Sarepta tries to fine-tune its gene therapy approach – BioPharma Dive

Posted: June 23, 2020 at 12:56 am

Dive Brief:

Known for its muscular dystrophy drugs Exondys 51 and Vydonys 53, Sarepta has quickly become a leader in gene therapy as well.

The last couple of years have seen the Cambridge, Massachusetts-based biotech ink gene therapy manufacturing and development deals, including a partnership with Roche that could be worth more than $3 billion.

Sarepta now has six gene therapies in clinical testing and another half dozen in preclinical stages. Most recently, the company announced positive, albeit early, results from a study of its experimental treatment for limb-girdle muscular dystrophy, a potentially deadly genetic disease.

With gene therapy set to become a cornerstone of its business, Sarepta is trying to avoid some the challenges presented by current technologies.

For example, when the company teamed up with North Carolina-based StrideBio late last year, one of the main goals was to use the partner's technology to "address re-dosing challenges in patients who have received AAV-delivered gene therapy." The deal came just days after a study testing an experimental gene therapy from Solid Biosciences, one of Sarepta's rivals, was paused due to a patient experiencing an immune response and organ complications.

"If successful, the ability to re-dose will be an enormous leap forward in the science of gene therapy and provide invaluable benefits to patients beyond those we anticipate with one-time dosing," said Doug Ingram, Sarepta's CEO, in a June 18 statement announcing the Selecta deal.

Under terms of that deal, Sarepta will pay Selecta an initial, undisclosed amount. Selecta is eligible to receive pre-clinical milestone payments, and could take home additional development, regulatory and commercial milestones should Sarepta exercise its options to enter a licensing agreement.

Specifically, the deal with Selecta centers on gene therapies for Duchenne muscular dystrophy and certain limb-girdle muscular dystrophies.

With Codiac, Sarepta has offered up $72.5 million in upfront and near-term license payments plus research funding. In addition, Codiak is eligible for "significant" milestone payments, according to Sarepta.

The two-year deal gives Sarepta the option to license Codiak's technology for up to five neuromuscular targets. The companies said they will collaborate on the design of exosomes that can deliver and release gene therapy, gene editing or RNA payloads. If Sarepta exercises an option, Codiak will then be in charge of research until right before the candidate goes into in-human testing. Sarepta is responsible for clinical development and commercial activities.

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Sarepta tries to fine-tune its gene therapy approach - BioPharma Dive

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