MeiraGTx is a clinical-stage gene therapy company dedicated to developing and commercializing innovative gene therapy products aimed at transforming the lives of patients with acquired and inherited disorders. The company has a diverse pipeline that includes several ongoing clinical programs focused primarily on treating conditions related to the eye, salivary gland, and central nervous system. Key product candidates include AAV-CNGB3, AAV-CNGA3, and AAV-RPGR, among others. MeiraGTx operates in the United States, the United Kingdom, and the European Union, leveraging its expertise in viral vector design and gene therapy manufacturing. With a portfolio approach to technology development, the company plans to expand its focus to include additional gene therapy treatments for various serious diseases in the future.
Neuron23, Inc. develops and manufactures medicines for genetic disorders using advanced artificial intelligence technology and genetics. Founded in 2018 and headquartered in South San Francisco, California, with an additional office in Munich, Germany, the company focuses on creating therapeutics for neurological diseases, including Parkinson's and Alzheimer's, as well as immunological disorders. By leveraging recent advancements in human genetics alongside its AI-enabled drug discovery and biomarker platforms, Neuron23 aims to provide innovative treatments for patients suffering from these debilitating conditions, enhancing their quality of life through targeted and effective therapies.
DNAnexus, Inc. specializes in cloud-based genome informatics and data management tools tailored for enterprises and organizations involved in genomic medicine. Founded in 2009 and headquartered in Mountain View, California, the company provides a platform that facilitates the analysis of raw sequencing data, integrates genomic data with clinical information, and enhances collaboration among researchers. Key offerings include the MOSAIC Microbiome platform for microbiome research, DNAnexus Apollo for clinico-genomic data exploration, DNAnexus Titan for data analysis, and DNAnexus Portals, which create secure online workspaces for collaborative projects. Additionally, the Clinico-Genomic Data Solution aims to enhance disease-specific datasets through partnerships with healthcare providers, improving screening and treatment pathways for complex diseases. DNAnexus serves a diverse clientele, including biopharmaceutical companies, genome centers, and diagnostic test providers, and has established a strategic alliance with WuXi NextCODE Genomics. The company also maintains additional offices in San Francisco, London, and Prague.
Synthego is a genome engineering company that enables the acceleration of life science research and development in the pursuit of improved human health.
The company leverages machine learning, automation, and gene editing to build platforms for science at scale. With its foundations in engineering disciplines, the company’s full-stack platform vertically integrates proprietary hardware, software, bioinformatics, chemistries, and molecular biology to advance both basic research and therapeutic development programs.
By providing both commercial and academic researchers and therapeutic developers with unprecedented access to cutting-edge genome engineering, Synthego is at the forefront of innovation, accelerating the development of truly engineered biology.
The company was founded in 2012 and is based in Redwood City, California.
SilaGene, Inc. is a biotechnology company based in Hillsborough, New Jersey, founded in 2008. The company specializes in the development of U1 adaptors that hold therapeutic potential for various diseases, particularly in the treatment of different types of cancers. Its innovative technology serves as a gene silencing tool, which enhances the research of gene function in both cell cultures and in vivo settings. By addressing the limitations of existing gene silencing technologies, SilaGene aims to provide effective treatment options for patients.
A-Alpha Bio, Inc., founded in 2017 and based in Seattle, Washington, specializes in synthetic biology with a focus on drug development that targets multiple proteins. The company's flagship platform, AlphaSeq, offers innovative cell-based tools for quantitative and high-throughput measurement of protein interactions. This technology accelerates processes such as target discovery, library screening, and preclinical drug characterization, addressing significant bottlenecks in the industry. By providing biologics and molecular glue discovery and optimization services, A-Alpha Bio supports pharmaceutical companies in enhancing binding and specificity for various target proteins. The team comprises experts in synthetic biology, structural biology, and next-generation sequencing, all dedicated to advancing drug discovery methodologies.
