Versant Ventures

Granite Bio is a biotechnology company that specializes in the development of novel monoclonal antibodies aimed at treating inflammatory, autoimmune, and fibrotic disorders. The company's primary focus is on creating therapeutic antibodies that deplete pathogenic cells and selectively target key biological pathways associated with these diseases. By advancing innovative antibody treatments, Granite Bio aims to contribute significantly to the fields of biotechnology and pharmaceuticals, addressing critical needs in the life sciences sector.

Light Horse Therapeutics is a biotechnology company specializing in the application of gene editing to small-molecule drug discovery. It develops innovative methods to target the root causes of severe and life-threatening diseases, with an initial focus on challenging oncology targets. The company's advanced platform identifies critical functional domains within disease-related proteins, enabling the development of more effective therapies.

Borealis Biosciences is a discovery-stage biotechnology company focused on developing next-generation RNA-based therapies, particularly targeting kidney diseases. The company specializes in molecular and cellular biology, chemistry, and analytical capabilities, utilizing these strengths to identify and map target opportunities within specific patient subsets affected by kidney conditions. Borealis aims to enhance the delivery mechanisms of RNA medicines to distinct cell types, thereby addressing significant unmet medical needs in the treatment of kidney diseases.

Kate Therapeutics is a biotechnology company specializing in the development of adeno-associated virus (AAV)-based gene therapies aimed at treating genetically defined muscle and heart diseases. The company employs innovative technology platforms that enhance tissue-specific delivery and gene regulation, effectively addressing the limitations of existing gene therapies. By utilizing novel capsids, Kate Therapeutics achieves superior muscle transduction and liver de-targeting compared to naturally occurring serotypes, facilitating more effective tissue transduction at significantly lower viral doses. This advancement helps to overcome major challenges in muscle gene delivery, ultimately improving treatment outcomes for patients with these specific conditions.

Santa Ana Bio is a biotechnology company focused on developing precision biologics for autoimmune and inflammatory diseases. The company employs advanced technologies, including deep cell profiling, multi-omics platforms, and single-cell genomics, to create targeted therapies. By utilizing antibody engineering and sophisticated analytics, Santa Ana Bio aims to deliver personalized treatments that enhance patient outcomes and improve quality of life for those affected by autoimmune conditions.

iECURE is a biotechnology company dedicated to developing innovative gene editing therapies aimed at treating rare and life-threatening liver disorders, particularly in children. Its approach employs mutation-agnostic in vivo gene insertion techniques, which allow for targeted interventions in diseases characterized by metabolic "loss of function" disorders. By focusing on conditions with high unmet medical needs, iECURE aims to offer potential cures for devastating illnesses that currently lack effective treatment options. The company's commitment to advancing gene editing technology positions it as a key player in the field of genetic medicine.

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

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.

Nested Therapeutics is a stealth biotechnology company based in Cambridge, Massachusetts, founded in 2021. The company specializes in the development of precision oncology therapies aimed at targeting specific cancer genes. By identifying new driver mutations, Nested Therapeutics employs a platform that integrates insights from genomics, computational chemistry, proteomics, and artificial intelligence to discover novel small-molecule drugs. This innovative approach enables healthcare professionals to administer more effective precision medications, ultimately improving treatment outcomes for cancer patients.

Coda Biotherapeutics, Inc. is a biotechnology company focused on developing innovative therapies to modulate neuronal activity through its chemogenetic platform. Established in 2014 and based in San Francisco, the company employs gene therapy techniques to modify specific neuronal populations, enabling them to express engineered ligand-gated ion channels that are responsive to a proprietary small molecule. This interaction allows for precise, dose-dependent control of neuronal activity, facilitating the treatment of conditions such as neuropathic pain, epilepsy, and movement disorders. Coda's approach utilizes viral vectors to deliver these engineered neurotransmitter receptors directly to targeted neurons, enhancing the efficiency of treatment for severe nervous system disorders compared to traditional therapies.

iECURE is a biotechnology company dedicated to developing innovative gene editing therapies aimed at treating rare and life-threatening liver disorders, particularly in children. Its approach employs mutation-agnostic in vivo gene insertion techniques, which allow for targeted interventions in diseases characterized by metabolic "loss of function" disorders. By focusing on conditions with high unmet medical needs, iECURE aims to offer potential cures for devastating illnesses that currently lack effective treatment options. The company's commitment to advancing gene editing technology positions it as a key player in the field of genetic medicine.

