Versant Ventures

Jade Biosciences is developing transformative therapies to redefine the standard of care for inflammation and immunology indications.

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.

SixPeaks Bio focuses on developing innovative therapies aimed at promoting healthy weight loss for individuals struggling with obesity. The company is working on a pipeline of medicines that utilize dual-specific antibodies to target activin type IIA and B receptors. This approach aims to help patients achieve weight loss while preserving skeletal muscle mass, addressing a common concern associated with traditional weight-loss methods that often lead to muscle loss alongside fat reduction. Through its research and development efforts, SixPeaks Bio seeks to provide effective solutions for sustainable weight management and improved health outcomes.

LENZ Therapeutics is a late-stage biopharmaceutical company dedicated to the development and commercialization of innovative ophthalmic therapies aimed at improving vision. The company's primary focus is on addressing presbyopia, a common condition that leads to the gradual loss of near vision, affecting nearly all individuals over the age of 45. LENZ Therapeutics is currently advancing an aceclidine-based eye drop specifically designed to restore near vision, addressing a significant unmet need in the market. Through its efforts, the company aims to enhance the quality of life for individuals experiencing vision changes related to aging.

FireFly Bio focuses on developing Degrader Antibody Conjugates (DACs), a novel approach that merges the benefits of antibody-drug conjugates with selective protein degraders. This proprietary healthcare platform is designed to enhance cancer treatment by ensuring the precise delivery of therapeutic agents to targeted tissues. By leveraging advanced bioconjugate technologies, FireFly Bio aims to create more effective and targeted medicines that improve patient outcomes in the fight against cancer.

Enterprise Therapeutics Ltd. is a drug discovery company based in Brighton, United Kingdom, founded in 2014. It focuses on the research and development of innovative therapies for respiratory diseases, specifically targeting chronic obstructive pulmonary disease (COPD), cystic fibrosis, and asthma. The company aims to create disease-modifying therapies that address the underlying mechanisms of mucus congestion in the lungs, which can lead to breathing difficulties and increased susceptibility to lung infections. By developing these novel treatments, Enterprise Therapeutics seeks to enhance the quality of life for patients suffering from these chronic respiratory conditions.

Nouscom is a biotechnology company focused on developing an immunotherapy platform that creates modified viral vector vaccines for cancer treatment. The company utilizes a range of engineered viral vectors optimized for the effective expression of long strings of tumor neoantigens. Additionally, its platform includes tools designed to induce broad and potent antitumor T-cell responses, which aim to enhance cancer-specific cellular immunity in patients.

Gate Bioscience specializes in a small molecule modality to treat biology’s intractable diseases.

Tentarix creates innovative protein therapeutics with uniquely combined bioactivities that leverage synthetic biology. The company develops a protein engineering platform designed for multispecific therapies.

Nexo Therapeutics is a biotechnology company focused on developing small-molecule therapies for cancer patients who have few effective treatment options. The company utilizes a specialized platform that integrates covalent ligand discovery with chemical biology to create innovative oncology drugs aimed at challenging targets that have been difficult to address in the past. By advancing its pipeline of novel therapies, Nexo Therapeutics seeks to provide new avenues for treatment, ultimately enhancing the care available to individuals battling cancer and other related diseases.

Kate Therapeutics is a biotechnology company dedicated to developing adeno-associated virus (AAV)-based gene therapies for genetically defined muscle and heart diseases. The company employs advanced technology platforms to enhance tissue-specific delivery and gene regulation, overcoming significant challenges associated with current gene therapies. By utilizing innovative capsids, Kate Therapeutics achieves superior muscle transduction and effectively de-targets the liver, allowing for more efficient tissue transduction at lower viral doses. This approach addresses key limitations in muscle gene delivery, aiming to improve treatment outcomes for patients with muscle and heart conditions.

Abdera Therapeutics is an oncology company focused on developing targeted alpha therapies (TATs) for patients suffering from relapsed, refractory, and metastatic cancers. The company specializes in targeted radiotherapies that employ purpose-built vectors to deliver high-energy radioisotopes directly to tumors and metastatic lesions. This innovative approach aims to provide more effective treatment options for patients, capitalizing on the significant therapeutic and commercial potential of TATs. With the nuclear medicine market projected to grow substantially, Abdera's targeted therapies are positioned to play a crucial role in the evolving landscape of cancer treatment.

Abdera Therapeutics is an oncology company focused on developing targeted alpha therapies (TATs) for patients suffering from relapsed, refractory, and metastatic cancers. The company specializes in targeted radiotherapies that employ purpose-built vectors to deliver high-energy radioisotopes directly to tumors and metastatic lesions. This innovative approach aims to provide more effective treatment options for patients, capitalizing on the significant therapeutic and commercial potential of TATs. With the nuclear medicine market projected to grow substantially, Abdera's targeted therapies are positioned to play a crucial role in the evolving landscape of cancer treatment.

Santa Ana Bio is a biotechnology company focused on developing precision therapies for autoimmune and inflammatory diseases. By utilizing advanced technologies such as deep cell profiling, single-cell genomics, and multi-omics platforms, the company designs targeted biologics that aim to provide effective and personalized treatment options. Through its innovative approach, Santa Ana Bio seeks to enhance patient outcomes and improve quality of life for those affected by autoimmune conditions.

