetically defined cancers, especially for patients with limited existing treatment options. Founded in 2014 and headquartered in Cambridge, Massachusetts, Black Diamond Therapeutics was previously known as ASET Therapeutics until its name change in January 2018.

SeLux Diagnostics, Inc. is a biotechnology company based in Charlestown, Massachusetts, founded in 2014. It specializes in developing a next-generation phenotyping platform for antibiotic susceptibility testing and diagnostics of infectious diseases. The company utilizes a synthetic amplifier technology that enhances the speed and complexity of antibiotic susceptibility testing, facilitating quicker and more accurate selection of personalized antimicrobial therapies for patients. By streamlining the identification of effective treatments, SeLux Diagnostics aims to improve patient care, reduce hospital stays, and tackle the growing issue of antibiotic resistance by minimizing the reliance on broad-spectrum antibiotics.

WhiteSwell Limited is a Galway-based company focused on advancing the treatment of acute decompensated heart failure (ADHF), a condition that leads to significant hospitalizations and emergency room visits. Founded in 2014, WhiteSwell has developed a unique catheter-based medical device that targets the lymphatic system, enabling the removal of excess fluid from both interstitial and intravascular spaces without compromising renal function. By creating a low-pressure zone at the thoracic duct outlet, the device reduces resistance to lymphatic drainage, thereby improving clinical outcomes for patients suffering from this serious condition. The company also has offices in Shefayim, Israel, and Palo Alto, California, emphasizing its commitment to addressing the critical need for effective and safe treatment options for ADHF, which is associated with high mortality rates.

Stoke Therapeutics, Inc. is an early-stage biopharmaceutical company headquartered in Bedford, Massachusetts, focused on developing novel antisense oligonucleotide medicines to address the underlying causes of severe genetic diseases. The company employs its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) technology to enhance gene expression by targeting RNA splicing, thereby selectively restoring protein levels. Its lead product candidate, STK-001, is currently in clinical testing for the treatment of Dravet syndrome, a serious and progressive genetic epilepsy. Established in 2014, Stoke Therapeutics also collaborates with Invitae Corporation to provide genetic testing services, further supporting its mission to advance gene-targeted therapies for patients with severe genetic conditions.

Lyra Therapeutics, Inc. is a clinical-stage therapeutics company focused on the development and commercialization of novel integrated drug and delivery solutions for the localized treatment of patients with ear, nose and throat diseases. The company’s proprietary technology platform, XTreo, is designed to precisely and consistently deliver medicines directly to the affected tissue for sustained periods with a single administration. The company’s initial product candidates, LYR-210 and LYR-220, are bioresorbable polymeric matrices designed to be administered in a brief, non-invasive, in-office procedure and intended to deliver up to six months of continuous drug therapy to the sinonasal passages for the treatment of CRS. The therapeutic embedded within LYR-210 and LYR-220 is mometasone furoate, which is the active ingredient in various FDA-approved drugs and has a well-established efficacy and safety profile.

Precision BioSciences, Inc. is a biotechnology company based in Durham, North Carolina, focused on genome editing and the development of therapeutic products. Utilizing its proprietary ARCUS genome editing platform, the company aims to create innovative solutions for human diseases and food applications. Precision BioSciences operates through two primary segments: Therapeutic and Food. The Therapeutic segment is engaged in developing allogeneic CAR T immunotherapies, with several candidates in clinical trials targeting various types of cancers, including acute lymphoblastic leukemia and multiple myeloma. Additionally, the company is involved in in vivo gene correction activities and has collaborations for developing treatments for chronic Hepatitis B. The Food segment concentrates on creating nutrition products. Founded in 2006, Precision BioSciences is dedicated to translating advanced gene editing technology into impactful products across the life sciences.

Eidos Therapeutics, Inc. is a clinical stage biopharmaceutical company based in San Francisco, California, that specializes in developing treatments for diseases related to transthyretin (TTR) amyloidosis. The company is advancing AG10, an orally administered small molecule currently in phase 3 clinical trials, which aims to stabilize tetrameric TTR and interrupt the molecular processes leading to amyloidosis. Founded in 2013, Eidos Therapeutics operates as a subsidiary of BridgeBio Pharma, which focuses on creating genetically targeted therapies designed to address the underlying causes of various diseases. Through its innovative approach, Eidos Therapeutics seeks to provide potentially disease-modifying therapies to meet the significant medical needs associated with TTR-related conditions.

Crinetics Pharmaceuticals is a clinical-stage pharmaceutical company dedicated to the discovery and development of innovative therapeutics for rare endocrine diseases and endocrine-related tumors. The company's lead product candidate, paltusotine, is an oral selective nonpeptide somatostatin receptor type 2 biased agonist currently undergoing Phase I clinical trials for the treatment of acromegaly and neuroendocrine tumors. In addition to paltusotine, Crinetics is advancing other candidates, including CRN01941, an oral selective nonpeptide sst2 biased agonist for neuroendocrine tumors, an oral selective somatostatin type 5 receptor agonist for congenital hyperinsulinism, and an oral adrenocorticotropic hormone antagonist targeting Cushing’s disease and congenital adrenal hyperplasia. Founded in 2008 and headquartered in San Diego, California, Crinetics Pharmaceuticals aims to leverage its internal discovery efforts and objective hormonal biomarker endpoints to effectively navigate the development process and bring meaningful treatments to market.

TARIS Biomedical, Inc. is a therapeutically focused urology company based in Lexington, Massachusetts, established in 2008. The company specializes in developing innovative treatments for challenging bladder diseases, including bladder cancer and overactive bladder. TARIS's unique drug delivery system allows for the continuous release of medications within the bladder over extended periods, ranging from weeks to months. This system is designed to be deployed and retrieved through minimally invasive, in-office procedures, enabling a tailored approach to drug release that meets the specific needs of various bladder conditions. As of December 2019, TARIS Biomedical operates as a subsidiary of Janssen Research & Development, LLC.