Genome Medical, Inc. is a national telegenomics company that specializes in providing genetic consultation and medical consulting services. Incorporated in 2016 and based in South San Francisco, California, the company utilizes its Genome Care Delivery technology platform to deliver genetic solutions across various clinical areas, including cancer, cardiovascular health, reproductive health, pediatrics, and pharmacy. By leveraging a nationwide network of genetic specialists, Genome Medical offers expert virtual genetic care to individuals and families, enhancing their health and well-being. The company also assists healthcare providers and patients in navigating the expanding field of genetics, enabling them to interpret test results, assess disease risk, expedite diagnoses, make informed treatment choices, and reduce healthcare costs.
Juno Diagnostics, Inc. is a health technology company based in San Diego, California, focused on democratizing access to genetic information. Established in 2017, Juno specializes in cell-free DNA-based prenatal testing and is also engaged in the development of genetic testing tools for various applications, including epigenetics, inherited diseases, and cancer diagnostics. The company aims to create simple and cost-effective devices that facilitate point-of-need genetic testing, thereby enhancing accessibility to vital health information.
Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for various diseases, particularly those affecting muscle and the central nervous system (CNS). Founded in 2019 and based in Waltham, Massachusetts, the company specializes in creating rationally designed adeno-associated virus (AAV) vectors. By integrating synthetic and systems biology with high-throughput screening and advanced resolution techniques, Affinia Therapeutics aims to address significant unmet medical needs. The company’s innovative approach allows for the development of novel gene therapies with enhanced properties, targeting the treatment of devastating diseases and improving the lives of affected individuals.
Forge Biologics, Inc. is a contract development and manufacturing organization based in Columbus, Ohio, focused on developing and manufacturing gene therapies and genetic medicines. The company specializes in viral vector gene therapies aimed at treating genetic diseases, including a novel approach combining adeno-associated virus (AAV) with umbilical cord transplants for infantile Krabbe disease, a severe neurodegenerative condition. Founded in 2019, Forge Biologics partners with scientists, healthcare professionals, and biotech and pharmaceutical companies to facilitate the transition of gene therapy programs from preclinical development to clinical and commercial-scale production. By providing end-to-end capabilities, the company aims to accelerate the availability of transformative medicines for patients suffering from rare genetic disorders.
Scribe Therapeutics Inc. is a biotechnology company focused on developing innovative therapeutics using CRISPR technology to address genetic disorders. Founded in 2017 and based in Berkeley, California, Scribe specializes in engineering CRISPR enzymes known as X-Editing (XE) molecules, which enhance the efficacy, specificity, and deliverability of genome editing compared to existing methods. The company's proprietary platform enables the creation of custom-engineered enzymes and delivery systems, facilitating precision targeting within the genome. Scribe Therapeutics aims to establish CRISPR-based therapies as a standard in clinical care, expanding access to transformative treatments for a wide range of conditions, including immuno-oncology and degenerative disorders. Through its commitment to overcoming the limitations of current genome editing technologies, Scribe Therapeutics is poised to make substantial contributions to human therapeutics.
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.
Beam Therapeutics Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing precision genetic medicines using its innovative base editing technology. This technology allows for targeted alterations of single bases in the genome without creating double-stranded breaks in DNA, representing a potential advancement in genetic therapies. The company's pipeline includes programs aimed at treating serious diseases such as sickle cell disease, beta-thalassemia, pediatric T-cell acute lymphoblastic leukemia, and pediatric acute myeloid leukemia, as well as conditions like alpha-1 antitrypsin deficiency and certain central nervous system disorders. Beam Therapeutics is committed to providing life-long cures through its various approaches, which include gene correction, gene modification, gene activation, gene silencing, and multiplex editing. Founded in 2017, Beam continues to build collaborations to enhance its research and development efforts.
