CODA Biotherapeutics’ revolutionary chemogenetic platform aims to control the activity of cells to treat disease with chemogenetics, the goal is to modify a target cell population using gene therapy to express a tunable “switch” protein. Cells modified with the “switch” can be activated or inactivated in a dose-dependent manner by a subsequently administered small molecule therapeutic, an effect that should only occur in the modified cells.
iECURE is a biotechnology company focused on developing gene editing therapies aimed at treating rare and life-threatening liver disorders, particularly in children. The company employs mutation-agnostic in vivo gene insertion techniques, which allow for targeted therapeutic interventions without being limited by specific genetic mutations. Its initial programs concentrate on addressing metabolic "loss of function" disorders, which often have significant unmet medical needs. Through its innovative approach, iECURE aims to provide effective treatments for devastating diseases that currently lack viable options.
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 is a gene editing company focused on targeted DNA integration to address severe diseases. The company employs technology that enables the insertion of new genetic sequences to replace defective genes, repair damaged portions of genes, and insert therapeutic genetic material at specific locations within the genome. This precise approach leverages natural cellular DNA repair mechanisms to enhance the efficiency of gene integration, aiming to correct the underlying causes of various diseases. Founded in 2019 and based in South San Francisco, California, Graphite Bio was previously known as Integral Medicines, Inc. and rebranded in August 2020. The company is committed to advancing gene replacement therapies and potential cures for individuals affected by serious health conditions.
Graphite Bio is a gene editing company focused on targeted DNA integration to address severe diseases. The company employs technology that enables the insertion of new genetic sequences to replace defective genes, repair damaged portions of genes, and insert therapeutic genetic material at specific locations within the genome. This precise approach leverages natural cellular DNA repair mechanisms to enhance the efficiency of gene integration, aiming to correct the underlying causes of various diseases. Founded in 2019 and based in South San Francisco, California, Graphite Bio was previously known as Integral Medicines, Inc. and rebranded in August 2020. The company is committed to advancing gene replacement therapies and potential cures for individuals affected by serious health conditions.
CODA Biotherapeutics’ revolutionary chemogenetic platform aims to control the activity of cells to treat disease with chemogenetics, the goal is to modify a target cell population using gene therapy to express a tunable “switch” protein. Cells modified with the “switch” can be activated or inactivated in a dose-dependent manner by a subsequently administered small molecule therapeutic, an effect that should only occur in the modified cells.
Passage Bio is a genetic medicines company based in Philadelphia, Pennsylvania, specializing in the development of therapies for rare monogenic central nervous system (CNS) diseases. Founded 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 managing clinical development, regulatory affairs, manufacturing, and commercialization of its product candidates. The company also engages with the Orphan Disease Center for natural history studies, key opinion leader involvement, and patient advocacy.
Passage Bio is a genetic medicines company based in Philadelphia, Pennsylvania, specializing in the development of therapies for rare monogenic central nervous system (CNS) diseases. Founded 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 managing clinical development, regulatory affairs, manufacturing, and commercialization of its product candidates. The company also engages with the Orphan Disease Center for natural history studies, key opinion leader involvement, and patient advocacy.
Gotham Therapeutics is a biotechnology company based in New York that focuses on developing a novel class of drugs aimed at targeting epitranscriptomics machinery. Established in 2017, the company seeks to create new treatment options for patients afflicted with various conditions, including cancers, autoimmune disorders, and neurodegenerative diseases. By altering the activity of proteins that modify messenger ribonucleic acid (mRNA), Gotham Therapeutics aims to advance therapeutic strategies and improve patient outcomes in these challenging medical areas.
CODA Biotherapeutics’ revolutionary chemogenetic platform aims to control the activity of cells to treat disease with chemogenetics, the goal is to modify a target cell population using gene therapy to express a tunable “switch” protein. Cells modified with the “switch” can be activated or inactivated in a dose-dependent manner by a subsequently administered small molecule therapeutic, an effect that should only occur in the modified cells.
Therachon is a global biotechnology company focused on developing medicines for rare, genetic diseases that currently have no available treatments. The company’s lead pipeline candidate, TA-46, is a novel protein therapy in development for achondroplasia, the most common form of short-limbed dwarfism. Therachon is headquartered in Basel, Switzerland, with research labs in Nice, France.
Therachon is a global biotechnology company focused on developing medicines for rare, genetic diseases that currently have no available treatments. The company’s lead pipeline candidate, TA-46, is a novel protein therapy in development for achondroplasia, the most common form of short-limbed dwarfism. Therachon is headquartered in Basel, Switzerland, with research labs in Nice, France.
