Generation Bio tees up $125M IPO to push next-gen gene therapies

Another preclinical-stage biotech is taking to the public markets—Generation Bio filed for a $125 million IPO to advance a pair of gene therapies for liver disease and push one of them into the clinic.

The deal comes on the heels of a $110 million venture round tagged to bankroll its lead programs: liver-targeted therapies for hemophilia A and phenylketonuria (PKU). Together, the funds will allow Generation Bio to finish IND-enabling studies for both programs as well as kick off a clinical trial for one, according to a securities filing.

That phase 1 study is still a ways away, though: “[All] of our programs are currently in the early stage of development, including our programs in PKU and hemophilia A, and we have not yet identified a product candidate for any of our programs,” the company said in the filing.

RELATED: Generation Bio grabs a $110M round to ramp up work on next-gen gene therapies

Generation Bio plans to gather enough data to pick its candidates over the course of this year, with IND-enabling studies slated for 2021 and IND applications for 2022.

“We anticipated submitting IND applications for additional programs in 2023 and beyond,” the company said in the filing.

The company’s pipeline includes treatments for diseases of the eye as well as three other liver disease programs, including treatments for Wilson disease and Gaucher disease. Its most advanced retina disease program targets Leber congenital amaurosis 10, or LCA10, a rare type of blindness caused by mutations in the CEP290 gene. That’s the same form of LCA that Editas Medicines and Allergan are aiming at with a CRISPR-based gene editing treatment.

RELATED:  Editas, Allergan kick off long-awaited in vivo CRISPR trial

Generation Bio’s programs are based on its non-viral gene therapy platform that uses lipid nanoparticles to deliver closed-ended DNA (ceDNA) to diseased tissue.

“Our vision is to develop re-dosable, long-lasting gene therapies manufactured at a scale that leaves no patient or family behind,” said Geoff McDonough, M.D., president and CEO of Generation Bio, earlier this year.