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Find Novel Drugs for Genetic Disease under Development in UNITED KINGDOM

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            Lead Product(s): OTL-203

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 15, 2020

            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.

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            Lead Product(s): OTL-203

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 29, 2020

            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.

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            Lead Product(s): OTL-102

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 29, 2020

            Details:

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