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

Capsida is a biotechnology company focused on developing targeted gene therapies for monogenic and sporadic disorders affecting both the central nervous system and other areas of the body. The company utilizes a proprietary adeno-associated virus (AAV) engineering platform, which creates capsids specifically designed to target particular tissues and cells within affected organs. This advanced approach not only aims to enhance the efficacy of the therapies by ensuring they reach the intended sites of action, but also minimizes the impact on non-target tissues, thereby improving patient health outcomes in the treatment of life-threatening genetic disorders.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that focuses on targeted DNA integration to address severe genetic diseases. The firm employs advanced technology to enable the precise insertion of genetic sequences, effectively correcting mutations, replacing malfunctioning genes, and introducing therapeutic genetic materials into specific regions of the genome. By harnessing high-efficiency targeted gene integration, Graphite Bio aims to develop novel therapies that could potentially cure a wide array of serious and life-threatening conditions. Formerly known as Integral Medicines, Inc., the company rebranded in August 2020 and has operated since its incorporation in 2019. Graphite Bio is committed to the pioneering of a precision gene editing approach designed to fulfill the medical challenge of accurately "finding and replacing" genes.

Monte Rosa Therapeutics is a biotechnology company dedicated to developing innovative cancer therapeutics through the modulation of protein degradation pathways. Specializing in molecular glue degraders (MGDs), the company utilizes the body's natural mechanisms for protein destruction to selectively degrade proteins that are relevant to therapeutic outcomes. Employing its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, Monte Rosa identifies target proteins for degradation by MGDs from its extensive library of over 50,000 molecules. The company's most advanced product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for the treatment of MYC-driven tumors. Monte Rosa Therapeutics aims to deliver pioneering therapies for cancer and potentially other diseases through its innovative approaches.

Century Therapeutics, Inc. is a biotechnology company focused on developing innovative cell therapies derived from induced pluripotent stem cells (iPSCs) to treat hematologic and solid malignancies. Founded in 2018 and headquartered in Philadelphia, Pennsylvania, the company utilizes a specialized platform that integrates gene editing and protein engineering to create allogeneic therapies. This platform allows for the generation of master cell banks of modified immune effector cells, specifically engineered to optimize performance and reduce the risk of rejection by the host immune system. By employing advanced technologies such as CRISPR-mediated gene editing and proprietary chimeric antigen receptors, Century Therapeutics aims to address significant unmet medical needs in cancer and other diseases.

Monte Rosa Therapeutics is a biotechnology company dedicated to developing innovative cancer therapeutics through the modulation of protein degradation pathways. Specializing in molecular glue degraders (MGDs), the company utilizes the body's natural mechanisms for protein destruction to selectively degrade proteins that are relevant to therapeutic outcomes. Employing its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, Monte Rosa identifies target proteins for degradation by MGDs from its extensive library of over 50,000 molecules. The company's most advanced product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for the treatment of MYC-driven tumors. Monte Rosa Therapeutics aims to deliver pioneering therapies for cancer and potentially other diseases through its innovative approaches.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that focuses on targeted DNA integration to address severe genetic diseases. The firm employs advanced technology to enable the precise insertion of genetic sequences, effectively correcting mutations, replacing malfunctioning genes, and introducing therapeutic genetic materials into specific regions of the genome. By harnessing high-efficiency targeted gene integration, Graphite Bio aims to develop novel therapies that could potentially cure a wide array of serious and life-threatening conditions. Formerly known as Integral Medicines, Inc., the company rebranded in August 2020 and has operated since its incorporation in 2019. Graphite Bio is committed to the pioneering of a precision gene editing approach designed to fulfill the medical challenge of accurately "finding and replacing" genes.

Monte Rosa Therapeutics is a biotechnology company dedicated to developing innovative cancer therapeutics through the modulation of protein degradation pathways. Specializing in molecular glue degraders (MGDs), the company utilizes the body's natural mechanisms for protein destruction to selectively degrade proteins that are relevant to therapeutic outcomes. Employing its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, Monte Rosa identifies target proteins for degradation by MGDs from its extensive library of over 50,000 molecules. The company's most advanced product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for the treatment of MYC-driven tumors. Monte Rosa Therapeutics aims to deliver pioneering therapies for cancer and potentially other diseases through its innovative approaches.

Aligos Therapeutics, Inc. is a clinical-stage biopharmaceutical company founded in 2018 and based in South San Francisco, California. The company is dedicated to developing innovative therapeutics to meet unmet medical needs in viral and liver diseases. Its primary focus is on chronic hepatitis B (CHB), with a robust pipeline that includes several drug candidates: ALG-010133, a synthetic oligonucleotide currently in Phase I clinical trials; ALG-000184, a capsid assembly modulator; ALG-020572, another oligonucleotide; and ALG-125097, an siRNA drug candidate. Additionally, Aligos is advancing ALG-055009, a small molecule THR-ß agonist aimed at treating non-alcoholic steatohepatitis (NASH). Aligos aims to leverage its expertise in liver diseases and viral infections to develop targeted antiviral therapies and establish itself as a leader in this therapeutic area.

