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Find Drugs for Genetic Disease in Preclinical Development

A collaboration with SCORR Marketing

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            Lead Product(s): BV-CYP01

            Therapeutic Area: Genetic Disease

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: AskBio

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Acquisition April 22, 2020

            Details:

            The acquisition adds BrainVectis's Huntington’s disease drug candidate BV-CYP01 to AskBio’s program.

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            Lead Product(s): ARCT-810

            Therapeutic Area: Genetic Disease

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 13, 2020

            Details:

            Two clinical trials for company's ARCT-810, being developed to treat ornithine transcarbamylase (OTC) deficiency have been approved by U.S FDA, and New Zealand Medicines, and Medsafe.

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            Lead Product(s): APR-OD031

            Therapeutic Area: Genetic Disease

            Highest Development Status: Preclinical Product Type: Undisclosed

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 26, 2020

            Details:

            First mix of amino acids having a pharmacological primary mode of action designed to reduce and control Phenylalanine fluctuations.

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            Lead Product(s): PR001

            Therapeutic Area: Genetic Disease

            Highest Development Status: Preclinical Product Type: Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 12, 2020

            Details:

            The Company also announced that the FDA has granted Rare Pediatric Disease Designation for PR001 for the treatment of neuronopathic Gaucher disease (nGD), the most severe form of the condition.

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            Lead Product(s): ETX001

            Therapeutic Area: Genetic Disease

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 08, 2020

            Details:

            Paper demonstrates first in class TMEM16A chloride channel potentiators, to accelerate mucociliary clearance.