RA Capital

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Trace Neuroscience is a biopharmaceutical company dedicated to advancing genetic medicine for patients with neurodegenerative diseases. It specializes in developing targeted antisense oligonucleotide therapies, with its lead product aimed at restoring UNC13A protein function in individuals with Amyotrophic Lateral Sclerosis (ALS) caused by TDP-43 mislocalization. This therapy corrects UNC13A mRNA splicing, potentially improving muscle function and extending survival for these patients.

Gameto is a biotechnology company focused on advancing female reproductive health through innovative therapies. Recognizing the significant lack of treatment options in this underserved area, Gameto employs cutting-edge scientific techniques to develop solutions that enhance the quality of life for women. The company utilizes cellular engineering to create a platform that produces engineered ovarian and endometrial cell lines capable of mimicking the functions of natural cells, including hormone production and response. This platform underpins a portfolio of cellular therapeutics aimed at addressing various female reproductive diseases. Gameto's first initiative, Fertilo, targets improvements in in vitro fertilization (IVF) and egg freezing processes, striving to make them shorter, safer, and more effective. Additionally, the company is developing Deovo, an organoid model of the female reproductive system, and Ameno, a cell-based therapy designed to mitigate health issues related to primary ovarian insufficiency and menopause. Through these efforts, Gameto aims to transform the landscape of female reproductive health care.

Ajax Therapeutics is a New York City-based biotechnology company that applies computational chemistry and structure-based technologies to develop novel small molecules for treating hematologic malignancies.

Lexeo Therapeutics is a fully integrated biotechnology company focused on developing adeno-associated virus (AAV)-mediated gene therapies. Its lead programs target both rare and non-rare monogenic diseases, with a preclinical pipeline that spans monogenic, hereditary, and acquired conditions across populations with unmet medical need. The company aims to advance clinical programs toward commercialization while maintaining an ongoing research collaboration with Weill Cornell Medicine’s Department of Genetic Medicine to inform and accelerate its preclinical and clinical work. By leveraging AAV-based approaches and collaborations, Lexeo Therapeutics seeks to transform treatment options for patients with genetic and complex diseases.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

Avidity Biosciences is a biopharmaceutical company developing antibody oligonucleotide conjugates to enable tissue-selective delivery and precision RNA therapies for serious diseases. Its AOC platform combines monoclonal antibody tissue targeting with oligonucleotide therapies to access previously undruggable tissues and genetic drivers. The lead candidate, AOC 1001, targets myotonic dystrophy type 1, and its muscle programs address muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. The pipeline also includes immune and other cell-type programs, including Del-zota for DMD, Del-desiran for DM1, and Del-brax for FSHD. Avidity is based in La Jolla, California.

BioAge Labs develops medicines to treat aging and age-related diseases using its proprietary BioAge platform, which combines systems biology and AI. Its pipeline includes BGE-117 for multiple aging-related conditions, BGE-175 for immune aging and respiratory infections, and azelaprag for obesity in the elderly.

PepGen Ltd. is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the next generation of nucleic acid therapeutics, particularly in the treatment of severe neuromuscular and neurologic diseases. Incorporated in 2018, PepGen has developed its Enhanced Delivery Oligonucleotide (EDO) platform, which is designed to improve the uptake and activity of conjugated oligonucleotide therapeutics. The company's innovative EDO peptides enhance tissue penetration, cellular uptake, and nuclear delivery, demonstrating their ability to transport oligonucleotides effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. PepGen aims to unlock the clinical potential of these transformative therapeutics, with a particular emphasis on the effective delivery of antisense oligonucleotides, and is advancing its novel conjugate therapeutics toward clinical application.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Rampart Bioscience develops gene medicines to provide long-lasting treatments for a wide range of diseases. It employs proprietary development and delivery platforms to create optimized therapeutics in a revocable, non-viral format, advancing gene-based therapies with improved safety and controllability. The company integrates capabilities from gene delivery, protein sciences, and clinical translation to address genetically driven diseases and translate scientific advances into clinical options.

