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, Inc. is a clinical-stage pharmaceuticals company based in Boston, Massachusetts, founded in 2018. The company specializes in developing innovative therapies aimed at addressing cardiometabolic disorders by enhancing or restoring cellular energy production. Imbria's research focuses on the mechanisms underlying these conditions, particularly in areas such as non-obstructive hypertrophic cardiomyopathy, stable angina, and heart failure with preserved ejection fraction. By translating scientific insights into practical treatments, Imbria Pharmaceuticals aims to meet significant unmet medical needs and improve the lives of patients affected by these life-altering disorders. Through its commitment to understanding cellular metabolism and mitochondrial function, the company works to advance healthcare solutions in the cardiometabolic space.

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

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.

Surrozen, Inc. is a clinical-stage biopharmaceutical company based in South San Francisco, California, focused on developing targeted regenerative antibodies to repair damaged tissues and improve organ function. The company utilizes antibody platforms that act as Wnt and R-spondin mimetics to stimulate tissue regeneration in a variety of organs. Its key programs include SZN-043, which aims to promote hepatocyte regeneration in liver diseases, and SZN-1326, designed to repair diseased epithelial tissue for patients with inflammatory bowel disease. Surrozen's research targets several disease areas, including conditions affecting the intestine, liver, retina, cornea, lung, kidney, cochlea, skin, pancreas, and central nervous system, by engaging the body’s natural biological repair mechanisms. The company was incorporated in 2015 and continues to advance its innovative therapeutic approaches.

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.

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

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

Verdiva Bio is a biopharmaceutical company specializing in the development of innovative therapies for patients with obesity and cardiometabolic disorders. The company focuses on creating more patient-friendly treatment options, with a particular emphasis on gut-brain biology and amylin molecules, aiming to improve efficacy, tolerability, and weight loss outcomes.

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

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

ADCendo ApS is a biotechnology company focused on developing innovative anti-cancer drugs through the use of antibody-drug conjugates (ADCs). The company targets novel receptors that are overexpressed in various cancer types, addressing significant unmet medical needs in the field of oncology. Its ADCs aim to provide targeted therapy for patients suffering from aggressive cancers such as soft-tissue sarcoma, osteosarcoma, glioblastoma multiforme, and triple-negative breast cancer, as well as other carcinomas including those of the breast, colon, pancreas, prostate, ovary, and kidney. Through its research and development efforts, ADCendo strives to create effective treatment options for patients facing challenging cancer diagnoses.

Synapticure is a virtual clinical care platform dedicated to supporting patients with neurodegenerative diseases, including dementia, Parkinson's, and ALS, throughout the United States. Founded by patients, the company offers comprehensive care services that include access to disease-specific neurologists, speech and language therapy, and 24/7 care coordination. Utilizing advances in science, technology, and computational analytics, Synapticure ensures that patients receive tailored treatment and support in their own homes. The platform integrates medical, dental, vision, and life insurance plans to facilitate easy access to a range of healthcare services, including behavioral health support such as psychiatry and therapy, all aimed at enhancing the quality of life for individuals facing these challenging conditions.

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

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.

Evommune, Inc. is a biotechnology company based in Los Altos, California, that focuses on developing and manufacturing tissue-based medicines aimed at treating inflammatory diseases. Founded in 2020, the company employs a proprietary tissue-based platform to accelerate the discovery of novel therapies, with a particular emphasis on immunology and dermatology. Evommune's approach seeks not only to alleviate the symptoms of inflammatory disorders but also to halt disease progression, thereby improving the quality of life for patients. Its pipeline includes initiatives targeting prevalent inflammatory conditions, with a commitment to advancing patient care by creating best-in-class treatment options. Through innovative strategies, Evommune aims to drive meaningful advancements in the field of inflammatory disease management.

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.

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 Trillium™ technology, designed to provide functional tricuspid valve replacement through cross-caval anchoring and a unique multi-valve design. This technology aims to regulate blood flow effectively, thereby enhancing both the lifespan and quality of life for patients. Innoventric has successfully completed over ten implantations of the Trillium™ technology as part of its first clinical trial in Europe and maintains a robust patent portfolio covering its key innovations in tricuspid treatment.

