Sarepta and GenEdit Expand Partnership to Transcend Gene Therapy Challenges


Sarepta Therapeutics, a precision genetics medicine company based in Cambridge, Massachusetts, and San Francisco-based GenEdit, which focuses on gene editing for tissue-specific delivery, announced a research agreement on Tuesday to develop gene editing therapeutics. The agreement is focused on genetic neuromuscular diseases, but the implications of the research could extend to a variety of conditions previously considered untreatable.

Sarepta and GenEdit are a hand-in-glove match for this type of research. Sarepta has over 40 years of experience in medical research and drug development. Now, Sarepta focuses on treatments for rare muscular diseases, and gene editing is the latest avenue of potential therapies it is pursuing. GenEdit’s NanoGalaxy™ platform uses non-viral, non-lipid polymer nanoparticles that target specific types of muscle tissue. The NanoGalaxy platform also models and screens polymers, eliminating the ones that won’t work and identifying the ones most likely to be effective in the body. Together, these two companies are combining their strengths to find treatment options for rare neuromuscular diseases.

The announcement is a milestone in gene editing research, but Sarepta and GenEdit have already been working together for a while. Beginning in 2020, the two companies began in vivo testing of GenEdit’s polymer nanoparticles. The tests were designed to see if gene editing polymers could be delivered systemically—through an IV, for example—and still deliver the target pharmaceutical to specific muscle tissue. The results of the in vivo study were successful.

GenEdit CEO Kunwoo Lee_courtesy GenEditIn an interview with BioSpace, GenEdit CEO and co-founder Dr. Kunwoo Lee, Ph.D., explained that delivery has been the most difficult obstacle facing gene editing.

“Delivery to muscle tissue has been a huge challenge. Currently, the only delivery system we have is AAV [adeno-associated virus], but that goes to the whole body and is not specific to muscle, and it’s difficult to manufacture. However, with the NanoGalaxy platform, we are seeing a potential way to solve this difficult challenge. There has never been a non-viral delivery system that has achieved specific muscle delivery. But with Sarepta’s expertise, we have shown that it is possible, and we are looking forward to next steps.”

One of those next steps was to set up this agreement to conduct further research. As part of the agreement, Sarepta obtains exclusive option rights to license polymer nanoparticles developed by GenEdit in the collaboration for up to four neuromuscular indications selected by Sarepta. GenEdit may also receive $57 million in near-term payments and is eligible for future development, regulatory and commercial milestones, along with tiered royalties.

“Sarepta is committed to the development of therapies for rare neuromuscular diseases, and we look forward to continuing to work with the team at GenEdit to advance effective gene editing-based treatments for these patients,” Sarepta president and CEO Doug Ingram said in a statement.

And what’s the next step after that? Lee told BioSpace he hopes that gene editing will help treat not just neuromuscular diseases, but a variety of other genetic conditions too.

“Eventually, we want to be able to deliver therapies to the heart and other types of muscles,” he said. “In this collaboration, we’re focusing on muscle, but our polymers could potentially deliver various genetic medicines to the nervous system and the immune system.”

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