Sarepta and Aldevron Partnering on Gene Therapy Program for DMD and LGMD

Ana Pena, PhD avatar

by Ana Pena, PhD |

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Sarepta partnership

Sarepta Therapeutics has entered a long-term, strategic partnership with Aldevron to advance its gene therapy program for Duchenne mulcular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD).

Aldevron, which produces biological products for the biotech industry, will be responsible for supplying plasmid DNA to support Sarepta’s gene therapy pipeline.

“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,” Michael Chambers, CEO and co-founder of Aldevron, said in a press release.

Plasmid DNA is a circular molecule of DNA, commonly used in gene editing to deliver a specific DNA sequence to cells — in this case, a functional version of the faulty gene carried by patients.

Aldevron has committed to produce Good Manufacturing Practice (GMP)-grade plasmids for Sarepta’s microdystrophin DMD gene therapy program.

GMP is a system of regulations and guidelines for ensuring that products are consistently produced and controlled according to quality standards. Aldevron has specialized in GMP manufacturing and is known for inventing the GMP-Source system, “a faster, cost-effective alternative to GMP production,” the company states on its website.

Sarepta currently has one microdystrophin gene therapy candidate for DMD under evaluation in a Phase 1/2 clinical trial (NCT03375164) at Nationwide Children’s Hospital in Columbus, Ohio.

The treatment consists of a viral carrier (called rAAVrh74.MHCK7.micro-dystrophin) that delivers a shorter but functional version of the DMD gene, the gene defective in people with DMD. This shorter gene provides instructions for the production of a smaller form of the dystrophin protein, called microdystrophin, which, despite being shorter, works properly.

A single dose of the treatment will be infused into the blood of 12 patients, divided in two age groups: 3 months to 3 years, and 4 to 7 years.

In July 2018, this trial was put on hold by the U.S. Food and Drug Administration, due to concerns about the raw materials used to make the therapy. After addressing these problems, the study resumed in September and is currently ongoing.

So far, results from the trial show that the gene therapy candidate is able to improve the functional performance of DMD boys, without causing serious adverse events.

The company will also provide plasmid source material for gene therapy candidates targeting five types of LGMD (LGMD2E, LGMD2D, LGMD2C, LGMD2B, and LGMD2L) as well as Sarepta’s future programs intended for other muscular and nervous system diseases.

“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.