RA Capital Management

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

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.

Ajax Therapeutics is a biotechnology company based in New York City that focuses on developing innovative small molecule therapies for hematologic malignancies. The company utilizes computational chemistry and structure-based technologies to create targeted treatments that address critical cytokine signaling pathways involved in these diseases. By integrating insights from disease mechanisms, genetics, and structural biology with an advanced computational drug discovery platform, Ajax Therapeutics aims to design precise therapeutics that meet significant unmet medical needs for patients suffering from hematologic malignancies.

Bluejay Therapeutics is a biopharmaceutical company dedicated to developing cures for infectious diseases, with a primary focus on chronic Hepatitis B. This condition poses a significant global health challenge, and the company aims to address the urgent need for effective treatments. Bluejay is advancing two innovative therapeutic approaches: best-in-class fully human IgG1 anti-HBs monoclonal antibodies and first-in-class oral small molecule inhibitors targeting HBsAg. These strategies are designed to reduce hepatitis B surface antigen levels and restore adaptive immunity, potentially leading to a functional cure for patients. Additionally, the company’s research encompasses therapeutic options for chronic Hepatitis D, further solidifying its commitment to improving immunity and recovery for individuals affected by serious viral and liver diseases.

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.

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.

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.

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.

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.

Star Therapeutics is a biotechnology company focused on developing innovative therapies for a wide range of rare diseases. The company aims to identify diseases that share common biological characteristics and create novel therapeutics that can effectively treat multiple conditions with a single treatment. This approach not only addresses the needs of patients suffering from rare diseases but also presents a unique opportunity for pharmaceutical companies to broaden their therapeutic offerings within a streamlined pipeline. By concentrating on life-changing therapies, Star Therapeutics aspires to make a significant impact on the lives of millions of patients.

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

Spyglass Pharma specializes in developing innovative treatments for chronic ophthalmic diseases, leveraging advanced technology to enhance patient care and vision. The company focuses on creating ophthalmic devices and a drug delivery platform that addresses unmet needs in areas such as cataracts and lens replacement surgeries. Its delivery system is designed to be implanted alongside intraocular lenses during standard cataract procedures, allowing surgeons to administer multi-year therapies for chronic conditions. This approach aims to improve the accessibility and affordability of quality eye treatments for patients, ensuring better outcomes in ophthalmic care.

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.

Alentis Therapeutics AG is a clinical-stage biopharmaceutical company based in Basel, Switzerland, focused on developing innovative therapies for advanced liver diseases, including liver fibrosis, cirrhosis, and liver cancer. Established in 2019, Alentis is recognized for its pioneering work in targeting CLDN1+ cancers through the use of anti-CLDN1 antibody-drug conjugates and other therapeutic modalities. The company's research platform incorporates clinically relevant read-outs and single-cell RNA sequencing of patient liver tissues, which aids in understanding and treating fibrotic diseases. Alentis aims to modify and reverse disease progression, ultimately addressing significant health challenges associated with liver-related conditions.

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.

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.

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.

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.

DICE Therapeutics is a biopharmaceutical company that utilizes its proprietary technology platform to develop a pipeline of novel oral therapeutic candidates aimed at treating chronic diseases, particularly in the field of immunology. The company focuses on creating oral therapeutics that target well-established proteins, intending to achieve efficacy similar to existing systemic biologics. DICE's DELSCAPE platform is specifically designed to discover selective oral small molecules capable of modulating protein-protein interactions effectively. Its lead candidate, S011806, is an oral antagonist targeting interleukin-17, a key player in various immunological conditions. In addition to S011806, DICE is developing other oral therapeutic candidates that target α4ß7 integrin and αVß1/αVß6 integrin, aimed at treating inflammatory bowel disease and idiopathic pulmonary fibrosis, respectively.

Exo is a medical device startup focused on developing a handheld ultrasound platform for imaging and therapeutic applications. The company aims to provide affordable and user-friendly medical imaging solutions for healthcare professionals globally. By integrating advanced nano-materials, innovative sensor technologies, and sophisticated signal processing with the cost efficiencies of semiconductor manufacturing, Exo significantly lowers the costs associated with medical imaging. This technology allows healthcare providers to make vital, real-time decisions that enhance patient outcomes. Exo's commitment to high-quality, accessible imaging represents a significant advancement in medical technology, making effective diagnostic tools available at an unprecedented price point.

