Sarepta and Genevant Enter Research Collaboration for LNP-Based Gene Editing Therapeutics

Rare Daily Staff

Sarepta Therapeutics and Genevant Sciences have entered into a research collaboration and option agreement for the delivery of lipid nanoparticle-based gene editing therapeutics in Sarepta’s pipeline for neuromuscular diseases.

The alliance will assess the use of Sarepta’s proprietary gene editing technology and Genevant’s proprietary lipid nanoparticle (LNP) delivery platform for multiple neuromuscular targets.

Under the terms of the agreement, Genevant will design and collaborate with Sarepta in the development of muscle targeted LNPs to be applied to gene editing targets in early stage development. Sarepta will have rights to an exclusive license to Genevant’s LNP technology for up to four neuromuscular indications, including Duchenne muscular dystrophy. Genevant may receive approximately $50 million in near-term payments and is also eligible for significant future development, regulatory and commercial milestones and tiered royalties ranging from the mid-single to low-double digits on future product sales.

LNPs offer the potential for a non-viral approach to gene editing and can provide both optimal uptake into desired cells and efficient release, resulting in functional delivery of gene editing cargo, such as CRISPR-Cas, to target tissues.

Sarepta is pursuing a variety of approaches to genetic medicine including exon skipping, gene therapies and gene editing in pursuit of cures for rare diseases. Gene editing has the potential to revolutionize the treatment of diseases caused by genetic mutations—including rare neuromuscular diseases—by permanently altering genes that lead to disease.

“As Sarepta works to advance precision genetic medicine across multiple modalities, we’ve invested in partnering and research efforts focused on improving the utility and benefit of gene-based medicines and providing the greatest possible outcome to patients,” said Doug Ingram, president and CEO of Sarepta Therapeutics. “This includes advancing our pre-clinical gene editing program, looking at both viral and non-viral methods to produce a functional gene in order to treat a broad range of neuromuscular diseases.”

Genevant is a leader in LNP delivery of nucleic acids, and is the delivery technology behind the first nucleic acid-LNP product to have achieved FDA approval—Alnylam Pharmaceuticals’ Onpattro for the treatment of polyneuropathy caused by hereditary transthyretin-mediated amyloidosis in adult patients.

“Efficient, optimized delivery is often the difference between successful and unsuccessful nucleic acid drug development, and we are excited to bring our experience to Sarepta’s gene editing programs in neuromuscular disease where new options – and new approaches – are desperately needed.” said Pete Lutwyche, president and CEO of Genevant Sciences.

Photo: Doug Ingram, president and CEO of Sarepta Therapeutics