Coda Biotherapeutics, Inc. is a biotechnology company focused on developing innovative therapies to modulate neuronal activity through its chemogenetic platform. Established in 2014 and based in San Francisco, the company employs gene therapy techniques to modify specific neuronal populations, enabling them to express engineered ligand-gated ion channels that are responsive to a proprietary small molecule. This interaction allows for precise, dose-dependent control of neuronal activity, facilitating the treatment of conditions such as neuropathic pain, epilepsy, and movement disorders. Coda's approach utilizes viral vectors to deliver these engineered neurotransmitter receptors directly to targeted neurons, enhancing the efficiency of treatment for severe nervous system disorders compared to traditional therapies.

Passage Bio, Inc. is a genetic medicines company based in Philadelphia, Pennsylvania, dedicated to developing transformative therapies for rare monogenic central nervous system (CNS) diseases. Established in 2017, the company aims to create a portfolio of five AAV-delivered therapeutics targeting conditions such as GM1 Gangliosidosis, Frontotemporal dementia, and Krabbe Disease. Passage Bio collaborates closely with the University of Pennsylvania, leveraging its Gene Therapy Program for preclinical work while taking responsibility for the clinical development, regulatory processes, manufacturing, and commercialization of its product candidates. The partnership also involves research and development collaborations that support the advancement of innovative treatments for these rare disorders.

Repare Therapeutics Inc. is a precision oncology company based in Montréal, Canada, that specializes in discovering and developing targeted therapeutics through a synthetic lethality approach. Utilizing its proprietary SNIPRx platform, which integrates genome-wide analysis and CRISPR technology, Repare systematically identifies and advances cancer therapies aimed at genomic instability, particularly in DNA damage repair. The company's lead candidate, RP-3500, is an oral small molecule inhibitor designed for solid tumors with specific genomic alterations related to DNA damage repair. In addition, Repare is developing innovative programs such as a CCNE1-SL inhibitor for tumors with CCNE1 amplification and a Polymerase Theta program targeting BRCA mutations and other genomic changes. Founded in 2016, Repare Therapeutics is focused on addressing the unique vulnerabilities of tumor cells in genetically defined patient populations to enhance cancer treatment outcomes.

Century Therapeutics, Inc. is a biotechnology company focused on developing innovative cell therapies derived from induced pluripotent stem cells (iPSCs) to treat hematologic and solid malignancies. Founded in 2018 and headquartered in Philadelphia, Pennsylvania, the company utilizes a specialized platform that integrates gene editing and protein engineering to create allogeneic therapies. This platform allows for the generation of master cell banks of modified immune effector cells, specifically engineered to optimize performance and reduce the risk of rejection by the host immune system. By employing advanced technologies such as CRISPR-mediated gene editing and proprietary chimeric antigen receptors, Century Therapeutics aims to address significant unmet medical needs in cancer and other diseases.

Passage Bio, Inc. is a genetic medicines company based in Philadelphia, Pennsylvania, dedicated to developing transformative therapies for rare monogenic central nervous system (CNS) diseases. Established in 2017, the company aims to create a portfolio of five AAV-delivered therapeutics targeting conditions such as GM1 Gangliosidosis, Frontotemporal dementia, and Krabbe Disease. Passage Bio collaborates closely with the University of Pennsylvania, leveraging its Gene Therapy Program for preclinical work while taking responsibility for the clinical development, regulatory processes, manufacturing, and commercialization of its product candidates. The partnership also involves research and development collaborations that support the advancement of innovative treatments for these rare disorders.

Coda Biotherapeutics, Inc. is a biotechnology company focused on developing innovative therapies to modulate neuronal activity through its chemogenetic platform. Established in 2014 and based in San Francisco, the company employs gene therapy techniques to modify specific neuronal populations, enabling them to express engineered ligand-gated ion channels that are responsive to a proprietary small molecule. This interaction allows for precise, dose-dependent control of neuronal activity, facilitating the treatment of conditions such as neuropathic pain, epilepsy, and movement disorders. Coda's approach utilizes viral vectors to deliver these engineered neurotransmitter receptors directly to targeted neurons, enhancing the efficiency of treatment for severe nervous system disorders compared to traditional therapies.

