Amicus bets on gene therapy with Celenex buy

Sep 20, 2018

NJ-based Amicus agreed to pay $100 million up front to buy privately-held Celenex, giving Amicus a portfolio of 10 experimental gene therapies.

The programs were developed at The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and The Ohio State University — Celenex was a spinoff from Nationwide Children’s Hospital.

The gene therapies are all for lysosomal storage disorders (LSDs), a focus for Amicus. The three lead programs are for Batten disease— a rare and life-threatening disorder.

Amicus could pay another $277 million in downstream payments if all Celenex’s programs successfully advance.

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