Audentes Therapeutics is a clinical-stage biotechnology company dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases caused by single gene defects. The company is advancing several therapies, including AT132, currently in Phase I/II clinical trials for X-linked myotubular myopathy, and AT342, also in Phase I/II studies for Crigler-Najjar syndrome. Additionally, Audentes is exploring AT845 in preclinical studies for Pompe disease and AT307 for the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia. The company is also working on vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Collaborations include a partnership with Genethon for XLMTM treatments and an agreement with the University of Pennsylvania for Crigler-Najjar syndrome products. Founded in 2012 and headquartered in San Francisco, California, Audentes Therapeutics operates as a subsidiary of Astellas US Holding, Inc.
Therachon is a global biotechnology company focused on developing medicines for rare, genetic diseases that currently have no available treatments. The company’s lead pipeline candidate, TA-46, is a novel protein therapy in development for achondroplasia, the most common form of short-limbed dwarfism. Therachon is headquartered in Basel, Switzerland, with research labs in Nice, France.
GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, specializing in the discovery and development of gene therapy treatments for retinal degenerative diseases and neurodegenerative disorders of the central nervous system. The company utilizes two primary technology platforms: mitochondrial targeting sequences and optogenetics, aiming to preserve or restore vision in patients affected by severe retinal diseases. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III clinical trials for treating Leber hereditary optic neuropathy associated with ND4 gene mutations, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. Additionally, GenSight is advancing various preclinical programs targeting both ophthalmic and neurodegenerative conditions, reflecting its commitment to innovative solutions for patients with significant vision impairment or blindness. GenSight Biologics was founded in 2012.
CRISPR Therapeutics AG is a gene editing company dedicated to developing transformative gene-based medicines for serious human diseases using its proprietary CRISPR/Cas9 technology. This revolutionary platform allows for precise modifications to genomic DNA, enabling the company to target a variety of disease areas, including hemoglobinopathies, oncology, regenerative medicine, and rare diseases. The lead product candidate, CTX001, is an ex vivo CRISPR gene-edited therapy aimed at treating transfusion-dependent beta thalassemia and severe sickle cell disease by engineering patients' hematopoietic stem cells to produce elevated levels of fetal hemoglobin. Additionally, CRISPR Therapeutics is advancing other therapies, including CTX110, an allogeneic CAR-T therapy for certain malignancies, and additional programs targeting conditions such as multiple myeloma, solid tumors, diabetes, and various genetic disorders. The company collaborates with strategic partners such as Bayer Healthcare, Vertex Pharmaceuticals, and ViaCyte, and is headquartered in Zug, Switzerland.
CRISPR Therapeutics AG is a gene editing company dedicated to developing transformative gene-based medicines for serious human diseases using its proprietary CRISPR/Cas9 technology. This revolutionary platform allows for precise modifications to genomic DNA, enabling the company to target a variety of disease areas, including hemoglobinopathies, oncology, regenerative medicine, and rare diseases. The lead product candidate, CTX001, is an ex vivo CRISPR gene-edited therapy aimed at treating transfusion-dependent beta thalassemia and severe sickle cell disease by engineering patients' hematopoietic stem cells to produce elevated levels of fetal hemoglobin. Additionally, CRISPR Therapeutics is advancing other therapies, including CTX110, an allogeneic CAR-T therapy for certain malignancies, and additional programs targeting conditions such as multiple myeloma, solid tumors, diabetes, and various genetic disorders. The company collaborates with strategic partners such as Bayer Healthcare, Vertex Pharmaceuticals, and ViaCyte, and is headquartered in Zug, Switzerland.
Audentes Therapeutics is a clinical-stage biotechnology company dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases caused by single gene defects. The company is advancing several therapies, including AT132, currently in Phase I/II clinical trials for X-linked myotubular myopathy, and AT342, also in Phase I/II studies for Crigler-Najjar syndrome. Additionally, Audentes is exploring AT845 in preclinical studies for Pompe disease and AT307 for the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia. The company is also working on vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Collaborations include a partnership with Genethon for XLMTM treatments and an agreement with the University of Pennsylvania for Crigler-Najjar syndrome products. Founded in 2012 and headquartered in San Francisco, California, Audentes Therapeutics operates as a subsidiary of Astellas US Holding, Inc.
CRISPR Therapeutics AG is a gene editing company dedicated to developing transformative gene-based medicines for serious human diseases using its proprietary CRISPR/Cas9 technology. This revolutionary platform allows for precise modifications to genomic DNA, enabling the company to target a variety of disease areas, including hemoglobinopathies, oncology, regenerative medicine, and rare diseases. The lead product candidate, CTX001, is an ex vivo CRISPR gene-edited therapy aimed at treating transfusion-dependent beta thalassemia and severe sickle cell disease by engineering patients' hematopoietic stem cells to produce elevated levels of fetal hemoglobin. Additionally, CRISPR Therapeutics is advancing other therapies, including CTX110, an allogeneic CAR-T therapy for certain malignancies, and additional programs targeting conditions such as multiple myeloma, solid tumors, diabetes, and various genetic disorders. The company collaborates with strategic partners such as Bayer Healthcare, Vertex Pharmaceuticals, and ViaCyte, and is headquartered in Zug, Switzerland.
