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            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 II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 03, 2024

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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 II Product Type: Cell and Gene therapy

            Recipient: Andelyn Biosciences

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership March 25, 2024

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            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 II Product 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

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            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: Undisclosed Product 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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