RA Capital Management

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 biotechnology company based in New York City that focuses on developing innovative small molecule therapies for hematologic malignancies. The company utilizes computational chemistry and structure-based technologies to design drugs that target critical cytokine signaling pathways involved in these cancers. By integrating insights from disease biology, genetics, and structural biology with an advanced computational drug discovery platform, Ajax Therapeutics aims to create precisely designed therapeutics that address significant unmet medical needs for patients suffering from hematologic malignancies.

Lexeo Therapeutics is a biotechnology company specializing in adeno-associated virus (AAV)-mediated therapies for both rare and common diseases caused by single gene mutations. Headquartered in New York, the company maintains an integrated pipeline developed in collaboration with Weill Cornell Medicine's Department of Genetic Medicine. Lexeo focuses on advancing its clinical programs towards commercialization while continuing to research new therapies for various patient populations and unmet medical needs.

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, Inc. is a biopharmaceutical company based in La Jolla, California, specializing in the development of oligonucleotide-based therapies, particularly through its innovative Antibody Oligonucleotide Conjugates (AOC) platform. This technology aims to address the limitations of traditional oligonucleotide therapies by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide treatments. Avidity's lead product candidate, AOC 1001, targets myotonic dystrophy type 1, a rare genetic muscle disease. In addition to this, the company is advancing multiple programs focused on muscle-related conditions, including muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Furthermore, Avidity is expanding its research to include immune and other cell types, positioning itself at the forefront of biopharmaceutical innovation for serious diseases.

BioAge Labs, Inc. is a biotechnology company focused on developing therapies to treat aging and age-related diseases. The company utilizes a proprietary systems biology and artificial intelligence platform to identify significant drug targets that influence aging, leveraging extensive datasets to uncover the molecular drivers of age-related pathologies. Among its key offerings is BGE-117, a hypoxia-inducible factor prolyl hydroxylase inhibitor that activates HIF-1 target genes involved in various biological processes, including tissue regeneration and vascular remodeling. Another notable product is BGE-175, an orally administered inhibitor targeting the prostaglandin D2 DP1 signaling pathway, which is linked to an increased risk of mortality and infections, including those associated with immune aging. Founded in 2015 and headquartered in Richmond, California, BioAge Labs is dedicated to harnessing the biology of human aging to create innovative therapies for metabolic and age-related diseases.

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 is a biotechnology company focused on developing gene medicines that aim to deliver long-lasting treatments for various diseases. The company has created proprietary development and delivery platforms that generate optimized therapeutics in a revocable, non-viral format. By integrating expertise from multiple scientific disciplines, including gene delivery, protein sciences, and clinical translation, Rampart Bioscience works to assist patients with genetically driven diseases. Their innovative approach seeks to enhance the effectiveness and safety of gene therapies, ultimately improving patient outcomes.

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, Inc. is a gene therapy company based in Dallas, Texas, focused on developing adeno-associated virus (AAV)-based therapies for monogenic diseases of the central nervous system (CNS). Founded in 2019, the company aims to create curative treatments for severe and life-threatening conditions. Its pipeline includes several product candidates, such as 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 collaborates with The University of Texas Southwestern Medical Center to enhance its development and commercialization efforts, leveraging expertise in gene therapy to advance its mission of eradicating monogenic CNS diseases.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

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 Ltd. is a clinical-stage genetic medicine company based in Singapore, focused on developing innovative treatments for genetically defined diseases using its proprietary PRISM platform. This platform enables the precise design, optimization, and production of stereopure oligonucleotides, which are designed to target genetic defects by either reducing harmful protein expression or converting dysfunctional proteins into functional ones. The company's primary focus is on neurology, particularly within the central and neuromuscular systems. WaVe Life Sciences collaborates with major pharmaceutical companies, including Pfizer and Takeda, to advance the research and development of its oligonucleotide therapeutics. Founded in 2012, the company aims to deliver transformative therapies to improve patient outcomes in both rare and prevalent disorders.

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.

Ansa Biotechnologies, Inc. is a biotechnology company based in Berkeley, California, focused on developing innovative DNA synthesis technology utilizing enzymes. Founded in 2018, the company aims to transform the traditional methods of DNA production, which have remained largely unchanged for decades. Ansa's enzymatic approach promises to be faster, more accurate, and environmentally friendly compared to conventional chemical methods. This advanced technology facilitates the synthesis of gene-length oligonucleotides, enabling medical researchers to create synthetic genes efficiently. Ansa Biotechnologies' offerings are designed to support a wide range of applications in biological research, diagnostics, therapeutics, and biomanufacturing, ultimately accelerating innovation in the field of biotechnology.

