Generation Bio, a preclinical biotech developing gene therapies for rare diseases, filed last Friday with the SEC to raise up to $125 million in an initial public offering.
The company's pipeline includes eight programs for rare and prevalent diseases of the liver and retina, initially prioritizing rare monogenic diseases that have well-established biomarkers and clear clinical and regulatory pathways. Its most advanced liver disease programs are in phenylketonuria and hemophilia A, which are in the preclinical stage of development and being assessed in IND-enabling studies. Its most advanced retina disease programs are in Leber’s Congenital Amaurosis and Stargardt disease, which are in the lead optimization stage and are being assessed in animal models.
The Cambridge, MA-based company was founded in 2016 and plans to list on the Nasdaq under the symbol GBIO. Generation Bio filed confidentially on April 10, 2020. J.P. Morgan, Jefferies, and Cowen are the joint bookrunners on the deal. No pricing terms were disclosed.