Belharra Therapeutics is a privately held drug discovery company that is disrupting the drug discovery paradigm with its novel photoaffinity-based chemoproteomics platform. Belharra scientists can identify small molecule drug candidates for any binding site, on any protein, in any conformational state, and in any cell type using its chemoproteomics platform. Belharra is based in the San Francisco Bay Area, with its primary lab and offices in San Diego.

Launchpad Therapeutics operates as an antibody-focused precision oncology company.

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.

Vector BioPharma's cutting-edge gene precision delivery technology will transform the safety, efficacy, and specificity of biologics.

Tempest Therapeutics is a biotechnology company based in South San Francisco, California, that specializes in developing small molecule therapeutics for cancer treatment. Founded in 2011, the company focuses on innovative therapies that enhance anti-tumor immunity and directly target tumor cells. Its pipeline includes several clinical-stage candidates, notably TPST-1120, which blocks the PPAR-alpha pathway to stimulate immune response, and TPST-8844, designed to inhibit an enzyme that allows tumor cells to evade immune detection. Additionally, Tempest Therapeutics is working on compounds that target prostaglandins to counteract immune suppression. By pursuing these mechanisms, the company aims to create effective treatments that harness the body's own immune system to combat cancer.

Cimeio Therapeutics develops curative treatments for genetic diseases, hematologic malignancies, and severe autoimmune disorders. The technology platform developed by Cimeio is based on the design and expression of modified variants of naturally occurring cell surface proteins in HSCs. The SCIP platform could allow more patients facing debilitating and fatal diseases to receive a life-saving HSC transplant. Its mission is to significantly improve the eligibility and outcomes of hematopoietic stem cell transplants and adoptive cell therapy.

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

Nested Therapeutics is a Cambridge-based VC-backed stealth biotech company. The company's focus is on finding and creating new precision oncology small-molecule drugs targeted toward cancer genes. Nested Therapeutics was established in 2021 in Cambridge, Massachusetts.

Coda Biotherapeutics, Inc. is a biotechnology company focused on developing innovative treatments for neurological disorders. The company specializes in chemogenetic technology, which allows for precise modulation of neuronal activity through gene therapy. By using viral vectors, Coda Biotherapeutics introduces engineered neurotransmitter receptors into specific neuronal populations. These receptors are designed to respond to a proprietary small molecule, enabling targeted control of neuronal functions. This approach aims to effectively treat conditions such as neuropathic pain, epilepsy, and movement disorders, offering a potential advancement over traditional therapies. Founded in 2014, Coda Biotherapeutics is headquartered in San Francisco, California.

SanReno Therapeutics specializes in the development, manufacturing, and commercialization of therapies for kidney diseases in several regions including the People's Republic of China, Hong Kong, Macau, Taiwan, and Singapore. Established through a joint venture involving Chinook Therapeutics, Frazier Healthcare Partners, and Pivotal bioVenture Partners China, the company is dedicated to researching and producing innovative biotech solutions aimed at improving the quality of life for patients suffering from kidney-related conditions.

Tentarix creates innovative protein therapeutics with uniquely combined bioactivities that leverage synthetic biology. The company develops a protein engineering platform designed for multispecific therapies.

Lycia Therapeutics is a biotechnology firm focused on discovering and developing first-in-class therapies. The company utilizes its innovative lysosomal targeting chimeras (LYTACs) platform to create therapeutics that target and degrade extracellular and membrane-bound proteins associated with challenging diseases, including cancer and autoimmune disorders. By addressing these difficult-to-treat conditions, Lycia Therapeutics aims to provide medical practitioners with effective treatment options to improve patient outcomes.

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.

Turnstone Biologics Inc. is a biotechnology company based in Ottawa, Canada, with an additional office in New York. Founded in 2015, it is dedicated to developing innovative viral immunotherapies aimed at improving survival rates for cancer patients. The company's lead product, RIVAL-01, utilizes a vaccinia virus backbone to deliver three key immunomodulators: Flt3 ligand, anti-CTLA-4 antibody, and IL-12 cytokine. These components are designed to enhance immune activity and reprogram the tumor microenvironment for optimal tumor eradication. Additionally, Turnstone Biologics is advancing next-generation tumor-infiltrating lymphocyte (TIL) therapies, which involve selecting and expanding the most effective T cells from a patient’s tumor to enhance the body’s immune response against solid tumors. Through these approaches, Turnstone aims to deliver breakthrough immunotherapies for cancer treatment.

LENZ Therapeutics is a late-stage biopharmaceutical company dedicated to the development and commercialization of innovative ophthalmic therapies aimed at improving vision. The company's primary focus is on addressing presbyopia, a common condition that leads to the gradual loss of near vision, affecting nearly all individuals over the age of 45. LENZ Therapeutics is currently advancing an aceclidine-based eye drop specifically designed to restore near vision, addressing a significant unmet need in the market. Through its efforts, the company aims to enhance the quality of life for individuals experiencing vision changes related to aging.

