Press release
Sarepta Therapeutics and Genevant Sciences Announce Research Collaboration for Lipid Nanoparticle-Based Gene Editing Therapeutics
-- Alliance will assess the use of Sarepta’s proprietary gene editing technology and Genevant’s proprietary LNP delivery platform for multiple neuromuscular
About this update from Sarepta Therapeutics, Inc.
[{"type":"text","content":"-- Alliance will assess the use of Sarepta’s proprietary gene editing technology and Genevant’s proprietary LNP delivery platform for multiple neuromuscular targets -- -- Sarepta to have options for an exclusive license to Genevant’s LNP technology for four neuromuscular indications -- -- Genevant may receive approximately $50 million in near-term payments and is also eligible for significant future milestones and royalties -- CAMBRIDGE, Mass., VANCOUVER, British Columbia, and BASEL, Switzerland, Jan. 13, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, and Genevant Sciences, a leading nucleic acid delivery company with world-class platforms and the industry’s most robust and expansive lipid nanoparticle (LNP) patent estate, today announced a research collaboration and option agreement for the delivery of LNP-gene editing therapeutics in Sarepta’s pipeline for neuromuscular diseases. 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. 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. 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. 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. “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-bas...