Sarepta plans gene editing center; files DMD therapy for review

Genetic disease-focused firms need infrastructure says Sarepta, which shared plans for a gene editing center of excellence at the JP Morgan healthcare conference.

The gene editing center will be in Durham, North Carolina according to CEO Douglas Ingram, who said investing in capacity and knowhow is key to Sarepta’s longevity.

“If one is going to be an enduring genetic medicine company, we are going to need infrastructure,” he said, citing partnerships as an important part of the expansion.

Image: iStock/vchal

“We’re deepening our relationship with Dr. Charlie Gersbach at Duke University and getting even deeper into the research around gene editing as a research platform.

“We’ve also entered into a relationship with a gene editing pioneer, that’s Amy Wagers at Harvard, who has a very specific approach to gene editing with respect to stem cells or satellite cells.”

Manufacturing partnerships

The plan to invest in a gene editing center follows a busy period of expansion for Sarepta.

Last year, for example, the firm built a gene therapy development center of excellence in Columbus, Ohio.

It also expanded commercial gene therapy manufacturing capacity in collaboration with various partners.

Ingram said: “We completed with Thermo Fisher our single-use site in Lexington, Massachusetts for micro-dystrophin, and we built even more capacity than that with Catalent, Paragon.”

He added Sarepta is conducting engineering runs to support Study 301 – a trial of SRP-9001 (AAVrh74.MHCK7.micro-dystrophin) that is due to start this year.

Hybrid model

The collaborations are part of a “hybrid manufacturing” approach Sarepta adopted in 2018.

The idea is to build internal expertise in adeno-associated virus (AAV)-based manufacturing while partnering with contractors.

In related news, this month French CDMO Yposkesi said it would make AAV material for Sarepta and development partner Genethon.

By 2023 Yposkesi plans to double its manufacturing footprint with a new plant. The firm told us the intention is to service part of the Genethon and Sarepta contract at the new facility.

Pipeline update

Sarepta also revealed it has filed Casimersen (SRP-4045) for US Food and Drug Administration (FDA) review.

Ingram said, “We commenced our rolling submission for the approval of Casimersen, which, if successful, will treat another 8% or so of the Duchenne muscular dystrophy [DMD] community. It is our goal to obtain that approval in 2020.”

He explained Sarepta opted for a rolling submission because additional CMC and specification work is required.

“We didn’t want to wait for that to get done and what and then submit. We wanted to start a rolling submission. So we’ve got to get that work done. That will get done in the near future.”