[{"orgOrder":0,"company":"Genethon","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Musculoskeletal","country":"FRANCE","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"GNT0004","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Genethon \/ Sarepta Therapeutics","highestDevelopmentStatusID":"6","companyTruncated":"Genethon \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Genethon","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"GNT-003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Genethon \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Genethon \/ Not Applicable"},{"orgOrder":0,"company":"Genethon","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"GNT0004","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Genethon \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Genethon \/ Not Applicable"},{"orgOrder":0,"company":"Genethon","sponsor":"Hansa Biopharma","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"GNT-0003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Genethon \/ Hansa Biopharma","highestDevelopmentStatusID":"8","companyTruncated":"Genethon \/ Hansa Biopharma"}]

Find Clinical Drug Pipeline Developments & Deals by Genethon

Menu
Xls
Filters Filter
×
FILTER:
filter
Company Name
    filter

    Year

      filter

      DEALS // DEV.

        filter

        Country

          filter
          Sponsor
            filter

            Therapeutic Area

              filter

              Study Phase

                filter

                Deal Type

                  filter

                  Product Type

                    filter

                    Dosage Form

                      filter

                      Lead Product

                        filter

                        Target

                          Loading...

                          Therapeutic Area by Lead Product

                          Study Phase by Lead Product

                          Company by Lead Product

                          Top Deals by Deal Size (USD bn)

                          01

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          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

                          blank

                          02

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          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

                          blank

                          03

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          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

                          blank

                          04

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          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

                          blank