Integrated Manufacturing

Learn More

Developing novel gene and cell therapies

Learn More

Fostering next-generation gene therapy delivery

Learn More

Putting advocacy into action

Learn More

Our inspiration

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 Q1 2020. 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