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

Enara Bio is a biotechnology company based in Oxford, United Kingdom, focused on developing vaccines and immunotherapies for cancer treatment and prevention. Founded in 2016, the company specializes in identifying and utilizing novel antigens derived from endogenous retroviral DNA sequences to create therapeutic cancer vaccines. Enara Bio's technology platform enables the exploration of dark antigens, assessing their tumor specificity and immunogenic potential, which is crucial for effective cancer therapies. The company's innovative approach targets the interaction between T-cells and cancer cells, aiming to create targeted immunotherapies that can benefit a broad patient population.

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.

Elektrofi is a preclinical-stage company based in Boston, Massachusetts, focused on advancing drug delivery methods to enhance patient experiences. Established in 2016, the company has developed a formulation system capable of processing a diverse array of therapeutic molecules, including large and complex proteins such as monoclonal antibodies, bispecifics, and fusion proteins. Its innovative technology aims to improve subcutaneous administration, allowing drug manufacturers to create breakthrough therapies that empower patients with more control over how they receive essential medications.

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

Magenta Medical Ltd. is a privately held company based in Kadima, Israel, founded in 2012. The company specializes in developing innovative medical devices aimed at treating acute decompensated heart failure. Its flagship product is a temporary venous catheter-based therapy that includes a self-expanding heart pump. This device targets critical issues related to acute heart failure, particularly renal venous congestion, which adversely affects both cardiac and renal function. By enabling physicians to more effectively manage fluid and salt removal in patients, Magenta Medical's technology enhances patient safety and supports kidney function, offering a promising solution for better management of heart failure.

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.

ADCendo ApS is a biotechnology company focused on developing innovative anti-cancer drugs through the use of antibody-drug conjugates (ADCs). The company targets novel receptors that are overexpressed in various cancer types, addressing significant unmet medical needs in the field of oncology. Its ADCs aim to provide targeted therapy for patients suffering from aggressive cancers such as soft-tissue sarcoma, osteosarcoma, glioblastoma multiforme, and triple-negative breast cancer, as well as other carcinomas including those of the breast, colon, pancreas, prostate, ovary, and kidney. Through its research and development efforts, ADCendo strives to create effective treatment options for patients facing challenging cancer diagnoses.

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

Bicycle Therapeutics is a clinical-stage biopharmaceutical company focused on developing a novel class of medicines known as Bicycles, which are synthetic short peptides designed to stabilize their structural geometry through two loops. The company primarily targets oncology indications with significant unmet medical needs. Its lead product candidate, BT1718, is a Bicycle Toxin Conjugate currently undergoing Phase I/IIa clinical trials aimed at tumors expressing Membrane Type 1 matrix metalloprotease. Other oncology candidates include BT5528, also in Phase I/IIa trials targeting EphA2, and BT8009, which is in preclinical studies for Nectin-4. Additionally, Bicycle Therapeutics is developing THR-149, a plasma kallikrein inhibitor that has completed Phase I trials for diabetic macular edema. The company collaborates with various biopharmaceutical organizations to advance programs in areas like anti-infectives and cardiovascular health. Founded in 2009, Bicycle Therapeutics is headquartered in Cambridge, United Kingdom.

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

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

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.

iTeos Therapeutics is a biopharmaceutical company dedicated to developing immuno-oncology therapies for cancer patients. Founded in 2011, the company operates from headquarters in Cambridge, Massachusetts, and Gosselies, Belgium. iTeos focuses on discovering and advancing small molecule immunomodulators that target the tumor microenvironment, aiming to enhance the body's immune response against cancer. Its pipeline includes EOS-850, a small molecule antagonist of the adenosine A2a receptor, and EOS-448, an antagonist of TIGIT, both currently in Phase 1/2a clinical trials. iTeos' approach is built on the expertise of Ludwig Cancer Research, aiming to improve clinical outcomes by restoring the immune system's ability to fight cancer.

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.

