RA Capital Management

Jade Biosciences is a biopharmaceutical company dedicated to developing innovative therapies for unmet needs in autoimmune diseases. Its lead asset, JADE-001, targets the APRIL pathway for treating IgA nephropathy, with initial clinical trials planned. Additionally, Jade has two preclinical antibody discovery programs, JADE-002 and JADE-003. The company was founded upon assets licensed from Paragon Therapeutics.

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

Vaxess Technologies, Inc. is a biotechnology company based in Boston, Massachusetts, founded in 2011. The company specializes in the development and commercialization of silk biomaterial technologies, particularly the MIMIX microneedle platform, which facilitates transdermal delivery of vaccines and therapeutics. This innovative sustained release patch technology enhances vaccine administration by simulating a natural infection, promoting a robust and prolonged immune response, thereby improving vaccine effectiveness. Additionally, Vaxess’s MATRIX formulation and drying platform creates thermostable vaccines, therapeutics, and diagnostic products that do not require refrigeration, making them suitable for distribution in low-resource settings. Through these advanced solutions, Vaxess aims to improve patient outcomes in oncology care and infectious diseases while reducing treatment burdens for healthcare practitioners.

Edgewise Therapeutics, founded in 2017 and based in Boulder, Colorado, is a clinical-stage biopharmaceutical company dedicated to developing precision medicine therapies for severe, rare muscle disorders. The company's core expertise lies in muscle biology and small molecule engineering, which it employs through its proprietary drug discovery platform. This platform uses custom-built systems to measure integrated muscle function, enabling the identification of small molecule therapies targeting key proteins in muscle tissue. Edgewise's pipeline focuses on addressing genetically defined muscle disorders such as Duchenne muscular dystrophy, Becker muscular dystrophy, and limb-girdle muscular dystrophies.

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

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.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Be Biopharma, Inc. is a biopharmaceutical company focused on developing therapeutics derived from engineered B-cells in the immune system. Founded in 2020 and based in Cambridge, Massachusetts, Be Biopharma aims to address the limitations of existing cell and gene therapies, such as the need for chemotherapy prior to treatment and the challenges of providing multiple doses. The company employs precision genome editing to create durable and effective cell therapies, targeting a range of diseases, including cancer, autoimmune disorders, and infectious diseases. By enhancing the functionality of B-cell medicines, Be Biopharma seeks to improve treatment outcomes for conditions like Hemophilia B and other serious health issues.

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 to treat acute myeloid leukemia (AML) and is currently in preclinical development. VOR33 eHSCs are engineered to lack CD33, a protein commonly expressed by AML cells, thereby allowing for selective targeting of cancer cells while sparing normal cells. This innovative approach aims to minimize the toxicities associated with traditional immunotherapies, such as bispecific antibodies and CAR T cells, which often affect both cancerous and healthy cells. Vor Biopharma's mission is to provide safer and more effective treatment options for patients with hematological malignancies by creating therapies that protect healthy, functional cells from depletion during targeted cancer treatments.

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

Indapta Therapeutics is a privately held biotechnology company that focuses on the development of a natural killer (NK) cell therapy platform aimed at treating various blood and solid tumor cancers. The company specializes in commercializing a proprietary, off-the-shelf, non-engineered, and allogeneic G-NK cell designed to enhance the immune response and significantly improve the cytotoxicity of antibody therapies. By harnessing the potent antibody-dependent cellular cytotoxicity (ADCC) activity of these NK cells, Indapta Therapeutics enables healthcare providers to offer innovative treatment options for patients battling cancer.

ADCendo ApS is a biotech company focused on the development of innovative anti-cancer therapies, specifically through the creation of antibody-drug conjugates (ADCs). These conjugates are designed to target receptors that are overexpressed in various forms of cancer, addressing significant unmet medical needs in the treatment landscape. ADCendo's approach aims to enhance targeted therapy for patients suffering from conditions such as soft-tissue sarcoma, osteosarcoma, glioblastoma multiforme, and triple-negative breast cancer, among others. By developing new medicines that effectively target carcinomas—including breast, colon, pancreatic, prostate, ovarian, and renal cancers—ADCendo seeks to improve treatment outcomes for cancer patients.

Cidara Therapeutics, Inc. is a biotechnology company based in San Diego, California, established in 2012. It specializes in the discovery, development, and commercialization of innovative anti-infective therapies aimed at treating serious diseases. The company's lead product candidate, rezafungin acetate, is an echinocandin-class antifungal designed to address serious invasive fungal infections such as candidemia and invasive candidiasis, which are associated with high mortality rates. Additionally, Cidara is advancing its Cloudbreak platform, which focuses on developing antiviral Fc-conjugates for the prevention and treatment of various viral infections, including influenza, RSV, HIV, and coronaviruses. Through its unique therapeutic approaches, Cidara Therapeutics aims to enhance treatment paradigms and improve patient outcomes in the face of life-threatening illnesses.

Synapticure is a virtual clinical care platform dedicated to supporting patients with neurodegenerative diseases, including dementia, Parkinson's, and ALS, across the United States. Founded by individuals with personal experiences in these conditions, the company offers a comprehensive suite of services that includes access to specialized neurologists, speech-language therapy, and 24/7 care coordination. Synapticure leverages advances in science and technology to ensure patients receive tailored treatment and support, fostering a holistic approach to care. By integrating various healthcare services, the platform simplifies access to essential medical resources, including behavioral health support, all designed to enhance the quality of life for those affected by neurodegenerative disorders.

Aclaris Therapeutics is a clinical-stage biopharmaceutical company based in the United States, specializing in the development of innovative therapies for dermatological and immuno-inflammatory conditions. Founded by the creators of Vicept Therapeutics, the company is dedicated to addressing significant unmet needs in these fields, focusing on areas where no FDA-approved treatments exist or where treatment options are limited. Aclaris operates primarily in two segments: therapeutics and contract research. The therapeutics segment is engaged in the identification and development of novel drug candidates, while the contract research segment generates revenue through laboratory services. Among its developmental candidates is Zunsemetinib, an oral MK2 inhibitor aimed at treating immuno-inflammatory diseases.

