CRISPR Therapeutics to Present Oral Presentation at the American Society of Gene & Cell Therapy (ASGCT) 2024 Annual Meeting

ZUG, Switzerland and BOSTON, April 22, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced 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) 2024 Annual Meeting, taking place May 7 – 11, 2024, in Baltimore, MD and virtually.

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 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.

About CRISPR Therapeutics 
Since its inception over a decade ago, CRISPR Therapeutics has transformed from a research-stage company advancing programs in the field of gene editing, to a company that recently celebrated the historic approval of the first-ever CRISPR-based therapy and has a diverse portfolio of product candidates across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine, cardiovascular, autoimmune, and rare diseases. CRISPR Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic in 2018 to investigate the treatment of sickle cell disease or transfusion-dependent beta thalassemia, and beginning in late 2023, CASGEVY (exagamglogene autotemcel) was approved in some countries to treat eligible patients with either of those conditions. The Nobel Prize-winning CRISPR science has revolutionized biomedical research and represents a powerful, clinically validated approach with the potential to create a new class of potentially transformative medicines. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer and Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California, and business offices in London, United Kingdom. To learn more, visit www.crisprtx.com.