Therachon is a biotechnology company specializing in the development of treatments for rare genetic diseases that lack available therapies. Primarily based in Basel, Switzerland, with research labs in Nice, France, the company is advancing its lead candidate, TA-46, a novel protein therapy aimed at treating achondroplasia, the most prevalent form of short-limbed dwarfism. In addition to its focus on achondroplasia, Therachon is also committed to developing therapeutics targeting rare gastrointestinal and musculoskeletal disorders, with the goal of enabling medical professionals to effectively manage conditions such as short bowel syndrome.

Aligos Therapeutics, Inc. is a clinical-stage biopharmaceutical company founded in 2018 and based in South San Francisco, California. The company is dedicated to developing innovative therapeutics to meet unmet medical needs in viral and liver diseases. Its primary focus is on chronic hepatitis B (CHB), with a robust pipeline that includes several drug candidates: ALG-010133, a synthetic oligonucleotide currently in Phase I clinical trials; ALG-000184, a capsid assembly modulator; ALG-020572, another oligonucleotide; and ALG-125097, an siRNA drug candidate. Additionally, Aligos is advancing ALG-055009, a small molecule THR-ß agonist aimed at treating non-alcoholic steatohepatitis (NASH). Aligos aims to leverage its expertise in liver diseases and viral infections to develop targeted antiviral therapies and establish itself as a leader in this therapeutic area.

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

Repare Therapeutics Inc. is a precision oncology company based in Montréal, Canada, that specializes in discovering and developing targeted therapeutics through a synthetic lethality approach. Utilizing its proprietary SNIPRx platform, which integrates genome-wide analysis and CRISPR technology, Repare systematically identifies and advances cancer therapies aimed at genomic instability, particularly in DNA damage repair. The company's lead candidate, RP-3500, is an oral small molecule inhibitor designed for solid tumors with specific genomic alterations related to DNA damage repair. In addition, Repare is developing innovative programs such as a CCNE1-SL inhibitor for tumors with CCNE1 amplification and a Polymerase Theta program targeting BRCA mutations and other genomic changes. Founded in 2016, Repare Therapeutics is focused on addressing the unique vulnerabilities of tumor cells in genetically defined patient populations to enhance cancer treatment outcomes.

Therachon is a biotechnology company specializing in the development of treatments for rare genetic diseases that lack available therapies. Primarily based in Basel, Switzerland, with research labs in Nice, France, the company is advancing its lead candidate, TA-46, a novel protein therapy aimed at treating achondroplasia, the most prevalent form of short-limbed dwarfism. In addition to its focus on achondroplasia, Therachon is also committed to developing therapeutics targeting rare gastrointestinal and musculoskeletal disorders, with the goal of enabling medical professionals to effectively manage conditions such as short bowel syndrome.

Audentes Therapeutics, a clinical stage biotechnology company based in San Francisco, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

Therachon is a biotechnology company specializing in the development of treatments for rare genetic diseases that lack available therapies. Primarily based in Basel, Switzerland, with research labs in Nice, France, the company is advancing its lead candidate, TA-46, a novel protein therapy aimed at treating achondroplasia, the most prevalent form of short-limbed dwarfism. In addition to its focus on achondroplasia, Therachon is also committed to developing therapeutics targeting rare gastrointestinal and musculoskeletal disorders, with the goal of enabling medical professionals to effectively manage conditions such as short bowel syndrome.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on the discovery and development of gene therapies for retinal degenerative diseases and neurodegenerative disorders of the central nervous system. Founded in 2012, the company utilizes innovative technologies, including mitochondrial targeting sequences and optogenetics, to develop treatments that aim to preserve or restore vision in patients with severe vision impairments. Its lead product candidates include GS010, an AAV2-based gene therapy that is currently undergoing Phase III clinical trials for Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is in Phase I/II trials for retinitis pigmentosa and geographic atrophy associated with dry age-related macular degeneration. GenSight Biologics is also advancing other product candidates in preclinical development targeting various ophthalmic and neurodegenerative conditions.

CRISPR Therapeutics AG is a biotechnology company based in Zug, Switzerland, dedicated to developing transformative gene-based medicines for serious human diseases using CRISPR/Cas9 technology. This revolutionary gene-editing platform allows for precise changes to genomic DNA. The company's portfolio includes therapeutic programs targeting hemoglobinopathies, oncology, regenerative medicine, and rare diseases. Their lead product candidate is CTX001, a gene-edited therapy for transfusion-dependent beta thalassemia or severe sickle cell disease, which has been approved as Casgevy in collaboration with Vertex Pharmaceuticals. Additionally, CRISPR Therapeutics is developing CTX110, an allogeneic CAR-T therapy targeting CD19-positive malignancies, and other programs like CTX120 for multiple myeloma and CTX130 for solid tumors and hematologic malignancies. The company also engages in regenerative medicine programs for diabetes and genetic disease treatments such as glycogen storage disease Ia, Duchenne muscular dystrophy, and cystic fibrosis. CRISPR Therapeutics has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and ViaCyte, Inc.

