ctLNP-siRNA / Generation Bio - LARVOL DELTA

Generation Bio Reports Business Highlights and Fourth Quarter and Full Year 2024 Financial Results (GlobeNewswire) - "Generation Bio has designed a ctLNP that is specifically targeted to T cells to achieve selective delivery of siRNA and knockdown of targets in vivo, overcoming significant historical challenges for the development of siRNA therapeutics in T cells. The company has shown that its T cell-selective LNP containing an unoptimized siRNA achieved efficient delivery and approximately 98% knockdown of the B2M protein in human T cells in both in vitro and mouse studies....Generation Bio plans to announce the target and indication for its lead ctLNP-siRNA program in mid-2025 and submit an investigational new drug (IND) application in the second half of 2026....Cash, cash equivalents and marketable securities were $185.2 million as of December 31, 2024, compared with $264.4 million in cash...The company continues to believe that its cash, cash equivalents and marketable securities will fund its operating expenditures and capital expenditure requirements into the second half of 2027."

Commercial • IND • Pipeline update • Preclinical • Immunology