Amicus joins gene therapy surge with $100M pipeline purchase

John Crowley
Amicus CEO John Crowley (Amicus)

Amicus Therapeutics has paid $100 million to buy Celenex. The takeover gives Amicus a ready-made pipeline of 10 gene therapies, including two that are in clinical trials.

New Jersey’s Amicus has spent the past few years working on drugs that stabilize lysosomal storage disorders (LSDs) such as Fabry disease and Pompe disease, leading to the FDA approval of Galafold. With those drugs now on the market or making their way through the clinic, Amicus has struck a deal to establish itself at the forefront of efforts to cure, not just stabilize, LSDs.

The deal sees Amicus pay $100 million upfront for Celenex, a biotech founded by movie producer Gordon Gray and his wife Kristen to advance gene therapies that may treat their daughters and other people with Batten disease. The Gray’s pursuit of treatments for Batten led them to the Center for Gene Therapy at Nationwide Children’s Hospital in Ohio, which was working on adeno-associated virus (AAV) gene therapies designed to treat the genetic nervous system disorder.


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Two of those gene therapies, which target the CLN6 and CLN3 forms of Batten, are now in the clinic, making them the most advanced assets in Amicus’ acquired pipeline. Further back, Amicus now has a CLN8 Batten program in preclinical development and gene therapies targeting Niemann Pick C, Wolman disease, Tay Sachs and other LSDs at the research stage. All the assets use AAVs.

As the gene therapies advance through development, Amicus will hand over up to $15 million in milestones. A further $262 million is tied to regulatory submission and approval milestones. While that means the value of the deal could swell over time, Amicus expects to pay $75 million, at most, over the next four years.

The financial commitments have bought Amicus a spot in the blossoming gene therapy sector. Having spent years on the sidelines, gene therapy has moved toward the mainstream over the past year on the back of Spark’s approval, Novartis’ big bet on AveXis and a general uptick in dealmaking. 

Shares in Amicus rose 6% in premarket trading following news of the takeover.

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