Zura Bio is a clinical-stage biotechnology company that develops innovative therapies for immune and inflammatory disorders. The company focuses on advancing its programs through phase 2 development, specifically targeting auto-immune illnesses. One of its key assets, ZB-168, is an anti-IL7R inhibitor designed to address disorders linked to the biological pathways of IL7 and TSLP, building on data from phase 1b studies related to type 1 diabetes. Additionally, Zura Bio is progressing the phase 2 clinical development of torudokimab, a human-affinity monoclonal antibody that neutralizes IL33. Through these efforts, Zura Bio aims to create effective therapeutic solutions for patients suffering from various 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.

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 develops innovative cell and gene therapies aimed at enhancing adoptive immunotherapy for cancer patients. Founded in 2015 and based in Cambridge, Massachusetts, the company focuses on creating next-generation therapies that utilize pharmacologic operating systems to provide precise control over protein activity within cells. This technology allows for the development of adoptive cell therapies with advanced functionalities that can be managed by physicians using simple, safe, and orally active medications. By offering improved control over treatment, Obsidian Therapeutics aims to provide better outcomes for patients compared to existing cell therapy options.

Surrozen, Inc. is a clinical-stage biopharmaceutical company based in South San Francisco, California, focused on developing targeted regenerative antibodies to repair damaged tissues and improve organ function. The company utilizes antibody platforms that act as Wnt and R-spondin mimetics to stimulate tissue regeneration in a variety of organs. Its key programs include SZN-043, which aims to promote hepatocyte regeneration in liver diseases, and SZN-1326, designed to repair diseased epithelial tissue for patients with inflammatory bowel disease. Surrozen's research targets several disease areas, including conditions affecting the intestine, liver, retina, cornea, lung, kidney, cochlea, skin, pancreas, and central nervous system, by engaging the body’s natural biological repair mechanisms. The company was incorporated in 2015 and continues to advance its innovative therapeutic approaches.

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.

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

Geron Corporation is a late-stage clinical biopharmaceutical company headquartered in Menlo Park, California, that specializes in developing therapies for hematologic myeloid malignancies. The company's lead product, imetelstat, is a first-in-class telomerase inhibitor currently undergoing Phase 2/3 clinical trials. This investigational drug aims to inhibit the uncontrolled proliferation of malignant progenitor cells, which can lead to dysfunctional blood cell production, thereby facilitating the recovery of normal blood cell development. Founded in 1990, Geron focuses on advancing treatments for conditions such as myelodysplastic syndromes and myelofibrosis, a rare blood cancer affecting bone marrow. The company also generates revenue through collaboration agreements, milestones, royalties, and licensing arrangements.

Vesta Healthcare is a technology and clinical services organization that focuses on enhancing the connection between caregivers and the broader care team. The company provides 24/7 telehealth support for caregivers and care recipients, particularly targeting high-need, frail senior populations. By partnering with home care agencies, health plans, and healthcare providers, Vesta Healthcare develops value-based population health programs that prioritize clinical quality, improved health outcomes, and personalized engagement. Originally established as Hometeam Technologies, Inc. in 2008, the company rebranded to Vesta Healthcare in January 2020 and is headquartered in New York, New York.

Regulus Therapeutics Inc. is a clinical stage biopharmaceutical company based in San Diego, California, specializing in the discovery and development of therapies that target microRNAs for various diseases. The company is advancing its lead product candidates, RG-012 and RGLS4326, which are in Phase II and Phase Ib clinical trials, respectively. RG-012 targets miR-21 for the treatment of Alport syndrome, a severe kidney disease, while RGLS4326 targets miR-17 for autosomal dominant polycystic kidney disease. In addition to these candidates, Regulus is developing a pipeline of preclinical drug products, including RGLS5579, aimed at inhibiting miR-10b, as well as programs targeting hepatitis B virus and non-alcoholic steatohepatitis. Founded in 2007, Regulus Therapeutics is at the forefront of leveraging the therapeutic potential of microRNA, a significant scientific breakthrough in recent years.

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.

Avidity Biosciences, Inc. is a biotechnology company focused on developing oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These innovative therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. This approach allows Avidity to target previously undruggable tissue and cell types, thereby addressing the underlying genetic drivers of various diseases. The company's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, a rare muscle disease. Additionally, Avidity is advancing multiple programs aimed at treating muscle-related conditions such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Founded in 2012 and based in La Jolla, California, Avidity is also exploring therapeutic applications in immune and other cell types.

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.