Synthego Corporation is a genome engineering company that specializes in accelerating life science research and development to enhance human health. Established in 2012 and headquartered in Redwood City, California, Synthego utilizes an innovative platform that integrates hardware, software, bioinformatics, chemistries, and molecular biology. This full-stack approach provides researchers with tools for gene editing, including engineered cells, CRISPR kits, and bioinformatics for guide RNA optimization and analysis. The products support various applications in basic research, therapeutic development, disease modeling, and diagnostics. Synthego serves a diverse range of customers, including biotechnology firms, universities, and therapeutic developers worldwide, distributing its offerings online and through partners in multiple countries. By leveraging automation and machine learning, Synthego enables genetic engineers and medical professionals to conduct research that is both rapid and cost-effective.

Metagenomi, Inc. is a genetic medicines company focused on developing innovative gene editing systems to address genetic diseases. Incorporated in 2016 and headquartered in Emeryville, California, Metagenomi utilizes metagenomics and machine learning to discover novel genome editing technologies. By sourcing cellular machinery from previously uncharacterized organisms, the company creates a proprietary toolbox that includes programmable nucleases, base editors, and advanced integration systems like prime editing and CRISPR-associated transposases. These systems are designed to be ultra-small, efficient, and highly specific, minimizing the risk of immune responses. Metagenomi aims to revolutionize gene editing techniques and deliver curative therapeutics to patients worldwide.

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, Inc., established in 2017 and headquartered in San Diego, California, specializes in developing innovative genetic analysis tools for research and diagnostic markets. The company's core business revolves around its disruptive DNA sequencing technology, which encompasses surface chemistry, sequencing chemistry, detection methods, and data analysis. Element Biosciences aims to enhance accessibility to next-generation sequencing by offering a modular, high-performing platform that delivers high-quality data and workflow flexibility, thereby reducing run and capital costs.

Larimar Therapeutics, Inc. is a clinical-stage biotechnology company based in Bala Cynwyd, Pennsylvania, dedicated to developing treatments for complex rare diseases. Its primary focus is on Friedreich's ataxia, a rare and progressive genetic disorder, for which it is advancing its lead compound, CTI-1601, through a Phase 1 clinical program. CTI-1601 is designed as a recombinant fusion protein that delivers human frataxin, an essential protein, directly to the mitochondria using a novel cell-penetrating peptide technology. In addition to Friedreich's ataxia, Larimar aims to leverage its intracellular delivery platform to create additional fusion proteins targeting other rare diseases associated with deficiencies in bioactive compounds.

Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for the treatment of severe diseases affecting the muscle and central nervous system (CNS). Established in 2019 and based in Waltham, Massachusetts, the company specializes in creating adeno-associated virus (AAV) vectors and utilizes a platform that integrates synthetic and systems biology with high-throughput screening techniques. This innovative approach allows Affinia Therapeutics to design novel gene therapies with enhanced properties tailored to address significant unmet medical needs. The company aims to provide healthcare professionals with advanced therapeutic options for patients suffering from debilitating conditions.

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.

Scribe Therapeutics Inc. is a biotechnology company focused on developing innovative therapies utilizing engineered CRISPR technologies to address genetic disorders. Founded in 2018 and based in Berkeley, California, Scribe Therapeutics is known for its proprietary X-Editing (XE) molecules, which are advanced CRISPR enzymes designed to enhance efficacy, specificity, and deliverability compared to existing genome editing methods. The company's platform aims to overcome the limitations of current technologies by creating custom-engineered enzymes and delivery systems that facilitate precise genetic modifications. Scribe Therapeutics is committed to establishing CRISPR-based therapies as a new standard of clinical care, with the potential to significantly impact the treatment landscape for a variety of diseases, including those related to immuno-oncology, degenerative disorders, and enzyme replacement therapy.

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

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

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 biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

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 genetic medicines company based in Cambridge, Massachusetts, focused on innovative therapies for cardiovascular disease. Founded in 2018, the company aims to transform the management of cardiovascular conditions from chronic treatments to single-course gene editing medicines. Verve Therapeutics employs advanced gene-editing technology and human genetic analysis to develop targeted therapies that reduce the risk of coronary artery diseases. Its initial programs focus on the PCSK9 and ANGPTL3 genes, which are crucial for lowering harmful blood lipids like low-density lipoprotein cholesterol. The company collaborates with strategic partners, including Beam Therapeutics for delivery technologies and Verily for gene editing delivery vehicles, enhancing its capabilities in the biotechnology and healthcare sectors.

Locanabio, Inc., based in San Diego, California, is a biotechnology company specializing in RNA-targeted gene therapies. Founded in 2016, the company focuses on developing treatments for severe neurodegenerative, neuromuscular, and retinal diseases. Locanabio's platform is designed to modify disease-causing RNA, offering therapeutic candidates distinct from traditional DNA-targeted approaches. The company aims to address a spectrum of underserved diseases, including those affecting the nervous system, muscles, and eyes.

