Led · Equity · Nov 2025

MyoGene Bio is a privately held biotechnology company focused on next-generation genetic therapies for Duchenne and other muscle diseases. Its lead program, MyoDys45-55, employs CRISPR-based editing to delete exons 45-55 of the DMD gene, recreating an in-frame variant that produces a highly functional dystrophin protein similar to that seen in mild Becker muscular dystrophy. This single-edit strategy could be applicable to roughly 50% of Duchenne patients and offers the potential for a one-time, permanent correction, unlike traditional gene-replacement approaches whose durability remains uncertain. The company plans to use newly raised funds to compare the functionality of its corrected dystrophin to other variants and to conduct additional safety characterization studies as it moves toward clinical trials. MyoGene has historically relied on non-dilutive grants and patient-advocacy capital to finance its work and intends to leverage the latest commitments to secure further awards. Including the most recent tranche, the company has attracted $1.42 million in total support from patient-advocacy organizations. No commercial revenue or clinical-stage milestones were disclosed in the article.

Led · Equity · Feb 2024

Kinea Bio is pioneering a dual adeno-associated virus (AAV) vector platform, SIMPLI-GT, designed to deliver therapeutic genes that exceed standard AAV packaging limits. Its lead candidate, KNA-155, aims to restore full-length dysferlin in patients with dysferlinopathy, a progressive muscular dystrophy with no approved treatments. To boost muscle targeting and safety, the company has licensed Solid Biosciences’ next-generation myotropic capsid, AAV-SLB101, as the delivery backbone for systemic administration. Preclinical work has already shown promising efficacy, and upcoming IND-enabling dose-finding and GLP toxicology studies will establish safety, biodistribution and therapeutic windows. The Jain Foundation has committed up to $1.1 million in milestone-based funding to support these studies, giving Kinea Bio initial capital to accelerate toward the clinic. Beyond dysferlinopathy, the firm plans to leverage its SIMPLI-GT platform for additional severe genetic diseases, such as Duchenne muscular dystrophy, through strategic collaborations and continued innovation.

Participated · Seed · Jan 2023

Myosana has built a muscle-specific non-viral gene delivery platform designed to target muscle tissue with large gene capacity, enable repeat dosing, and avoid immune responses associated with viral vectors. The company’s lead therapeutic approach is delivery of full-length dystrophin as a potential therapy for Duchenne muscular dystrophy. Management says the platform could be applied across a broad range of neuromuscular and cardiac diseases to increase longevity and improve quality of life. Myosana plans to identify its first development candidate for Duchenne by 2025 and management has cited an 18–24 month timeline for candidate nomination tied to platform optimization. The company recently completed a seed financing of over $5 million to support those development activities. Myosana’s leadership includes cofounders Stan Froehner, Ph.D., and Nick Whitehead, Ph.D., and newly appointed CEO Dr. Matthew Lumley, a physician-scientist with experience in rare disease clinical development. Myosana Therapeutics is developing a pioneering non-viral gene delivery platform that specifically targets skeletal and cardiac muscle cells to deliver full-length genes, including dystrophin, regardless of gene size. The platform is intended to overcome limitations of AAV vectors such as micro-dystrophin delivery and immunogenicity. The company’s initial focus is on disease-modifying therapeutics for Duchenne muscular dystrophy, with potential applicability to other neuromuscular genetic diseases. Myosana will use the seed funding to advance development of its non-viral full-length dystrophin gene therapy. The company is a University of Washington spinout led by founders Stan Froehner and Nick Whitehead. As part of the transaction, CureDuchenne’s Debra Miller will join Myosana’s board.