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 treatment options, including both oral and injectable therapies, specifically targeting incretin, non-incretin, and combination approaches. By integrating proprietary health technology tools, Metsera aims to provide personalized care that meets the evolving demands of weight loss treatment. Its commitment to addressing multiple therapeutic targets positions the company to significantly impact the future of obesity management and related metabolic diseases.

Alentis Therapeutics AG is a clinical-stage biopharmaceutical company based in Basel, Switzerland, specializing in the development of innovative therapies for advanced liver diseases and cancers associated with Claudin-1 (CLDN1) expression. Founded in 2019, Alentis focuses on addressing conditions such as liver fibrosis, cirrhosis, and liver cancer through the use of anti-CLDN1 antibody-drug conjugates and other targeted treatments. The company's unique approach combines clinically relevant read-outs with advanced techniques like single-cell RNA sequencing of patient liver tissues, aiming to reverse disease progression and improve patient outcomes. Alentis Therapeutics is recognized for its pioneering role in exploring anti-CLDN1 therapeutics, contributing to advancements in the treatment of fibrotic diseases and CLDN1-positive tumors.

Innoventric is a medical device company based in Ness-Ziona, Israel, focused on developing innovative percutaneous solutions for Tricuspid Regurgitation and right heart disease. Founded in 2017, the company has made significant advancements with its proprietary Trillium™ technology, designed to treat Tricuspid Valve Regurgitation. Innoventric's device features a functional tricuspid valve replacement that employs cross-caval anchoring and a multi-valve design, aimed at enhancing blood flow regulation. The company has conducted over ten implantations of the Trillium™ technology as part of its first human clinical trial in Europe. Additionally, Innoventric maintains a comprehensive patent portfolio for the Trillium™ and related technologies, positioning itself as a key player in the structural heart medical device sector.

Eledon Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in Irvine, California, specializing in the development of innovative treatments for patients with disorders of the ear, nose, and throat, as well as for those undergoing organ or cellular transplantation and individuals with autoimmune and neurodegenerative diseases. The company’s lead product, OP0201, is a surfactant-based nasal aerosol designed for patients at risk for or suffering from otitis media. Additionally, Eledon has developed a foam-based drug delivery technology, OP0101 and OP0102, aimed at delivering medications to the ear, nose, and sinus cavities. The company also focuses on the CD40L pathway, with its main compound in development, tegoprubart, being an IgG1 anti-CD40L antibody that shows promise for treating autoimmune diseases and supporting organ transplants. Eledon Pharmaceuticals was formerly known as Novus Therapeutics, Inc. before its rebranding in January 2021.

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

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

Be Biopharma, Inc. is a biopharmaceutical company focused on developing therapeutics derived from engineered B-cells in the immune system. Founded in 2020 and based in Cambridge, Massachusetts, Be Biopharma aims to address the limitations of existing cell and gene therapies, such as the need for chemotherapy prior to treatment and the challenges of providing multiple doses. The company employs precision genome editing to create durable and effective cell therapies, targeting a range of diseases, including cancer, autoimmune disorders, and infectious diseases. By enhancing the functionality of B-cell medicines, Be Biopharma seeks to improve treatment outcomes for conditions like Hemophilia B and other serious health issues.

Enara Bio Limited is a biotechnology company based in Oxford, United Kingdom, specializing in the development of vaccines and immunotherapies for cancer treatment and prevention. Founded in 2016 and previously known as ErVaxx Limited, the company focuses on creating targeted cancer immunotherapies by exploring novel antigens derived from endogenous retroviral DNA sequences. Enara Bio's technology platform enables the identification and assessment of tumor specificity and immunogenic potential, facilitating improved strategies for combating various cancers. Through its innovative approach, the company aims to provide effective therapeutic options for a broad patient population.

Aktis Oncology is a biotechnology company focused on the discovery and development of a new class of targeted radiopharmaceuticals aimed at treating various solid tumor cancers. The company has developed proprietary platforms to create tumor-targeting agents optimized for alpha radiotherapy. These agents are engineered for high tumor penetration and prolonged residence time, allowing for effective tumor elimination while reducing the side effects typically associated with cancer treatments. Additionally, Aktis Oncology's innovative approach enables clinicians to visualize and confirm target engagement before administering therapeutic radioisotopes, enhancing treatment precision and efficacy.

Superluminal Medicines is a biotechnology company specializing in generative biology and chemistry. It employs a unique platform that combines deep biological and chemical expertise, machine learning, and proprietary big data infrastructure to rapidly generate candidate-ready compounds for drug discovery and development. By integrating high-throughput experimental data with advanced algorithms, the company aims to accelerate the identification and optimization of novel drug candidates, improving efficiency and success rates in small molecule drug development compared to traditional methods.

Halda Therapeutics is a research-stage drug discovery company dedicated to developing next-generation precision medicine. The company employs a unique therapeutic modality that serves as a platform for addressing various disease states. By focusing on the discovery of novel therapies that modify disease-causing pathways, Halda Therapeutics aims to enable healthcare providers to offer more targeted treatments for their patients. Through its innovative approach, the company seeks to advance the field of precision medicine and improve patient outcomes.

IDRx is a clinical-stage biopharmaceutical company focused on developing precision therapies for oncology, specifically targeting gastrointestinal stromal tumors. The company specializes in designing precision combination therapies that utilize engineered drug combinations to address key tumor escape mechanisms and enhance the duration of therapeutic responses. Through its innovative approach, IDRx aims to transform cancer treatment by enabling healthcare professionals to create targeted cancer medicines tailored to individual patient needs.

Eliem Therapeutics, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, that specializes in developing therapeutic drugs for hyperexcitability disorders affecting the nervous system. Founded in 2018, the company focuses on creating innovative treatments for conditions such as chronic pain, depression, epilepsy, and anxiety, which significantly impact millions of individuals. Eliem utilizes its expertise in neuroscience and translational medicine to advance a promising pipeline of drug candidates, including its lead candidate, ETX-155, aimed at addressing major depressive disorder and focal onset seizures. The company's mission centers on meeting unmet medical needs and improving patients' quality of life through its novel therapies.

