Generation Bio develops a non-viral platform designed to enable re-dosable, long-lasting gene therapies. Its core technologies are a cell-targeted lipid nanoparticle delivery system (ctLNP), a proprietary closed-ended DNA construct (ceDNA), and a high-capacity capsid-free biologics manufacturing process. The platform is intended to enable repeat dosing, treat patients with pre-existing immunity to AAV, deliver large genes, and scale to larger global patient populations. The company is advancing two lead liver-targeted programs for hemophilia A and phenylketonuria (PKU) toward IND-enabling studies and clinical development. Generation Bio is also developing gene therapies for skeletal muscle and eye diseases. The company was founded by Atlas Venture and is headquartered in Cambridge, Mass. Generation Bio develops the proprietary GeneWave™ technology to create genetic medicines that can be titrated to effect and re-dosed to provide durable transgene expression over a patient’s lifetime. The platform is designed to overcome the immunogenicity limitations of viral-vector gene therapies and has demonstrated durable, dose-dependent expression and re-dosing in vivo. The company is advancing first-in-class programs aimed initially at rare liver diseases and is developing approaches to deliver ceDNA to other tissues including the eye, central nervous system and lungs. Proceeds from the recent financing will support IND-enabling studies for its first two therapeutic candidates and advancement of additional programs. Generation Bio was founded in 2016 and was launched by Atlas Venture, and it is headquartered in Cambridge, Mass.

Total raised

$210M

Funding rounds

2

Latest round

Series C

Latest activity

Jan 2020