Venture philanthropy organisation Solve FSHD has announced an investment of $3m in Armatus Bio to support the development of ARM-201, an adeno-associated virus (AAV)-delivered microRNA (miRNA) therapy for facioscapulohumeral muscular dystrophy (FSHD).
The investment is part of a strategic effort to combat FSHD, a widespread form of muscular dystrophy.
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The funding will expedite the progress of the therapy targeting the toxic double homeobox 4 (DUX4) protein, which is implicated in muscle deterioration in FSHD patients.
ARM-201’s miRNA payload aims to decrease DUX4 expression, potentially halting muscle weakening and atrophy, eliminating further degeneration, and minimising inflammation and oxidative stress associated with FSHD.
The therapy employs AAV-SLB101, a capsid licensed from Solid Biosciences, engineered to aim at integrin receptors, and in preclinical trials, it has demonstrated improvement in cardiac and skeletal muscle transduction while minimising liver exposure.
It has demonstrated tolerability with strong transduction and expression levels in a Duchenne muscular dystrophy trial.
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By GlobalDataFollowing a pre-investigational new drug (IND) meeting with the US Food and Drug Administration, Armatus plans to use the investment for IND-enabling activities, setting the stage for regulatory submission and the initiation of clinical trials for FSHD.
Solve FSHD, established by Canadian entrepreneur Chip Wilson, aims to expedite research to find a cure for FSHD. The Wilson family has pledged $100m to support projects aligned with this mission.
In July 2023, Solve FSHD awarded four grants to enhance the understanding of FSHD and foster the development of therapeutic solutions for this condition.