MMI S.r.l. is an Italian company that specializes in the manufacture and development of advanced micro instruments designed for surgical interventions. Founded in 2015 and based in Calci, Italy, MMI focuses on creating wristed micro instruments and their associated fabrication processes. The company also offers a proprietary robotic platform that enhances the capabilities of these instruments, facilitating complex reconstruction procedures following traumatic injuries and tumor removals in areas such as the breast, head, and bones. MMI aims to support surgeons globally by providing innovative tools that improve surgical outcomes while also reducing costs for patients and healthcare systems.

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.

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 is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing nucleic acid therapeutics, particularly antisense oligonucleotides. Founded in 2018, the company aims to enhance the delivery and efficacy of these therapies through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative platform utilizes engineered peptides designed to improve tissue penetration, cellular uptake, and nuclear delivery of oligonucleotides. PepGen's technology has shown promise in preclinical studies, demonstrating the ability to transport therapeutics effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. The company's mission is to unlock the full potential of nucleic acid therapeutics to transform the treatment landscape for severe neuromuscular and neurological diseases.

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

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.

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.

Claris Biotherapeutics develops a topical formulation intended to cure corneal wounds, ulcers, and scars. The formulation creates a recombinant hepatocyte growth factor which accelerates the healing process, providing healthcare providers noninvasive therapeutic treatment.

Forward Therapeutics is a biopharmaceutical company focused on developing innovative oral small molecule therapies to treat chronic inflammatory disorders. The company's platform, driven by leading scientific expertise, enables the discovery and development of advanced treatments that aim to significantly improve patient outcomes and address unmet medical needs in this field.

Rampart Bioscience specializes in the development of gene medicines aimed at providing long-lasting treatments for a broad array of diseases. The company has created proprietary development and delivery platforms that enable the production of optimized therapeutics in a non-viral format. By integrating expertise from various scientific disciplines, such as gene delivery, protein sciences, and clinical translation, Rampart Bioscience focuses on assisting patients with genetically driven conditions, ultimately enhancing the effectiveness and safety of therapeutic interventions.

Aiolos Bio is a biotechnology company specializing in the development of therapies for respiratory diseases and immune conditions. Its primary focus is on advancing AIO-001, a monoclonal antibody therapy for moderate-to-severe asthma, designed for dosing every 6 months. The company, founded by industry veterans, aims to address unmet treatment needs and improve patient outcomes by providing more efficacious and less burdensome therapies.

Amplifier Therapeutics is a biopharmaceutical company dedicated to developing novel therapies targeting AMP-activated protein kinase (AMPK), a key regulator of cellular energy balance. The company's focus is on treating age-related disorders, including metabolic conditions, cardiovascular diseases, kidney diseases, and certain types of cancer, by activating AMPK to restore normal cellular function.

Commons Clinic is a multi-specialty healthcare facility located in Austin, Texas, founded in 2021. The clinic aims to deliver seamless healthcare treatment by coordinating elite physicians and providing ambulatory services. It offers comprehensive specialty and surgical care programs focusing on pain management, orthopedics, women's health, and men's health. By emphasizing efficient and improved medical care, Commons Clinic strives to enhance the overall patient experience and health outcomes.

Hyku is a biotechnology company focused on developing small molecule therapeutics that target disease-causing proteins through precise covalent binding. Utilizing its innovative platform, Hyku identifies molecules that covalently bind to non-cysteine amino acids, facilitating the creation of novel therapies. These therapies aim to address significant challenges in drug development, including issues related to selectivity, druggability, and resistance. By pushing the boundaries of covalent medicine, Hyku seeks to create effective treatments that can improve patient outcomes and advance the field of therapeutic development.

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

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

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.

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.

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

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

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, established in 2019, focused on developing adeno-associated virus (AAV)-based gene therapies for monogenic diseases affecting the central nervous system (CNS). The company’s product pipeline includes several therapies currently in development, 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 Gene Therapies aims to translate innovative treatments from research to clinical practice efficiently, leveraging a strategic partnership with The University of Texas Southwestern Medical Center to enhance its development and commercialization capabilities. The company's mission is to create curative medicines that target both rare and prevalent CNS conditions, thereby addressing severe and life-threatening diseases.