Element Biosciences, Inc. is a life science company based in San Diego, California, that specializes in developing innovative genetic analysis tools for research and diagnostics. Established in 2017, the company focuses on creating a modular and high-performing DNA sequencing platform designed to provide high-quality data and workflow flexibility. Their proprietary technology enhances the signal-to-noise ratio and incorporates advancements in surface chemistry, instrumentation, and biochemistry. This approach significantly reduces both operational and capital costs while ensuring superior sequencing data quality, thereby making next-generation sequencing technology more accessible to medical researchers and improving their research capabilities.

NiKang Therapeutics is an early-stage biotech company focused on discovering and developing innovative small molecule oncology medicines to help patients with unmet medical needs. Its discovery approach is informed by target structure biology and capitalizes on structure-based drug design enabling a rapid and efficient discovery and development of proprietary drug candidates with the most desirable pharmacological features into clinical studies.

Ceribell, Inc. is a medical technology company that designs and manufactures devices for electroencephalography (EEG) data acquisition and interpretation, aimed at improving the diagnosis and management of neurological conditions. Its flagship product, the Ceribell EEG System, includes a flexible EEG Headband that accommodates various hair types and head sizes, alongside a portable EEG Recorder that ensures clinical-quality data and on-device display. The system is designed to be set up by any healthcare provider within six minutes, making EEG testing more accessible and efficient. Notably, it features a unique function that converts brainwaves into sound, allowing for quicker detection of seizures without the need for specialized technicians. This innovation facilitates earlier diagnosis and targeted treatment for patients, particularly for non-convulsive seizures, thereby reducing the risk of complications and improving overall patient outcomes. Established in 2014 and based in Mountain View, California, Ceribell is committed to enhancing care in hospital ICUs, inpatient units, and emergency departments.

Century Therapeutics is a biotechnology company focused on developing advanced cell therapies using induced pluripotent stem cells (iPSCs) to treat various cancers, including hematologic and solid tumors. Founded in 2018 and based in Philadelphia, Pennsylvania, the company utilizes a unique allogeneic iPSC-derived cell therapy platform that integrates gene editing, protein engineering, and robust manufacturing capabilities. This innovative approach allows for the generation of modified immune effector cells, such as natural killer (NK) and T cells, designed to meet significant unmet medical needs. By employing techniques like CRISPR-mediated gene editing and proprietary chimeric antigen receptors, Century Therapeutics aims to enhance cell product performance and minimize rejection by the host immune system, thereby improving the efficacy of cancer treatments.

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.

Xilio Therapeutics is a clinical-stage biotechnology company focused on developing innovative immunotherapies to enhance the immune system's ability to combat cancer. The company specializes in tumor-selective immunotherapies that leverage its proprietary technology to maximize the effectiveness of existing immuno-oncology treatments while minimizing side effects outside the tumor. Its lead product, XTX201, is designed to induce immune activity specifically within tumors, demonstrating improved antitumor efficacy. Xilio Therapeutics aims to deliver significant improvements in patient outcomes by utilizing its platform to create a pipeline of novel therapies, including tumor-activated cytokines, antibodies, and immune cell engagers, all targeted to optimize therapeutic effects within the tumor microenvironment. Founded in 2015 and based in Waltham, Massachusetts, the company was previously known as Akrevia Therapeutics before rebranding in March 2020.

Cyteir Therapeutics, Inc. is a clinical-stage biotechnology company based in Lexington, Massachusetts, focused on developing innovative therapies that leverage the biology of DNA repair for cancer treatment and synthetic lethality in autoimmune diseases. The company utilizes its expertise in DNA damage response biology to create a pipeline of small molecule therapeutics aimed at selectively targeting vulnerabilities in cancer cells and inducing their self-destruction through DNA damage overload. Cyteir's lead compound, CYT-0851, is an oral investigational drug designed to inhibit RAD51-mediated homologous recombination, which is crucial for repairing double-strand DNA breaks. The company is dedicated to addressing a variety of hematological malignancies, solid tumors, and chronic autoimmune disorders through its integrated drug development platform. Founded in 2012, Cyteir is committed to advancing next-generation therapies that can improve patient outcomes in challenging disease states.