Neuron23, Inc. develops and manufactures medicines for genetic disorders using advanced artificial intelligence technology and genetics. Founded in 2018 and headquartered in South San Francisco, California, with an additional office in Munich, Germany, the company focuses on creating therapeutics for neurological diseases, including Parkinson's and Alzheimer's, as well as immunological disorders. By leveraging recent advancements in human genetics alongside its AI-enabled drug discovery and biomarker platforms, Neuron23 aims to provide innovative treatments for patients suffering from these debilitating conditions, enhancing their quality of life through targeted and effective therapies.
Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic disorder primarily affecting boys. The company's lead candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in muscle tissues. In addition to SGT-001, Solid Biosciences is advancing Anti-LTBP4, a monoclonal antibody designed to mitigate fibrosis and inflammation by targeting the LTBP4 protein. The company also works on developing biomarkers and wearable assistive devices under the Solid Suit program, which aims to provide therapeutic benefits to patients. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to further develop and commercialize gene therapies for DMD. The company’s initiatives address a critical need, as there are currently no effective cures or satisfactory treatments for this progressive and life-threatening condition.
AavantiBio, Inc. is dedicated to developing gene transfer therapy and gene editing technologies aimed at improving the lives of individuals afflicted by rare and fatal diseases. The company primarily focuses on Friedreich’s Ataxia, a rare inherited genetic disorder that leads to significant cardiac and central nervous system dysfunction. Founded in 2019 and based in Gainesville, Florida, AavantiBio is committed to advancing innovative gene therapies to address unmet medical needs in the treatment of rare genetic diseases and autoimmune conditions. Through its research and development efforts, the company seeks to transform patient care and enhance treatment options for those affected by these serious health challenges.
Taysha Gene Therapies is a company focused on developing adeno-associated virus (AAV) based gene therapies aimed at treating monogenic diseases of the central nervous system. Founded in 2019 and based in Dallas, Texas, the company is advancing several therapeutic candidates, including TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha Gene Therapies collaborates with The University of Texas Southwestern Medical Center to enhance the development and commercialization of its innovative treatments, with a mission to eradicate severe and life-threatening CNS diseases through curative gene therapies. The company's expertise in drug development and its partnership with a leading gene therapy program enable it to effectively progress its pipeline of potential therapies.
Forge Biologics, Inc. is a contract development and manufacturing organization based in Columbus, Ohio, focused on developing and manufacturing gene therapies and genetic medicines. The company specializes in viral vector gene therapies aimed at treating genetic diseases, including a novel approach combining adeno-associated virus (AAV) with umbilical cord transplants for infantile Krabbe disease, a severe neurodegenerative condition. Founded in 2019, Forge Biologics partners with scientists, healthcare professionals, and biotech and pharmaceutical companies to facilitate the transition of gene therapy programs from preclinical development to clinical and commercial-scale production. By providing end-to-end capabilities, the company aims to accelerate the availability of transformative medicines for patients suffering from rare genetic disorders.
Frequency Therapeutics is a clinical-stage biotechnology company based in Woburn, Massachusetts, dedicated to leveraging the body's natural biology to repair or reverse damage from various degenerative diseases. The company employs a unique Progenitor Cell Activation approach, utilizing combinations of small molecules to stimulate progenitor cells to regenerate functional tissue. Its lead product candidate, FX-322, is currently undergoing phase 2a clinical trials aimed at addressing the underlying causes of sensorineural hearing loss. In addition to hearing loss, Frequency Therapeutics is developing therapies for a variety of degenerative conditions, including multiple sclerosis and ailments affecting muscle, skin, gastrointestinal tract, and bone health. The company has also established a collaboration agreement with Astellas Pharma for the development and commercialization of FX-322. Founded in 2014, Frequency Therapeutics is focused on disease modification through innovative therapeutic strategies.
Genome Medical, Inc. is a national telegenomics company that specializes in providing genetic consultation and medical consulting services. Incorporated in 2016 and based in South San Francisco, California, the company utilizes its Genome Care Delivery technology platform to deliver genetic solutions across various clinical areas, including cancer, cardiovascular health, reproductive health, pediatrics, and pharmacy. By leveraging a nationwide network of genetic specialists, Genome Medical offers expert virtual genetic care to individuals and families, enhancing their health and well-being. The company also assists healthcare providers and patients in navigating the expanding field of genetics, enabling them to interpret test results, assess disease risk, expedite diagnoses, make informed treatment choices, and reduce healthcare costs.