Bright Peak Therapeutics is a biotechnology company focused on developing a range of immunotherapies aimed at treating cancer and autoimmune diseases. The company specializes in creating immuno-cytokines that possess pharmacological properties enabling tissue and cell-specific targeting of cytokine payloads. This innovative approach is designed to enhance the efficacy of treatments, ultimately improving patient outcomes and quality of life. By leveraging its expertise in cytokine therapeutics, Bright Peak aims to advance the field of immunotherapy and provide effective solutions for challenging medical conditions.

ADCendo ApS is a biotech company focused on the development of innovative anti-cancer therapies, specifically through the creation of antibody-drug conjugates (ADCs). These conjugates are designed to target receptors that are overexpressed in various forms of cancer, addressing significant unmet medical needs in the treatment landscape. ADCendo's approach aims to enhance targeted therapy for patients suffering from conditions such as soft-tissue sarcoma, osteosarcoma, glioblastoma multiforme, and triple-negative breast cancer, among others. By developing new medicines that effectively target carcinomas—including breast, colon, pancreatic, prostate, ovarian, and renal cancers—ADCendo seeks to improve treatment outcomes for cancer patients.

Gameto is a biotechnology company focused on advancing female reproductive health through innovative therapies. Recognizing the significant lack of treatment options in this underserved area, Gameto employs cutting-edge scientific techniques to develop solutions that enhance the quality of life for women. The company utilizes cellular engineering to create a platform that produces engineered ovarian and endometrial cell lines capable of mimicking the functions of natural cells, including hormone production and response. This platform underpins a portfolio of cellular therapeutics aimed at addressing various female reproductive diseases. Gameto's first initiative, Fertilo, targets improvements in in vitro fertilization (IVF) and egg freezing processes, striving to make them shorter, safer, and more effective. Additionally, the company is developing Deovo, an organoid model of the female reproductive system, and Ameno, a cell-based therapy designed to mitigate health issues related to primary ovarian insufficiency and menopause. Through these efforts, Gameto aims to transform the landscape of female reproductive health care.

Bicycle Therapeutics plc is a clinical-stage biopharmaceutical company based in Cambridge, United Kingdom, specializing in the development of a novel class of medicines known as Bicycles. These are synthetic short peptides designed to form two loops that stabilize their structure. The company's lead product candidate, BT1718, is a Bicycle Toxin Conjugate (BTC) currently undergoing Phase I/IIa clinical trials targeting tumors that express Membrane Type 1 matrix metalloprotease. Other oncology candidates include BT5528, also a BTC in Phase I/IIa trials targeting EphA2, and BT8009, which is in preclinical studies targeting Nectin-4. Additionally, Bicycle is advancing THR-149, a plasma kallikrein inhibitor that has completed Phase I trials for diabetic macular edema. The company collaborates with various biopharmaceutical organizations to address therapeutic areas such as anti-infective, cardiovascular, ophthalmology, and respiratory indications. Bicycle Therapeutics was incorporated in 2009 and aims to address significant unmet medical needs in oncology and beyond.

Vaxess Technologies, Inc. is a biotechnology company based in Boston, Massachusetts, founded in 2011. The company specializes in the development and commercialization of silk biomaterial technologies, particularly the MIMIX microneedle platform, which facilitates transdermal delivery of vaccines and therapeutics. This innovative sustained release patch technology enhances vaccine administration by simulating a natural infection, promoting a robust and prolonged immune response, thereby improving vaccine effectiveness. Additionally, Vaxess’s MATRIX formulation and drying platform creates thermostable vaccines, therapeutics, and diagnostic products that do not require refrigeration, making them suitable for distribution in low-resource settings. Through these advanced solutions, Vaxess aims to improve patient outcomes in oncology care and infectious diseases while reducing treatment burdens for healthcare practitioners.

AltruBio is a biopharmaceutical company dedicated to the development of targeted antibody therapeutics aimed at treating cancer and immune-related inflammatory diseases. With its research and development foundation established in Taipei, Taiwan, since 2000, the company focuses on addressing unmet medical needs through innovative therapeutic solutions. AltruBio has developed an efficient discovery platform that identifies and validates novel antibody therapeutics. The company is actively advancing several potential therapeutic antibodies that target various conditions, including autoimmune diseases and multiple forms of cancer, such as pancreatic, stomach, colorectal, ovarian, and lung cancers. By leveraging its expertise in antibody-drug conjugate therapy and developing specialized linker payloads, AltruBio strives to improve patient outcomes and enhance recovery from immune-mediated diseases.

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 design drugs that target critical cytokine signaling pathways involved in these cancers. By integrating insights from disease biology, genetics, and structural biology with an advanced computational drug discovery platform, Ajax Therapeutics aims to create precisely designed therapeutics that address significant unmet medical needs for patients suffering from hematologic malignancies.

iTeos Therapeutics is a clinical-stage biopharmaceutical company focused on developing immuno-oncology therapeutics aimed at improving outcomes for cancer patients. Founded in 2011 and headquartered in Gosselies, Belgium, with additional operations in Cambridge, Massachusetts, the company is advancing a pipeline of innovative drug candidates. Notable among these is EOS-850, a small molecule antagonist targeting the adenosine A2a receptor, currently undergoing an open-label Phase 1/2a clinical trial in adult patients. Another key candidate is EOS-448, an antagonist of TIGIT, which is also in Phase 1/2a trials. iTeos leverages expertise in tumor immunology to create small molecule immunomodulators designed to counteract cancer-induced immune suppression, thereby enhancing the effectiveness of existing treatments and emerging immunotherapies.

Bluejay Therapeutics is a private biopharmaceutical company dedicated to developing curative treatments for infectious diseases, with an initial focus on chronic Hepatitis B. This disease remains prevalent worldwide and has significant unmet medical needs. The company is pursuing two innovative approaches: fully human IgG1 anti-HBs monoclonal antibodies and first-in-class HBsAg oral small molecule inhibitors. These therapies aim to reduce hepatitis B surface antigen levels and restore adaptive immunity, potentially achieving a functional cure for patients.

