RA Capital Management

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.

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.

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.

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.

Cidara Therapeutics is a biotechnology company based in San Diego, California, established in 2012. The company specializes in the discovery and development of innovative anti-infective therapies for serious diseases, particularly those that affect individuals with compromised immune systems. Its lead candidate, rezafungin acetate, is an antifungal agent designed to treat and prevent severe invasive fungal infections, such as candidemia and invasive candidiasis, which carry significant mortality risks. In addition to its antifungal efforts, Cidara is advancing its proprietary Cloudbreak platform, which focuses on developing antiviral Fc-conjugates aimed at preventing and treating various viral infections, including influenza, RSV, HIV, and coronavirus. Cidara's mission is to transform treatment and prevention strategies for life-threatening illnesses.

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.

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.

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 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.

Aktis Oncology is a biotechnology company focused on developing targeted radiopharmaceuticals for the treatment of various solid tumor cancers. The company has created proprietary platforms that generate tumor-targeting agents optimized for alpha radiotherapy. These agents are engineered to achieve high penetration into tumors and prolonged residence time, allowing for effective tumor elimination while reducing side effects associated with treatment. Additionally, Aktis Oncology's innovative approach enables clinicians to visualize and confirm target engagement before administering therapeutic radioisotopes, enhancing the precision of cancer treatment.

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

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

CellCentric Ltd. is a biotechnology company based in Cambridge, United Kingdom, focused on developing innovative cancer therapeutic products. Established in 2003, the company specializes in small-molecule inhibitors that target p300 and CBP proteins, as well as a deubiquitinase enzyme closely linked to prostate cancer. This enzyme modulates the androgen receptor pathway, which is critical in addressing resistance mechanisms encountered with existing prostate cancer treatments. CellCentric's therapies aim to treat various cancers, including castrate-resistant prostate cancer, lung cancer, bladder cancer, non-small cell lung cancer, breast cancer, and colon cancer. By targeting specific pathways and mechanisms, CellCentric seeks to provide effective treatment options for patients with challenging cancer types.

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

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.

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 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.

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

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

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.

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

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.

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

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.

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 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.

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

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

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.

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 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.

Harpoon Therapeutics is a clinical-stage immunotherapy company focused on developing innovative T cell engagers to harness the body's immune system for treating cancer and other diseases. Utilizing its proprietary TriTAC platform, the company is working on a pipeline of engineered proteins designed to direct T cells to target and eliminate specific cancer cells. The lead product candidate, HPN424, is currently undergoing Phase I clinical trials for metastatic castration-resistant prostate cancer. Additionally, Harpoon is developing HPN536, which is in Phase I/IIa trials for ovarian cancer and other tumors expressing MSLN, as well as HPN217 for multiple myeloma and HPN328 for small cell lung cancer. Founded in 2015 and headquartered in South San Francisco, California, Harpoon Therapeutics has established a collaboration agreement with AbbVie Biotechnology Ltd to further its research efforts.

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, Inc. is a clinical-stage biopharmaceutical development company based in Sioux Falls, South Dakota, with an additional office in Cambridge, Massachusetts. Founded in 2014, the company focuses on developing immunotherapies for various human diseases using its proprietary DiversitAb platform. This innovative platform enables the rapid production of fully targeted human polyclonal antibodies from genetically engineered transchromosomic cattle, eliminating the need for human plasma or serum. SAB Biotherapeutics' product pipeline includes SAB-142 for Type 1 diabetes, SAB-176 for seasonal influenza, and SAB-301 for Middle East Respiratory Syndrome Coronavirus (MERS-CoV). The company's approach aims to address public health challenges, rare diseases, and pandemic threats through advanced antibody science.

Adela specializes in developing advanced technologies for the early detection of cancer and other serious health conditions through routine blood tests. The company's genome-wide methylome enrichment platform efficiently captures comprehensive data from the entire methylome, enabling the identification of highly informative methylated regions of the genome. This targeted approach facilitates the detection and classification of tumors using plasma cell-free DNA methylomes. Adela's innovations not only aid in cancer detection but also extend to prenatal diagnostics, cardiology, and the monitoring of immune responses, thereby enhancing overall healthcare outcomes.

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.

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

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.

Inhibrx, Inc. is a clinical-stage biotechnology company based in La Jolla, California, dedicated to developing a diverse pipeline of novel biologic therapeutic candidates. The company employs advanced protein engineering techniques, including its proprietary sdAb platform, to target complex disease biology, with a focus on oncology, orphan diseases, and infectious diseases. Inhibrx's lead candidates include INBRX-109, a multivalent agonist in Phase 1 trials for solid tumors; INBRX-105, an antagonist of PD-L1 also in Phase 1 trials; INBRX-101, an Fc-fusion protein for alpha-1 antitrypsin deficiency; and INBRX-103, a monoclonal antibody targeting CD47. Additionally, the company is developing preclinical programs such as INBRX-106, an agonist of OX40, and INBRX-111, an antibody targeting Pseudomonas aeruginosa. Inhibrx has established collaborations with organizations like Celgene and Bluebird Bio and has received funding from various granting agencies, including the NIH and CARB-X.

