Gene Therapy, Startups, Venture

Hauling in a $100M mega-round, gene therapy startup Generation Bio builds alternative to potentially risky viral vectors

A gene therapy company headed up by bigwigs and backed early on by Atlas has pulled in a mega-round of $100 million. The 2-year-old startup captured investors’ attention by suggesting it knows a better way to do gene transfer than the potentially toxic viral vector strategies of late.

Generation Bio is the Boston/Cambridge upstart, and it’s a company that’s strayed from the pack when it comes to the how of gene therapy. While its rivals in the field are buckling down on viral vehicles like adeno associated viruses (AAV), Generation Bio is taking a different route.

Robert Kotin

Founded in part by ex-NIH, ex-Voyager executive Robert Kotin, whom you may know as one of the world’s top experts on viral transport, the new company centers on a tech they’re calling close-ended DNA (ceDNA). This can move from the cytoplasm of the cell to the nucleus without an AAV carrying it along. Once inside the nucleus, it can create high levels of gene expression capable of addressing a broad range of genetic diseases.

Generation Bio says the hope is that this tech can address the emerging concern revolving around viral vectors. Well-known gene therapy companies like Spark and Solid Biosciences are using AAVs as vectors, a method that, when used in high doses, has recently been called into question by a pioneer in the field, Penn professor James Wilson.

Wilson surprised the gene therapy community in January when he quit the advisor board of Solid and raised alarms about the potential toxicity of high-dose AAV studies.

Generation Bio is capitalizing on the uncertainty clouding the leading tech in the field by portraying their therapies as potentially safer.

Last month, the company’s CEO Geoff McDonough told Endpoints the company would start by using a lipid nanoparticle to deliver ceDNA to the liver, where it will make systemic proteins to fight diseases.

“These therapies are designed to overcome the immunogenicity associated with viral-vector gene therapies, which has historically limited the number of patients that can be treated and prevented re-dosing,” Generation Bio stated in a press release. “Since its founding in 2016, Generation Bio has demonstrated durable, dose-dependent expression and the ability to increase expression in the liver with re-dosing in vivo with the GeneWave platform.”

Investors appear to be intrigued. The $100 million financing was a Series B led by Fidelity Management & Research Company, with participation from Invus, Deerfield Management Company, Casdin Capital, Foresite Capital, and Leerink Partners’ Affiliates.

This round comes less than two months after Generation Bio announced its $25 million Series A backed by Atlas.

The new money will be used to take two therapeutic candidates through IND-enabling studies.

“This will be a critically important year for us as we develop the GeneWave platform and work towards our first development candidates to address rare diseases of the liver,” McDonough said in a statement. “The round will also support the development of approaches to deliver ceDNA to other tissues such as the eye, the central nervous system and the lungs.”


Image: Generation Bio’s president and CEO Geoff McDonough. Generation Bio


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