Gene therapy startup Generation Bio has lost no time raising the resources it needs to take its programs forward into the clinic, closing a $100 million second-round financing just over a month after its $25 million series A.
The Atlas Venture-backed biotech, which launched with the aim of developing genetic therapies that can be titrated to a dose and redosed like a conventional drug—says it will use the proceeds to move its first two candidates through Investigational New Drug (IND) enabling studies.
“We have not selected development candidates at this point,” Generation Bio’s CEO Geoff McDonough—who was formerly at the helm of rare disease specialist Sobi—told FierceBiotech.
The company is developing gene therapies under the GeneWave banner that use closed-ended DNA (ceDNA) rather than viruses to deliver therapeutic proteins, which could sidestep safety issues such as immune reactions.
“We are working towards declaring two liver-directed GeneWave candidates in the next 18 months based on completing additional preclinical work in relevant disease models in mice and in non-human primates,” he added. The company intends to subsequently move these development candidates into pre-IND studies.
“Disease programs we are exploring include intrinsic diseases of the liver such as progressive familial intrahepatic cholestasis, phenylketonuria, and glycogen storage disease 1a,” said McDonough, who added that the biotech is also looking at diseases in which the liver can secrete transgene-derived proteins to address systemic diseases such as lysosomal storage diseases and hemophilia.
2018 will be a critically important year for the Cambridge, Massachusetts-based biotech, according to the CEO, who said the funding round will help it advance earlier stage programs aimed at delivering ceDNA to other tissues such as the eye, the central nervous system and the lungs.
The series B financing was led by Fidelity Management & Research, with Invus, Deerfield Management, Casdin Capital, Foresite Capital and Leerink Partners’ affiliates also taking part.
“We are delighted to have the support of this group of expert, long-term investors who share our vision to create a generation of people living unaffected by genetic disease,” said McDonough.