- Sarepta Therapeutics is officially taking onboard an experimental gene therapy developed by scientists at Nationwide Children's Hospital in Ohio, announcing Wednesday it had licensed the treatment for a type of muscular dystrophy.
- With the licensing agreement, Sarepta has followed through on a deal it struck with Nationwide two years ago to secure an exclusive option for the gene therapy. Wednesday's agreement was spurred by the completion of preclinical and safety studies that Sarepta said had demonstrated "early proof of concept" for the treatment.
- The gene therapy is designed to treat a common form of limb-girdle muscular dystrophy, a severe neuromuscular disease that leads to progressive weakness in the limbs and around the hips and shoulders. The type targeted by Nationwide's treatment, Type 2A, is caused by mutations in the CAPN-3 gene and is considered the most common form of limb-girdle.
Sarepta can claim one of the larger pipelines in biotech, with 42 different programs listed by the developer. Twenty-six of those are gene replacement therapies for a range of muscular dystrophies as well as lysosomal storage disorders and diseases of the nervous system. Most, however, are in the preclinical or early clinical stages of development.
Nationwide has been a common partner and source for those programs, including the Duchenne muscular dystrophy treatment that is currently the most valued and closely watched gene therapy Sarepta is developing. The Ohio hospital is a hub for gene therapy research, having spun out several biotech companies, including AveXis, the developer of the spinal muscular atrophy treatment Novartis now sells as Zolgensma, and Myonexus, a startup acquired by Sarepta in 2019.
The hospital has also built out its manufacturing capabilities, launching a subsidiary called Andelyn Biosciences that aims to produce gene therapy components for biotech and pharmaceutical companies.
Zarife Sahenk, a neruologist at Nationwide, led preclinical research supporting the limb-girdle gene therapy Sarepta has now licensed.
The candidate treatment is built around the same type of uninfectious virus that Sarepta uses to deliver functional genes with its Duchenne treatment and five others it's developing for different forms of limb-girdle. In licensing the treatment, Sarepta hopes to "build off the knowledge we gained" from those other programs, said Louise Rodino-Klapac, the company's chief scientific officer and the former head of a gene therapy lab at Nationwide, in a statement.
Sarepta estimates that more than 70% of limb-girdle muscular dystrophy cases would be addressed by the six gene therapies it now owns and is developing. The most advanced of those candidates is known as SRP-9003 and targets Type 2E. Early results from a handful of patients show treatment led to increased production of the protein missing in the disease, and resulted in functional gains.