Undisclosed hemophilia A therapeutic / Generation Bio - LARVOL DELTA

Generation Bio Outlines 2022 Strategic Priorities and Reports Fourth Quarter and Full Year 2021 Financial Results (GlobeNewswire) - "Generation Bio Co...outlined its strategic priorities for 2022 and reported fourth quarter and full year 2021 financial results....Generation Bio plans to continue to optimize the biodistribution profile of its liver ctLNP in NHP to support the selection of a development candidate for its hemophilia A program and to advance additional liver programs."

Preclinical • Genetic Disorders • Hemophilia

Generation Bio Provides Update on Preclinical Studies for Hemophilia A Program (GlobeNewswire) - "Generation Bio Co...today provided an update on factor VIII expression from a series of mouse and companion non-human primate (NHP) studies in hemophilia A, as well as an update on the development of its non-viral genetic medicine platform....In mouse studies, RES-derived ceDNA delivered by ctLNP generated peak mean human factor VIII expression of 205% of normal at 2.0 mg/kg. This is compared to peak mean human factor VIII expression of 23% of normal at 2.0 mg/kg using ceDNA produced by the company’s prior Sf9-based manufacturing process....Additional optimization is needed to translate the improvement in potency and reduction in variability observed in mice to NHP, and to support nomination of a development candidate for the company’s hemophilia A program....The company expects to announce updates from its pipeline programs in 2022 and to provide timing for its first IND submission in the future."

Preclinical • Genetic Disorders • Hemophilia

Generation Bio Announces Plan to Scale Next-Generation Rapid Enzymatic Manufacturing Process Across Portfolio and Provides Pipeline Update (GlobeNewswire) - "Company to transition all portfolio programs to rapid enzymatic synthesis (RES), enabling improved quality, scale and speed of closed-ended DNA manufacturing...IND submission for hemophilia A now planned for 2023; factor VIII expression data with RES material in non-human primates expected year-end 2021."

IND • Preclinical • Genetic Disorders • Hemophilia

Generation Bio Reports Third Quarter 2020 Business Updates and Financial Results (GlobeNewswire) - "Generation Bio Co....today the company reported recent business highlights and third quarter financial results....Selected Anticipated Company Milestones: Development candidate selection for hemophilia A...in 2021; Initiation of IND-enabling studies for hemophilia A...in 2021."

Preclinical • Hemophilia