4D Molecular Therapeutics is a clinical-stage gene therapy company based in Emeryville, California, that specializes in developing targeted gene therapy products using evolved adeno-associated virus vectors. The company has a diverse portfolio focusing on three main therapeutic areas: ophthalmology, cardiology, and pulmonology. Key product candidates include 4D-125, currently in a Phase I/II clinical trial for treating X-linked retinitis pigmentosa; 4D-110, in a Phase I trial for choroideremia; and 4D-310, also in a Phase I/II trial for Fabry disease. Additionally, 4D Molecular Therapeutics is developing investigational new drug candidates such as 4D-150 for wet age-related macular degeneration and 4D-710 for cystic fibrosis lung disease. The company aims to address unmet medical needs through its innovative gene therapies.
DNAnexus, Inc. specializes in cloud-based genome informatics and data management tools tailored for enterprises and organizations involved in genomic medicine. Founded in 2009 and headquartered in Mountain View, California, the company provides a platform that facilitates the analysis of raw sequencing data, integrates genomic data with clinical information, and enhances collaboration among researchers. Key offerings include the MOSAIC Microbiome platform for microbiome research, DNAnexus Apollo for clinico-genomic data exploration, DNAnexus Titan for data analysis, and DNAnexus Portals, which create secure online workspaces for collaborative projects. Additionally, the Clinico-Genomic Data Solution aims to enhance disease-specific datasets through partnerships with healthcare providers, improving screening and treatment pathways for complex diseases. DNAnexus serves a diverse clientele, including biopharmaceutical companies, genome centers, and diagnostic test providers, and has established a strategic alliance with WuXi NextCODE Genomics. The company also maintains additional offices in San Francisco, London, and Prague.
ArcherDX, Inc. is a genomics company based in Boulder, Colorado, focused on advancing molecular pathology through next-generation sequencing technology. Established in 2013, the company develops and commercializes a range of research products, including DNA-based VariantPlex, RNA-based FusionPlex, ctDNA-based LiquidPlex, and RNA-based Immunoverse, aimed at optimizing therapy and monitoring cancer. ArcherDX also creates in-vitro diagnostic products and offers customizable assay design services to clinical and biopharmaceutical clients, enabling the development of new applications tailored to specific biomarker targets. The Archer platform utilizes proprietary Anchored Multiplexed PCR (AMP™) chemistry and sophisticated bioinformatics to enhance genetic mutation detection significantly. The company's products and services are utilized by academic and reference laboratories, biopharmaceutical firms, and contract research organizations, with ongoing efforts to gain regulatory approval for various companion diagnostic assays. As of October 2020, ArcherDX operates as a subsidiary of Invitae Corporation.
Avidity Biosciences is a biopharmaceutical company specializing in the development of oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. Avidity's lead product candidate, AOC 1001, targets myotonic dystrophy type 1, a rare genetic muscle disorder. The company's development pipeline also includes programs focused on treating various muscle diseases, such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Additionally, Avidity is exploring therapeutic applications for immune and other cell types, leveraging its proprietary AOC platform to reach previously undruggable tissues and effectively target the genetic underpinnings of these conditions. Founded in 2012, Avidity Biosciences is headquartered in La Jolla, California.
Frequency Therapeutics is a clinical-stage biotechnology company based in Woburn, Massachusetts, dedicated to leveraging the body's natural biology to repair or reverse damage from various degenerative diseases. The company employs a unique Progenitor Cell Activation approach, utilizing combinations of small molecules to stimulate progenitor cells to regenerate functional tissue. Its lead product candidate, FX-322, is currently undergoing phase 2a clinical trials aimed at addressing the underlying causes of sensorineural hearing loss. In addition to hearing loss, Frequency Therapeutics is developing therapies for a variety of degenerative conditions, including multiple sclerosis and ailments affecting muscle, skin, gastrointestinal tract, and bone health. The company has also established a collaboration agreement with Astellas Pharma for the development and commercialization of FX-322. Founded in 2014, Frequency Therapeutics is focused on disease modification through innovative therapeutic strategies.