ExcepGen Ltd is a biotechnology company focused on developing full-length human proteins aimed at advancing drug discovery. Established in 2017 and based in London, United Kingdom, the company utilizes engineered human cell lines to produce these proteins. ExcepGen specializes in mRNA therapeutics, utilizing proprietary technology to adapt cellular responses by modulating both undesirable and beneficial pathways. This approach enhances the yield and functionality of therapeutic mRNA without relying on workarounds. By enabling laboratories to accelerate development times and improve high-throughput screening processes, ExcepGen contributes to more effective decision-making in drug pipeline development.

Taysha Gene Therapies develops adeno-associated virus-based gene therapies to treat monogenic diseases of the central nervous system. Its pipeline includes treatments for GM2 gangliosidosis, CLN1 disease, Rett syndrome, SLC6A1 haploinsufficiency disorder, and Surfeit locus 1 deficiency. The company collaborates with The University of Texas Southwestern Medical Center to develop and commercialize transformative gene therapy treatments.

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

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.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

NAPIGEN is a biotechnology company focused on advancing genome engineering across a range of organisms, including plants, microbes, and animals. The company's innovative technology specifically targets mitochondria and chloroplasts, which are organelles that contain their own DNA and play crucial roles in cellular energy production. By harnessing the potential of these organelles, NAPIGEN aims to develop solutions that enhance the growth and efficiency of various biological systems, ultimately contributing to a more convenient and sustainable society.

Wave Life Sciences is a clinical-stage biotechnology company focused on RNA medicines, designing, optimizing, and producing stereopure oligonucleotides through its PRISM platform. The company aims to address genetically defined diseases by reducing harmful protein expression or correcting dysfunctional proteins, employing modalities such as RNA editing, antisense silencing, and RNA interference. Its programs span neurological and neuromuscular conditions, reflecting an emphasis on central nervous system targets. Wave collaborates with Pfizer and Takeda on research, development, and commercialization of stereopure oligonucleotide therapeutics and antisense therapies. Founded in 2012 and based in Singapore, the company seeks to unlock the broader potential of RNA medicines to improve patient outcomes.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

Founded in 2018, Ansa Biotechnologies develops an innovative DNA synthesis technology based on enzymes. This novel approach aims to accelerate biological research and engineering by producing gene-length oligonucleotides more efficiently than traditional chemical methods.

Synthego is a biotechnology company specializing in genome engineering. It offers integrated hardware, software, bioinformatics, chemistries, and molecular biology platforms to advance basic research and therapeutic development. Its products include engineered cells, CRISPR kits, and bioinformatics tools for gene knockout, target validation, and disease modeling. Synthego serves scientists globally, with sales through distributors in multiple countries and online.

Metagenomi is a genetic medicines company headquartered in Emeryville, California. It leverages metagenomics and machine learning to discover novel genome editing systems from diverse organisms, then convert these natural systems into compact, highly efficient, and specific tools aimed at reducing immune responses. The company maintains a proprietary, metagenomics-derived genome editing toolbox that includes programmable nucleases, base editors, small RNA-mediated integration systems (RIGS), large template RIGS, and CRISPR-associated transposases (CASTs). Its development focus is in vivo gene editing with a wholly owned pipeline, while exploring technology out-licensing for ex vivo cell therapies to pursue curative therapeutics for genetic diseases.

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.

GentiBio, Inc. is a biotherapeutics company that specializes in developing engineered regulatory T cells, known as EngTregs, aimed at treating autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and headquartered in Boston, Massachusetts, with additional locations in Israel and Seattle, Washington, GentiBio employs a proprietary platform that combines autologous and allogeneic technologies. This platform enhances the ability to restore immune tolerance and addresses significant limitations found in existing regulatory T-cell therapeutics. By focusing on the underlying mechanisms of immune system disorders, GentiBio aims to provide innovative therapeutic solutions that tackle the root causes of diseases caused by dysregulated immune responses. The company is co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of advanced immunotherapy development.

