Editas Sells off iNK Preclinical Cell Therapy Program, Gene-Editing Technologies to Shoreline

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The company is reprioritizing to focus on its clinical stage programs, which ran into a number of setbacks in 2022.

Shoreline Biosciences’ is acquiring Editas Medicines’ cell therapy programs, including its induced pluripotent stem cell (iPSC) derived natural killer cell (iNK) preclinical programs, and licensing its SLEEK (SeLection by Essential-gene Exon Knock-in) and AsCas12a gene editing technologies for oncologic and other indications.1

“The acquisition of our allogeneic iNK franchise by Shoreline is highly aligned with our strategic portfolio reprioritization, as it allows us to sharpen our efforts on advancing current clinical stage trials and focus our resources on in vivo fit-for-purpose therapeutic construction and development,” Gilmore O’Neill, MB, MMSc, president and chief executive officer, Editas Medicine, said in a statement.1 “Shoreline is a leader in developing next generation iNK and macrophage cell therapies, and we believe they are the right company to move these assets toward clinical applications.”

Shoreline is developing cell therapies based on iPSCs that utilize proprietaryiNK and macrophage (iMACs) platforms. Aside from proprietary programs, Shoreline has also partnered with Kite, a Gilead Company, and BeiGene to advance other programs toward the clinic.

“Our goal is to win the war on cancer, and through this agreement with Editas, we have strategically enhanced our ability to execute upon our mission. The addition of Editas Medicine’s novel gene editing SLEEK technology, combined with the use of a high efficiency and high fidelity proprietary CRISPR enzyme, and the other assets from Editas Medicine’s iNK franchise, strengthens our portfolio and ability to create next generation immunotherapies for patients with cancer,” Kleanthis G. Xanthopoulos, PhD,chairman and chief executive officer, Shoreline Biosciences, added to the statement.1 “We look forward to advancing our pipeline towards the clinic, including these new assets and technologies from Editas.”

WATCH NOW: Akshay Sharma, MBBS, on Investigating CRISPR/Cas9 Edited Cells for Sickle Cell Disease

The announcement comes after Editas, the only licensed company for the Broad Institute's CRISPR/Cas9 and Cas12a technologies, experienced multiple struggles with itsclinical stage gene-edited cell therapy pipeline candidates in 2022. The biggest setback was Editas’ announcement in November 2022 that it was pausing development of its lead clinical program EDIT-101, which was being evaluated in the phase 1/2 BRILLIANCE trial (NCT03872479) after disappointing efficacy in treating Leber congenital amaurosis 10.2 Another trial, the RUBY trial (NCT04853576) of EDIT-301 for treating sickle cell disease was paused before dosing due to the need for an improved potency assay before collection of efficacy data.3 However, the hold was lifted in the second half of the year and has since shown an ability to reduce vaso-occlusive events (VOEs) in the first 2 participants dosed.4

“The results from the BRILLIANCE trial provide a proof of concept and important learnings for our inherited retinal disease programs. We’ve demonstrated that we can safely deliver a CRISPR-based gene editing therapeutic to the retina and have clinically meaningful outcomes," O'Neill had said at the time.“While we will not progress EDIT-101 on our own and have made the decision to pause enrollment, we have the patient community top of mind and are looking for a collaboration partner to advance this program.”

REFERENCES
1. Editas Medicine and Shoreline Biosciences Enter into Definitive Agreement for Shoreline to Acquire Editas’ iNK Cell Franchise and Related Gene Editing Technologies. News release. Editas Medicine. January 19, 2023.
2. Editas Medicine announces clinical data demonstrating proof of concept of EDIT-101 from phase 1/2 BRILLIANCE trial. News release. Editas Medicines. November 17, 2022. https://finance.yahoo.com/news/editas-medicine-announces-clinical-data-121500871.html
3. Editas Medicine announces clinical achievements in the development of EDIT-301 for sickle cell disease. News release. Editas Medicine, Inc. July 27, 2022. https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-announces-clinical-achievements-development-edit
4. Editas Medicine announces positive safety and efficacy data from the first two patients treated in the RUBY trial Of EDIT-301 for the treatment of severe sickle cell disease. News release. Editas Medicine. December 6, 2022. https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-announces-positive-safety-and-efficacy-data
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