2
ALL2
LocanabioYear
2
ALL1
20221
2020DEALS // DEV.
2
ALL1
Deals1
DevelopmentsCountry
2
ALL2
U.S.A2
ALL1
Inapplicable1
Vida Ventures LLCTherapeutic Area
2
ALL2
NeurologyStudy Phase
2
ALL1
Preclinical1
DiscoveryDeal Type
2
ALL1
Inapplicable1
Series B FinancingProduct Type
2
ALL1
Cell and Gene therapy1
UndisclosedDosage Form
2
ALL1
Oral1
UndisclosedLead Product
2
ALL1
C9orf2 -ALS1
UndisclosedTarget
2
ALL1
Hexanucleotide1
UndisclosedLead Product(s) : Undisclosed
Therapeutic Area : Neurology
Study Phase : Preclinical
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
February 05, 2022
Lead Product(s) : Undisclosed
Therapeutic Area : Neurology
Highest Development Status : Preclinical
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : C9orf2 -ALS
Product Type : Cell and Gene therapy
Upfront Cash : Undisclosed
December 14, 2020