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Find Clinical Drug Pipeline Developments & Deals by AskBio

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            Lead Product(s): MMA-101,ImmTOR

            Therapeutic Area: Genetic Disease Product Name: MMA-101

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

            Partner/Sponsor/Collaborator: Selecta Biosciences

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 19, 2020

            Details:

            MMA-101 is an AAV-based gene therapy in development for the treatment of isolated methylmalonic acidemia due to methylmalonyl-CoA mutase gene mutations. AskBio and Selecta expect to start a Phase 1 trial of MMA-101 and ImmTOR for patients with MMA in the first half of 2021.

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            Lead Product(s): AAV2/8LSPhGAA

            Therapeutic Area: Genetic Disease Product Name: AAV2/8LSPhGAA

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Bayer HealthCare

            Deal Size: $4,000.0 million Upfront Cash: $2,000.0 million

            Deal Type: Acquisition October 26, 2020

            Details:

            AskBio's development portfolio includes investigational pre-clinical and clinical stage candidates for the treatment of neuromuscular, central nervous system, cardiovascular and metabolic diseases.

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            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Recipient: University of North Carolina

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 21, 2020

            Details:

            The study shows that a CRISPR-Cas9-based gene therapy designed to increase the production of ubiquitin protein ligase E3A (UBE3A) — the enzyme lacking in Angelman syndrome — partially or completely corrected physical and behavioral deficits in a mouse model of the disease.

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            Lead Product(s): MMA-101,ImmTOR

            Therapeutic Area: Genetic Disease Product Name: MMA-101

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

            Recipient: Selecta Biosciences

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 20, 2020

            Details:

            U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to MMA-101 for the treatment of isolated methylmalonic acidemia (MMA) due to methylmalonyl-CoA mutase (MMUT) gene mutations.

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            Lead Product(s): AAV2/8LSPhGAA

            Therapeutic Area: Genetic Disease Product Name: AAV2/8LSPhGAA

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Scottish Enterprise

            Deal Size: $2.5 million Upfront Cash: Undisclosed

            Deal Type: Funding September 02, 2020

            Details:

            This grant has been awarded to AskBio Europe to develop solutions to these industry-wide challenges. AskBio team will further enhance its Pro10™ platform, a simplified and cost-effective AAV manufacturing process that can be scaled and applied throughout the AskBio group.

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            Lead Product(s): AAV gene therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Recipient: University of North Carolina

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement May 26, 2020

            Details:

            Collaboration builds upon preclinical research that demonstrates potential for gene therapy to correct deficiency in UBE3A gene.

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            Lead Product(s): Gene therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Recipient: University of North Carolina

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement March 18, 2020

            Details:

            Collaboration builds upon preclinical research that demonstrates potential for gene therapy to correct deficiency in UBE3A gene .

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            Lead Product(s): NAN-101

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: Undisclosed

            Highest Development Status: Phase I Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 04, 2020

            Details:

            AskBio dosed first patient in a Phase 1 clinical trial of NAN-101. The aim of the study is to identify potential therapeutic targets to restore heart function.