ExcepGen Ltd is a biotechnology company focused on developing full-length human proteins for drug discovery. Founded in 2017 and based in London, the company employs engineered human cell lines to produce these proteins, facilitating research and development in therapeutics. By utilizing proprietary technology, ExcepGen aims to enhance the effectiveness of mRNA therapeutics, optimizing cellular responses to improve therapeutic outcomes. This approach allows laboratories to minimize development time, accelerate high-throughput screening processes, and make informed decisions regarding their therapeutic pipelines. With a commitment to addressing fundamental challenges in drug discovery, ExcepGen is positioned to contribute significantly to the advancement of innovative treatments.

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.

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.

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

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.

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.

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.

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

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.

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.

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.

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

DBV Technologies S.A. is a clinical-stage biopharmaceutical company based in Montrouge, France, dedicated to developing epicutaneous immunotherapy products aimed at treating food allergies. The company's lead candidate, Viaskin Peanut, has completed Phase III clinical trials for peanut allergies in children and adults. Additionally, DBV is developing Viaskin Milk, currently in Phase I/II trials for cow's milk protein allergy and related conditions, and Viaskin Egg, which is in pre-clinical stages for hen's egg allergy. The company also works on a booster vaccine for Bordetella pertussis and has earlier-stage research programs targeting respiratory syncytial virus, Crohn's disease, celiac disease, and type I diabetes. DBV Technologies collaborates with Nestlé Health Science to create MAG1C, a diagnostic patch test for non-IgE mediated cow's milk protein allergy in infants and toddlers. Founded in 2002, the company aims to provide safe and effective treatments for food allergies through its innovative Viaskin technology platform, which delivers biologically active compounds via the skin to activate the immune system.

Concert Pharmaceuticals is a clinical-stage biopharmaceutical company focused on discovering and developing novel small molecule drugs for autoimmune and central nervous system disorders. The company employs an innovative precision deuterium chemistry platform to enhance the properties of established drug molecules, which enables the creation of new chemical entities with potential efficacy and safety advantages. Notable product candidates include CTP-543, currently in Phase III trials for alopecia areata, CTP-692 for schizophrenia in Phase II trials, and AVP-786, which targets neurological and psychiatric disorders in Phase III trials. Concert Pharmaceuticals also engages in strategic collaborations with various pharmaceutical companies to support its development efforts. Founded in 2006 and based in Lexington, Massachusetts, the company aims to streamline research and development processes, reducing associated risks and costs while advancing its therapeutic pipeline.

Celcuity Inc. is a clinical-stage biotechnology company based in Minneapolis, Minnesota, focused on advancing cancer treatment through innovative diagnostics. The company's CELsignia diagnostic platform utilizes living tumor cells to uncover specific cellular processes that drive cancer in individual patients. This approach enables the identification of new cancer sub-types that traditional molecular diagnostics may overlook, ultimately expanding therapeutic options. Celcuity is developing the CELsignia HSF test to diagnose two new sub-types of HER2-negative breast cancer and the CELsignia MP test for diagnosing 11 sub-types across various cancers, including breast, lung, colon, ovarian, kidney, and bladder. The company is also advancing its lead therapeutic candidate, gedatolisib, a pan-PI3K and mTOR inhibitor, currently being evaluated in a Phase 3 clinical trial for patients with advanced HR+/HER2- breast cancer.

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.

Triana Biomedicines is focused on discovering and developing molecular glues, a novel therapeutic approach aimed at regulating challenging disease targets. The company employs a sophisticated drug discovery engine that combines high-resolution structural insights, advanced in silico tools, and custom chemical libraries. This platform is designed to stabilize existing protein interactions or create new ones, thereby influencing the functionality of disease targets. Triana has successfully validated its technology and is currently advancing multiple research programs across various disease areas, positioning itself to contribute significantly to therapeutic innovation.

Incyclix Bio is a biotechnology company based in Durham, North Carolina, focused on the development of small molecule inhibitors targeting cyclin-dependent kinases (CDKs) for the treatment of advanced and resistant cancers. Founded in 2020 by a team including Patrick Roberts, Jay Strum, John Bisi, and Fred Eshelman, the company aims to provide potent and selective inhibition of challenging CDK targets. This approach enables pharmaceutical companies and medical practitioners to access innovative therapeutic options, thereby enhancing treatment opportunities for cancer patients facing difficult cases. Through its research and development efforts, Incyclix Bio seeks to contribute to the advancement of cancer treatment solutions.

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

Electra Therapeutics is a clinical-stage biotechnology company focused on developing therapies that target signal regulatory proteins (SIRP) for treating immunological diseases and cancer. The company has one program in clinical development and two additional preclinical programs. Its lead product candidate, ELA026, is a monoclonal antibody designed to target SIRP on the surface of myeloid and T cells, effectively depleting pathological immune cells. ELA026 is currently being investigated for secondary hemophagocytic lymphohistiocytosis (sHLH), a severe hyperinflammatory condition for which no approved treatment exists.