RayzeBio, Inc. is a biotechnology company based in San Diego, California, founded in 2020. It specializes in developing tumor-targeted small molecule medicines that utilize the therapeutic potential of radioisotopes, particularly Actinium-225, an alpha-emitting radioisotope aimed at treating solid tumors. RayzeBio is focused on enhancing cancer treatment outcomes by creating a robust pipeline of radiopharmaceutical candidates targeting validated oncology drug targets. The company's portfolio includes a range of drug candidates at various stages of development, from discovery to late-stage clinical programs, addressing significant market opportunities in the field of cancer therapeutics.

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.

Stablix Therapeutics is a biotechnology company specializing in Targeted Protein Stabilization (TPS). The company has developed a platform that produces heterobifunctional small molecules, known as RESTORACS, which selectively recruit deubiquitinase enzymes to remove ubiquitin from targeted proteins. This process enhances the stability and activity of these proteins. Stablix focuses on utilizing its innovative technology to create therapeutic programs aimed at treating rare diseases, cancer, and immunological disorders. By restoring protein function, Stablix aims to provide more effective treatment options for complex medical conditions.

Capsida is a biotechnology company that specializes in tackling monogenetic and sporadic CNS and non-CNS disorders. It features a proprietary adeno-associated virus (AAV) engineering platform that generates capsids optimized to target specific tissues and cells in the diseased organ.

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

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

Monte Rosa Therapeutics is a biotechnology company focused on developing innovative cancer therapeutics that utilize molecular glue degraders (MGDs) to modulate protein degradation pathways. By leveraging the body's natural mechanisms for protein destruction, these small molecule drugs selectively target and degrade disease-causing proteins. The company employs its QuEEN platform, which combines artificial intelligence and proprietary experimental tools, to identify relevant proteins for degradation. With a diverse library of over 50,000 MGD molecules, Monte Rosa's lead candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for treating MYC-driven tumors. The company aims to advance its pipeline of pioneering therapies for cancer and other diseases.

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

Pipeline Therapeutics is a developer of regenerative therapies designed to promote functional recovery of neurological diseases. The company's therapies leverage the natural repair process in several different nervous system cell types to target diseases such as multiple sclerosis, providing patients with a treatment to repair damage to the nervous system.

Gritstone bio is a biotechnology company focused on developing personalized immunotherapies for cancer and infectious diseases. The company initially concentrated on tumor-specific neoantigens but has expanded its programs to include viral antigens found on the surfaces of infected cells. This approach leverages the immune system's ability to recognize abnormal cell targets, which is crucial for both anti-tumor and anti-viral immunity. Gritstone bio is building a robust pipeline of immunotherapy product candidates, including GRANITE, SLATE, and CORAL, designed to address various solid tumors. Through these developments, Gritstone bio aims to contribute to the evolving landscape of cancer treatment and enhance patient outcomes.

RayzeBio, Inc. is a biotechnology company based in San Diego, California, founded in 2020. It specializes in developing tumor-targeted small molecule medicines that utilize the therapeutic potential of radioisotopes, particularly Actinium-225, an alpha-emitting radioisotope aimed at treating solid tumors. RayzeBio is focused on enhancing cancer treatment outcomes by creating a robust pipeline of radiopharmaceutical candidates targeting validated oncology drug targets. The company's portfolio includes a range of drug candidates at various stages of development, from discovery to late-stage clinical programs, addressing significant market opportunities in the field of cancer therapeutics.

RayzeBio, Inc. is a biotechnology company based in San Diego, California, founded in 2020. It specializes in developing tumor-targeted small molecule medicines that utilize the therapeutic potential of radioisotopes, particularly Actinium-225, an alpha-emitting radioisotope aimed at treating solid tumors. RayzeBio is focused on enhancing cancer treatment outcomes by creating a robust pipeline of radiopharmaceutical candidates targeting validated oncology drug targets. The company's portfolio includes a range of drug candidates at various stages of development, from discovery to late-stage clinical programs, addressing significant market opportunities in the field of cancer therapeutics.

Monte Rosa Therapeutics is a biotechnology company focused on developing innovative cancer therapeutics that utilize molecular glue degraders (MGDs) to modulate protein degradation pathways. By leveraging the body's natural mechanisms for protein destruction, these small molecule drugs selectively target and degrade disease-causing proteins. The company employs its QuEEN platform, which combines artificial intelligence and proprietary experimental tools, to identify relevant proteins for degradation. With a diverse library of over 50,000 MGD molecules, Monte Rosa's lead candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for treating MYC-driven tumors. The company aims to advance its pipeline of pioneering therapies for cancer and other diseases.

Lava Therapeutics B.V. is a biotechnology company based in Utrecht, the Netherlands, founded in 2016. The company specializes in developing proprietary bispecific antibodies aimed at engaging gamma-delta T cells for the treatment of both hematological and solid cancers. Its innovative platform, known as the Gammabody platform, is designed to create next-generation bispecific antibodies that activate the immune system to recognize and eliminate tumor cells. By harnessing the potential of γδ T cells, Lava Therapeutics aims to produce potent, safe, and cost-effective biopharmaceuticals that enhance the body's ability to combat cancer, thereby transforming the landscape of cancer treatment.