TScan Therapeutics is a biopharmaceutical company dedicated to discovering and developing innovative T cell receptor (TCR) engineered therapies to combat various cancers. Based in Waltham, Massachusetts, the company focuses on TCR-T therapies for both liquid cancers and solid tumors. Its lead candidates, TSC-100 and TSC-101, are designed to address hematologic malignancies, specifically targeting residual leukemia and preventing relapse after hematopoietic stem cell transplantation. In addition, TScan is advancing a multiplexed TCR-T therapy program aimed at treating a range of solid tumors. The company's proprietary platform identifies clinically relevant T cell antigens and minimizes off-target effects, enhancing the precision of its therapies. Founded in 2018, TScan Therapeutics is committed to improving outcomes for patients with serious diseases.

DICE Therapeutics is a biopharmaceutical company that utilizes its proprietary technology platform to develop a pipeline of novel oral therapeutic candidates aimed at treating chronic diseases, particularly in the field of immunology. The company focuses on creating oral therapeutics that target well-established proteins, intending to achieve efficacy similar to existing systemic biologics. DICE's DELSCAPE platform is specifically designed to discover selective oral small molecules capable of modulating protein-protein interactions effectively. Its lead candidate, S011806, is an oral antagonist targeting interleukin-17, a key player in various immunological conditions. In addition to S011806, DICE is developing other oral therapeutic candidates that target α4ß7 integrin and αVß1/αVß6 integrin, aimed at treating inflammatory bowel disease and idiopathic pulmonary fibrosis, respectively.

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.

Edgewise Therapeutics is a clinical-stage biopharmaceutical company based in Boulder, Colorado, that specializes in developing small molecule therapies for severe, rare musculoskeletal diseases. Founded in 2017, the company employs a precision medicine approach to create innovative treatments targeting various muscle disorders, including Duchenne and Becker muscular dystrophies, as well as limb girdle muscular dystrophies. Utilizing its proprietary muscle-focused drug discovery platform, Edgewise integrates expertise in muscle biology and small molecule engineering to identify and develop therapies that address genetically defined muscle disorders. The company emphasizes a holistic drug discovery method that assesses integrated muscle function, enabling it to create orally bioavailable therapies aimed at improving outcomes for patients with significant unmet medical needs in neuromuscular and cardiac 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.

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.

Silverback Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Seattle, Washington, that specializes in developing tissue-targeted therapeutics aimed at treating cancer, chronic viral infections, and other serious diseases. The company’s lead product candidate, SBT6050, is currently undergoing a Phase I/Ib clinical trial; it is a TLR8 agonist conjugated to a HER2-directed monoclonal antibody, targeting tumors such as breast, gastric, and non-small cell lung cancers. Additionally, Silverback is advancing SBT6290, which is in preclinical development and targets Nectin4, found in bladder, triple-negative breast, head and neck, and non-small cell lung cancers. The company is also developing SBT8230, aimed at treating chronic hepatitis B virus infection, and exploring other agents that utilize its proprietary technology to modulate pathways in oncology and fibrosis indications. Silverback's innovative ImmunoTAC platform enables the design of therapies that are systemically delivered but act specifically at disease sites, unlocking previously inaccessible pathways for treatment.

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.

Kinnate Biopharma Inc. is a biopharmaceutical company based in San Diego, California, focused on developing small molecule kinase inhibitors for the treatment of genomically defined cancers. Founded in 2018, the company aims to create precision oncology therapeutics to address the needs of underserved patient populations. Kinnate's lead product candidate, KIN002787, is a rapidly accelerated fibrosarcoma inhibitor currently in preclinical development for lung cancer, melanoma, and other solid tumors. Additionally, the company is working on KIN003, which targets specific alterations in the FGFR2 and FGFR3 genes, and has other small molecule research programs, including a Cyclin-Dependent Kinase 12 inhibitor. Kinnate Biopharma employs expertise in structure-based drug discovery and translational research to advance its pipeline of candidates aimed at treating cancers associated with specific oncogenic alterations.

