Solve FSHD Invests $3 Million in Armatus Bio’s AAV-miRNA Therapy for Muscular Dystrophy

Venture philanthropy organization Solve FSHD has announced a $3 million investment in Armatus Bio to advance the development of ARM-201, an adeno-associated virus (AAV)-delivered microRNA (miRNA) therapy targeting facioscapulohumeral muscular dystrophy (FSHD).

This investment underscores a strategic push to combat FSHD, a prevalent form of muscular dystrophy. The funding will accelerate the progress of ARM-201, a therapy specifically designed to target the toxic double homeobox 4 (DUX4) protein, which plays a key role in the muscle deterioration observed in FSHD patients. The miRNA payload within ARM-201 aims to reduce DUX4 expression, potentially halting the characteristic muscle weakening and atrophy associated with the disease. This could lead to the prevention of further degeneration and a reduction in inflammation and oxidative stress linked to FSHD.

ARM-201 utilizes AAV-SLB101, a viral capsid licensed from Solid Biosciences. This capsid has been engineered to target integrin receptors and, in preclinical studies, has shown promising results in improving gene delivery to both cardiac and skeletal muscle while minimizing exposure to the liver. Notably, AAV-SLB101 has demonstrated tolerability along with strong gene transfer and expression levels in a previous clinical trial for Duchenne muscular dystrophy.

Following a pre-investigational new drug (IND) meeting with the U.S. Food and Drug Administration, Armatus Bio intends to use this $3 million investment from Solve FSHD to conduct essential IND-enabling activities. This critical step will pave the way for a regulatory submission and the subsequent initiation of clinical trials for their FSHD therapy.

Solve FSHD was established by Canadian entrepreneur Chip Wilson with the primary goal of accelerating research towards a cure for FSHD. The Wilson family has committed a substantial $100 million to support projects that align with this important mission.