Amicus Makes Its Gene Therapy Move, Focusing On CNS Lysosomal Storage Disorders
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.
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While Amicus will transfer the gene therapy portfolio’s R&D risk to Caritas, it retains a financial interest in many of the spinout’s programs. Pompe candidate AT-GAA accepted for FDA review.
The big pharma could not confirm whether AveXis co-founder and CSO Brian Kaspar and his brother, SVP of R&D Allan Kaspar, were fired. Instead, Novartis noted that the withholding of preclinical safety data for Zolgensma was limited to the action of a few employees. NIBR preclinical safety chief takes key role at AveXis
The VC firm known for its new company formation efforts will launch 10-12 biopharma, device and diagnostic ventures with its fifth fund. Also, EpimAb raises a $74m series B round, three more drug developers join the IPO queue and Amicus leads recent follow-on offerings.