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

Chinook Therapeutics is a clinical-stage biotechnology company based in Seattle, Washington, that specializes in developing precision medicines for kidney diseases. The company's lead program is atrasentan, an investigational endothelin receptor antagonist currently undergoing Phase III trials for IgA nephropathy and other glomerular diseases. Additionally, Chinook is evaluating BION-1301, an anti-APRIL monoclonal antibody, in a Phase Ib trial for IgA nephropathy. The company is also advancing CHK-336, a preclinical candidate targeting an undisclosed ultra-orphan kidney disease, along with research initiatives focused on other rare and severe chronic kidney conditions, including polycystic kidney disease. Chinook Therapeutics aims to address significant unmet medical needs in the field of nephrology through its innovative product pipeline.

T-knife is a developer of T cell receptors intended for T cell therapy of cancer.The company uses a humanized TCR mice platform to carry human TCRαβ gene loci and recombine a broad repertoire of human TCRs, enabling the efficient generation and virtual selection of cell receptors for any human tumor antigen.

Matterhorn Biosciences is a biotechnology company dedicated to developing cellular therapies that target the MR1 molecule, which displays cancer-specific metabolites on the surface of cancer cells. The company's approach involves creating M-TCR cell therapies that utilize T cells designed to recognize and attack these metabolites, facilitating the destruction of tumors across various tissue types. By leveraging a library of T-cell receptors that can be linked to MR1-specific T cells, Matterhorn aims to enhance the ability of the immune system to identify and eliminate a wide range of cancerous cells. This innovative focus positions Matterhorn at the forefront of cancer treatment research, aiming to provide effective therapeutic options for patients facing challenging diagnoses.

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.

Monte Rosa Therapeutics is a biotechnology company focused on developing innovative cancer therapeutics that utilize molecular glue degraders (MGDs) to modulate protein degradation pathways. By leveraging the body's natural mechanisms for protein destruction, these small molecule drugs selectively target and degrade disease-causing proteins. The company employs its QuEEN platform, which combines artificial intelligence and proprietary experimental tools, to identify relevant proteins for degradation. With a diverse library of over 50,000 MGD molecules, Monte Rosa's lead candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for treating MYC-driven tumors. The company aims to advance its pipeline of pioneering therapies for cancer and other diseases.

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

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

Aligos Therapeutics, Inc. is a clinical stage biopharmaceutical company that was founded in 2018 with the mission to become a world leader in the treatment of viral infections and liver diseases. Aligos is focused on the development of targeted antiviral therapies for chronic hepatitis B (CHB) and coronaviruses as well as leveraging its expertise in liver diseases to create targeted therapeutics for nonalcoholic steatohepatitis (NASH). Aligos’ strategy is to harness the deep expertise and decades of drug development experience its workforce has in liver disease, particularly viral hepatitis, to rapidly advance its pipeline of potentially best-in-class molecules.

VectivBio AG is a clinical-stage biotechnology company based in Basel, Switzerland, founded in 2019 as a spinout from Therachon. The company specializes in developing innovative treatments for severe rare diseases with significant unmet medical needs. Its primary product candidate, Apraglutide, is a long-acting synthetic analog of glucagon-like peptide-2 (GLP-2) designed to enhance the intestine's ability to absorb fluids and nutrients, thereby reducing the reliance on parenteral support. VectivBio aims to deliver transformational therapies that significantly improve the lives of patients and their families, rather than offering only incremental benefits over existing treatments.

Pipeline Therapeutics is a developer of regenerative therapies designed to promote functional recovery of neurological diseases. The company's therapies leverage the natural repair process in several different nervous system cell types to target diseases such as multiple sclerosis, providing patients with a treatment to repair damage to the nervous system.

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

Coda Biotherapeutics, Inc. is a biotechnology company focused on developing innovative treatments for neurological disorders. The company specializes in chemogenetic technology, which allows for precise modulation of neuronal activity through gene therapy. By using viral vectors, Coda Biotherapeutics introduces engineered neurotransmitter receptors into specific neuronal populations. These receptors are designed to respond to a proprietary small molecule, enabling targeted control of neuronal functions. This approach aims to effectively treat conditions such as neuropathic pain, epilepsy, and movement disorders, offering a potential advancement over traditional therapies. Founded in 2014, Coda Biotherapeutics is headquartered in San Francisco, California.

Anokion, a spin-off from the Ecole Polytechnique Fédérale de Lausanne (EPFL), is focused on applying the company’s antigen-specific immune tolerance technology to reduce the immunogenicity of therapeutic proteins and to treat autoimmune and allergic diseases. As a platform technology, Anokion’s approach to antigen-specific tolerance can be translated to virtually any protein in numerous clinical indications.

Passage Bio is a fully integrated gene therapy company with a mission to develop a portfolio of five life-transforming AAV-delivered therapeutics for the treatment of rare monogenic central nervous system diseases. The company is based in Philadelphia, PA and has a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program (GTP) as well as the Orphan Disease Center (ODC). Pursuant to the research collaboration, GTP conducts IND-enabling preclinical work, and Passage Bio is responsible for clinical development, regulatory, manufacturing and commercialization of all product candidates. The ODC is responsible for natural history studies, KOL engagement, and patient advocacy outreach.