PepGen Ltd. is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the next generation of nucleic acid therapeutics, particularly in the treatment of severe neuromuscular and neurologic diseases. Incorporated in 2018, PepGen has developed its Enhanced Delivery Oligonucleotide (EDO) platform, which is designed to improve the uptake and activity of conjugated oligonucleotide therapeutics. The company's innovative EDO peptides enhance tissue penetration, cellular uptake, and nuclear delivery, demonstrating their ability to transport oligonucleotides effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. PepGen aims to unlock the clinical potential of these transformative therapeutics, with a particular emphasis on the effective delivery of antisense oligonucleotides, and is advancing its novel conjugate therapeutics toward clinical application.

Element Biosciences develops innovative genetic analysis tools for research and diagnostic markets. Founded in 2017, the company specializes in a disruptive DNA sequencing technology that offers high-quality data, workflow flexibility, and affordable access to next-generation sequencing.

Larimar Therapeutics is a clinical-stage biotechnology company dedicated to developing treatments for complex rare diseases. Its lead compound, CTI-1601, is currently in Phase 1 clinical trials as a potential treatment for Friedreich's ataxia, a rare and progressive genetic disease.

Affinia Therapeutics develops adeno-associated virus (AAV) gene therapies for muscular and central nervous system diseases. The company uses an AAV vector design platform built on synthetic and systems biology, high-throughput screening, and tissue- and single-cell-resolution analyses to create vectors with improved properties. It leverages sequencing, artificial intelligence, and structural modeling to enable rational design of therapies. Headquartered in Waltham, Massachusetts, Affinia Therapeutics was founded in 2019 and targets diseases with significant unmet needs in muscle and CNS.

Forge Biologics, Inc. is a contract development and manufacturing organization (CDMO) specializing in gene therapies and genetic medicines. Founded in 2019 and based in Columbus, Ohio, the company focuses on developing and manufacturing therapeutics, particularly for viral vector gene therapies aimed at treating genetic diseases. Notably, it offers a novel approach combining adeno-associated virus (AAV) and umbilical cord transplant to address infantile Krabbe disease, a severe neurodegenerative disorder. Additionally, Forge Biologics collaborates with scientists, physicians, biotech and pharmaceutical companies, and patient groups to facilitate the progression of gene therapy programs from preclinical development through to clinical and commercial-scale manufacturing. The company is committed to enabling access to transformative medicines for patients suffering from rare genetic diseases.

Founded in 2018, Scribe Therapeutics specializes in engineering and developing in vivo therapies based on CRISPR technology to rewrite and repair genetic disorders. Its proprietary platform, X-Editing (XE), enables greater efficacy, specificity, and deliverability compared to existing CRISPR technologies.

Graphite Bio is a clinical-stage gene editing company focused on targeted DNA integration to treat serious diseases. Its platform aims to precisely insert, repair, or replace genetic sequences by integrating therapeutic cargoes at predefined genomic locations, enabling correction of mutations or replacement of disease-causing genes while preserving normal regulatory control. Based in South San Francisco, the company pursues high-efficiency precision gene editing to address underlying causes of disease.

Century Therapeutics is a biotechnology company developing allogeneic cell therapies derived from induced pluripotent stem cells to treat cancer and related diseases. Its platform enables engineering of iPSCs into NK and T cells with master cell banks of modified cells that can be expanded and differentiated into standardized, off-the-shelf therapies. The company combines CRISPR-mediated gene editing, proprietary chimeric antigen receptors, Allo-Evasion technology, and manufacturing capabilities to enable scalable production of homogeneous cell products for hematologic and solid tumors. While focused on oncology, its platform also targets autoimmune and inflammatory diseases with the goal of improving cell therapy performance and reducing host immune rejection.

