Business
Metagenomi Reports Business Updates and Third Quarter 2024 Financial Results
Initiating IND-enabling activities for MGX-001 development candidate (DC) in hemophilia A; Achieved durable Factor VIII activity levels over twelve months in
About this update from Metagenomi Therapeutics, Inc.
[{"type":"text","content":"Initiating IND-enabling activities for MGX-001 development candidate (DC) in hemophilia A; Achieved durable Factor VIII activity levels over twelve months in nonhuman primate (NHP) study On track for one to two DC nominations in 2025 from wave 1 Ionis collaboration programs focusing on cardiometabolic indications Novel adenine base editor (ABE) and ultra small SMART editing platform demonstrated highly efficient and precise editing and wide targetability Well capitalized with $274.6 million in cash, cash equivalents and available-for-sale marketable securities at the end of Q3 2024; cash runway anticipated to support operating plans into 2027 EMERYVILLE, Calif., Nov. 13, 2024 (GLOBE NEWSWIRE) -- Metagenomi, Inc. (Nasdaq: MGX) (Metagenomi), a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary gene editing toolbox, today provided a business update and reported third quarter 2024 financial results. “Our strong pace of innovation and execution continued in the third quarter, highlighted by the nomination of our first DC, MGX-001, intended as a one-time curative gene editing therapeutic for both adults and children with hemophilia A,” said Brian C. Thomas, PhD, CEO and founder of Metagenomi. “Supporting this DC nomination, we achieved successful proof-of-concept in NHPs, demonstrating site specific integration and durable Factor VIII activity levels through 12 months. This preclinical study provided an important validation of our gene editing platform, supporting our potential to overcome a key limitation of gene therapies that have struggled to achieve long term persistence of Factor VIII expression in patients. Pre-clinical data from our hemophilia A program has been accepted for oral presentation at the American Society of Hematology meeting in December and we remain on track to file an IND for MGX-001 in 2026.” “Metagenomi’s collaboration with Ionis is progressing, with one to two planned DC nominations in 2025, focused on cardiometabolic development programs, where gene editing could represent a transformative option for patients. Our gene editing toolbox continues to differentiate on multiple fronts. We recently unveiled our novel ABE platform which we believe has superior targetability, precision and efficiency. Additionally, our ultra small SMART editing system...