Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Lead Product(s) : GS-100
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Grace Science Announces First Patient Treated in Phase 1/2/3 Trial of AAV9 Gene Therapy
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.
Product Name : Undisclosed
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
May 03, 2024
Lead Product(s) : GS-100
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : GS-100
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Recipient : Andelyn Biosciences
Deal Size : Undisclosed
Deal Type : Partnership
Andelyn Biosciences Partners for AAV Gene Therapy Manufacturing for NGLY1 Deficiency
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 NGLY1 Deficiency.
Product Name : Undisclosed
Product Type : Cell and Gene therapy
Upfront Cash : Undisclosed
March 25, 2024
Lead Product(s) : GS-100
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Recipient : Andelyn Biosciences
Deal Size : Undisclosed
Deal Type : Partnership
Lead Product(s) : GS-100
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Grace Science Selected by FDA for START Pilot Program for GS-100 Gene Therapy
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.
Product Name : Undisclosed
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
March 06, 2024
Lead Product(s) : GS-100
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : GS-100
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency
Details : Undisclosed
Product Name : Undisclosed
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
January 10, 2024
Lead Product(s) : GS-100
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : GS-100
Therapeutic Area : Genetic Disease
Study Phase : Undisclosed
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Undisclosed
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
June 22, 2021
Lead Product(s) : GS-100
Therapeutic Area : Genetic Disease
Highest Development Status : Undisclosed
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable