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Solid Bio Adds Friedreich Ataxia to Pipeline With Aavanti Acquisition

Solid has also pivoted from developing SGT-001 to SGT-003 for treating Duchene muscular dystrophy.

Wet AMD Gene Therapy Reduces Anti-VEGF Treatment Burden

Participants in the AAVIATE trial had up to an 85% decrease in treatment burden 6 months after treatment with RGX-314.

Advancing Neonatal Cell Therapy Research

Newborn Cell Therapies Group at Monash University has received a $50,000 grant from Lions Cord Blood foundation.

Allogeneic Anti-CD7 CAR-T Demonstrates Promising Results in Hematological Malignancies

Among the 11 patients evaluated for efficacy, 9 patients (81.8%) had objective responses at 28 days.

Omidubicel Improves Survival in Hematologic Malignancies With HSCT

Disease-free survival was 56% in 105 participants over 3 years.

BioMarin Resubmits BLA for Hemophilia A Gene Therapy Val-Rox

A prior resubmission in June 2022 was delayed with requests for additional durability and safety data.