Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Lead Product(s) : Undisclosed
Therapeutic Area : Neurology
Study Phase : Preclinical
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : Undisclosed
Molecule Type : Large molecule
Upfront Cash : Not Applicable
May 02, 2022
Lead Product(s) : Undisclosed
Therapeutic Area : Neurology
Highest Development Status : Preclinical
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : C9orf2 -ALS
Molecule Type : Cell and Gene therapy
Upfront Cash : Undisclosed
December 14, 2020
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