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Lead Product(s): Miglustat,Trehalose API
Therapeutic Area: Genetic Disease Product Name: BBDF 101
Highest Development Status: Preclinical Product Type: Small molecule
Partner/Sponsor/Collaborator: Beyond Batten Disease Foundation
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration March 02, 2021
Details:
Janssen will provide drug supply for the clinical trials and Extended Access Program, and right of reference to data within the Zavesca New Drug Application (NDA) to support the development of BBDF-101.
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Lead Product(s): Branaplam
Therapeutic Area: Genetic Disease Product Name: LMI070
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 October 21, 2020
Details:
In preclinical models, branaplam has been shown to reduce levels of mutant huntingtin protein. In addition, during the investigation of branaplam in spinal muscular atrophy (SMA), it was also observed to reduce huntingtin messenger RNA (mRNA) in SMA patients.
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Lead Product(s): VS-01
Therapeutic Area: Genetic Disease Product Name: VS-01
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 October 19, 2020
Details:
VS-01 is an innovative liposomal-based detoxification therapy that acts as a clearance enhancer for a large spectrum of toxic metabolites accumulated during liver and kidney failures.
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Lead Product(s): APR-OD031
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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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