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Find Clinical Drug Pipeline Developments & Deals by Benitec Biopharma

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            Lead Product(s): BB-301

            Therapeutic Area: Genetic Disease Product Name: BB-301

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 23, 2020

            Details:

            The BB-301 Tissue Transduction Study is the first of three studies to be conducted in large animals, with the overall goal of providing data to submit an investigational new drug application to the U.S. Food and Drug Administration, a mandatory step to conduct clinical trials.

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            Lead Product(s): BB-301

            Therapeutic Area: Genetic Disease Product Name: BB-301

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 08, 2020

            Details:

            BB-301 is an internally optimized, AAV-based gene therapy agent that can both silence the expression of mutated, disease-causing genes (to slow, or halt, the underlying mechanism of disease progression) and replace the mutant genes with normal, "wild type" genes.