Bolt Biotherapeutics, Inc. is a biotechnology company focused on developing innovative immunotherapy solutions for cancer treatment. The company employs its proprietary Boltbody platform, which features immune-stimulating antibody conjugates (ISAC) that link tumor-targeting antibodies with powerful immune stimulants. This approach aims to activate the immune system against tumors, effectively transforming cold tumors into immunologically active ones. Bolt Biotherapeutics is advancing several candidates in its pipeline, including BDC-1001, a monotherapy targeting HER2-expressing solid tumors, and BDC-3042, among others. Founded in 2015 and headquartered in Redwood City, California, the company leverages its expertise in myeloid biology and cancer drug development to create targeted therapies that harness both innate and adaptive immune responses.

Everest Medicines Limited is a clinical-stage biopharmaceutical company based in Shanghai, China, founded in 2017. The company focuses on licensing, developing, and commercializing innovative therapies to meet critical unmet medical needs in Greater China and other Asia Pacific markets. Its diverse portfolio includes eight clinical-stage drug candidates aimed at treating various conditions such as oncology, immunology, cardio-renal diseases, and infectious diseases. Everest Medicines aims to partner with innovative global companies to bring transformative pharmaceutical solutions to the Emerging Market, leveraging its expertise in clinical development, regulatory affairs, and business operations. The company's pipeline features several notable candidates, including Nefecon, EVER001, PTX-COVID19-B, and Eravacycline, among others.

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.

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.

Arcutis Biotherapeutics is a clinical-stage biopharmaceutical company dedicated to developing and commercializing treatments for immune-mediated dermatological diseases. Founded in 2016 and based in Westlake Village, California, the company is focused on addressing unmet medical needs within the field of immuno-dermatology. Its lead product candidate, a topical cream formulation of roflumilast known as ARQ-151, is undergoing Phase III clinical trials for plaque psoriasis and atopic dermatitis. Additionally, Arcutis is working on several other candidates, including ARQ-154, a topical foam for seborrheic dermatitis and scalp psoriasis, ARQ-252, a selective inhibitor for hand eczema and other inflammatory conditions, and ARQ-255, designed to penetrate deeper into the skin for alopecia areata treatment. The company aims to leverage advances in immunology and inflammation to create effective therapies for challenging skin diseases.

Sojournix, Inc. is a biotechnology company developing innovative medicines for women's health and endocrine disorders.

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.

Synthorx, Inc. is a biopharmaceutical company based in La Jolla, California, focused on developing innovative cytokine Synthorin programs for the treatment of cancer and autoimmune disorders. The company's lead product candidate, THOR-707, is a variant of recombinant human IL-2 being explored for various solid tumors, both as a standalone treatment and in combination with immune checkpoint inhibitors. Additionally, Synthorx is working on IL-2 Synthorin for autoimmune conditions, IL-10 Synthorin for immuno-oncology, and IL-15 Synthorins to enhance immune regulation. The company employs advanced synthetic biology techniques, utilizing a novel DNA base pair to create proteins with enhanced specificity, safety, and efficacy. These developments enable the manufacturing of improved therapeutics that can better modulate pharmacological properties compared to traditional biologics. Synthorx, which was established in 2014 and previously known as Alinos, Inc., operates as a subsidiary of Sanofi and holds exclusive rights to its innovative synthetic biology technology derived from research at The Scripps Research Institute.

Arvinas, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, that specializes in the discovery, development, and commercialization of therapies designed to degrade disease-causing proteins. The company’s lead product candidates include ARV-110, a proteolysis-targeting chimera (PROTAC) currently in phase I clinical trials for treating metastatic castration-resistant prostate cancer, and ARV-471, which targets the estrogen receptor for patients with metastatic ER-positive/HER2-negative breast cancer. Additionally, Arvinas is developing other PROTACs aimed at degrading various androgen receptor mutations and has initiatives focused on treating neurodegenerative diseases, including tauopathies. Arvinas collaborates with major pharmaceutical companies such as Pfizer, Genentech, Roche, and Bayer to advance its innovative therapeutic approaches. Founded in 2015, Arvinas is dedicated to improving the lives of patients with debilitating and life-threatening conditions through its unique protein degradation technology.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Spero Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative treatments for multi-drug resistant (MDR) bacterial infections and rare diseases. The company’s leading product candidate, tebipenem pivoxil hydrobromide, is an oral antibiotic designed to treat MDR Gram-negative infections in adults. Additionally, Spero is advancing SPR206, an intravenous agent targeting MDR Gram-negative infections, and SPR720, an oral antibiotic aimed at treating pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics collaborates with various organizations, including Meiji Seika Pharma for tebipenem HBr, Everest Medicines for SPR206 in Asia, and the Bill & Melinda Gates Medical Research Institute for SPR720. Founded in 2013, Spero leverages a strong team in chemistry and microbiology to address significant unmet medical needs in infectious disease treatment.

