Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Lead Product(s) : GNT-0003
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase II
Sponsor : Hansa Biopharma
Deal Size : Not Applicable
Deal Type : Not Applicable
Genethon/Hansa Start Phase 2 Trial Of Imlifidase Pre-Treatment for Crigler-Najjar
Details : GNT-0003 is being developed for crigler-najjar syndrome. The drug candidate is administered through intravenous route. Direct administration of an AAV vector carrying a normal copy of UGT1A1.
Product Name : Undisclosed
Product Type : Cell and Gene therapy
Upfront Cash : Not Applicable
December 03, 2024
Lead Product(s) : GNT-0003
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II
Sponsor : Hansa Biopharma
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : GNT0004
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
First Results Of Gene Therapy GNT0004 for Duchenne Muscular Dystrophy Presented
Details : GNT0004 is an AAV8 vector-based gene therapy containing a shortened version of the DMD gene encoding dystrophin, targeting treatment for patients with Duchenne muscular dystrophy.
Product Name : GNT0004
Product Type : Cell and Gene therapy
Upfront Cash : Not Applicable
April 23, 2024
Lead Product(s) : GNT0004
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : GNT-003
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Product Name : GNT-003
Product Type : Cell and Gene therapy
Upfront Cash : Not Applicable
January 10, 2023
Lead Product(s) : GNT-003
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : GNT0004
Therapeutic Area : Musculoskeletal
Study Phase : Preclinical
Sponsor : Sarepta Therapeutics
Deal Size : Undisclosed
Deal Type : Collaboration
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.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Undisclosed
January 09, 2020
Lead Product(s) : GNT0004
Therapeutic Area : Musculoskeletal
Highest Development Status : Preclinical
Sponsor : Sarepta Therapeutics
Deal Size : Undisclosed
Deal Type : Collaboration
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