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, Inc. is a genetic medicines company focused on developing innovative gene editing systems to address genetic diseases. Incorporated in 2016 and headquartered in Emeryville, California, Metagenomi utilizes metagenomics and machine learning to discover novel genome editing technologies. By sourcing cellular machinery from previously uncharacterized organisms, the company creates a proprietary toolbox that includes programmable nucleases, base editors, and advanced integration systems like prime editing and CRISPR-associated transposases. These systems are designed to be ultra-small, efficient, and highly specific, minimizing the risk of immune responses. Metagenomi aims to revolutionize gene editing techniques and deliver curative therapeutics to patients worldwide.

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 Corporation is a genome engineering company that specializes in accelerating life science research and development to enhance human health. Established in 2012 and headquartered in Redwood City, California, Synthego utilizes an innovative platform that integrates hardware, software, bioinformatics, chemistries, and molecular biology. This full-stack approach provides researchers with tools for gene editing, including engineered cells, CRISPR kits, and bioinformatics for guide RNA optimization and analysis. The products support various applications in basic research, therapeutic development, disease modeling, and diagnostics. Synthego serves a diverse range of customers, including biotechnology firms, universities, and therapeutic developers worldwide, distributing its offerings online and through partners in multiple countries. By leveraging automation and machine learning, Synthego enables genetic engineers and medical professionals to conduct research that is both rapid and cost-effective.

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, Inc., established in 2017 and headquartered in San Diego, California, specializes in developing innovative genetic analysis tools for research and diagnostic markets. The company's core business revolves around its disruptive DNA sequencing technology, which encompasses surface chemistry, sequencing chemistry, detection methods, and data analysis. Element Biosciences aims to enhance accessibility to next-generation sequencing by offering a modular, high-performing platform that delivers high-quality data and workflow flexibility, thereby reducing run and capital costs.

Akouos, Inc. is a biotechnology company dedicated to developing precision genetic medicine aimed at restoring, improving, and preserving hearing for individuals with various forms of hearing loss. Utilizing a proprietary adeno-associated viral vector library and an innovative delivery approach, Akouos focuses on gene therapies tailored to genetically-defined patient populations, including those affected by specific genetic mutations, ototoxic drug exposure, and age-related hearing loss. The company's lead product candidate, AK-OTOF, targets hearing loss associated with mutations in the OTOF gene. Founded in 2016 and based in Boston, Massachusetts, Akouos collaborates with strategic partners, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing effective treatments for sensorineural hearing loss.

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, Inc. is an early-stage biopharmaceutical company focused on developing novel antisense oligonucleotide medicines aimed at addressing the underlying causes of severe genetic diseases. Founded in 2014 and headquartered in Bedford, Massachusetts, the company utilizes its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach to enhance gene expression through RNA splicing. Its lead product candidate, STK-001, is currently in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. Stoke Therapeutics has also partnered with Invitae Corporation to offer complementary genetic testing services.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

Akouos, Inc. is a biotechnology company dedicated to developing precision genetic medicine aimed at restoring, improving, and preserving hearing for individuals with various forms of hearing loss. Utilizing a proprietary adeno-associated viral vector library and an innovative delivery approach, Akouos focuses on gene therapies tailored to genetically-defined patient populations, including those affected by specific genetic mutations, ototoxic drug exposure, and age-related hearing loss. The company's lead product candidate, AK-OTOF, targets hearing loss associated with mutations in the OTOF gene. Founded in 2016 and based in Boston, Massachusetts, Akouos collaborates with strategic partners, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing effective treatments for sensorineural hearing loss.

Precision BioSciences, Inc. is a biotechnology company focused on genome editing, headquartered in Durham, North Carolina. The company utilizes its proprietary ARCUS technology to develop therapeutic products aimed at treating human diseases and improving food and agricultural solutions. Precision BioSciences operates through two segments: Therapeutic and Food. The Therapeutic segment is particularly dedicated to advancing allogeneic CAR T immunotherapies for cancer treatment, including candidates like PBCAR0191 and PBCAR20A, which target specific tumor antigens. The company is also involved in in vivo gene correction and is exploring treatments for conditions such as acute lymphoblastic leukemia and chronic lymphocytic leukemia. Additionally, the Food segment develops innovative nutrition products. Precision BioSciences has formed strategic collaborations, including agreements with Shire Plc and Gilead Sciences, to enhance its research and development capabilities. Founded in 2006, the company aims to harness its next-generation gene editing technology to address pressing health challenges.

Dyne Therapeutics, Inc. is a biotechnology company based in Waltham, Massachusetts, dedicated to developing targeted therapies for genetically driven muscle diseases. Founded in 2017, the company focuses on advancing treatments for conditions such as myotonic dystrophy type 1, Duchenne muscular dystrophy, and facioscapulohumeral muscular dystrophy, as well as rare skeletal, cardiac, and metabolic muscle diseases. Utilizing its proprietary FORCE platform, Dyne Therapeutics delivers nucleic acids and other therapeutic molecules to muscle tissues with high precision, aiming to restore muscle strength and improve patients' quality of life. The company is currently advancing its product candidates, DYNE-101 and DYNE-251, which are in phase 1/2 clinical trials.