AAVLife, founded in 2014 and based in Paris, France, specializes in gene therapy services and conducts clinical trials focused on Friedreich’s Ataxia Cardiomyopathy. The company's innovative approach utilizes an adeno-associated virus to deliver a normal gene into cardiac tissue, aiming to restore cardiac function and reverse heart enlargement observed in mouse models of the disease. AAVLife is advancing its research to determine appropriate dosing and administration methods for upcoming clinical trials. Plans include conferring with regulatory authorities to establish requirements for toxicity studies and trial design. The aim is to initiate a clinical trial to evaluate the gene therapy for heart disease linked to Friedreich’s Ataxia, following an observational study. This genetic disorder typically manifests during childhood or adolescence and predominantly affects cardiac health, leading to significant mortality in early adulthood. AAVLife is dedicated to improving patient outcomes and values its connections with patient communities and related organizations.
Audentes Therapeutics is a clinical-stage biotechnology company dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases caused by single gene defects. The company is advancing several therapies, including AT132, currently in Phase I/II clinical trials for X-linked myotubular myopathy, and AT342, also in Phase I/II studies for Crigler-Najjar syndrome. Additionally, Audentes is exploring AT845 in preclinical studies for Pompe disease and AT307 for the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia. The company is also working on vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Collaborations include a partnership with Genethon for XLMTM treatments and an agreement with the University of Pennsylvania for Crigler-Najjar syndrome products. Founded in 2012 and headquartered in San Francisco, California, Audentes Therapeutics operates as a subsidiary of Astellas US Holding, Inc.
GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, specializing in the discovery and development of gene therapy treatments for retinal degenerative diseases and neurodegenerative disorders of the central nervous system. The company utilizes two primary technology platforms: mitochondrial targeting sequences and optogenetics, aiming to preserve or restore vision in patients affected by severe retinal diseases. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III clinical trials for treating Leber hereditary optic neuropathy associated with ND4 gene mutations, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. Additionally, GenSight is advancing various preclinical programs targeting both ophthalmic and neurodegenerative conditions, reflecting its commitment to innovative solutions for patients with significant vision impairment or blindness. GenSight Biologics was founded in 2012.
OpGen, Inc. is a precision medicine company based in Gaithersburg, Maryland, that specializes in developing molecular diagnostics and informatics aimed at combating infectious diseases. The company offers a range of products, including the Acuitas AMR Gene Panel, which detects bacterial nucleic acids and antimicrobial resistance markers, and QuickFISH and PNA FISH tests for identifying pathogens in blood cultures. Additionally, OpGen provides the Acuitas Lighthouse informatics system, a cloud-based platform that integrates clinical lab results with patient data to help manage multidrug-resistant organisms in healthcare settings. Through its strategic collaboration with the New York State Department of Health, OpGen is working on solutions to detect and manage antimicrobial-resistant infections in healthcare facilities. The company's innovative technologies are utilized by academic, government, and research institutions globally for applications such as strain typing and genome assembly. OpGen aims to enhance patient care and outcomes while addressing the growing challenge of antibiotic resistance.
Helicos BioSciences Corporation is a life sciences company that specializes in advanced genetic analysis technology. It has developed the True Single Molecule Sequencing (tSMS) technology, which allows for the rapid analysis of large amounts of genetic material by directly sequencing individual molecules of DNA and RNA. This innovative approach enables precise sequencing and offers significant advantages in research, drug discovery, and clinical diagnostics. The Helicos Genetic Analysis Platform includes the HeliScope Single Molecule Sequencer, which performs a series of biochemical reactions on single DNA or RNA molecules, and the HeliScope Analysis Engine, an image analysis computer system that processes the sequencing data. The platform also utilizes specialized reagents and disposable supplies essential for the sequencing process. Through its unique technology, Helicos aims to enhance the understanding of genetic information and improve applications in various fields of life sciences.