DTx Pharma, LLC is a biotechnology company headquartered in San Diego, California, specializing in the development and commercialization of fatty acid-conjugated peptide therapeutics. Founded in 2017, the company focuses on delivering RNA-based medicines to treat patients with rare and chronic diseases, including retinitis pigmentosa, Duchenne muscular dystrophy, and various central nervous system disorders. DTx Pharma's innovative delivery technology addresses challenges faced by previous-generation platforms, such as poor pharmacokinetics and limited cellular uptake, thereby enhancing the efficiency of nucleic acid drug delivery across diverse tissues and cell types. The company aims to establish RNA therapeutics as a leading option for personalized treatment in multiple therapeutic areas.

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

Design Therapeutics, Inc. is a clinical-stage biotechnology company headquartered in Solana Beach, California, focused on developing innovative therapies for serious degenerative disorders caused by nucleotide repeat expansions. The company specializes in a novel class of small-molecule gene-targeted chimera therapeutics known as GeneTACTM molecules, which aim to modify disease progression by addressing the root causes of these conditions. Its lead program targets Friedreich’s ataxia, while additional efforts are directed toward conditions such as Fuchs endothelial corneal dystrophy and other degenerative diseases like Fragile X syndrome and myotonic dystrophy. Since its incorporation in 2017, Design Therapeutics has been dedicated to advancing its therapeutic candidates to potentially transform treatment options for patients suffering from these inherited disorders.

Verve Therapeutics is a biotechnology company developing genetic medicines for cardiovascular disease, aiming to transform treatment from chronic management to single-course gene editing therapies. It leverages human genetics analysis and gene-editing technology, with expertise in cardiovascular medicine, human genetics, gene editing, delivery technologies, drug development, and commercialization.

Locanabio is a biotechnology company developing RNA-targeted gene therapies for underserved diseases, with a focus on neuromuscular, neurodegenerative, and retinal conditions. It employs an RNA-targeting platform to modify disease-causing RNA and correct aberrant transcripts, offering therapies distinct from DNA-based approaches. The company identifies target RNA sequences within transcripts and programs therapies to advance candidates for rare genetic neuromuscular, neurodegenerative, and retinal diseases. Located in San Diego, Locanabio aims to translate RNA-directed interventions into treatments for patients with severe conditions.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

PepGen Ltd. is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the next generation of nucleic acid therapeutics, particularly in the treatment of severe neuromuscular and neurologic diseases. Incorporated in 2018, PepGen has developed its Enhanced Delivery Oligonucleotide (EDO) platform, which is designed to improve the uptake and activity of conjugated oligonucleotide therapeutics. The company's innovative EDO peptides enhance tissue penetration, cellular uptake, and nuclear delivery, demonstrating their ability to transport oligonucleotides effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. PepGen aims to unlock the clinical potential of these transformative therapeutics, with a particular emphasis on the effective delivery of antisense oligonucleotides, and is advancing its novel conjugate therapeutics toward clinical application.

Metagenomi is a genetic medicines company headquartered in Emeryville, California. It leverages metagenomics and machine learning to discover novel genome editing systems from diverse organisms, then convert these natural systems into compact, highly efficient, and specific tools aimed at reducing immune responses. The company maintains a proprietary, metagenomics-derived genome editing toolbox that includes programmable nucleases, base editors, small RNA-mediated integration systems (RIGS), large template RIGS, and CRISPR-associated transposases (CASTs). Its development focus is in vivo gene editing with a wholly owned pipeline, while exploring technology out-licensing for ex vivo cell therapies to pursue curative therapeutics for genetic diseases.

AavantiBio, Inc. is a biopharmaceutical company focused on developing gene transfer and gene editing therapies aimed at treating rare genetic diseases. Incorporated in 2019 and based in Gainesville, Florida, the company primarily concentrates on Friedreich’s Ataxia, a rare inherited disorder that leads to significant cardiac and central nervous system dysfunction. AavantiBio is committed to transforming the lives of patients suffering from this condition by advancing innovative therapies that address unmet medical needs in the field of genetic disorders. The company’s efforts are directed toward both treatment development and research, with the goal of providing effective solutions for patients facing the challenges of rare diseases.

