Integrated Manufacturing
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Pipeline
Our clinical-stage therapies are designed to deliver functional copies of affected genes to cells using gene transfer technologies.
EB-101
Preparing to enter Phase 3 development in Q4 2019. Autologous gene-corrected cell therapy for patients with RDEB.
ABO-102
In Phase 1/2 development. One-time, AAV-based gene therapy for patients with Sanfilippo syndrome type A (MPS IIIA).
ABO-101
In Phase 1/2 development. One-time, AAV-based gene therapy for patients with Sanfilippo syndrome type B (MPS IIIB).