Genetic Disease | Preclinical Developments

BrainVectis

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FRANCE France

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AskBio Acquires BrainVectis to Expand its Clinical Pipeline for Neurodegenerative Diseases

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.

Arcturus Therapeutics

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United States

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Arcturus Therapeutics Announces Allowance of IND & Approval of Clinical Trial Application (CTA) for ARCT-810

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.

APR Applied Pharma Research

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Switzerland

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FDA Grants Orphan Drug Designation to APR-OD031 for the Treatment of Phenylketonuria (PKU)

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.

Prevail Therapeutics

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United States

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Prevail Therapeutics’ PR001 Gets Orphan Drug Designation By FDA

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.