Repare Therapeutics Inc. is a precision oncology company based in Montréal, Canada, specializing in the discovery and development of novel therapeutics through a synthetic lethality approach. Utilizing its proprietary SNIPRx platform, which combines genome-wide and CRISPR-enabled technology, the company systematically identifies targeted cancer therapies addressing genomic instability, particularly in DNA damage repair. Its lead candidate, RP-3500, is an oral small molecule inhibitor designed to treat solid tumors with specific DNA damage repair-related genomic alterations. Additionally, Repare is advancing a CCNE1-SL inhibitor program targeting tumors with CCNE1 amplification, along with a Polymerase Theta program focused on BRCA mutations and related genomic alterations. Founded in 2016, the company aims to improve cancer treatment by leveraging innovative drug discovery methods and addressing the unique vulnerabilities of tumor cells.

Chinook Therapeutics is a clinical-stage biotechnology company based in Seattle, Washington, that specializes in developing precision medicines for kidney diseases. The company's lead program is atrasentan, an investigational endothelin receptor antagonist currently undergoing Phase III trials for IgA nephropathy and other glomerular diseases. Additionally, Chinook is evaluating BION-1301, an anti-APRIL monoclonal antibody, in a Phase Ib trial for IgA nephropathy. The company is also advancing CHK-336, a preclinical candidate targeting an undisclosed ultra-orphan kidney disease, along with research initiatives focused on other rare and severe chronic kidney conditions, including polycystic kidney disease. Chinook Therapeutics aims to address significant unmet medical needs in the field of nephrology through its innovative product pipeline.

Oyster Point Pharma is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for ocular surface diseases. The company's primary focus is on its lead product candidate, OC-01, a nasal spray that acts as a selective nicotinic acetylcholine receptor agonist. OC-01 aims to address the signs and symptoms of dry eye disease, a chronic condition that affects over 30 million Americans. By activating the trigeminal parasympathetic pathway, OC-01 seeks to restore tear film homeostasis and stimulate the natural production of tears through the Lacrimal Functional Unit.

Passage Bio is a fully integrated gene therapy company with a mission to develop a portfolio of five life-transforming AAV-delivered therapeutics for the treatment of rare monogenic central nervous system diseases. The company is based in Philadelphia, PA and has a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program (GTP) as well as the Orphan Disease Center (ODC). Pursuant to the research collaboration, GTP conducts IND-enabling preclinical work, and Passage Bio is responsible for clinical development, regulatory, manufacturing and commercialization of all product candidates. The ODC is responsible for natural history studies, KOL engagement, and patient advocacy outreach.

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

Akero Therapeutics is a clinical-stage biotechnology company based in South San Francisco, California, that specializes in developing treatments for serious metabolic diseases, particularly nonalcoholic steatohepatitis (NASH). Founded in 2017 and formerly known as Pippin Pharmaceuticals, the company is dedicated to addressing the significant unmet medical needs associated with NASH, a condition characterized by liver inflammation and damage, which can lead to severe complications such as fibrosis and liver failure. Akero's lead product candidate, AKR-001, is a long-acting Fc fusion modified FGF21 protein designed to restore metabolic balance by targeting both liver and adipose tissues, thereby reducing liver fat and suppressing inflammation and fibrosis. This innovative approach builds upon two decades of research into FGF21 biology, positioning Akero as a frontrunner in the quest for effective NASH therapies.

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

Gotham Therapeutics is a biotechnology company based in New York that focuses on developing a novel class of drugs targeting epitranscriptomics machinery. Founded in 2017, the company aims to provide new treatment options for patients with cancers, autoimmune disorders, and neurodegenerative diseases. Its approach involves creating small molecules that modify the activity of messenger ribonucleic acid (mRNA) to influence protein transcription. This innovative strategy allows Gotham Therapeutics to potentially optimize therapeutic outcomes for individuals suffering from serious health conditions.

Coda Biotherapeutics, Inc. is a biotechnology company focused on developing innovative treatments for neurological disorders. The company specializes in chemogenetic technology, which allows for precise modulation of neuronal activity through gene therapy. By using viral vectors, Coda Biotherapeutics introduces engineered neurotransmitter receptors into specific neuronal populations. These receptors are designed to respond to a proprietary small molecule, enabling targeted control of neuronal functions. This approach aims to effectively treat conditions such as neuropathic pain, epilepsy, and movement disorders, offering a potential advancement over traditional therapies. Founded in 2014, Coda Biotherapeutics is headquartered in San Francisco, California.

Therachon is a biotechnology company dedicated to developing treatments for rare genetic diseases that currently lack available therapies. Based in Basel, Switzerland, with research facilities in Nice, France, Therachon focuses on creating innovative therapeutics for conditions such as achondroplasia, the most prevalent form of short-limbed dwarfism, through its lead candidate, TA-46. The company also addresses other rare gastrointestinal and musculoskeletal disorders, aiming to provide effective solutions for severe medical conditions like short bowel syndrome.

