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Details:
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.
Lead Product(s): GS-100
Therapeutic Area: Genetic Disease Product Name: GS-100
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Recipient: Andelyn Biosciences
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Partnership March 25, 2024
Details:
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.
Lead Product(s): GS-100
Therapeutic Area: Genetic Disease Product Name: GS-100
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 05, 2024
Details:
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.
Lead Product(s): AAV9 Gene Therapy
Therapeutic Area: Genetic Disease Product Name: GS-100
Highest Development Status: UndisclosedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 22, 2021
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