Gracell Biotechnologies Inc. is a clinical-stage biopharmaceutical company based in Shanghai, China, focused on discovering and developing cell therapies for cancer treatment. The company’s lead product candidates include GC012F, a dual-target autologous CAR-T therapy for multiple myeloma, and GC019F, which targets adult B cell acute lymphoblastic leukemia and is also being investigated for relapsed or refractory B cell non-Hodgkin’s lymphoma. Additionally, Gracell is developing GC007F for B cell non-Hodgkin's lymphoma and GC027, an allogeneic CAR-T product candidate for adult T cell acute lymphoblastic leukemia. The company also has GC007g, a donor-derived therapy for relapsed or refractory B cell acute lymphoblastic leukemia, and a pipeline of earlier stage candidates targeting other cancer types, including ovarian cancer and breast cancer. Founded in 2017, Gracell aims to provide innovative cellular therapeutics that enhance treatment options for patients suffering from various hematological malignancies and solid tumors.

CG Oncology is a clinical-stage biopharmaceutical company dedicated to developing innovative immunotherapies for cancer treatment, particularly focusing on bladder cancer. The company specializes in oncolytic immunotherapies, with its lead candidate, cretostimogene grenadenorepvec, designed as a targeted, intravesical immunotherapy. Currently, CG Oncology is conducting multiple clinical trials, including two phase three trials aimed at high-risk patients with non-muscle invasive bladder cancer (NMIBC) who are unresponsive to BCG therapy, as well as an additional phase two study in combination with a checkpoint inhibitor. The company aims to provide effective bladder-sparing treatment options for patients while advancing the development of its proprietary therapies.

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

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.

Carmot Therapeutics, Inc. is a clinical-stage biotechnology company based in Berkeley, California, with an additional office in San Francisco. Founded in 2008, the company focuses on discovering and developing therapies for metabolic diseases, including obesity and diabetes. Carmot employs a proprietary drug discovery approach known as Chemotype Evolution, which enables the exploration of novel chemical and biological frontiers. The company is advancing a portfolio of clinical-stage drug candidates, including CT-388, a once-weekly injectable dual GLP-1/GIP receptor agonist; CT-996, a once-daily oral GLP-1 receptor agonist; and CT-868, a once-daily injectable dual GLP-1/GIP receptor agonist for treating type 1 diabetes in patients with overweight or obesity. These innovative therapies aim to enhance glucose clearance and promote weight loss, addressing the fundamental causes of metabolic diseases.

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.

Cortica Inc. is a company dedicated to providing neurological therapies for children facing autism and other neurodevelopmental challenges. Founded in 2014 and based in San Diego, California, Cortica offers tailored treatment programs that are informed by a comprehensive understanding of each child's neurobiology and developmental profile. The company operates multiple centers across California, including locations in Carlsbad, Irvine, San Rafael, Torrance, and Westlake Village. Cortica's services encompass a wide range of assessments and therapies, including diagnoses for autism, ADHD, and developmental delays. Treatment options available include gene sequencing, metabolic testing, electroencephalography, nutrition evaluation, medication management, and various therapeutic interventions such as ABA therapy, parent coaching, speech-language therapy, occupational therapy, physical therapy, and music therapy. The company provides these services through in-home, in-clinic, and telehealth modalities.

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.

ADCendo ApS is a biotechnology company focused on developing innovative anti-cancer drugs through the use of antibody-drug conjugates (ADCs). The company targets novel receptors that are overexpressed in various cancer types, addressing significant unmet medical needs in the field of oncology. Its ADCs aim to provide targeted therapy for patients suffering from aggressive cancers such as soft-tissue sarcoma, osteosarcoma, glioblastoma multiforme, and triple-negative breast cancer, as well as other carcinomas including those of the breast, colon, pancreas, prostate, ovary, and kidney. Through its research and development efforts, ADCendo strives to create effective treatment options for patients facing challenging cancer diagnoses.

Capacity Bio is a therapeutics company dedicated to advancing the field of mitophagy, which involves the regulation of mitochondrial quality control. The company is developing a comprehensive platform aimed at creating innovative mitophagy therapeutics. By focusing on fundamental biological processes, Capacity Bio seeks to provide radical therapeutic benefits that can restore mitochondrial function in various diseases, thereby addressing critical healthcare challenges.