Medicenna Therapeutics Corp. is a clinical-stage immunotherapy company based in Toronto, Canada, focused on the development and commercialization of novel cytokine-based therapies for cancer treatment. The company specializes in Empowered Cytokines (EC) and Superkines, including its lead product, MDNA55, which is currently undergoing Phase 2b clinical trials for recurrent glioblastoma. Medicenna's pipeline also features MDNA57, targeting solid tumors; MDNA109, an IL-2 agonist for cancer immunotherapy; MDNA209, an IL-2 antagonist for autoimmune diseases; MDNA413, a dual IL-4/IL-13 antagonist for various solid tumors and inflammatory conditions; and MDNA132, an IL-13 agonist aimed at treating solid tumors. Its innovative technology platform allows for precise delivery of therapeutic agents while minimizing damage to healthy cells, positioning Medicenna as a significant player in the immuno-oncology space. The company was founded in 2012 and continues to expand its research and development efforts to address a wide range of cancers and related diseases.

Zura Bio is a clinical-stage biotechnology company dedicated to developing innovative therapies for immune and inflammatory disorders. It focuses on advancing its pipeline of treatments through phase 2 development programs, including ZB-168 and torudokimab, which target autoimmune illnesses. ZB-168 is an anti-IL7R inhibitor designed to address disorders linked to IL7 and TSLP biological pathways, building on encouraging phase 1b data related to type 1 diabetes. Torudokimab, a human-affinity monoclonal antibody, is in phase 2 clinical development and works by neutralizing IL33. Through these initiatives, Zura Bio aims to create effective therapeutic options for patients suffering from complex immune-related conditions.

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

Eliem Therapeutics, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, that specializes in developing therapeutic drugs for hyperexcitability disorders affecting the nervous system. Founded in 2018, the company focuses on creating innovative treatments for conditions such as chronic pain, depression, epilepsy, and anxiety, which significantly impact millions of individuals. Eliem utilizes its expertise in neuroscience and translational medicine to advance a promising pipeline of drug candidates, including its lead candidate, ETX-155, aimed at addressing major depressive disorder and focal onset seizures. The company's mission centers on meeting unmet medical needs and improving patients' quality of life through its novel therapies.

TORL BioTherapeutics is a clinical-stage biopharmaceutical company dedicated to developing innovative, antibody-based therapies aimed at enhancing the lives of cancer patients. The company specializes in antibody-drug conjugates and other biologics, focusing on oncologic diseases with significant unmet medical needs. TORL BioTherapeutics employs a strategy that involves target identification and early discovery work, allowing them to selectively advance drug programs through novel licensing agreements. This approach enables their researchers to develop proprietary drugs with unique and optimized profiles, contributing to the advancement of cancer treatment options.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on developing precision oncology therapeutics. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond positively to specific treatments, thereby enhancing the effectiveness of its drug development efforts. Acrivon's pipeline features its lead program, ACR-368 (prexasertib), which is currently in Phase 2 trials, alongside several preclinical programs targeting key components of DNA damage response and cell cycle regulation, including WEE1 and PKMYT1. By concentrating on complex oncology drug targets that are often not addressed by traditional therapies, Acrivon aims to expedite the advancement of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials.

ARTARA THERAPEUTICS is a clinical-stage healthcare company focused on developing transformative therapies for individuals with rare and specialty diseases. Its lead program, TARA-002, is an investigational cell therapy designed to treat lymphatic malformations and non-muscle invasive bladder cancer. Additionally, the company is advancing intravenous choline chloride, an investigational phospholipid substrate replacement therapy aimed at addressing intestinal failure-associated liver disease. Based in New York, ARTARA THERAPEUTICS is dedicated to providing innovative solutions for patients with limited treatment options, leveraging a team that values creativity and diverse perspectives to expedite the development of its therapies.

Obsidian Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing next-generation cell and gene therapies aimed at enhancing adoptive immunotherapy for cancer patients. Its innovative approach utilizes pharmacologic operating systems that enable precise control over protein activity within cells. This technology allows for the creation of adoptive cell therapies with novel functionalities, which can be regulated by treating physicians using simple and safe orally active medications. Through these advancements, Obsidian Therapeutics seeks to improve upon existing cell therapies and expand treatment options for a broader patient demographic.

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

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

CervoMed is a clinical-stage company focused on developing therapeutic treatments for neurodegenerative diseases, particularly in the early stages of these illnesses. The firm specializes in creating drugs aimed at addressing conditions affecting the central nervous system, with a primary emphasis on Alzheimer's disease. CervoMed's innovative approach includes a drug that promotes functional recovery in patients experiencing acute neurological injuries, thereby enhancing the ability of healthcare professionals to effectively manage brain diseases and improve episodic memory functions in affected individuals.

Avalo Therapeutics is a clinical-stage biopharmaceutical company that focuses on developing highly targeted therapeutics for immune dysregulation and other areas of significant unmet clinical need. The company employs a precision medicine approach in its research and development efforts. Its lead asset, AVTX-009, is an anti-IL-1B monoclonal antibody designed to address inflammatory diseases. In addition to AVTX-009, Avalo's pipeline includes quisovalimab, an anti-LIGHT monoclonal antibody, and AVTX-008, a BTLA agonist fusion protein, which further exemplify its commitment to advancing innovative treatments for complex medical conditions.

Nkarta Therapeutics is a biopharmaceutical company focused on developing engineered natural killer (NK) cell therapies for treating various cancers and autoimmune diseases. Unlike T-cell therapies, NK cells have an inherent ability to recognize and destroy abnormal cells without genetic alteration, offering promise for diverse tumor types with potentially better tolerated side effects. Nkarta's approach involves leveraging its NK expansion platform technology alongside proprietary cell engineering techniques to generate a substantial supply of enhanced NK cells that can persistently target and eliminate cancer cells.

