Forge Biologics Reports Positive Clinical Data on Brain Development and Motor Function from the RESKUE Novel Phase 1/2 Gene Therapy Trial in Patients with Krabbe Disease at the SSIEM Annual Symposium
The data will be presented on August 31, 2022, at the annual symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM) in Freiburg, Germany.
- The data will be presented on August 31, 2022, at the annual symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM) in Freiburg, Germany.
- The first patients data from the RESKUE clinical trial demonstrate that intravenous FBX-101 following UCBT has been safe and well tolerated through Day 180.
- Notably, the data demonstrate an absence of humoral immune response against the vector and significantly increased GALC enzyme activity.
- Through Day 180, the patient exhibited improved motor activity and normal brain development compared to previously reported transplanted patients with Krabbe disease.