18
Participants
Start Date
September 1, 2020
Primary Completion Date
January 31, 2026
Study Completion Date
June 30, 2030
4D-310
4D-310 is a novel adeno-associated virus (AAV) gene therapy comprised of two active components: the capsid (4D-C102) and the transgene cassette, which encodes a codon-optimized full length human GLA transgene driven by the CAG promoter. 4D-310 has been engineered so that it cannot replicate (replication incompetent).
Children's Hospital of Pittsburgh of UPMC, Pittsburgh
Lysosomal & Rare Disorders Research & Treatment Center, Inc, Fairfax
Emory University, Atlanta
University of California at San Diego, La Jolla
Lead Sponsor
4D Molecular Therapeutics
INDUSTRY