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

Cybin is a biopharmaceutical company dedicated to advancing research and development in psychedelic and medicinal mushrooms. It focuses on creating safe and effective therapeutics for mental health issues, with an emphasis on psilocybin-based products. The company is actively launching these products in jurisdictions where they are permitted and is engaged in clinical studies across North America and other regions. Through strategic partnerships with academic and institutional entities, Cybin seeks to develop novel compounds and delivery mechanisms, aiming to provide innovative treatments for various psychiatric and neurological conditions.

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

Lexeo Therapeutics is a biotechnology company specializing in adeno-associated virus (AAV)-mediated therapies for both rare and common diseases caused by single gene mutations. Headquartered in New York, the company maintains an integrated pipeline developed in collaboration with Weill Cornell Medicine's Department of Genetic Medicine. Lexeo focuses on advancing its clinical programs towards commercialization while continuing to research new therapies for various patient populations and unmet medical needs.

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

ProfoundBio is a clinical-stage biotechnology company dedicated to creating novel antibody-based therapeutics aimed at treating cancer. The company utilizes innovative proprietary technology platforms to develop a diverse pipeline of antibody-drug conjugate (ADC) candidates that target both solid tumors and hematological malignancies. These drug candidates are currently in various stages of discovery, preclinical, and clinical development. ProfoundBio's focus on advancing targeted therapeutics leverages the immune system's capabilities to potentially eradicate cancer, offering improved treatment options with curative potential for patients.

BioAge Labs, Inc. is a biotechnology company focused on developing therapies to treat aging and age-related diseases. The company utilizes a proprietary systems biology and artificial intelligence platform to identify significant drug targets that influence aging, leveraging extensive datasets to uncover the molecular drivers of age-related pathologies. Among its key offerings is BGE-117, a hypoxia-inducible factor prolyl hydroxylase inhibitor that activates HIF-1 target genes involved in various biological processes, including tissue regeneration and vascular remodeling. Another notable product is BGE-175, an orally administered inhibitor targeting the prostaglandin D2 DP1 signaling pathway, which is linked to an increased risk of mortality and infections, including those associated with immune aging. Founded in 2015 and headquartered in Richmond, California, BioAge Labs is dedicated to harnessing the biology of human aging to create innovative therapies for metabolic and age-related diseases.

Mineralys Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapies for hypertension. The company is advancing its lead product candidate, MLS-101, a highly selective and potent aldosterone synthase inhibitor licensed from Mitsubishi Tanabe Pharma Corporation. Mineralys aims to provide a targeted treatment for patients with uncontrolled hypertension, particularly those who do not achieve adequate blood pressure control despite using multiple antihypertensive medications. By addressing the underlying factors associated with elevated aldosterone levels, Mineralys seeks to improve treatment outcomes for individuals suffering from this condition.

PepGen Ltd. is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the next generation of nucleic acid therapeutics, particularly in the treatment of severe neuromuscular and neurologic diseases. Incorporated in 2018, PepGen has developed its Enhanced Delivery Oligonucleotide (EDO) platform, which is designed to improve the uptake and activity of conjugated oligonucleotide therapeutics. The company's innovative EDO peptides enhance tissue penetration, cellular uptake, and nuclear delivery, demonstrating their ability to transport oligonucleotides effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. PepGen aims to unlock the clinical potential of these transformative therapeutics, with a particular emphasis on the effective delivery of antisense oligonucleotides, and is advancing its novel conjugate therapeutics toward clinical application.

Tyra Biosciences, Inc. is a clinical-stage biotechnology company based in Carlsbad, California, dedicated to developing precision oncology therapies targeted at overcoming drug resistance in cancer treatment. Founded in 2018, the company utilizes its proprietary SNÅP platform to facilitate rapid and precise drug design by generating iterative molecular snapshots. This innovative approach allows Tyra to create next-generation therapies specifically aimed at addressing acquired resistance in tumors. The company's primary focus is on developing selective inhibitors of the Fibroblast Growth Factor Receptor (FGFR) family, which are implicated in approximately 7% of all cancers. Tyra's lead product candidate, TYRA-300, is designed to selectively inhibit FGFR3, initially targeting patients with metastatic urothelial carcinoma of the bladder and urinary tract.

Astria Therapeutics is a biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative therapies for patients affected by rare allergic and immunological diseases. The company focuses on creating life-changing treatments, with its lead program, STAR-0215, being a monoclonal antibody designed to inhibit plasma kallikrein, currently in preclinical development for hereditary angioedema. Additionally, Astria is advancing STAR-0310, another monoclonal antibody, which targets the OX40 receptor and is also in preclinical development for atopic dermatitis, a condition characterized by impaired skin barrier function and significant itching. Through its targeted approach, Astria Therapeutics aims to address unmet medical needs in the field of immunology.

Dianthus Therapeutics is a clinical-stage biotechnology company focused on developing innovative treatments for severe autoimmune diseases through targeted therapies. The company's lead candidate, DNTH103, is a monoclonal antibody designed to selectively inhibit a specific component of the immune system, offering potential advantages over existing complement therapies. This treatment aims to improve safety and efficacy for patients suffering from serious autoimmune and inflammatory conditions. Dianthus is committed to advancing its pipeline of next-generation complement therapeutics, supported by a team of experienced professionals in the biotech and pharmaceutical sectors. The company envisions that DNTH103 could be administered as a self-injection for certain patients, enhancing convenience and accessibility in treatment.

Vaxart, Inc. is a clinical-stage biotechnology company based in South San Francisco, California, specializing in the discovery and development of oral recombinant protein vaccines using its proprietary vaccine platform. The company is focused on creating oral vaccines that can generate broad and durable immune responses against various infectious diseases. Its product pipeline includes an oral tablet vaccine for norovirus, which has completed Phase I clinical trials, a seasonal influenza vaccine currently in Phase II trials, and vaccines targeting respiratory syncytial virus and coronavirus. Additionally, Vaxart is developing therapeutic vaccines for human papillomavirus-related conditions. The company aims to improve vaccination rates by offering a convenient, room temperature-stable tablet format, which is easier to distribute and administer compared to traditional injectable vaccines. Vaxart also has a research collaboration with Janssen Vaccines & Prevention B.V. to explore its oral vaccine platform for a universal influenza vaccine program.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Arvinas, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, that specializes in the discovery, development, and commercialization of innovative therapies aimed at degrading disease-causing proteins. The company's lead product candidates include ARV-110, a proteolysis targeting chimera (PROTAC) currently in phase I clinical trials for metastatic castration-resistant prostate cancer, and ARV-471, which targets the estrogen receptor for patients with metastatic ER-positive/HER2-negative breast cancer. In addition to these, Arvinas is developing various other PROTACs aimed at degrading clinically relevant androgen receptor mutations and treatments for neurodegenerative diseases, including tauopathies. The company has established collaborations with prominent pharmaceutical firms such as Pfizer Inc., Genentech, and Bayer AG, enhancing its research capabilities and reach within the biopharmaceutical landscape. Founded in 2015, Arvinas aims to improve patient outcomes by eliminating harmful proteins from the body through its proprietary technology.