CRISPR Therapeutics AG is a biotechnology company based in Zug, Switzerland, dedicated to developing transformative gene-based medicines for serious human diseases using CRISPR/Cas9 technology. This revolutionary gene-editing platform allows for precise changes to genomic DNA. The company's portfolio includes therapeutic programs targeting hemoglobinopathies, oncology, regenerative medicine, and rare diseases. Their lead product candidate is CTX001, a gene-edited therapy for transfusion-dependent beta thalassemia or severe sickle cell disease, which has been approved as Casgevy in collaboration with Vertex Pharmaceuticals. Additionally, CRISPR Therapeutics is developing CTX110, an allogeneic CAR-T therapy targeting CD19-positive malignancies, and other programs like CTX120 for multiple myeloma and CTX130 for solid tumors and hematologic malignancies. The company also engages in regenerative medicine programs for diabetes and genetic disease treatments such as glycogen storage disease Ia, Duchenne muscular dystrophy, and cystic fibrosis. CRISPR Therapeutics has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and ViaCyte, Inc.

Audentes Therapeutics, a clinical stage biotechnology company based in San Francisco, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

CRISPR Therapeutics AG is a biotechnology company based in Zug, Switzerland, dedicated to developing transformative gene-based medicines for serious human diseases using CRISPR/Cas9 technology. This revolutionary gene-editing platform allows for precise changes to genomic DNA. The company's portfolio includes therapeutic programs targeting hemoglobinopathies, oncology, regenerative medicine, and rare diseases. Their lead product candidate is CTX001, a gene-edited therapy for transfusion-dependent beta thalassemia or severe sickle cell disease, which has been approved as Casgevy in collaboration with Vertex Pharmaceuticals. Additionally, CRISPR Therapeutics is developing CTX110, an allogeneic CAR-T therapy targeting CD19-positive malignancies, and other programs like CTX120 for multiple myeloma and CTX130 for solid tumors and hematologic malignancies. The company also engages in regenerative medicine programs for diabetes and genetic disease treatments such as glycogen storage disease Ia, Duchenne muscular dystrophy, and cystic fibrosis. CRISPR Therapeutics has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and ViaCyte, Inc.

AAVLife is a gene therapy company established in 2014 and headquartered in Paris, France. The firm focuses on developing treatments for Friedreich’s Ataxia Cardiomyopathy, a serious genetic disorder that leads to progressive neurological and cardiac symptoms, often manifesting in childhood or adolescence. AAVLife's innovative research utilizes adeno-associated virus (AAV) technology to deliver a normal gene to cardiac tissue, which has shown promise in reversing heart dysfunction in preclinical mouse models. The company's efforts have included planning clinical trials and collaborating with regulatory authorities to address safety and design aspects of the studies. AAVLife aims to advance its gene therapy approach to improve cardiac health in individuals affected by this life-threatening condition, emphasizing its commitment to patient communities and the organizations that support them.

Audentes Therapeutics, a clinical stage biotechnology company based in San Francisco, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

Veracyte, Inc. is a genomic diagnostics company based in South San Francisco, California, that specializes in providing advanced diagnostic solutions for cancer and other diseases. Its product offerings include the Afirma Genomic Sequencing Classifier, which helps identify benign thyroid nodules, the Percepta Genomic Sequencing Classifier for lung cancer, and the Envisia Genomic Classifier for distinguishing idiopathic pulmonary fibrosis from other interstitial lung diseases. Additionally, Veracyte offers the Prosigna Breast Cancer Prognostic Gene Signature Assay, which assesses the risk of distant recurrence in breast cancer patients. The company's tests leverage advances in genomic science to reduce the need for invasive procedures, thereby facilitating timely and accurate treatment decisions. Veracyte has established collaborations with Johnson & Johnson Innovation and Loxo Oncology to enhance its diagnostic capabilities and develop targeted therapies for genetically defined cancers. Since its incorporation in 2006 and subsequent name change in 2008, Veracyte has aimed to improve patient care through innovative genomic testing.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on the discovery and development of gene therapies for retinal degenerative diseases and neurodegenerative disorders of the central nervous system. Founded in 2012, the company utilizes innovative technologies, including mitochondrial targeting sequences and optogenetics, to develop treatments that aim to preserve or restore vision in patients with severe vision impairments. Its lead product candidates include GS010, an AAV2-based gene therapy that is currently undergoing Phase III clinical trials for Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is in Phase I/II trials for retinitis pigmentosa and geographic atrophy associated with dry age-related macular degeneration. GenSight Biologics is also advancing other product candidates in preclinical development targeting various ophthalmic and neurodegenerative conditions.

