Sarepta, Aldevron Partnering to Develop Gene Therapies for CMT, Other Rare Diseases

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Aldevron has signed a long-term agreement with Sarepta Therapeutics to provide the gene-transfer vehicles used in Sarepta’s gene therapies that are being tested in ongoing and future clinical trials, as well as for future commercialization.

Sarepta Therapeutics is a leading pharmaceutical developing precision genetic therapies for rare diseases, and has begun developing a gene therapy program for Charcot-Marie-Tooth (CMT) disease, as well as Sanfilippo syndrome type A (also called mucopolysaccharidosis type 3A), Pompe disease, and other disorders affecting the central nervous system.

“Our greatest satisfaction comes in helping companies whose research is making an impact on the lives of patients and we are proud to partner with Sarepta, a company dedicated to extending and saving lives,” said Michael Chambers, CEO and co-founder of Aldevron, said in a press release.

“Aldevron has made significant investments in people, processes and facilities to support the pre-clinical, clinical and commercial production of new, genetically-based therapies that have significant potential in transforming disease.”

Recently, Sarepta acquired the exclusive rights to neutotrophin 3 (NT-3), a gene therapy candidate for the treatment of CMT neuropathies developed by researchers at Nationwide Children’s Hospital.

NT-3 belongs to the nerve growth factor family, meaning it contributes to the growth and survival of neuronal cells in both the central and peripheral nervous systems.

A plasmid containing the NT-3 gene construct promoted nerve regeneration and sensory improvement in a mouse model of CMT type 1A.

Gene therapies use small circular, closed DNA molecules called plasmids as gene-transfer vehicles.

Under the terms of the agreement, Aldevron will provide GMP-Source plasmids, or Good Manufacturing Practice plasmids, that ensure the material is of high quality, allowing a controlled composition of the DNA used in the therapy.

“One of our highest priorities is building a robust supply chain and scalable manufacturing that can accelerate and ensure robust patient access to our pipeline of promising gene therapies on an accelerated timeline,” said Doug Ingram, Sarepta’s president and CEO.

“Aldevron, one of the top plasmid DNA manufacturers in the world, is an important partner in fulfilling our strategic objectives. This agreement is anticipated to provide sufficient plasmid supply to support our ambitious development and commercial gene therapy objectives.”

Sarepta has been developing gene therapies for neuromuscular disorders, including limb-girdle muscular dystrophy and with Duchenne muscular dystrophy.

One of its therapies for DMD is currently being tested in a Phase 1/2 trial.