Rampart Bioscience is a biotechnology company focused on developing gene medicines that aim to deliver long-lasting treatments for various diseases. The company has created proprietary development and delivery platforms that generate optimized therapeutics in a revocable, non-viral format. By integrating expertise from multiple scientific disciplines, including gene delivery, protein sciences, and clinical translation, Rampart Bioscience works to assist patients with genetically driven diseases. Their innovative approach seeks to enhance the effectiveness and safety of gene therapies, ultimately improving patient outcomes.

Harpoon Therapeutics, Inc. is a clinical-stage immunotherapy company based in South San Francisco, California, focused on developing a novel class of T cell engagers to harness the body's immune system for treating cancer and other diseases. The company utilizes its proprietary TriTAC (Tri-specific T cell Activating Construct) platform to create engineered proteins that direct T cells to target and kill cells expressing specific antigens. Harpoon's lead product candidate, HPN424, is currently undergoing Phase I clinical trials for metastatic castration-resistant prostate cancer. Additionally, the company is advancing other candidates, including HPN536, which is in Phase I/IIa trials for ovarian cancer and MSLN-expressing tumors, as well as HPN217 for multiple myeloma and HPN328 for small cell lung cancer. Harpoon Therapeutics was founded in 2015 and is committed to addressing unmet medical needs in oncology through innovative therapies.

Tourmaline Bio is a clinical biotechnology company focused on developing transformative medicines aimed at improving the lives of patients suffering from immune diseases. The company is in the late stages of clinical development and is centered on its lead product candidate, TOUR006, a fully human monoclonal antibody designed to selectively target interleukin-6 (IL-6), a significant proinflammatory cytokine implicated in various autoimmune and inflammatory disorders. The IL-6 antibody class has a well-established history, with over two decades of clinical and commercial experience, having successfully treated more than a million patients with diverse autoimmune and inflammatory conditions. Through its innovative research, Tourmaline Bio seeks to address the unmet medical needs of patients affected by life-altering immune diseases.

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

Adela is a company focused on developing innovative technologies for the early detection of cancer and other serious health conditions through routine blood testing. Utilizing a genome-wide methylome enrichment platform, Adela captures and analyzes information from the entire methylome, effectively identifying and targeting highly informative methylated regions of the genome for sequencing. This approach enables the detection of minimal residual disease and aids in tumor classification using plasma cell-free DNA methylomes. Adela's technology is designed to assist healthcare professionals in cancer detection, as well as in prenatal diagnostics, cardiology, and the monitoring of immune responses. Through its advancements, Adela aims to improve diagnostic accuracy and patient outcomes in the realm of high-morbidity and high-mortality conditions.

Vaxess Technologies, Inc. is a biotechnology company based in Boston, Massachusetts, founded in 2011. The company specializes in the development and commercialization of silk biomaterial technologies, particularly the MIMIX microneedle platform, which facilitates transdermal delivery of vaccines and therapeutics. This innovative sustained release patch technology enhances vaccine administration by simulating a natural infection, promoting a robust and prolonged immune response, thereby improving vaccine effectiveness. Additionally, Vaxess’s MATRIX formulation and drying platform creates thermostable vaccines, therapeutics, and diagnostic products that do not require refrigeration, making them suitable for distribution in low-resource settings. Through these advanced solutions, Vaxess aims to improve patient outcomes in oncology care and infectious diseases while reducing treatment burdens for healthcare practitioners.

Mariana Oncology is a biotechnology company focused on developing innovative radiopharmaceuticals aimed at targeting cancer cells with radioactive drugs. The company employs a multidisciplinary approach that includes ligand discovery, radiochemistry, radiobiology, and translational and clinical research in oncology. By building a diverse pipeline of treatments for various solid tumor types, Mariana Oncology seeks to enhance the efficacy of radio medicines in cancer therapy, ultimately aiding medical professionals in their efforts to improve patient outcomes.

Nimbus Therapeutics, LLC is a biotechnology company based in Cambridge, Massachusetts, focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. Founded in 2009, the company utilizes a computational technology-driven approach to advance a pipeline of novel small-molecule therapeutics. Nimbus Therapeutics specializes in designing allosteric inhibitors, notably targeting Acetyl-CoA Carboxylase for conditions such as non-alcoholic steatohepatitis and hepatocellular carcinoma. The company has established strategic partnerships with organizations like Charles River Laboratories and Celgene Corporation to enhance its drug development efforts. In March 2015, Nimbus Discovery rebranded itself as Nimbus Therapeutics to better reflect its transition into a clinical-stage enterprise.

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

Superluminal Medicines is a biotechnology company specializing in generative biology and chemistry. It employs a unique platform that combines deep biological and chemical expertise, machine learning, and proprietary big data infrastructure to rapidly generate candidate-ready compounds for drug discovery and development. By integrating high-throughput experimental data with advanced algorithms, the company aims to accelerate the identification and optimization of novel drug candidates, improving efficiency and success rates in small molecule drug development compared to traditional methods.

ExcepGen Ltd is a biotechnology company focused on developing full-length human proteins aimed at advancing drug discovery. Established in 2017 and based in London, United Kingdom, the company utilizes engineered human cell lines to produce these proteins. ExcepGen specializes in mRNA therapeutics, utilizing proprietary technology to adapt cellular responses by modulating both undesirable and beneficial pathways. This approach enhances the yield and functionality of therapeutic mRNA without relying on workarounds. By enabling laboratories to accelerate development times and improve high-throughput screening processes, ExcepGen contributes to more effective decision-making in drug pipeline development.