OpGen, Inc. is a precision medicine company focused on developing molecular diagnostics and informatics to address infectious diseases. Headquartered in Gaithersburg, Maryland, OpGen offers a range of products designed to provide clinicians with rapid and actionable information regarding life-threatening infections, ultimately aiming to enhance patient outcomes and reduce the spread of multidrug-resistant organisms (MDROs). Its key offerings include the Acuitas AMR Gene Panel, which detects bacterial nucleic acids and genetic markers of antimicrobial resistance, and the QuickFISH and PNA FISH products for pathogen identification in blood cultures. Additionally, OpGen provides the Acuitas Lighthouse informatics system, a cloud-based platform that integrates clinical lab results with patient and hospital data to support the management of antimicrobial-resistant infections. The company collaborates with the New York State Department of Health to develop solutions for detecting and tracking such infections in healthcare settings. Established in 2001, OpGen continues to innovate in addressing the critical challenge of antibiotic resistance through its advanced molecular microbiology solutions.

Helicos BioSciences Corporation (Helicos) is a life sciences company, which has developed technology focused on the research, drug discovery and clinical diagnostics markets. Its True Single Molecule Sequencing (tSMS) technology enables rapid analysis of large quantities of genetic material by directly sequencing single molecules of deoxyribonucleic acid (DNA) or single DNA copies of ribonucleic acid (RNA) (cDNA) and its approach of direct sequencing of RNA. Its Helicos Genetic Analysis Platform is designed to obtain sequencing information by repetitively performing a cycle of biochemical reactions on individual DNA or RNA molecules and imaging the results after each cycle. The platform consists of an instrument called the HeliScope Single Molecule Sequencer, an image analysis computer tower called the HeliScope Analysis Engine, associated reagents, which are chemicals used in the sequencing process and disposable supplies.

Groove Biopharma focuses on the generation of novel microRNA (miRNA) therapeutics with improved properties, including higher potency and enhanced pharmacodynamic activity. Endogenous microRNAs (miRNAs) are involved in extensive regulatory networks for many target genes and pathways. Groove Biopharma’s core technology addresses a new avenue of nucleotide therapeutic drug discovery via modulation of cellular miRNA. Using exclusive technology, Groove Biopharma is generating a series of lead candidates with the potential to be best-in-class therapeutics. By incorporating proprietary structural modifications into miRNA therapeutics, the Groove Biopharma technology platform presents promising potential for treating a wide range of diseases, including cancer, fibrosis, and infectious disease.

OpGen, Inc. is a precision medicine company focused on developing molecular diagnostics and informatics to address infectious diseases. Headquartered in Gaithersburg, Maryland, OpGen offers a range of products designed to provide clinicians with rapid and actionable information regarding life-threatening infections, ultimately aiming to enhance patient outcomes and reduce the spread of multidrug-resistant organisms (MDROs). Its key offerings include the Acuitas AMR Gene Panel, which detects bacterial nucleic acids and genetic markers of antimicrobial resistance, and the QuickFISH and PNA FISH products for pathogen identification in blood cultures. Additionally, OpGen provides the Acuitas Lighthouse informatics system, a cloud-based platform that integrates clinical lab results with patient and hospital data to support the management of antimicrobial-resistant infections. The company collaborates with the New York State Department of Health to develop solutions for detecting and tracking such infections in healthcare settings. Established in 2001, OpGen continues to innovate in addressing the critical challenge of antibiotic resistance through its advanced molecular microbiology solutions.

Okairos AG is a biopharmaceutical company headquartered in Basel, Switzerland, with operations in Rome and Naples, Italy. The company specializes in the discovery and development of genetic T-cell vaccines, focusing on conditions such as hepatitis C virus. Okairos also excels in gene delivery technologies, providing expertise in scalable viral-vector manufacturing processes. This enables other biotech firms to transition genetic vaccines and medicinal products from preclinical stages to commercial readiness, utilizing advanced good manufacturing practice (GMP) facilities. Previously a subsidiary of Istituto Di Ricerche Di Biologia Molecolare P. Angeletti Spa, Okairos is dedicated to advancing innovative therapeutic solutions in the healthcare sector.

