2seventy bio achieves a preclinical $15M milestone from hemophilia A pact

2seventy bio (TSVT) announced that it has achieved a preclinical $15M milestone as part of the joint research and development collaboration focused on an in vivo gene editing treatment for hemophilia A. The milestone is a result of delivering key pre-clinical proof of concept data that the Company plans to present during a virtual Research and Development deep dive on May 19. "Hemophilia A is a lifelong condition that is detected early; the best solution for the condition is to create permanent FVIII production for the lifetime of patients which is why we believe integrating the corrective transgene into the genome is the best approach for treatment," said Philip Gregory, D.Phil., chief scientific officer, 2seventy bio. "We are excited about the continued progress in this program based on our internally developed in vivo gene editing platform. This program represents a natural extension of our gene editing capabilities which are also being applied to our oncology programs, including our bbT369 clinical program for non-Hodgkin lymphoma." The collaboration agreement with Novo Nordisk builds upon the original research collaboration signed between bluebird bio and Novo Nordisk (NVO) in 2019 and expanded between 2seventy bio and Novo Nordisk in 2021. It is focused on identifying a development gene therapy candidate for people with hemophilia A, a genetic bleeding disorder resulting from defective Factor VIII. The collaboration utilizes 2seventy bio’s internally developed in vivo mRNA platform as well as megaTAL technology that has now shown the potential to provide a specific and efficient way to silence, edit, or insert genetic components.