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Find Novel Drugs for Genetic Disease under Development in UNITED KINGDOM

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            Lead Product(s): Cell-penetrating peptides conjugates

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Peptide

            Partner/Sponsor/Collaborator: RA Capital Management

            Deal Size: $45.0 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing December 09, 2020

            Details:

            PepGen will use the funding to advance into the clinic their next-generation cell-penetrating peptides conjugated to phosphorodiamidate morpholino oligomers (PPMOs) that are designed to correct genetic defects in diseases with high unmet medical need.

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            Lead Product(s): OTL-203

            Therapeutic Area: Genetic Disease Product Name: OTL-203

            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 September 28, 2020

            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 in Milan, Italy.

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            Lead Product(s): OTL-203

            Therapeutic Area: Genetic Disease Product Name: OTL-203

            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 September 01, 2020

            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.

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            Lead Product(s): OTL-203

            Therapeutic Area: Genetic Disease Product Name: OTL-203

            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 August 24, 2020

            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’.

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            Lead Product(s): OTL-203

            Therapeutic Area: Genetic Disease Product Name: OTL-203

            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 July 20, 2020

            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 type I (MPS-I).