COMPASS Pathways is a mental health care company based in Cheshire, United Kingdom, with operations in the United States. The company is focused on developing innovative therapies for treatment-resistant depression (TRD) using its proprietary psilocybin formulation, COMP360. Currently undergoing Phase IIb clinical trials, COMP360 aims to provide a new treatment option for patients who have not responded to traditional depression therapies. COMPASS Pathways emphasizes the combination of psilocybin-assisted therapy with psychological support to enhance treatment efficacy. The company is committed to addressing the significant unmet needs in mental health care by advancing evidence-based solutions for those suffering from mental health challenges.

AN2 Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Menlo Park, California, focused on the research, development, and commercialization of innovative medicines for infectious diseases. Founded in 2017, the company aims to address significant unmet medical needs through its development of novel therapeutics. Its lead product candidate, epetraborole, is a once-daily oral treatment designed for patients suffering from chronic non-tuberculous mycobacterial lung disease. AN2 Therapeutics is committed to leveraging advanced biomedical expertise to create transformative solutions for patients dealing with serious and rare infectious diseases. The company has also established a strategic partnership with Brii Biosciences to enhance its development efforts.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, focused on developing adeno-associated virus (AAV)-based therapies for monogenic diseases of the central nervous system (CNS). Founded in 2019, the company aims to create curative treatments for severe and life-threatening conditions. Its pipeline includes several product candidates, such as TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha collaborates with The University of Texas Southwestern Medical Center to enhance its development and commercialization efforts, leveraging expertise in gene therapy to advance its mission of eradicating monogenic CNS diseases.

Gracell Biotechnologies Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative cell therapies for cancer treatment. Based in Shanghai, China, Gracell's lead product candidates include GC012F, a dual-targeting autologous CAR-T therapy for multiple myeloma, and GC019F, which targets adult B cell acute lymphoblastic leukemia. Both candidates are currently undergoing Phase I clinical trials. Additionally, the company is advancing GC007F for B cell non-Hodgkin's lymphoma and GC027 for adult T cell acute lymphoblastic leukemia, among others. Gracell also has a range of earlier-stage product candidates addressing various cancer types, such as ovarian cancer and breast cancer. Founded in 2017, the company aims to provide effective cellular therapeutics to improve outcomes for patients with hematological malignancies and solid tumors.

CG Oncology, formerly known as Cold Genesys, is a privately held clinical-stage biopharmaceutical company specializing in the development of innovative oncolytic immunotherapies aimed at treating cancer. The company focuses on creating therapies that offer alternatives to conventional cancer treatments, particularly for patients with bladder cancer. Its lead candidate, cretostimogene grenadenorepvec, is a targeted intravesical immunotherapy agent designed to be delivered directly to the bladder. Currently, CG Oncology is conducting multiple clinical trials, including two phase three trials addressing high-risk and intermediate-risk non-muscle invasive bladder cancer (NMIBC), as well as a phase two study in combination with a checkpoint inhibitor for high-risk NMIBC. Through its research and clinical endeavors, CG Oncology aims to provide effective and less invasive treatment options for bladder cancer patients.

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

Boundless Bio, Inc. is a clinical-stage biopharmaceutical company based in La Jolla, California, focused on developing innovative therapies for the treatment of aggressive cancers. The company specializes in targeting extrachromosomal DNA (ecDNA), a key factor in the amplification of oncogenes that affects over 14% of cancer patients. By investigating the biology of ecDNA, Boundless Bio aims to create transformative treatments for previously untreatable cancers. Its lead therapeutic candidate, BBI-355, is an oral selective inhibitor of checkpoint kinase 1 (CHK1) designed to manage the replication and transcription of ecDNA in cancer cells. Founded in 2018, Boundless Bio is committed to addressing the significant unmet medical needs of patients with oncogene amplified tumors.

Convergent Therapeutics is a clinical-stage biotechnology company specializing in the development of radiopharmaceutical therapies for prostate cancer. The company's lead product, CONV 01-α, is a monoclonal antibody conjugated with the radioactive alpha particle emitter 225Ac, designed to bind specifically to the prostate-specific membrane antigen (PSMA). This innovative therapy allows for the internalization of the radioactive payload directly into prostate cancer cells, enhancing treatment efficacy. Developed through proprietary technology by Dr. Neil Bander at Weill Cornell Medicine, CONV 01-α is protected by multiple U.S. and foreign patents. If it receives FDA approval, it will become the first antibody to target a radioisotope for prostate cancer treatment and the first drug to utilize 225Ac in this context. Convergent Therapeutics aims to leverage its therapeutic platform to explore targeted combination strategies against validated and novel cancer antigens, ultimately enhancing treatment options for patients.

Alentis Therapeutics AG is a clinical-stage biopharmaceutical company based in Basel, Switzerland, specializing in the development of innovative therapies for advanced liver diseases and cancers associated with Claudin-1 (CLDN1) expression. Founded in 2019, Alentis focuses on addressing conditions such as liver fibrosis, cirrhosis, and liver cancer through the use of anti-CLDN1 antibody-drug conjugates and other targeted treatments. The company's unique approach combines clinically relevant read-outs with advanced techniques like single-cell RNA sequencing of patient liver tissues, aiming to reverse disease progression and improve patient outcomes. Alentis Therapeutics is recognized for its pioneering role in exploring anti-CLDN1 therapeutics, contributing to advancements in the treatment of fibrotic diseases and CLDN1-positive tumors.

