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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

In Phase 3 development. 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).

Manufacturing at The Elisa Linton Center for Rare Disease Therapies