Ivantis Inc. is a medical device company based in Irvine, California, focused on developing innovative treatments for primary open-angle glaucoma. Founded in 2007, the company specializes in a minimally invasive intracanalicular scaffold that alleviates high intraocular pressure by restoring the natural outflow pathway in Schlemm's canal. This device aims to provide a less invasive and more effective solution for the over 60 million individuals worldwide affected by this condition. In addition to its primary focus on glaucoma, Ivantis intends to explore innovative treatments for other ophthalmic diseases as it expands its product offerings. The company also provides a Session Persistence Server to support clinical trials, ensuring data protection for telnet task workers and terminal emulation users across various devices and operating systems.

ShockWave Medical, Inc. is a medical device company based in Santa Clara, California, focused on developing and commercializing intravascular lithotripsy technology to treat calcified plaque associated with peripheral vascular, coronary vascular, and heart valve diseases. The company offers a range of catheters, including M5 for above-the-knee peripheral artery disease, C2 for coronary artery disease, and S4 for below-the-knee peripheral artery disease. Its Lithoplasty™ technology utilizes sonic pressure waves to facilitate the treatment of rigid vascular and valvular lesions, allowing for improved angioplasty outcomes and reduced reliance on stents. This innovative approach enhances vessel preparation before procedures such as drug eluting balloon placement and trans-catheter aortic valve replacement. ShockWave Medical serves a diverse clientele, including interventional cardiologists, vascular surgeons, and interventional radiologists, through a network of sales representatives, managers, and distributors across the United States and Europe. Incorporated in 2009, the company is committed to transforming cardiovascular care and improving patient outcomes.

G1 Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of innovative small molecule therapeutics for cancer treatment. The company is advancing several key drug candidates, including trilaciclib, an intravenous cyclin-dependent kinases (CDK) 4/6 inhibitor currently in Phase 1b/2 trials for extensive-stage small cell lung cancer (SCLC) and Phase 2 trials for first-line SCLC and metastatic triple-negative breast cancer. Additionally, G1 Therapeutics is developing lerociclib, an oral CDK4/6 inhibitor in Phase 1b/2a trials for breast cancer and Phase 1b trials for non-small cell lung cancer, as well as rintodestrant, an oral selective estrogen receptor degrader in Phase I/2 trials. The company partners with Quantum Leap Healthcare Collaborative to explore trilaciclib for neoadjuvant treatment of locally advanced breast cancer. Founded in 2008 and based in Research Triangle Park, North Carolina, G1 Therapeutics aims to address unmet needs in oncology through its novel therapeutic approaches.

Kala Pharmaceuticals, Inc. is a biopharmaceutical company headquartered in Watertown, Massachusetts, that specializes in developing therapies for diseases affecting mucosal tissues using its proprietary nanoparticle-based Mucus Penetrating Particles (MPP) technology. This innovative approach enhances drug distribution and pharmacokinetics by delivering biocompatible, drug-loaded particles that uniformly coat mucosal surfaces. The company's lead product candidate, KPI-121 0.25%, has successfully completed two Phase III clinical trials aimed at providing temporary relief for dry eye disease. Additionally, INVELTYS, another product, has also completed two Phase III trials for treating inflammation and pain following ocular surgery. Kala is advancing its pipeline, including KPI-285, a receptor tyrosine kinase inhibitor for retinal diseases, which is currently in preclinical studies. The company aims to address significant clinical needs while exploring research collaborations to enhance its product offerings.