Veracyte, Inc. is a genomic diagnostics company based in South San Francisco, California, that specializes in providing advanced diagnostic solutions for cancer and other diseases. Its product offerings include the Afirma Genomic Sequencing Classifier, which helps identify benign thyroid nodules, the Percepta Genomic Sequencing Classifier for lung cancer, and the Envisia Genomic Classifier for distinguishing idiopathic pulmonary fibrosis from other interstitial lung diseases. Additionally, Veracyte offers the Prosigna Breast Cancer Prognostic Gene Signature Assay, which assesses the risk of distant recurrence in breast cancer patients. The company's tests leverage advances in genomic science to reduce the need for invasive procedures, thereby facilitating timely and accurate treatment decisions. Veracyte has established collaborations with Johnson & Johnson Innovation and Loxo Oncology to enhance its diagnostic capabilities and develop targeted therapies for genetically defined cancers. Since its incorporation in 2006 and subsequent name change in 2008, Veracyte has aimed to improve patient care through innovative genomic testing.

Groove Biopharma focuses on the generation of novel microRNA (miRNA) therapeutics with improved properties, including higher potency and enhanced pharmacodynamic activity. Endogenous microRNAs (miRNAs) are involved in extensive regulatory networks for many target genes and pathways. Groove Biopharma’s core technology addresses a new avenue of nucleotide therapeutic drug discovery via modulation of cellular miRNA. Using exclusive technology, Groove Biopharma is generating a series of lead candidates with the potential to be best-in-class therapeutics. By incorporating proprietary structural modifications into miRNA therapeutics, the Groove Biopharma technology platform presents promising potential for treating a wide range of diseases, including cancer, fibrosis, and infectious disease.

Fluidigm Corporation develops and markets innovative technologies and tools for life sciences research globally. The company specializes in integrated fluidic circuits that control fluids at the nanoscale, enabling a range of applications in biological research. Its product offerings include analytical systems such as the Helios CyTOF system, the Hyperion imaging system, and the Biomark HD system, along with various assays and reagents designed for immuno-oncology and genetic analysis. Fluidigm also provides preparatory instruments and single-cell microfluidics, such as the C1 and Polaris systems. The company serves a diverse clientele, including academic institutions, clinical research laboratories, biopharmaceutical, biotechnology, and agricultural biotechnology companies, focusing on non-diagnostic and non-clinical applications. Established in 1999 as Mycometrix Corporation, it adopted the name Fluidigm Corporation in 2001 and is headquartered in South San Francisco, California.

Helicos BioSciences Corporation (Helicos) is a life sciences company, which has developed technology focused on the research, drug discovery and clinical diagnostics markets. Its True Single Molecule Sequencing (tSMS) technology enables rapid analysis of large quantities of genetic material by directly sequencing single molecules of deoxyribonucleic acid (DNA) or single DNA copies of ribonucleic acid (RNA) (cDNA) and its approach of direct sequencing of RNA. Its Helicos Genetic Analysis Platform is designed to obtain sequencing information by repetitively performing a cycle of biochemical reactions on individual DNA or RNA molecules and imaging the results after each cycle. The platform consists of an instrument called the HeliScope Single Molecule Sequencer, an image analysis computer tower called the HeliScope Analysis Engine, associated reagents, which are chemicals used in the sequencing process and disposable supplies.

PhaseRx is a Seattle-based biopharmaceutical startup focused on developing intracellular enzyme replacement therapies for inherited liver diseases using its proprietary Hybrid mRNA Technology platform. This platform enables the synthesis of missing enzymes within cells, targeting three urea cycle disorders: ornithine transcarbamylase deficiency (OTCD), argininosuccinate lyase deficiency (ASL deficiency), and argininosuccinate synthetase deficiency (ASS1 deficiency). The company was founded by industry veterans and renowned academics, attracting top-tier venture capital investments.

OpGen, Inc. is a precision medicine company focused on developing molecular diagnostics and informatics to address infectious diseases. Headquartered in Gaithersburg, Maryland, OpGen offers a range of products designed to provide clinicians with rapid and actionable information regarding life-threatening infections, ultimately aiming to enhance patient outcomes and reduce the spread of multidrug-resistant organisms (MDROs). Its key offerings include the Acuitas AMR Gene Panel, which detects bacterial nucleic acids and genetic markers of antimicrobial resistance, and the QuickFISH and PNA FISH products for pathogen identification in blood cultures. Additionally, OpGen provides the Acuitas Lighthouse informatics system, a cloud-based platform that integrates clinical lab results with patient and hospital data to support the management of antimicrobial-resistant infections. The company collaborates with the New York State Department of Health to develop solutions for detecting and tracking such infections in healthcare settings. Established in 2001, OpGen continues to innovate in addressing the critical challenge of antibiotic resistance through its advanced molecular microbiology solutions.

