[{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-102","moa":"Gene","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"1","companyTruncated":"Orchard Therapeutics \/ Inapplicable"}]

Find Novel Drugs for Genetic Disease under Development in UNITED KINGDOM

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

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          Not Confirmed

                          Details : EMA has granted Priority Medicines designation to OTL-203, an investigational ex vivo autologous hematopoietic stem cell gene therapy in development for the treatment of mucopolysaccharidosis type I at the San Raffaele Telethon Institute for Gene Therapy...

                          Product Name : OTL-203

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          September 28, 2020

                          Lead Product(s) : OTL-203

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          02

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          Not Confirmed

                          Details : New interim data from OTL-203, an investigational gene therapy for the treatment of mucopolysaccharidosis type I (MPS-I), will be shared as part of an invited oral presentation titled ‘Gene Therapy in Leucodystrophies and Other Metabolic Disorders’.

                          Product Name : OTL-203

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          August 24, 2020

                          Lead Product(s) : OTL-203

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          03

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          Not Confirmed

                          Details : Orchard Therapeutics has received both orphan drug designation and rare pediatric disease designation from the U.S FDA for OTL-203, an ex vivo autologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis...

                          Product Name : OTL-203

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          July 20, 2020

                          Lead Product(s) : OTL-203

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          04

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          Not Confirmed

                          Details : MPS-I clinical trial interim results demonstrated- all patients, treated with OTL-203, achieving hematological recovery in less than 45 days – a primary outcome measure – as well as supraphysiological levels of IDUA enzyme in the blood.

                          Product Name : OTL-203

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          May 15, 2020

                          Lead Product(s) : OTL-203

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          05

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          Not Confirmed

                          Details : Accepted abstracts include data on investigational gene therapies for neurometabolic conditions, including updated interim data from proof-of-concept trial in OTL-203 for Mucopolysaccharidosis Type I.

                          Product Name : OTL-203

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          April 29, 2020

                          Lead Product(s) : OTL-203

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          06

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          Not Confirmed

                          Details : Early clinical data support ex vivo hematopoietic stem cell gene therapy as a potentially promising treatment option for X-CGD.

                          Product Name : OTL-102

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          January 29, 2020

                          Lead Product(s) : OTL-102

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          07

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          Not Confirmed

                          Details : New preliminary analyses of radiological outcome measures suggest that treatment with OTL-203 leads to stabilization or improvement in disease-related neurological abnormalities, as measured by brain and spine MRI.

                          Product Name : OTL-203

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          January 09, 2020

                          Lead Product(s) : OTL-203

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Undisclosed

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank