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Details:
GNT0004 product is an AAV8 (adeno-associated virus) vector-based gene therapy containing a shortened, but functional version of DMD gene (hMD1) encoding dystrophin, the protein deficient in people suffering from Duchenne muscular dystrophy.
Lead Product(s): GNT0004
Therapeutic Area: Genetic Disease Product Name: GNT0004
Highest Development Status: Phase IProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 23, 2024
Details:
GNT-0003 is being developing a gene therapy treatment to treat patients with severe forms of CN syndrome by restoring expression of the UGT1A1 enzyme in the patient’s liver.
Lead Product(s): GNT-003
Therapeutic Area: Rare Diseases and Disorders Product Name: GNT-003
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 January 10, 2023
Details:
CDMO’s capacity to produce AAV materials at large-scale will support Genethon and Sarepta’s project to take micro-dystrophin to clinical trials this year.
Lead Product(s): GNT0004
Therapeutic Area: Musculoskeletal Product Name: Undisclosed
Highest Development Status: PreclinicalProduct Type: Undisclosed
Partner/Sponsor/Collaborator: Sarepta Therapeutics
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration January 09, 2020
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