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Details:
C9orf72-related Amyotrophic Lateral Sclerosis (ALS) is a genetic motor neuron disorder caused by a mutation in the C9orf72 gene, resulting in hexanucleotide (G4C2 and C4G2) repeat expansions, targets and destroys hexanucleotide repeats.
Lead Product(s): Undisclosed
Therapeutic Area: Neurology Product Name: Undisclosed
Highest Development Status: PreclinicalProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 02, 2022
Details:
Funding supports pre-clinical and clinical development of proprietary RNA-targeting systems delivered via gene therapy for treating genetic diseases such as Huntington’s disease, myotonic dystrophy type 1, genetic ALS, and retinal disease.
Lead Product(s): C9orf2 -ALS
Therapeutic Area: Neurology Product Name: C9orf2 -ALS
Highest Development Status: DiscoveryProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Vida Ventures LLC
Deal Size: $100.0 million Upfront Cash: Undisclosed
Deal Type: Series B Financing December 14, 2020
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