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Amicus Makes Its Gene Therapy Move, Focusing On CNS Lysosomal Storage Disorders

 
• By Jessica Merrill

The rare disease specialist gained 10 gene therapy programs with the acquisition of Celenex, including two in clinical-stage development for Batten disease, in exchange for $100m up front. President Bradley Campbell talked to Scrip about the process that led to the deal and next steps for development.

A strand of DNA with the letters or word within it to illustrate genetics, heredity and medical research

Amicus Therapeutics Inc. is poised to become a leader in gene therapy, with a focus on neurological lysosomal storage disorders (LSDs), via the acquisition of Celenex. The company announced the deal Sept. 20, which will add 10 gene therapy programs to the company's pipeline, including two in clinical-stage development for Batten disease, in exchange for $100m up front and milestones.

Privately held Celenex is progressing gene therapy programs developed from The Center for Gene Therapy at the Research Institute at Nationwide

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