Unicycive Therapeutics provides development programs that focus on treating kidney diseases with significant unmet medical needs. It aims to open the way for a broad range of disease-modifying therapies by modifying the intracellular and extracellular concentrations of these various electrolytes and ions in Chronic Kidney Disease and Gitelman’s Syndrome and by addressing the underlying mitochondrial pathology and inflammation associated with Acute Kidney Disease. Unicycive Therapeutics was founded in 2016 and is headquartered in Los Altos, California.

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

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

Apogee Therapeutics is a biotechnology company based in San Francisco, California, founded in 2022. The company focuses on developing differentiated biologics for the treatment of immunological and inflammatory disorders, specifically targeting atopic dermatitis and chronic obstructive pulmonary disease. Apogee Therapeutics aims to address high unmet medical needs by advancing novel therapies that utilize advanced antibody engineering to enhance therapeutic properties such as half-life. The company's key programs, APG777 and APG808, are designed to leverage established mechanisms of action to improve treatment outcomes for patients suffering from these conditions.

NewAmsterdam Pharma, established in 2019 and headquartered in Naarden, the Netherlands, is a late-stage biopharmaceutical company dedicated to improving patient care in cardio-metabolic disease populations. The company's primary focus is the development of transformative therapies, with its lead product, obicetrapib, being a next-generation, oral, low-dose CETP inhibitor designed to lower LDL-C levels more effectively than current treatments, particularly for patients with statin intolerance or Arteriosclerotic Cardiovascular Disease/Familial Hypercholesterolemia (ASCVD/FH) on maximally-tolerated statin therapy.

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 is a clinical-stage biopharmaceutical company dedicated to developing innovative immunotherapies for cancer treatment, particularly focusing on bladder cancer. The company specializes in oncolytic immunotherapies, with its lead candidate, cretostimogene grenadenorepvec, designed as a targeted, intravesical immunotherapy. Currently, CG Oncology is conducting multiple clinical trials, including two phase three trials aimed at high-risk patients with non-muscle invasive bladder cancer (NMIBC) who are unresponsive to BCG therapy, as well as an additional phase two study in combination with a checkpoint inhibitor. The company aims to provide effective bladder-sparing treatment options for patients while advancing the development of its proprietary therapies.

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

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.

Rivus Pharmaceuticals is a biopharmaceutical company focused on enhancing cardio-metabolic health through the development of controlled metabolic accelerators (CMAs). These innovative therapeutics aim to improve cellular metabolism and target the root causes of prevalent metabolic and cardiovascular diseases. By providing oral small-molecule treatments, Rivus addresses critical metabolic risk factors associated with cardiovascular mortality and morbidity, thereby contributing to better health outcomes for patients suffering from these conditions.

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

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

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

Carmot Therapeutics, Inc. is a clinical-stage biotechnology company based in Berkeley, California, with an additional office in San Francisco. Founded in 2008, the company focuses on discovering and developing therapies for metabolic diseases, including obesity and diabetes. Carmot employs a proprietary drug discovery approach known as Chemotype Evolution, which enables the exploration of novel chemical and biological frontiers. The company is advancing a portfolio of clinical-stage drug candidates, including CT-388, a once-weekly injectable dual GLP-1/GIP receptor agonist; CT-996, a once-daily oral GLP-1 receptor agonist; and CT-868, a once-daily injectable dual GLP-1/GIP receptor agonist for treating type 1 diabetes in patients with overweight or obesity. These innovative therapies aim to enhance glucose clearance and promote weight loss, addressing the fundamental causes of metabolic diseases.

MMI S.r.l. is an Italian company that specializes in the manufacture and development of advanced micro instruments designed for surgical interventions. Founded in 2015 and based in Calci, Italy, MMI focuses on creating wristed micro instruments and their associated fabrication processes. The company also offers a proprietary robotic platform that enhances the capabilities of these instruments, facilitating complex reconstruction procedures following traumatic injuries and tumor removals in areas such as the breast, head, and bones. MMI aims to support surgeons globally by providing innovative tools that improve surgical outcomes while also reducing costs for patients and healthcare systems.