On April 22, 2024 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported an oral presentation highlighting the Company’s lipid nanoparticle (LNP) approach for ocular editing will be presented at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2024 Annual Meeting, taking place May 7 – 11, 2024, in Baltimore, MD and virtually (Press release, CRISPR Therapeutics, APR 22, 2024, View Source [SID1234642193]).
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The abstract describes our proprietary capabilities to deliver to and edit genes in the eye, opening a potential new focus area. Multiple LNPs as well as modified gRNAs and mRNAs were screened to achieve maximal editing in vivo. These optimized components have been applied to target myocilin (MYOC). Mutations of MYOC in trabecular meshwork cells have been linked to severe glaucomatous conditions. In human primary trabecular meshwork cells, up to 95% MYOC editing and 85% protein knockdown were seen. This novel approach aims to facilitate glaucoma treatment using transient expression of editing machinery targeting MYOC.
Title: Development of an In Vivo Non-Viral Ocular Editing Platform and Application to Potential Treatments for Glaucoma
Session Type: In-Person Oral Presentation
Session Title: Ophthalmic and Auditory: Delivery Innovations
Abstract Number: 87
Location: Room 318 – 323
Session Date and Time: Wednesday, May 8, 2024, 1:30 p.m. – 3:15 p.m. ET
The accepted abstract is available online on the ASGCT (Free ASGCT Whitepaper) website. The data are embargoed until 6:00 a.m. ET on the presentation day, Wednesday May 8, 2024. A copy of the presentation will be available at www.crisprtx.com once the presentation concludes.