Deals, Gene Therapy

J&J snags rights to a gene therapy portfolio from little MeiraGTx, a spinoff from Sam Waksal’s Kadmon

After getting a quick lesson on little MeiraGTx’s gene therapy efforts — including their home made on/off switch — J&J is going all in. And like other deals it’s made recently, J&J’s research organization under Mathai Mammen is demonstrating its enthusiasm for small biotechs that are short on history and long on promise.

The pharma giant followed up on the initial research alliance they signed last fall with the biotech and snapped up commercial rights to a slate of clinical-stage, once-and-done development programs aimed at eye diseases. J&J will pay $100 million in a cash upfront for the deal and is putting another $340 million on the table in milestones.

Alexandria Forbes

J&J is also on the hook now for development costs for the portfolio, which includes candidates for inherited retinal diseases: achromatopsia caused by mutations in either CNGB3 or CNGA3 and X-linked retinitis pigmentosa. Tied in is a pact on collaborating on AAV manufacturing technology, which they will work on together.

J&J is tying itself up to a New York-based company that spun out of Kadmon, Sam Waksal’s comeback biotech after his prison sentence for insider trading — a case that also took celebrity homemaker Martha Stewart down with him. Waksal bowed out just as Kadmon went public, as he was barred from being an officer in a public company again.

J&J created an extensive network of dealmaking offices around the world several years ago, and it’s been paying some top prices to land new clinical-stage drugs to bring into the pipeline. That strategy was on display when J&J snagged the BCMA CAR-T at Shanghai’s Legend, with plans to go up against bluebird for the first-in-class title.

“Our gene regulation platform, based on RNA structure, was invented and developed entirely in-house at MeiraGTx and is a reflection of our commitment to ground-breaking science,” said MeiraGTx CEO Alexandria Forbes, a scientist who took a later spin at a hedge fund before helming her first biotech.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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