Synthego is a biotechnology company specializing in genome engineering. It offers integrated hardware, software, bioinformatics, chemistries, and molecular biology platforms to advance basic research and therapeutic development. Its products include engineered cells, CRISPR kits, and bioinformatics tools for gene knockout, target validation, and disease modeling. Synthego serves scientists globally, with sales through distributors in multiple countries and online.

GentiBio, Inc. is a biotherapeutics company that specializes in developing engineered regulatory T cells, known as EngTregs, aimed at treating autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and headquartered in Boston, Massachusetts, with additional locations in Israel and Seattle, Washington, GentiBio employs a proprietary platform that combines autologous and allogeneic technologies. This platform enhances the ability to restore immune tolerance and addresses significant limitations found in existing regulatory T-cell therapeutics. By focusing on the underlying mechanisms of immune system disorders, GentiBio aims to provide innovative therapeutic solutions that tackle the root causes of diseases caused by dysregulated immune responses. The company is co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of advanced immunotherapy development.

Element Biosciences develops innovative genetic analysis tools for research and diagnostic markets. Founded in 2017, the company specializes in a disruptive DNA sequencing technology that offers high-quality data, workflow flexibility, and affordable access to next-generation sequencing.

Akouos is a biotechnology company focused on precision genetic medicine to restore and preserve hearing. Based in Boston, it develops gene therapies using a proprietary adeno-associated viral vector platform and delivery approaches to treat sensorineural hearing loss in genetically defined populations. The company's lead program, AK-OTOF, targets hearing loss caused by mutations in the OTOF gene. Akouos pursues research and development across gene-based interventions aimed at improving auditory function and long-term sensory outcomes. It has collaborations with academic and industry partners, including Massachusetts Eye and Ear and Lonza, to advance discovery and manufacturing capabilities. Founded in 2016, Akouos addresses unmet clinical needs in auditory health by seeking to restore physiologic hearing through targeted genetic therapies.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Inozyme Pharma, Inc. is a biopharmaceutical company focused on developing therapies for rare diseases related to abnormal mineralization affecting the vasculature, soft tissue, and skeletal systems. The company's lead product candidate, INZ-701, is a recombinant protein designed to treat rare genetic conditions caused by ENPP1 and ABCC6 deficiencies, as well as calciphylaxis. Inozyme's therapies aim to address the underlying causes of these debilitating diseases, providing potentially disease-modifying treatments for conditions such as Generalized Arterial Calcification of Infancy and Autosomal Recessive Hypophosphatemic Rickets Type 2. The company has a licensing agreement with Yale University for certain therapeutic applications and is headquartered in Boston, Massachusetts. Established in 2015, Inozyme is dedicated to improving the quality of life for patients affected by severe metabolic disorders associated with mineral imbalances.

Stoke Therapeutics is a biopharmaceutical company developing novel antisense oligonucleotide medicines. These target RNA splicing to increase gene expression, aiming to treat severe genetic diseases. Its lead product candidate, STK-001, focuses on Dravet syndrome, a progressive genetic epilepsy.

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

Akouos is a biotechnology company focused on precision genetic medicine to restore and preserve hearing. Based in Boston, it develops gene therapies using a proprietary adeno-associated viral vector platform and delivery approaches to treat sensorineural hearing loss in genetically defined populations. The company's lead program, AK-OTOF, targets hearing loss caused by mutations in the OTOF gene. Akouos pursues research and development across gene-based interventions aimed at improving auditory function and long-term sensory outcomes. It has collaborations with academic and industry partners, including Massachusetts Eye and Ear and Lonza, to advance discovery and manufacturing capabilities. Founded in 2016, Akouos addresses unmet clinical needs in auditory health by seeking to restore physiologic hearing through targeted genetic therapies.

Precision BioSciences, founded in 2006, is a biotechnology company headquartered in Durham, North Carolina. It specializes in genome editing, developing therapeutic products using its proprietary ARCUS technology. The company focuses on creating allogeneic CAR T immunotherapies to treat cancers like acute lymphoblastic leukemia and non-hodgkin lymphoma, as well as genetic disorders.