Genome Medical, Inc. is a national telegenomics company that specializes in providing genetic consultation and medical consulting services. Incorporated in 2016 and based in South San Francisco, California, the company utilizes its Genome Care Delivery technology platform to deliver genetic solutions across various clinical areas, including cancer, cardiovascular health, reproductive health, pediatrics, and pharmacy. By leveraging a nationwide network of genetic specialists, Genome Medical offers expert virtual genetic care to individuals and families, enhancing their health and well-being. The company also assists healthcare providers and patients in navigating the expanding field of genetics, enabling them to interpret test results, assess disease risk, expedite diagnoses, make informed treatment choices, and reduce healthcare costs.
ArcherDX, Inc. is a genomics company based in Boulder, Colorado, focused on advancing molecular pathology through next-generation sequencing technology. Established in 2013, the company develops and commercializes a range of research products, including DNA-based VariantPlex, RNA-based FusionPlex, ctDNA-based LiquidPlex, and RNA-based Immunoverse, aimed at optimizing therapy and monitoring cancer. ArcherDX also creates in-vitro diagnostic products and offers customizable assay design services to clinical and biopharmaceutical clients, enabling the development of new applications tailored to specific biomarker targets. The Archer platform utilizes proprietary Anchored Multiplexed PCR (AMP™) chemistry and sophisticated bioinformatics to enhance genetic mutation detection significantly. The company's products and services are utilized by academic and reference laboratories, biopharmaceutical firms, and contract research organizations, with ongoing efforts to gain regulatory approval for various companion diagnostic assays. As of October 2020, ArcherDX operates as a subsidiary of Invitae Corporation.
BridgeBio is a biotechnology company dedicated to the discovery, development, and delivery of medicines for genetic diseases. The company has a diverse pipeline of 20 development programs, which range from early discovery to late-stage clinical trials. Among its notable candidates are BBP-265, an oral small molecule targeting transthyretin amyloidosis currently in Phase 3 trials, and infigratinib, a selective tyrosine kinase inhibitor aimed at treating FGFR-driven cancers and achondroplasia. Additionally, BridgeBio is developing BBP-631, a preclinical gene therapy for congenital adrenal hyperplasia, and BBP-454, a program focused on small molecule inhibitors for KRAS-driven cancers. The company collaborates with several prestigious institutions, including Stanford University and Johns Hopkins University, to enhance its research and development efforts. Founded in 2015 and headquartered in Palo Alto, California, BridgeBio aims to accelerate the development of therapies for Mendelian diseases, genetic dermatology, and oncology.
Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops novel antisense oligonucleotide medicines to treat the underlying causes of severe genetic diseases. Its lead product candidate, STK-001 used to treat Dravet syndrome, a severe and progressive genetic epilepsy. Stoke Therapeutics, Inc. has a partnership with Invitae Corporation to offer genetic testing. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was founded in 2014 and is headquartered in Bedford, Massachusetts.
Allogene Therapeutics is a clinical-stage biotechnology company focused on immuno-oncology, particularly the development of genetically engineered allogeneic T cell therapies for cancer treatment. The company is advancing a pipeline of allogeneic T cell product candidates that leverage validated gene editing and proprietary cell manufacturing technologies. Key offerings include UCART19, which targets relapsed or refractory acute lymphoblastic leukemia, alongside several preclinical allogeneic CAR T therapies aimed at various malignancies and autoimmune diseases. By using T cells derived from healthy donors, Allogene Therapeutics aims to provide off-the-shelf treatment options that enhance patient eligibility and scalability. The company seeks to redefine CAR T therapy through its innovative approaches, addressing conditions like lymphoma, leukemia, and solid tumors.
