Metagenomi reports Q3 EPS (51c), consensus (78c)

Cash, cash equivalents, and available-for-sale marketable securities were $274.6M as of September 30…”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 (MGX). “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 systems showed compatibility with single-AAV delivery, potentially unlocking novel in vivo extrahepatic therapeutic development opportunities in the neuromuscular space….Metagenomi continues to advance its wholly-owned lead program in hemophilia A, designed as a one-time curative treatment for both adults and children. Preclinical data utilizing a cynomolgus Factor VIII construct showed site-specific integration and durable FVIII activity levels in NHPs over 12 months and the study remains ongoing.