CureDuchenne

MyoGene Bio is a biotechnology company focused on developing innovative therapies for muscle diseases, with an initial emphasis on gene-editing treatments for Duchenne muscular dystrophy. The company employs advanced technologies to address the underlying causes of this condition, aiming to significantly improve disease progression and outcomes for patients. By targeting the root of the disorder, MyoGene Bio seeks to provide clinicians and patients with effective solutions that have a meaningful impact on the management of devastating muscle disorders.

Insmed is a global biopharmaceutical company dedicated to improving the lives of patients with serious and rare diseases. Its first approved product, ARIKAYCE®, treats Mycobacterium avium complex (MAC) lung disease in adults with limited treatment options. Insmed's pipeline includes INS1007 for non-cystic fibrosis bronchiectasis and other inflammatory diseases, and INS1009 for pulmonary arterial hypertension.

Myosana Therapeutics, Inc. is a biotechnology company focused on developing non-viral gene therapies aimed at treating muscle diseases, particularly Duchenne muscular dystrophy and other neuromuscular conditions. Founded in 2018 and based in Seattle, Washington, the company has created a novel gene delivery platform that specifically targets muscle cells to facilitate the production of proteins that are deficient due to genetic mutations. By delivering these proteins directly to the cell nuclei, Myosana Therapeutics seeks to slow the degeneration of skeletal muscles and reduce the impact of heart failure, ultimately improving the quality of life and longevity for affected individuals.

Code Biotherapeutics is a next-generation gene therapy company pioneering the development of targeted non-viral gene therapies to treat serious and life-threatening genetic diseases. Code Bio leverages its novel multivalent synthetic DNA delivery platform, 3DNA, which has been engineered to overcome many of the challenges inherent with viral-based gene therapies such as immunogenicity, size and delivery limitations, re-dosability, and manufacturing complexity. The company is advancing an internal pipeline focused on select rare disease programs as well as establishing partnerships to take forward programs in both rare and prevalent diseases.

Gennao Bio is a privately held company focused on developing innovative targeted nucleic acid therapeutics, primarily aimed at treating oncology and rare monogenic skeletal muscle diseases. The company utilizes its proprietary gene monoclonal antibody (GMAB) platform technology, which features a novel, cell-penetrating antibody that facilitates the non-covalent binding and delivery of various nucleic acid payloads to specific cells. This non-viral delivery system stands out from traditional gene therapy methods by allowing for multiple types of nucleic acids, enabling repeat dosing, and employing established manufacturing processes. Gennao Bio's approach aims to address significant unmet medical needs and improve patient outcomes in the relevant therapeutic areas.

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.

Code Biotherapeutics is a next-generation gene therapy company pioneering the development of targeted non-viral gene therapies to treat serious and life-threatening genetic diseases. Code Bio leverages its novel multivalent synthetic DNA delivery platform, 3DNA, which has been engineered to overcome many of the challenges inherent with viral-based gene therapies such as immunogenicity, size and delivery limitations, re-dosability, and manufacturing complexity. The company is advancing an internal pipeline focused on select rare disease programs as well as establishing partnerships to take forward programs in both rare and prevalent 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.

Mesentech Inc. is a regenerative medicine company based in Vancouver, Canada, focused on developing innovative therapies for bone-related diseases. Established in 2013, the company specializes in a prodrug platform that delivers therapeutics selectively to bone, addressing challenges associated with conventional drug delivery methods. Mesentech's lead product, MES1007, is a bone-targeted prodrug of a prostaglandin mimetic that aims to reverse bone loss associated with conditions such as osteoporosis and Duchenne muscular dystrophy. The company's proprietary technology enhances the delivery of small-molecule and biological drugs to the bone's surface and interior, allowing for more effective treatment while minimizing side effects. This approach is particularly significant given the inherent difficulty in accessing bone tissue due to its hard surface and low porosity. Mesentech's focus on selective drug delivery not only aims to improve treatment efficacy but also hopes to accelerate bone healing and address complications associated with low bone density disorders.

