Working together to deliver cell and gene therapies for people impacted by serious diseases

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Our inspiration

Pipeline

pz-cel (prademagene zamikeracel)

BLA submitted for investigational autologous, COL7A1 gene-corrected epidermal sheets for RDEB.

ABO-50X

Exploring the potential of ABO-50X for the treatment of genetic eye disorders.

Manufacturing at The Elisa Linton Center for Rare Disease Therapies