Audentes Therapeutics, a clinical-stage biotechnology company based in San Francisco, is dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapies, including AT132 and AT342, which are in Phase I/II clinical studies for treating X-linked myotubular myopathy and Crigler-Najjar syndrome, respectively. Additionally, it is exploring treatments for Pompe disease and catecholaminergic polymorphic ventricular tachycardia, along with vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Audentes has established collaborative agreements for research and development with Genethon and the University of Pennsylvania. Founded in 2012, Audentes Therapeutics became a subsidiary of Astellas US Holding in 2020 and operates as Astellas Gene Therapies, focusing on leveraging its expertise in gene therapy research and commercialization.

Aclaris Therapeutics started by the founders of Vicept Therapeutics, is a privately held specialty pharmaceutical dermatology company focused on the development of novel dermatologic therapies. It is committed to identifying, developing, and commercializing innovative therapies to address significant unmet needs in dermatology, both medical and aesthetic, and immunology. Aclaris is focused on market segments with no FDA-approved medications or where treatment gaps exist.

Protagonist Therapeutics is a clinical-stage biopharmaceutical company focused on developing peptide-based product candidates to meet unmet medical needs in hematology and gastroenterology. The company is advancing several key products, including PTG-300, an injectable hepcidin mimetic in Phase II clinical trials for treating beta-thalassemia-related anemia, and PTG-200, an antagonist peptide that has completed Phase I trials aimed at moderate-to-severe Crohn's disease. Additionally, PN-943, an oral integrin antagonist, is in Phase I trials for inflammatory bowel disease. Protagonist also explores both oral and injectable peptide candidates for various gastrointestinal conditions. The company has established a collaboration with Janssen Biotech for the development and commercialization of PTG-200. Founded in 2006, Protagonist Therapeutics is headquartered in Newark, California.

Merus B.V. is a clinical-stage biomedical company focused on the discovery and development of antibody-based biopharmaceuticals, specifically in the field of immuno-oncology. The company utilizes its proprietary Oligoclonics technology to produce a class of human antibodies, which allows for the creation of a mixture of therapeutic antibodies targeting a common antigen from a single cell clone. Additionally, Merus develops bispecific antibody therapeutics known as Biclonics, which are designed to engage multiple targets simultaneously. The company's pipeline includes several product candidates such as MCLA-128, MCLA-117, and MCLA-158, among others, all aimed at addressing various cancer indications.

Blueprint Medicines is a biopharmaceutical company dedicated to developing targeted therapies for cancers and rare diseases driven by abnormal kinase activation. Founded in 2008 and based in Cambridge, Massachusetts, the company utilizes its proprietary Insights-to-Validation Platform and chemical library to create small molecule kinase inhibitors that address specific genomic drivers. Key drug candidates include avapritinib for systemic mastocytosis and gastrointestinal stromal tumors, BLU-263 for indolent systemic mastocytosis, and pralsetinib for RET-altered cancers such as non-small cell lung cancer and medullary thyroid carcinoma. Additionally, fisogatinib is under investigation for advanced hepatocellular carcinoma, while BLU-782 targets fibrodysplasia ossificans progressiva. Blueprint Medicines collaborates with several pharmaceutical companies to enhance its research and development efforts.

Zipline Medical, Inc. is a healthcare company specializing in the development and commercialization of non-invasive skin closure solutions. Founded in 2009 and based in Campbell, California, the company focuses on its innovative PRELOC™ technology platform, which provides uniform closure forces while minimizing tension that can lead to scarring. The flagship product, Zip Surgical Skin Closure, is applicable across a range of medical specialties, including cardiology, orthopedics, dermatology, and emergency medicine. The technology aims to combine the speed of staples with the cosmetic advantages of sutures, addressing the clinical need for rapid and effective skin closure while reducing the risk of surgical site infections. By eliminating skin punctures and decreasing the risk of needlestick injuries, Zipline Medical enhances surgical outcomes and patient care in various surgical procedures. The company operates as a subsidiary of Stryker Corporation.

