RARE Daily

Armatus Bio Licenses Solid’s AAV Capsid to Develop an RNAi Therapy to treat FSHD

March 8, 2024

Rare Daily Staff

Solid Biosciences entered into a non-exclusive worldwide license and collaboration agreement with Armatus Bio for the use of Solid’s proprietary capsid AAV-SLB101 for the development and commercialization of Armatus’ vectorized RNAi candidate to treat facioscapulohumeral muscular dystrophy.

The AAV-SLB101 capsid has been shown in preclinical studies to have enhanced biodistribution and improved expression in muscle cells.

Under the terms of the agreement, Solid granted Armatus a non-exclusive worldwide license to utilize AAV-SLB101 for treatment of facioscapulohumeral muscular dystrophy (FSHD) and will provide Armatus AAV-SLB101 plasmid material, preclinical data characterizing AAV-SLB101, and manufacturing and regulatory know-how to enable development. In return, Solid will receive an upfront payment, payments upon the achievement of certain development and sales milestones, and tiered royalties on net sales for any products incorporating AAV-SLB101. Armatus will be responsible for the development and commercialization of licensed products incorporating AAV-SLB101.

“Our transaction with Armatus Bio is the first of what we intend to be a broad out-licensing of our capsid library to both companies and academic institutions,” said Bo Cumbo, president and CEO at Solid Biosciences. “We designed AAV-SLB101 for enhanced biodistribution to skeletal muscle, which is intended to help improve the efficacy and reduce the toxicity of AAV-delivered gene therapies for muscular disorders.”

FSHD is one of the most common forms of adult muscular dystrophy, affecting 1 in 8,000 people worldwide. Symptoms usually start before age 20 and may lead to patients being wheelchair-bound by age 50. There are no treatment options for FSHD, and patients manage symptoms through the use of assistive devices, physical therapy, and orthopedics.

Armatus is developing ARM-201, a potential best-in-class, single dose therapeutic used for the treatment of FSHD, a progressive genetic neuromuscular disorder caused by DUX4 overexpression. ARM-201’s proprietary miRNA payload seeks to reduce DUX4, and thus arrest muscle weakening and atrophy, prevent further degeneration, and ameliorate the amount of inflammation and oxidative stress caused by FSHD.

“The use of Solid’s unique AAV-SLB101 capsid will help enable optimal patient dosing for our FSHD program. The preclinical data presented to date for AAV-SLB101 demonstrate increased muscle transduction while simultaneously reducing biodistribution in the liver. These data, combined with the data we have generated for ARM-201 give us high confidence in the potential to reduce total AAV dose levels compared to first generation capsids,” said Brian Price, chief technology officer at Armatus Bio. “Further, we are encouraged by the strong safety profile demonstrated by AAV-SLB101, which has a cleared IND as the capsid in Solid’s SGT-003 construct for the treatment of DMD.”

Photo: Bo Cumbo, president and CEO at Solid Biosciences

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