Allogene Therapeutics is a clinical-stage biotechnology company focused on immuno-oncology, particularly the development of genetically engineered allogeneic T cell therapies for cancer treatment. The company is advancing a pipeline of allogeneic T cell product candidates that leverage validated gene editing and proprietary cell manufacturing technologies. Key offerings include UCART19, which targets relapsed or refractory acute lymphoblastic leukemia, alongside several preclinical allogeneic CAR T therapies aimed at various malignancies and autoimmune diseases. By using T cells derived from healthy donors, Allogene Therapeutics aims to provide off-the-shelf treatment options that enhance patient eligibility and scalability. The company seeks to redefine CAR T therapy through its innovative approaches, addressing conditions like lymphoma, leukemia, and solid tumors.
4D Molecular Therapeutics is a clinical-stage gene therapy company based in Emeryville, California, that specializes in developing targeted gene therapy products using evolved adeno-associated virus vectors. The company has a diverse portfolio focusing on three main therapeutic areas: ophthalmology, cardiology, and pulmonology. Key product candidates include 4D-125, currently in a Phase I/II clinical trial for treating X-linked retinitis pigmentosa; 4D-110, in a Phase I trial for choroideremia; and 4D-310, also in a Phase I/II trial for Fabry disease. Additionally, 4D Molecular Therapeutics is developing investigational new drug candidates such as 4D-150 for wet age-related macular degeneration and 4D-710 for cystic fibrosis lung disease. The company aims to address unmet medical needs through its innovative gene therapies.
Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. Operating in the United Kingdom, European Union, and the United States, Orchard employs an autologous ex vivo gene therapy approach to transform patients’ hematopoietic stem cells into gene-modified products for treatment through a single administration. Its portfolio includes Strimvelis, the first approved gene therapy for adenosine deaminase-severe combined immunodeficiency (ADA-SCID), along with several products in clinical development targeting metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has a robust preclinical pipeline addressing various mucopolysaccharidosis types. Orchard collaborates with leading institutions in gene therapy, enhancing its research capabilities and clinical programs. Founded in 2015, Orchard Therapeutics continues to demonstrate a commitment to transforming the lives of patients through its advanced therapies.
IDEAYA Biosciences is an oncology-focused biotechnology company based in South San Francisco, California, dedicated to the discovery and development of targeted therapeutics for genetically defined patient populations. The company specializes in precision medicine using molecular diagnostics and synthetic lethality approaches to address cancer treatment. Its lead product candidate, IDE196, is a protein kinase C inhibitor currently undergoing Phase 1/2 clinical trials for cancers associated with GNAQ or GNA11 gene mutations. IDEAYA's preclinical pipeline includes programs targeting various mechanisms related to DNA damage and repair, such as MAT2A for tumors with MTAP deletions, Pol-theta for homologous recombination deficiency, and PARG for BRCA2 mutation context. The company collaborates with Cancer Research UK and the University of Manchester to develop small molecule inhibitors of Poly (ADP-ribose) glycohydrolase, while also engaging in partnerships for clinical trials with Pfizer and GlaxoSmithKline. Founded in 2015, IDEAYA aims to advance innovative therapies for enhancing immunotherapy responses and improving patient outcomes in oncology.
Counsyl is a health technology company specializing in DNA screening for individuals and families. Its services aim to provide critical health information that can influence important decisions regarding behavior, prevention, and preparedness for future health challenges. By leveraging advanced technology, including custom-built robotics and a team of scientists and engineers, Counsyl processes blood and saliva samples with high precision and efficiency. The company has screened over 500,000 patients and collaborated with more than 6,000 healthcare professionals, striving to make DNA screening accessible to a broad audience. Counsyl’s innovative approach has earned recognition, including the Wall Street Journal’s Innovation Award for Medicine, and its Family Prep Screen has significantly aided individuals in understanding crucial genetic information prior to childbearing. The platform has expanded its offerings to include informed pregnancy and cancer screening, emphasizing the integration of sophisticated technology in real-time communication of medical results between patients and healthcare providers.