Fluidigm Corporation develops and markets innovative technologies and tools for life sciences research globally. The company specializes in integrated fluidic circuits that control fluids at the nanoscale, enabling a range of applications in biological research. Its product offerings include analytical systems such as the Helios CyTOF system, the Hyperion imaging system, and the Biomark HD system, along with various assays and reagents designed for immuno-oncology and genetic analysis. Fluidigm also provides preparatory instruments and single-cell microfluidics, such as the C1 and Polaris systems. The company serves a diverse clientele, including academic institutions, clinical research laboratories, biopharmaceutical, biotechnology, and agricultural biotechnology companies, focusing on non-diagnostic and non-clinical applications. Established in 1999 as Mycometrix Corporation, it adopted the name Fluidigm Corporation in 2001 and is headquartered in South San Francisco, California.

Pathwork Diagnostics, located in Redwood City, California, specializes in the development of molecular diagnostics for oncology. The company is known for its Pathwork Tissue of Origin Test, which was the first microarray-based gene expression test to receive FDA clearance. This innovative test assists in the identification of challenging tumors, including poorly differentiated, undifferentiated, and metastatic cancers. Pathwork Diagnostics offers lab services for various specimen types, including formalin-fixed, paraffin-embedded, and frozen samples, leveraging genomic information to enhance tumor classification and support oncological decision-making.

Helicos BioSciences Corporation (Helicos) is a life sciences company, which has developed technology focused on the research, drug discovery and clinical diagnostics markets. Its True Single Molecule Sequencing (tSMS) technology enables rapid analysis of large quantities of genetic material by directly sequencing single molecules of deoxyribonucleic acid (DNA) or single DNA copies of ribonucleic acid (RNA) (cDNA) and its approach of direct sequencing of RNA. Its Helicos Genetic Analysis Platform is designed to obtain sequencing information by repetitively performing a cycle of biochemical reactions on individual DNA or RNA molecules and imaging the results after each cycle. The platform consists of an instrument called the HeliScope Single Molecule Sequencer, an image analysis computer tower called the HeliScope Analysis Engine, associated reagents, which are chemicals used in the sequencing process and disposable supplies.

Fluidigm Corporation develops and markets innovative technologies and tools for life sciences research globally. The company specializes in integrated fluidic circuits that control fluids at the nanoscale, enabling a range of applications in biological research. Its product offerings include analytical systems such as the Helios CyTOF system, the Hyperion imaging system, and the Biomark HD system, along with various assays and reagents designed for immuno-oncology and genetic analysis. Fluidigm also provides preparatory instruments and single-cell microfluidics, such as the C1 and Polaris systems. The company serves a diverse clientele, including academic institutions, clinical research laboratories, biopharmaceutical, biotechnology, and agricultural biotechnology companies, focusing on non-diagnostic and non-clinical applications. Established in 1999 as Mycometrix Corporation, it adopted the name Fluidigm Corporation in 2001 and is headquartered in South San Francisco, California.

Fluidigm Corporation develops and markets innovative technologies and tools for life sciences research globally. The company specializes in integrated fluidic circuits that control fluids at the nanoscale, enabling a range of applications in biological research. Its product offerings include analytical systems such as the Helios CyTOF system, the Hyperion imaging system, and the Biomark HD system, along with various assays and reagents designed for immuno-oncology and genetic analysis. Fluidigm also provides preparatory instruments and single-cell microfluidics, such as the C1 and Polaris systems. The company serves a diverse clientele, including academic institutions, clinical research laboratories, biopharmaceutical, biotechnology, and agricultural biotechnology companies, focusing on non-diagnostic and non-clinical applications. Established in 1999 as Mycometrix Corporation, it adopted the name Fluidigm Corporation in 2001 and is headquartered in South San Francisco, California.

Fluidigm Corporation develops and markets innovative technologies and tools for life sciences research globally. The company specializes in integrated fluidic circuits that control fluids at the nanoscale, enabling a range of applications in biological research. Its product offerings include analytical systems such as the Helios CyTOF system, the Hyperion imaging system, and the Biomark HD system, along with various assays and reagents designed for immuno-oncology and genetic analysis. Fluidigm also provides preparatory instruments and single-cell microfluidics, such as the C1 and Polaris systems. The company serves a diverse clientele, including academic institutions, clinical research laboratories, biopharmaceutical, biotechnology, and agricultural biotechnology companies, focusing on non-diagnostic and non-clinical applications. Established in 1999 as Mycometrix Corporation, it adopted the name Fluidigm Corporation in 2001 and is headquartered in South San Francisco, California.