Bellicum Pharmaceuticals is a clinical-stage biopharmaceutical company based in Houston, Texas, focused on developing innovative cellular immunotherapies for the treatment of hematological cancers and solid tumors. The company is advancing its product candidates, including BPX-601, an autologous GoCAR-T therapy aimed at solid tumors expressing prostate stem cell antigen, and BPX-603, a dual-switch GoCAR-T therapy targeting solid tumors that express human epidermal growth factor receptor 2. Bellicum utilizes its proprietary Chemical Induction of Dimerization technology platform, which allows for real-time control of immune system components to enhance therapeutic efficacy. The company collaborates with various institutions, including Adaptimmune Therapeutics and Baylor College of Medicine, to further its research and development efforts. Founded in 2004, Bellicum Pharmaceuticals continues to make strides in cancer treatment through its novel immunotherapeutic approaches.

Civitas Therapeutics is a biopharmaceutical company dedicated to developing and commercializing innovative pulmonary delivery therapies. Utilizing its proprietary ARCUS technology, the company creates therapeutics aimed at treating central nervous system and respiratory disorders. Civitas Therapeutics serves patients across the United States, focusing on delivering transformative treatment options to improve health outcomes.

Coherus Biosciences is a biopharmaceutical company that specializes in the development, manufacture, and commercialization of biologic therapeutics, with a primary focus on oncology and inflammatory diseases. The company has established a portfolio of FDA-approved products, including UDENYCA, a biosimilar to Neulasta, and is preparing to launch YUSIMRY, a biosimilar to Humira, in the United States in 2023. Coherus's development efforts emphasize process science, analytical characterization, and clinical-regulatory development. The company's pipeline includes candidates for Immunology, ophthalmology, and oncology, particularly targeting anti-tumor necrosis factor therapies. By leveraging its diverse portfolio, Coherus aims to build a robust immuno-oncology franchise while generating revenue primarily from the U.S. market.

Zipline Medical, Inc. is a healthcare company specializing in the development and commercialization of non-invasive skin closure solutions. Founded in 2009 and based in Campbell, California, the company focuses on its innovative PRELOC™ technology platform, which provides uniform closure forces while minimizing tension that can lead to scarring. The flagship product, Zip Surgical Skin Closure, is applicable across a range of medical specialties, including cardiology, orthopedics, dermatology, and emergency medicine. The technology aims to combine the speed of staples with the cosmetic advantages of sutures, addressing the clinical need for rapid and effective skin closure while reducing the risk of surgical site infections. By eliminating skin punctures and decreasing the risk of needlestick injuries, Zipline Medical enhances surgical outcomes and patient care in various surgical procedures. The company operates as a subsidiary of Stryker Corporation.

Dicerna Pharmaceuticals is a biotechnology company dedicated to the discovery and development of innovative treatments for rare inherited diseases and other conditions affecting the liver. Utilizing its proprietary ribonucleic acid interference (RNAi) technology, specifically the GalXC platform, Dicerna focuses on silencing disease-causing genes to develop pharmaceuticals for various therapeutic areas, including liver diseases, cardiovascular diseases, and cancers. Key product candidates include nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B virus infection, and additional programs targeting undisclosed rare liver diseases. The company has established strategic collaborations with several major pharmaceutical firms to enhance its research and development efforts. Founded in 2006 and headquartered in Lexington, Massachusetts, Dicerna Pharmaceuticals aims to address significant unmet medical needs through its innovative approaches.

T2 Biosystems, Inc. is an in vitro diagnostics company focused on developing advanced diagnostic products for detecting pathogens and biomarkers. Utilizing its proprietary T2 Magnetic Resonance technology, the company offers rapid and accurate testing for various medical conditions directly from unpurified patient samples, including whole blood, plasma, and urine. Key products include the T2Dx Instrument for diagnosing sepsis and Lyme disease, the T2Candida Panel for identifying Candida species, and the T2Bacteria Panel for detecting bacterial pathogens linked to sepsis. Additionally, T2 Biosystems provides the T2Resistance Panel for identifying antibiotic resistance markers and is developing other diagnostic panels for multi-drug resistant pathogens and Lyme disease strains. The company collaborates with organizations like Canon U.S. Life Sciences and Allergan Sales to enhance its testing capabilities. Founded in 2006 and based in Lexington, Massachusetts, T2 Biosystems aims to improve patient care and reduce healthcare costs through its innovative diagnostic solutions.