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Find Clinical Drug Pipeline Developments & Deals by Grace Science
GS-100 is a recombinant, single-stranded AAV9 vector that encodes a full-length version of the human NGLY1 gene. It is being evaluated for the treatment of NGLY1 Deficiency.
The partnership aims to tech transfer and manufacture GS-100, a recombinant AAV9 vector and a suspension process AAV NGLY1 gene therapy for Phase I/II/III clinical trial material for the treatment of NGLY1 Deficiency.
GS-100 is a recombinant, single-stranded AAV9 vector that encodes a full-length version of the human NGLY1 gene. It is being evaluated for the treatment of NGLY1 Deficiency.
Grace Science is advancing GS-100 into a first-in-human clinical trial for NGLY1 deficiency. The viral vector development and clinical manufacturing services for GS-100 will be provided by Thermo Fisher Scientific at its site in Alachua, FL.