Akero Therapeutics is a clinical-stage biotechnology company based in South San Francisco, California, that specializes in developing treatments for serious metabolic diseases, particularly nonalcoholic steatohepatitis (NASH). Founded in 2017 and formerly known as Pippin Pharmaceuticals, the company is dedicated to addressing the significant unmet medical needs associated with NASH, a condition characterized by liver inflammation and damage, which can lead to severe complications such as fibrosis and liver failure. Akero's lead product candidate, AKR-001, is a long-acting Fc fusion modified FGF21 protein designed to restore metabolic balance by targeting both liver and adipose tissues, thereby reducing liver fat and suppressing inflammation and fibrosis. This innovative approach builds upon two decades of research into FGF21 biology, positioning Akero as a frontrunner in the quest for effective NASH therapies.

Lava Therapeutics B.V. is a biotechnology company based in Utrecht, the Netherlands, founded in 2016. The company specializes in developing proprietary bispecific antibodies aimed at engaging gamma-delta T cells for the treatment of both hematological and solid cancers. Its innovative platform, known as the Gammabody platform, is designed to create next-generation bispecific antibodies that activate the immune system to recognize and eliminate tumor cells. By harnessing the potential of γδ T cells, Lava Therapeutics aims to produce potent, safe, and cost-effective biopharmaceuticals that enhance the body's ability to combat cancer, thereby transforming the landscape of cancer treatment.

Enterprise Therapeutics Ltd. is a drug discovery company based in Brighton, United Kingdom, founded in 2014. It focuses on the research and development of innovative therapies for respiratory diseases, specifically targeting chronic obstructive pulmonary disease (COPD), cystic fibrosis, and asthma. The company aims to create disease-modifying therapies that address the underlying mechanisms of mucus congestion in the lungs, which can lead to breathing difficulties and increased susceptibility to lung infections. By developing these novel treatments, Enterprise Therapeutics seeks to enhance the quality of life for patients suffering from these chronic respiratory conditions.

Tempest Therapeutics is a biotechnology company based in South San Francisco, California, that specializes in developing small molecule therapeutics for cancer treatment. Founded in 2011, the company focuses on innovative therapies that enhance anti-tumor immunity and directly target tumor cells. Its pipeline includes several clinical-stage candidates, notably TPST-1120, which blocks the PPAR-alpha pathway to stimulate immune response, and TPST-8844, designed to inhibit an enzyme that allows tumor cells to evade immune detection. Additionally, Tempest Therapeutics is working on compounds that target prostaglandins to counteract immune suppression. By pursuing these mechanisms, the company aims to create effective treatments that harness the body's own immune system to combat cancer.

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

Quentis Therapeutics, Inc. is a biotechnology company based in New York, founded in 2016. The company specializes in developing immunotherapies that target endoplasmic reticulum (ER) stress response pathways within the tumor microenvironment. By addressing ER stress, Quentis aims to enhance the immune system's capacity to combat cancer, providing patients with innovative treatment options. Its therapeutic approach focuses on awakening anti-tumor immunity, thereby potentially improving outcomes for individuals facing various forms of cancer.

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

Aligos Therapeutics, Inc. is a clinical stage biopharmaceutical company that was founded in 2018 with the mission to become a world leader in the treatment of viral infections and liver diseases. Aligos is focused on the development of targeted antiviral therapies for chronic hepatitis B (CHB) and coronaviruses as well as leveraging its expertise in liver diseases to create targeted therapeutics for nonalcoholic steatohepatitis (NASH). Aligos’ strategy is to harness the deep expertise and decades of drug development experience its workforce has in liver disease, particularly viral hepatitis, to rapidly advance its pipeline of potentially best-in-class molecules.

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.

Oyster Point Pharma is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for ocular surface diseases. The company's primary focus is on its lead product candidate, OC-01, a nasal spray that acts as a selective nicotinic acetylcholine receptor agonist. OC-01 aims to address the signs and symptoms of dry eye disease, a chronic condition that affects over 30 million Americans. By activating the trigeminal parasympathetic pathway, OC-01 seeks to restore tear film homeostasis and stimulate the natural production of tears through the Lacrimal Functional Unit.

Nouscom is a biotechnology company focused on developing an immunotherapy platform that creates modified viral vector vaccines for cancer treatment. The company utilizes a range of engineered viral vectors optimized for the effective expression of long strings of tumor neoantigens. Additionally, its platform includes tools designed to induce broad and potent antitumor T-cell responses, which aim to enhance cancer-specific cellular immunity in patients.

Gritstone bio is a biotechnology company focused on developing personalized immunotherapies for cancer and infectious diseases. The company initially concentrated on tumor-specific neoantigens but has expanded its programs to include viral antigens found on the surfaces of infected cells. This approach leverages the immune system's ability to recognize abnormal cell targets, which is crucial for both anti-tumor and anti-viral immunity. Gritstone bio is building a robust pipeline of immunotherapy product candidates, including GRANITE, SLATE, and CORAL, designed to address various solid tumors. Through these developments, Gritstone bio aims to contribute to the evolving landscape of cancer treatment and enhance patient outcomes.

