PRME - Prime Medicine Presents First-ever Prime Editing Data in Non-human Primates Demonstrating Highly Efficient Ability of Prime Editors to Precisely Correct Disease-causing Mutation of GSD1b | Benzinga
Up to 83% of Hepatocytes (up to 50% whole liver) Precisely Edited for p.L358fs Mutation Following IV Infusion of Prime Editor in NHPs with No Safety Concerns Observed; Editing Well Above Anticipated Threshold Necessary to Reverse Disease Manifestations
Up to 56% Whole Liver Editing Observed in Humanized GSD1b Mouse Model
No Detectable Off-target Edits Observed Following Comprehensive in vitro Analysis
Data Presented Today in an Oral Presentation at ESGCT 2023
CAMBRIDGE, Mass., Oct. 27, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (NASDAQ:PRME), a biotechnology Company committed to delivering a new class of differentiated, one-time curative genetic therapies, today reported new preclinical data demonstrating the ability of liver-targeted Prime Editors to efficiently and precisely correct one of the most prevalent disease-causing mutations of glycogen storage disease 1b (GSD1b) in non-human primates (NHP) and mouse models. The data were presented today at the European Society of Gene and Cell Therapy (ESGCT) 2023 Congress in Brussels, Belgium.
"The data presented today are highly encouraging, both for patients and caregivers impacted by GSD1b, as well as for Prime Medicine and the field of gene editing," said Jeremy Duffield, M.D., Ph.D., Chief Scientific Officer of Prime Medicine. "These data are the first Prime Editing data in NHPs and provide further proof-of-concept for our Prime Editing approach to potentially address a wide range of diseases, in this case, by targeting a specific gene with a liver-directed LNP. We have designed our Prime Editors for GSD1b to correct the two most prevalent disease-causing mutations of the disease – p.L348fs and p.G339C – and are highly encouraged by the efficient and precise corrections we have observed across in vitro evaluations, in vivo rodent studies and now, NHP studies. Importantly, we continue to observe minimal to no detectable off-target edits with our Prime Editors, providing further confidence in the precision of this technology."
GSD1b is a rare, serious progressive disease that causes impaired glycogen metabolism and affects approximately 1,500 patients. It results from mutations in the glucose-6-phosphate transporter (G6PT), which is encoded by the gene SLC37A4. Deficiencies in this transporter result in hypoglycemia, or low blood glucose levels, which can be fatal if patients do not adhere to a strict dietary regimen, including consuming slow-release glucose and overnight feeding. P.L348fs and p.G339C mutations are known to be the most prevalent disease-causing mutations and are found in approximately 46-52% of the GSD1b patient population. According to scientific literature and Prime Medicine research, correcting SLC37A4 gene mutations in fewer than 10% of liver cells may be sufficient to reverse many manifestations of this disease.
To address the underlying genetic cause of GSD1b, Prime Medicine is advancing Prime Editors that are delivered to the liver by single intravenous infusion and designed to enable ...