Dyne Therapeutics is a clinical-stage biotechnology company developing therapies for genetically driven neuromuscular diseases. It aims to deliver disease-modifying therapeutics to skeletal, cardiac, and smooth muscle, addressing conditions such as muscular dystrophies and other muscle disorders. The company uses the FORCE platform to create targeted therapeutics with payloads designed to address the disease gene, connected via a linker to an antigen-binding fragment, enabling delivery to muscle tissue and the central nervous system. Its pipeline includes DYNE-101, DYNE-251, DYNE-302, and DYNE-401. Headquartered in Waltham, Massachusetts.

Dicerna Pharmaceuticals is a biopharmaceutical company that specializes in the discovery and development of RNA interference (RNAi)-based therapeutics. The company focuses on innovative treatments for rare inherited liver diseases, viral infections, chronic liver diseases, and cardiometabolic conditions. Utilizing its proprietary GalXC RNAi technology platform, Dicerna is advancing several key product candidates, including nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B, and programs targeting genetic liver diseases and cardiovascular conditions. Founded in 2007 and headquartered in Lexington, Massachusetts, Dicerna has established strategic collaborations with major pharmaceutical firms to enhance its research and development efforts.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

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.

AveXis, operating as Novartis Gene Therapies, Inc., is a biotechnology company focused on developing and commercializing gene therapies for rare and life-threatening neurological genetic diseases. Acquired by Novartis in 2018, the company specializes in innovative treatments such as motor neuron cell-targeted gene replacement therapy aimed at conditions like spinal muscular atrophy, Rett syndrome, and amyotrophic lateral sclerosis. These therapies are designed to assist healthcare institutions in addressing diseases that cause progressive muscle weakness, paralysis, and ultimately, death. Through its advancements in gene therapy, AveXis is dedicated to improving the lives of patients and families impacted by these severe genetic disorders.

Wave Life Sciences is a clinical-stage biotechnology company focused on RNA medicines, designing, optimizing, and producing stereopure oligonucleotides through its PRISM platform. The company aims to address genetically defined diseases by reducing harmful protein expression or correcting dysfunctional proteins, employing modalities such as RNA editing, antisense silencing, and RNA interference. Its programs span neurological and neuromuscular conditions, reflecting an emphasis on central nervous system targets. Wave collaborates with Pfizer and Takeda on research, development, and commercialization of stereopure oligonucleotide therapeutics and antisense therapies. Founded in 2012 and based in Singapore, the company seeks to unlock the broader potential of RNA medicines to improve patient outcomes.

ARCA biopharma, Inc. is a Colorado-based biopharmaceutical company developing genetically-targeted therapies for cardiovascular diseases. ARCA’s principal focus is to develop personalized therapies for the treatment of cardiovascular disease through the use of genetics. The Company’s business focus combines expertise in cardiovascular pathophysiology, molecular genetics and clinical development.

Calimmune is a clinical-stage gene therapy company focused on HIV, developing cell-based therapies to protect individuals with HIV from progression to AIDS. Its lead candidate, Cal-1, is a gene-based therapy designed to modify immune cells to reduce HIV entry and replication by lowering CCR5 and preventing viral fusion, with activity demonstrated in preclinical studies and evaluated in Phase I/II trials. The company is based in Tucson, Arizona, and aims to address HIV at various stages of infection and treatment history by delivering durable cellular therapies that strengthen the immune system.

Precision BioSciences, founded in 2006, is a biotechnology company headquartered in Durham, North Carolina. It specializes in genome editing, developing therapeutic products using its proprietary ARCUS technology. The company focuses on creating allogeneic CAR T immunotherapies to treat cancers like acute lymphoblastic leukemia and non-hodgkin lymphoma, as well as genetic disorders.