BridgeBio is a biotechnology company dedicated to the discovery, development, and delivery of medicines for genetic diseases. The company has a diverse pipeline of 20 development programs, which range from early discovery to late-stage clinical trials. Among its notable candidates are BBP-265, an oral small molecule targeting transthyretin amyloidosis currently in Phase 3 trials, and infigratinib, a selective tyrosine kinase inhibitor aimed at treating FGFR-driven cancers and achondroplasia. Additionally, BridgeBio is developing BBP-631, a preclinical gene therapy for congenital adrenal hyperplasia, and BBP-454, a program focused on small molecule inhibitors for KRAS-driven cancers. The company collaborates with several prestigious institutions, including Stanford University and Johns Hopkins University, to enhance its research and development efforts. Founded in 2015 and headquartered in Palo Alto, California, BridgeBio aims to accelerate the development of therapies for Mendelian diseases, genetic dermatology, and oncology.
Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic disorder primarily affecting boys. The company's lead candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in muscle tissues. In addition to SGT-001, Solid Biosciences is advancing Anti-LTBP4, a monoclonal antibody designed to mitigate fibrosis and inflammation by targeting the LTBP4 protein. The company also works on developing biomarkers and wearable assistive devices under the Solid Suit program, which aims to provide therapeutic benefits to patients. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to further develop and commercialize gene therapies for DMD. The company’s initiatives address a critical need, as there are currently no effective cures or satisfactory treatments for this progressive and life-threatening condition.
Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic disorder primarily affecting boys. The company's lead candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in muscle tissues. In addition to SGT-001, Solid Biosciences is advancing Anti-LTBP4, a monoclonal antibody designed to mitigate fibrosis and inflammation by targeting the LTBP4 protein. The company also works on developing biomarkers and wearable assistive devices under the Solid Suit program, which aims to provide therapeutic benefits to patients. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to further develop and commercialize gene therapies for DMD. The company’s initiatives address a critical need, as there are currently no effective cures or satisfactory treatments for this progressive and life-threatening condition.
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.
REGENXBIO Inc. is a clinical-stage biotechnology company focused on developing gene therapy product candidates that utilize its proprietary NAV Technology Platform, which is based on adeno-associated virus (AAV) gene delivery. This platform aims to address genetic defects and enable cells to produce therapeutic proteins or antibodies to combat various diseases. The company's lead candidate, RGX-314, is currently undergoing Phase I/IIa clinical trials for wet age-related macular degeneration. Other notable product candidates include RGX-121 for mucopolysaccharidosis type II, RGX-111 for mucopolysaccharidosis type I, RGX-181 for late infantile neuronal ceroid lipofuscinosis type II, and RGX-501 for homozygous familial hypercholesterolemia, all in various stages of clinical trials. Additionally, REGENXBIO licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies and has partnered with Neurimmune AG to develop vectorized antibodies for neurodegenerative diseases. Founded in 2008 and headquartered in Rockville, Maryland, the company was previously known as ReGenX Biosciences, LLC before rebranding in 2014.
Invitae is a genetic information company focused on integrating genetic testing into routine medical practice to enhance healthcare quality worldwide. It specializes in genetic diagnostics for hereditary disorders, offering a comprehensive service that consolidates a wide array of genetic tests into one platform, ensuring faster turnaround times and lower costs compared to traditional single-gene tests. Invitae's test offerings cover various hereditary conditions, including cancers, neurological disorders, cardiovascular diseases, pediatric and metabolic disorders. The company aims to make genetic testing more affordable and accessible, develop a secure infrastructure for genome management, and foster a global community for sharing genetic information to advance scientific research and medical practices. By lowering the barriers to obtaining diagnostic genetic information, Invitae seeks to improve healthcare outcomes for millions of individuals.