ADCendo ApS is a biotech company focused on the development of innovative anti-cancer therapies, specifically through the creation of antibody-drug conjugates (ADCs). These conjugates are designed to target receptors that are overexpressed in various forms of cancer, addressing significant unmet medical needs in the treatment landscape. ADCendo's approach aims to enhance targeted therapy for patients suffering from conditions such as soft-tissue sarcoma, osteosarcoma, glioblastoma multiforme, and triple-negative breast cancer, among others. By developing new medicines that effectively target carcinomas—including breast, colon, pancreatic, prostate, ovarian, and renal cancers—ADCendo seeks to improve treatment outcomes for cancer patients.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

Vaxess Technologies, Inc. is a biotechnology company based in Boston, Massachusetts, founded in 2011. The company specializes in the development and commercialization of silk biomaterial technologies, particularly the MIMIX microneedle platform, which facilitates transdermal delivery of vaccines and therapeutics. This innovative sustained release patch technology enhances vaccine administration by simulating a natural infection, promoting a robust and prolonged immune response, thereby improving vaccine effectiveness. Additionally, Vaxess’s MATRIX formulation and drying platform creates thermostable vaccines, therapeutics, and diagnostic products that do not require refrigeration, making them suitable for distribution in low-resource settings. Through these advanced solutions, Vaxess aims to improve patient outcomes in oncology care and infectious diseases while reducing treatment burdens for healthcare practitioners.

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 to treat acute myeloid leukemia (AML) and is currently in preclinical development. VOR33 eHSCs are engineered to lack CD33, a protein commonly expressed by AML cells, thereby allowing for selective targeting of cancer cells while sparing normal cells. This innovative approach aims to minimize the toxicities associated with traditional immunotherapies, such as bispecific antibodies and CAR T cells, which often affect both cancerous and healthy cells. Vor Biopharma's mission is to provide safer and more effective treatment options for patients with hematological malignancies by creating therapies that protect healthy, functional cells from depletion during targeted cancer treatments.

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.

CG Oncology, formerly known as Cold Genesys, is a privately held clinical-stage biopharmaceutical company specializing in the development of innovative oncolytic immunotherapies aimed at treating cancer. The company focuses on creating therapies that offer alternatives to conventional cancer treatments, particularly for patients with bladder cancer. Its lead candidate, cretostimogene grenadenorepvec, is a targeted intravesical immunotherapy agent designed to be delivered directly to the bladder. Currently, CG Oncology is conducting multiple clinical trials, including two phase three trials addressing high-risk and intermediate-risk non-muscle invasive bladder cancer (NMIBC), as well as a phase two study in combination with a checkpoint inhibitor for high-risk NMIBC. Through its research and clinical endeavors, CG Oncology aims to provide effective and less invasive treatment options for bladder cancer patients.

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

Vaxess Technologies, Inc. is a biotechnology company based in Boston, Massachusetts, founded in 2011. The company specializes in the development and commercialization of silk biomaterial technologies, particularly the MIMIX microneedle platform, which facilitates transdermal delivery of vaccines and therapeutics. This innovative sustained release patch technology enhances vaccine administration by simulating a natural infection, promoting a robust and prolonged immune response, thereby improving vaccine effectiveness. Additionally, Vaxess’s MATRIX formulation and drying platform creates thermostable vaccines, therapeutics, and diagnostic products that do not require refrigeration, making them suitable for distribution in low-resource settings. Through these advanced solutions, Vaxess aims to improve patient outcomes in oncology care and infectious diseases while reducing treatment burdens for healthcare practitioners.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Nimbus Therapeutics, LLC is a biotechnology company based in Cambridge, Massachusetts, focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. Founded in 2009, the company utilizes a computational technology-driven approach to advance a pipeline of novel small-molecule therapeutics. Nimbus Therapeutics specializes in designing allosteric inhibitors, notably targeting Acetyl-CoA Carboxylase for conditions such as non-alcoholic steatohepatitis and hepatocellular carcinoma. The company has established strategic partnerships with organizations like Charles River Laboratories and Celgene Corporation to enhance its drug development efforts. In March 2015, Nimbus Discovery rebranded itself as Nimbus Therapeutics to better reflect its transition into a clinical-stage enterprise.

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

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

DBV Technologies S.A. is a clinical-stage biopharmaceutical company headquartered in Montrouge, France, focused on developing innovative treatments for food allergies through epicutaneous immunotherapy. The company's lead candidate, Viaskin Peanut, has completed Phase III clinical trials for the treatment of peanut allergies in children, adolescents, and adults. Additionally, DBV Technologies is advancing Viaskin Milk, which is in Phase I/II trials for cow’s milk protein allergy and related conditions, and Viaskin Egg, currently in pre-clinical development for hen’s egg allergy. The company is also exploring a booster vaccine for Bordetella pertussis and has ongoing research programs targeting respiratory syncytial virus, Crohn’s disease, celiac disease, and type I diabetes. Notably, DBV Technologies collaborates with Nestlé Health Science to develop MAG1C, an atopy patch test aimed at diagnosing non-IgE mediated cow’s milk protein allergy in infants and toddlers. Founded by pediatricians and entrepreneurs in 2002, DBV Technologies aims to revolutionize the field of immunotherapy by safely activating the immune system through its proprietary Viaskin technology.

Mineralys Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapies for hypertension. The company is advancing its lead product candidate, MLS-101, a highly selective and potent aldosterone synthase inhibitor licensed from Mitsubishi Tanabe Pharma Corporation. Mineralys aims to provide a targeted treatment for patients with uncontrolled hypertension, particularly those who do not achieve adequate blood pressure control despite using multiple antihypertensive medications. By addressing the underlying factors associated with elevated aldosterone levels, Mineralys seeks to improve treatment outcomes for individuals suffering from this condition.

Concert Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on discovering and developing novel small-molecule drugs to treat autoimmune and central nervous system disorders. The company employs a unique precision deuterium chemistry platform to modify the properties of validated drug molecules, resulting in a diverse pipeline of new chemical entities. Key product candidates include CTP-543, which is in Phase III trials for alopecia areata, and CTP-692, undergoing Phase II trials for schizophrenia. Additionally, AVP-786, a combination therapy for neurologic and psychiatric disorders, is also in Phase III trials. Concert Pharmaceuticals aims to enhance drug safety and efficacy while minimizing research and development risks and costs. Established in 2006 and based in Lexington, Massachusetts, the company has formed strategic collaborations with several pharmaceutical partners to advance its innovative drug development efforts.