[{"orgOrder":0,"company":"Locanabio","sponsor":"Vida Ventures LLC","pharmaFlowCategory":"D","amount":"$100.0 million","upfrontCash":"Undisclosed","newsHeadline":"Locanabio Announces $100 Million Series B Financing to Advance Portfolio of Novel Rnatargeted Gene Therapies","therapeuticArea":"Neurology","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Neurology","graph2":"Discovery"},{"orgOrder":0,"company":"Locanabio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Locanabio to Present New Preclinical Data Demonstrating Application of CORRECTx\u2122 Platform for Treating Amyotrophic Lateral Sclerosis at American Society of Gene and Cell Therapy 25th Annual Meeting","therapeuticArea":"Neurology","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Neurology","graph2":"Preclinical"}]
Find Clinical Drug Pipeline Developments & Deals by Locanabio
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.
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.