Venatorx Pharmaceuticals, Inc. is a pharmaceutical company focused on the discovery and development of novel anti-infective agents aimed at treating multi-drug-resistant bacterial infections and challenging viral infections. Founded in 2010 and based in Malvern, Pennsylvania, the company specializes in developing antibacterial drugs to combat resistant infections commonly encountered in hospital and community settings, such as those caused by MRSA, Pseudomonas spp., and Salmonella spp. Venatorx employs innovative chemical approaches to create treatments that exhibit selective and potent activity against various resistant bacterial strains, thereby providing healthcare professionals with broader treatment options to address significant unmet medical needs.

Repare Therapeutics Inc. is a precision oncology company based in Montréal, Canada, specializing in the discovery and development of novel therapeutics through a synthetic lethality approach. Utilizing its proprietary SNIPRx platform, which combines genome-wide and CRISPR-enabled technology, the company systematically identifies targeted cancer therapies addressing genomic instability, particularly in DNA damage repair. Its lead candidate, RP-3500, is an oral small molecule inhibitor designed to treat solid tumors with specific DNA damage repair-related genomic alterations. Additionally, Repare is advancing a CCNE1-SL inhibitor program targeting tumors with CCNE1 amplification, along with a Polymerase Theta program focused on BRCA mutations and related genomic alterations. Founded in 2016, the company aims to improve cancer treatment by leveraging innovative drug discovery methods and addressing the unique vulnerabilities of tumor cells.

Tarveda Therapeutics is a developer of miniature drug conjugates designed for the treatment of patients with a wide range of solid tumors. The company's HSP90 targeted drug conjugate platform with lead drug candidate PEN-866 selectively binds in tumors to the activated form of Heat Shock Protein 90 (HSP90) and releases its potent topoisomerase 1 inhibitor payload, SN38 and it also develops a miniature drug conjugate in clinical evaluation for the treatment of patients with somatostatin receptor 2 (SSTR2) expressing neuroendocrine, small cell lung and other solid tumors, enabling companies to extend the lives of patients with hard to treat cancers while minimizing potential toxicities.

Jecure Therapeutics, Inc. is a biotechnology company based in San Diego, California, focused on developing small-molecule therapeutics for the treatment of non-alcoholic steatohepatitis (NASH) and liver fibrosis. Established in 2015 and operating as a subsidiary of Genentech, Inc. since 2018, the company specializes in innovative drug discovery programs aimed at addressing serious inflammatory diseases. Jecure's therapeutics work by blocking inflammasome activation and disrupting the inflammation processes that lead to liver cell injury and death. The company utilizes advanced methodologies, including SiRNA-based murine fibrosis models for target identification and validation, as well as unique in-vivo screening models that replicate key features of NASH. This approach enables the identification of drug candidates capable of intervening throughout the disease progression, facilitating a clearer path for translational development.

BlueRock Therapeutics is a biotech company that plans to develop induced pluripotent stem cell (iPSC) therapies to cure a range of diseases using an industry-leading platform. One of the initial programs is to regenerate heart muscle in patients who have had a heart attack (myocard infarction, MI) or are suffering from chronic heart failure, leading causes of morbidity and mortality worldwid.

Turnstone Biologics Inc. is a biotechnology company based in Ottawa, Canada, with an additional office in New York. Founded in 2015, it is dedicated to developing innovative viral immunotherapies aimed at improving survival rates for cancer patients. The company's lead product, RIVAL-01, utilizes a vaccinia virus backbone to deliver three key immunomodulators: Flt3 ligand, anti-CTLA-4 antibody, and IL-12 cytokine. These components are designed to enhance immune activity and reprogram the tumor microenvironment for optimal tumor eradication. Additionally, Turnstone Biologics is advancing next-generation tumor-infiltrating lymphocyte (TIL) therapies, which involve selecting and expanding the most effective T cells from a patient’s tumor to enhance the body’s immune response against solid tumors. Through these approaches, Turnstone aims to deliver breakthrough immunotherapies for cancer treatment.

Nouscom is a biotechnology company focused on developing an immunotherapy platform that creates modified viral vector vaccines for cancer treatment. The company utilizes a range of engineered viral vectors optimized for the effective expression of long strings of tumor neoantigens. Additionally, its platform includes tools designed to induce broad and potent antitumor T-cell responses, which aim to enhance cancer-specific cellular immunity in patients.

Benvenue Medical, Inc. is a company specializing in the design, manufacture, and marketing of minimally invasive expandable implant systems for the treatment of spinal disorders, particularly vertebral compression fractures and degenerative disc diseases. Its key products include the Kiva treatment system, which addresses spinal fractures in the thoracic and lumbar regions, and the Blazer – C system, designed for pathological vertebral compression fractures. Additionally, the company offers the Luna 3D expandable interbody fusion system, suitable for various surgical approaches. Headquartered in Santa Clara, California, Benvenue Medical serves customers across the United States, Canada, Belgium, France, and Germany. Founded in 2004, the company is committed to providing innovative solutions that enhance patient outcomes and restore spinal alignment.