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 Ltd. is a clinical-stage genetic medicine company based in Singapore, focused on developing innovative treatments for genetically defined diseases using its proprietary PRISM platform. This platform enables the precise design, optimization, and production of stereopure oligonucleotides, which are designed to target genetic defects by either reducing harmful protein expression or converting dysfunctional proteins into functional ones. The company's primary focus is on neurology, particularly within the central and neuromuscular systems. WaVe Life Sciences collaborates with major pharmaceutical companies, including Pfizer and Takeda, to advance the research and development of its oligonucleotide therapeutics. Founded in 2012, the company aims to deliver transformative therapies to improve patient outcomes in both rare and prevalent disorders.

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, Inc. is a clinical-stage gene therapy company based in Tucson, Arizona, specializing in innovative cell-based therapies for individuals living with human immunodeficiency virus (HIV). Founded in 2000, the company is focused on developing its lead therapeutic candidate, Cal-1, a gene-based stem cell therapy designed to control HIV infection and prevent the progression to AIDS. Cal-1 employs a dual mechanism to reduce the production of CCR5, a protein critical for HIV infection, and to hinder viral fusion, which facilitates the virus's entry into cells. This approach has demonstrated effectiveness against various strains of HIV in pre-clinical studies. Calimmune aims to address the diverse needs of individuals at different stages of HIV infection and with varying treatment histories, offering potential solutions that enhance immune function and improve quality of life for those affected by the virus.

Precision BioSciences, Inc. is a biotechnology company focused on genome editing, headquartered in Durham, North Carolina. The company utilizes its proprietary ARCUS technology to develop therapeutic products aimed at treating human diseases and improving food and agricultural solutions. Precision BioSciences operates through two segments: Therapeutic and Food. The Therapeutic segment is particularly dedicated to advancing allogeneic CAR T immunotherapies for cancer treatment, including candidates like PBCAR0191 and PBCAR20A, which target specific tumor antigens. The company is also involved in in vivo gene correction and is exploring treatments for conditions such as acute lymphoblastic leukemia and chronic lymphocytic leukemia. Additionally, the Food segment develops innovative nutrition products. Precision BioSciences has formed strategic collaborations, including agreements with Shire Plc and Gilead Sciences, to enhance its research and development capabilities. Founded in 2006, the company aims to harness its next-generation gene editing technology to address pressing health challenges.

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 Ltd. is a clinical-stage genetic medicine company based in Singapore, focused on developing innovative treatments for genetically defined diseases using its proprietary PRISM platform. This platform enables the precise design, optimization, and production of stereopure oligonucleotides, which are designed to target genetic defects by either reducing harmful protein expression or converting dysfunctional proteins into functional ones. The company's primary focus is on neurology, particularly within the central and neuromuscular systems. WaVe Life Sciences collaborates with major pharmaceutical companies, including Pfizer and Takeda, to advance the research and development of its oligonucleotide therapeutics. Founded in 2012, the company aims to deliver transformative therapies to improve patient outcomes in both rare and prevalent disorders.

Larimar Therapeutics, Inc. is a clinical-stage biotechnology company based in Bala Cynwyd, Pennsylvania, dedicated to developing treatments for complex rare diseases. Its primary focus is on Friedreich's ataxia, a rare and progressive genetic disorder, for which it is advancing its lead compound, CTI-1601, through a Phase 1 clinical program. CTI-1601 is designed as a recombinant fusion protein that delivers human frataxin, an essential protein, directly to the mitochondria using a novel cell-penetrating peptide technology. In addition to Friedreich's ataxia, Larimar aims to leverage its intracellular delivery platform to create additional fusion proteins targeting other rare diseases associated with deficiencies in bioactive compounds.

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, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on researching, developing, and commercializing gene therapies for severe genetic diseases and cancer. The company's gene therapy programs include LentiGlobin, aimed at treating β-thalassemia and sickle cell disease, and Lenti-D for cerebral adrenoleukodystrophy. In oncology, bluebird bio is developing chimeric antigen receptor T (CAR T) cell therapies, specifically bb2121 and bb21217, for multiple myeloma. The company has formed strategic collaborations with several organizations, including Bristol-Myers Squibb and Regeneron Pharmaceuticals, to advance gene therapies in oncology and immune cell therapies. Additionally, bluebird bio collaborates with various partners to explore T cell receptor product candidates and in vivo genome editing treatments for genetic disorders. Established in 1992 and originally known as Genetix Pharmaceuticals, the company transitioned to its current name in 2010.

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.