OpGen, Inc. is a precision medicine company based in Gaithersburg, Maryland, that specializes in developing molecular diagnostics and informatics aimed at combating infectious diseases. The company offers a range of products, including the Acuitas AMR Gene Panel, which detects bacterial nucleic acids and antimicrobial resistance markers, and QuickFISH and PNA FISH tests for identifying pathogens in blood cultures. Additionally, OpGen provides the Acuitas Lighthouse informatics system, a cloud-based platform that integrates clinical lab results with patient data to help manage multidrug-resistant organisms in healthcare settings. Through its strategic collaboration with the New York State Department of Health, OpGen is working on solutions to detect and manage antimicrobial-resistant infections in healthcare facilities. The company's innovative technologies are utilized by academic, government, and research institutions globally for applications such as strain typing and genome assembly. OpGen aims to enhance patient care and outcomes while addressing the growing challenge of antibiotic resistance.
Okairos AG is a biopharmaceutical company that specializes in the discovery and development of genetic T-cell vaccines, with a significant focus on combating the hepatitis C virus. The company is also involved in providing health and allied services. Headquartered in Basel, Switzerland, Okairos operates in Rome and Naples, Italy. It has expertise in gene delivery technologies and scalable viral-vector manufacturing processes, which facilitate the advancement of vaccines and advanced therapies. By utilizing state-of-the-art GMP facilities, Okairos supports biotech companies in transitioning their genetic vaccines and medicinal products from preclinical stages to commercial readiness.
Helicos BioSciences Corporation is a life sciences company that specializes in advanced genetic analysis technology. It has developed the True Single Molecule Sequencing (tSMS) technology, which allows for the rapid analysis of large amounts of genetic material by directly sequencing individual molecules of DNA and RNA. This innovative approach enables precise sequencing and offers significant advantages in research, drug discovery, and clinical diagnostics. The Helicos Genetic Analysis Platform includes the HeliScope Single Molecule Sequencer, which performs a series of biochemical reactions on single DNA or RNA molecules, and the HeliScope Analysis Engine, an image analysis computer system that processes the sequencing data. The platform also utilizes specialized reagents and disposable supplies essential for the sequencing process. Through its unique technology, Helicos aims to enhance the understanding of genetic information and improve applications in various fields of life sciences.
PhaseRx is a biopharmaceutical company based in Seattle that focuses on the development of innovative therapies for inherited enzyme deficiencies in the liver. Utilizing its proprietary Hybrid messenger RNA technology platform, PhaseRx aims to provide intracellular enzyme replacement therapy (i-ERT) by enabling the synthesis of essential enzymes directly within cells. The company is developing a portfolio of products targeting specific urea cycle disorders, including ornithine transcarbamylase deficiency, argininosuccinate lyase deficiency, and argininosuccinate synthetase deficiency. Founded by a team with expertise from the University of Washington and Fred Hutchinson Cancer Research Center, PhaseRx leverages novel polymer technology for the delivery of therapeutic agents, including siRNA and other macromolecular therapeutics.
OpGen, Inc. is a precision medicine company based in Gaithersburg, Maryland, that specializes in developing molecular diagnostics and informatics aimed at combating infectious diseases. The company offers a range of products, including the Acuitas AMR Gene Panel, which detects bacterial nucleic acids and antimicrobial resistance markers, and QuickFISH and PNA FISH tests for identifying pathogens in blood cultures. Additionally, OpGen provides the Acuitas Lighthouse informatics system, a cloud-based platform that integrates clinical lab results with patient data to help manage multidrug-resistant organisms in healthcare settings. Through its strategic collaboration with the New York State Department of Health, OpGen is working on solutions to detect and manage antimicrobial-resistant infections in healthcare facilities. The company's innovative technologies are utilized by academic, government, and research institutions globally for applications such as strain typing and genome assembly. OpGen aims to enhance patient care and outcomes while addressing the growing challenge of antibiotic resistance.
Pathwork Diagnostics, located in Redwood City, California, specializes in 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 identifying challenging tumors, including poorly differentiated, undifferentiated, and metastatic cancers. The test leverages genomic information from a tumor to enhance diagnostic precision and can be applied to various specimen types, including frozen tissue and formalin-fixed, paraffin-embedded samples. Through its advanced diagnostic solutions, Pathwork Diagnostics aims to improve clinical outcomes in cancer care.
Helicos BioSciences Corporation is a life sciences company that specializes in advanced genetic analysis technology. It has developed the True Single Molecule Sequencing (tSMS) technology, which allows for the rapid analysis of large amounts of genetic material by directly sequencing individual molecules of DNA and RNA. This innovative approach enables precise sequencing and offers significant advantages in research, drug discovery, and clinical diagnostics. The Helicos Genetic Analysis Platform includes the HeliScope Single Molecule Sequencer, which performs a series of biochemical reactions on single DNA or RNA molecules, and the HeliScope Analysis Engine, an image analysis computer system that processes the sequencing data. The platform also utilizes specialized reagents and disposable supplies essential for the sequencing process. Through its unique technology, Helicos aims to enhance the understanding of genetic information and improve applications in various fields of life sciences.