Dimension Therapeutics, Inc. is a gene therapy company based in Cambridge, Massachusetts, specializing in the development of innovative treatments for rare and metabolic diseases associated with the liver. Founded in 2013, the company has a robust pipeline of gene therapy programs, including DTX101 and DTX201 for hemophilia B and A, respectively. Additionally, its portfolio encompasses DTX301 for ornithine transcarbamylase deficiency, DTX401 for glycogen storage disease type Ia, DTX501 for phenylketonuria, DTX701 for Wilson disease, and DTX601 for citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, gaining access to a significant portfolio of patents related to adeno-associated virus (AAV) therapeutics. This collaboration supports the company's aim to advance its gene therapy platform and enhance its product development capabilities in the treatment of rare diseases. Dimension Therapeutics operates as a subsidiary of Ultragenyx Pharmaceutical Inc. since November 2017.

Natera, Inc. is a diagnostic and research company specializing in genetic testing services, particularly in the areas of preconception and prenatal diagnostics. Founded in 2003 and headquartered in San Carlos, California, Natera offers a range of products including the Panorama non-invasive prenatal test, which screens for chromosomal abnormalities in fetuses, and Vistara, a test for single-gene mutations. Additionally, the company provides Horizon carrier screening to assess carrier status for various genetic diseases, and Spectrum, which analyzes genetic conditions during in vitro fertilization. Natera's Anora product analyzes fetal chromosomes to understand miscarriage causes, while its non-invasive paternity tests determine parentage through fetal DNA analysis. The company's Signatera technology focuses on circulating tumor DNA to monitor cancer recurrence. Natera distributes its products through a direct sales force and a network of approximately 100 laboratory and distribution partners, both domestically and internationally. The company collaborates with BGI Genomics to develop genetic testing assays and has partnered with Foundation Medicine for personalized cancer monitoring assays.

Wave Life Sciences is a clinical-stage biotechnology company focused on RNA medicines, designing, optimizing, and producing stereopure oligonucleotides through its PRISM platform. The company aims to address genetically defined diseases by reducing harmful protein expression or correcting dysfunctional proteins, employing modalities such as RNA editing, antisense silencing, and RNA interference. Its programs span neurological and neuromuscular conditions, reflecting an emphasis on central nervous system targets. Wave collaborates with Pfizer and Takeda on research, development, and commercialization of stereopure oligonucleotide therapeutics and antisense therapies. Founded in 2012 and based in Singapore, the company seeks to unlock the broader potential of RNA medicines to improve patient outcomes.

Larimar Therapeutics is a clinical-stage biotechnology company dedicated to developing treatments for complex rare diseases. Its lead compound, CTI-1601, is currently in Phase 1 clinical trials as a potential treatment for Friedreich's ataxia, a rare and progressive genetic disease.

Dicerna Pharmaceuticals is a biopharmaceutical company that specializes in the discovery and development of RNA interference (RNAi)-based therapeutics. The company focuses on innovative treatments for rare inherited liver diseases, viral infections, chronic liver diseases, and cardiometabolic conditions. Utilizing its proprietary GalXC RNAi technology platform, Dicerna is advancing several key product candidates, including nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B, and programs targeting genetic liver diseases and cardiovascular conditions. Founded in 2007 and headquartered in Lexington, Massachusetts, Dicerna has established strategic collaborations with major pharmaceutical firms to enhance its research and development efforts.

Bluebird Bio is a clinical-stage biotechnology company focused on developing gene therapies to treat severe genetic and rare diseases. Its proprietary lentiviral vector platform enables the creation of potentially curative treatments by genetically modifying patients' cells to address the root cause of these conditions.

CellScape Corporation (formerly Saryna Medical Corp) is a privately funded company headquartered in Newark, California, in the heart of the biotech corridor of the San Francisco Bay Area. The Company has developed whole fetal cell technology to transform prenatal genetic testing using a simple blood test. Their vision is to make comprehensive prenatal genetic testing available to every woman without requiring an invasive procedure that might put the fetus at risk. Their leadership team is accomplished in bringing innovative, cost-effective medical and molecular diagnostics to market.