Myosana Therapeutics, Inc. is a biotechnology company focused on developing non-viral gene therapies aimed at treating muscle diseases, particularly Duchenne muscular dystrophy and other neuromuscular conditions. Founded in 2018 and based in Seattle, Washington, the company has created a novel gene delivery platform that specifically targets muscle cells to facilitate the production of proteins that are deficient due to genetic mutations. By delivering these proteins directly to the cell nuclei, Myosana Therapeutics seeks to slow the degeneration of skeletal muscles and reduce the impact of heart failure, ultimately improving the quality of life and longevity for affected individuals.

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.

Shape Therapeutics, Inc. is a biotechnology company focused on RNA-editing gene therapy for various medical conditions, including neurodegenerative disorders, oncology, metabolic diseases, and rare genetic disorders. Founded in 2018 and headquartered in Seattle, Washington, the company utilizes its proprietary RNAfix technology to edit RNA using the body’s natural cellular machinery. This approach minimizes risks associated with immunogenicity, cellular toxicity, and unintended DNA editing. Shape Therapeutics is dedicated to advancing its innovative platforms for payload, delivery, and manufacturing to enable the development of effective treatments, aiming to address critical health challenges and improve patient outcomes.

Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative treatments for severe, rare muscle disorders with significant unmet medical need. It employs a holistic drug discovery approach targeting the muscle as an organ, utilizing its proprietary platform to identify precision medicines regulating key proteins in muscle tissue.

4D Molecular Therapeutics, Inc. is a clinical-stage gene therapy company based in Emeryville, California, focused on developing innovative gene therapies for unmet medical needs. The company utilizes targeted and evolved adeno-associated virus (AAV) vectors to create its product candidates, which are concentrated in the therapeutic areas of ophthalmology, cardiology, and pulmonology. Key candidates in its portfolio include 4D-125, currently in a Phase I/II trial for X-linked retinitis pigmentosa, and 4D-110, undergoing Phase I trials for choroideremia. Additionally, 4D-310 is in Phase I/II trials for Fabry disease, while investigational drugs 4D-150 and 4D-710 are being developed for wet age-related macular degeneration and cystic fibrosis lung disease, respectively. Founded in 2013, 4D Molecular Therapeutics aims to advance its gene therapies to address significant health challenges.

Exonics Therapeutics, Inc. is a biotechnology company focused on developing gene editing technologies to address severe genetic neuromuscular disorders, particularly Duchenne muscular dystrophy (DMD). Founded in 2017 and headquartered in Boston, Massachusetts, with an additional office in Dallas, Texas, Exonics employs CRISPR/Cas9 technology to create potential one-time therapies that permanently correct mutations responsible for DMD. The company's innovative approach utilizes adeno-associated virus (AAV) vectors to deliver gene editing components that target and correct exon mutations, thereby facilitating the production of dystrophin, a crucial protein that stabilizes and protects muscle fibers. By enabling the restoration of dystrophin production, Exonics aims to significantly improve the quality of life for patients affected by this debilitating condition. The company operates as a subsidiary of Vertex Pharmaceuticals Incorporated.

TRiNDS, LLC is a specialized contract research organization based in Pennsylvania that focuses on therapeutic research in neuromuscular disorders and rare diseases. With over 15 years of experience, TRiNDS provides essential support in clinical operations, data management, biostatistics, and study measurements. The company collaborates with pharmaceutical and biotechnology industries, as well as academic investigators and clinical trial sites, to facilitate successful clinical trials and drug development programs. By offering tailored solutions and expertise, TRiNDS aims to enhance the quality of research and ultimately benefit the patient community.

Capricor Therapeutics is a clinical-stage biotechnology company focused on developing cell and exosome-based therapies for Duchenne muscular dystrophy and other high-need diseases. Its lead program CAP-1002 is an allogeneic cardiac-derived cell therapy evaluated for Duchenne muscular dystrophy and related cardiac conditions. The company also pursues CAP-2003 in preclinical development for inflammatory diseases and explores exosome-based approaches through its StealthX platform to advance next-generation therapeutics. Capricor aims to address unmet medical needs by translating cellular and exosome science into transformative treatments. The company was founded in 2005 and is headquartered in Los Angeles, California.

Entos develops next-generation genetic medicines using their proprietary Fusogenix proteolipid vehicle (PLV) drug delivery system. Fusogenix PLVs are formulated with FAST proteins to enable the delivery of nucleic acid into target cells through direct fusion.