Kyras Therapeutics Inc. is a biotechnology company based in New York, specializing in the development of drugs aimed at treating incurable cancers and developmental disorders. Founded in 2015, the company focuses on addressing conditions linked to mutations in RAS genes, which are responsible for a significant percentage of various cancers, including pancreatic, colorectal, and lung cancers. By targeting these mutations, Kyras Therapeutics aims to provide effective treatments that can potentially cure patients suffering from these challenging health issues.

Inception IBD is focused on translating academic discoveries in the field of IBD

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

Turnstone Biologics Inc. is a biotechnology company based in Ottawa, Canada, with an additional office in New York. Founded in 2015, it is dedicated to developing innovative viral immunotherapies aimed at improving survival rates for cancer patients. The company's lead product, RIVAL-01, utilizes a vaccinia virus backbone to deliver three key immunomodulators: Flt3 ligand, anti-CTLA-4 antibody, and IL-12 cytokine. These components are designed to enhance immune activity and reprogram the tumor microenvironment for optimal tumor eradication. Additionally, Turnstone Biologics is advancing next-generation tumor-infiltrating lymphocyte (TIL) therapies, which involve selecting and expanding the most effective T cells from a patient’s tumor to enhance the body’s immune response against solid tumors. Through these approaches, Turnstone aims to deliver breakthrough immunotherapies for cancer treatment.

Gritstone bio is a biotechnology company focused on developing personalized immunotherapies for cancer and infectious diseases. The company initially concentrated on tumor-specific neoantigens but has expanded its programs to include viral antigens found on the surfaces of infected cells. This approach leverages the immune system's ability to recognize abnormal cell targets, which is crucial for both anti-tumor and anti-viral immunity. Gritstone bio is building a robust pipeline of immunotherapy product candidates, including GRANITE, SLATE, and CORAL, designed to address various solid tumors. Through these developments, Gritstone bio aims to contribute to the evolving landscape of cancer treatment and enhance patient outcomes.

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

Therachon is a biotechnology company dedicated to developing treatments for rare genetic diseases that currently lack available therapies. Based in Basel, Switzerland, with research facilities in Nice, France, Therachon focuses on creating innovative therapeutics for conditions such as achondroplasia, the most prevalent form of short-limbed dwarfism, through its lead candidate, TA-46. The company also addresses other rare gastrointestinal and musculoskeletal disorders, aiming to provide effective solutions for severe medical conditions like short bowel syndrome.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on developing gene therapy treatments for retinal degenerative diseases and neurodegenerative conditions affecting the central nervous system. Utilizing its core technology platforms—Mitochondrial Targeting Sequence and Optogenetics—the company aims to preserve and restore vision in patients with severe retinal disorders. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III trials for treating Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. In addition to these, GenSight is advancing several products in preclinical stages targeting various ophthalmic and neurodegenerative diseases. Founded in 2012, the company is dedicated to innovative approaches that aim to prevent vision loss and improve the quality of life for affected patients.

PIQUR Therapeutics is a Swiss pharmaceutical company focusing on the discovery and development of innovative anti-cancer drugs based on lipid kinase (PI3K) and mTOR inhibition. PIQUR’s pipeline originates from one of the most promising research areas in oncology. Both PI3K and mTOR are clinically validated drug targets in oncology. PIQUR has a secured patent scope protecting many chemical compounds. PIQUR Therapeutics AG develops breakthrough targeted oncology and dermatology medicines for the treatment of cancers and genetic diseases. The company lead compound bimiralisib focuses on the dual inhibition of the PI3K/mTOR pathway. Both PI3K and mTOR are clinically validated drug targets in oncology and dermato-oncology. PIQUR Therapeutics was founded in 2011 and is based in Basel, Switzerland.

CRISPR Therapeutics is focused on the development of transformative medicines using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. They have licensed the foundational CRISPR/Cas9 patent estate for human therapeutic use from their scientific founder, Dr. Emmanuelle Charpentier, who co-invented the application of CRISPR/Cas9 for gene editing. CRISPR Therapeutics’ vision is to cure serious human diseases at the molecular level using CRISPR-Cas9. The company is headquartered in Basel, Switzerland and has operations in London, UK.

CRISPR Therapeutics is focused on the development of transformative medicines using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. They have licensed the foundational CRISPR/Cas9 patent estate for human therapeutic use from their scientific founder, Dr. Emmanuelle Charpentier, who co-invented the application of CRISPR/Cas9 for gene editing. CRISPR Therapeutics’ vision is to cure serious human diseases at the molecular level using CRISPR-Cas9. The company is headquartered in Basel, Switzerland and has operations in London, UK.

Benvenue Medical, Inc. is a company specializing in the design, manufacture, and marketing of minimally invasive expandable implant systems for the treatment of spinal disorders, particularly vertebral compression fractures and degenerative disc diseases. Its key products include the Kiva treatment system, which addresses spinal fractures in the thoracic and lumbar regions, and the Blazer – C system, designed for pathological vertebral compression fractures. Additionally, the company offers the Luna 3D expandable interbody fusion system, suitable for various surgical approaches. Headquartered in Santa Clara, California, Benvenue Medical serves customers across the United States, Canada, Belgium, France, and Germany. Founded in 2004, the company is committed to providing innovative solutions that enhance patient outcomes and restore spinal alignment.