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GS030-DP
go.drugbank.com/drugs/DB17847InvestigationalIt is a component of GS030, an investigational optogenetic treatment, in combination with a medical device GS030-MD.[A259816, L46701] … GS030-DP is an optogenetic gene therapy being investigated for the treatment of retinitis pigmentosa.
AAV1-FS344
go.drugbank.com/drugs/DB17855InvestigationalAAV1-FS344 is an investigational gene therapy developed by Milo Biotechnology to deliver follistatin, a potent myostatin inhibitor. … It is being investigated for the treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and inclusion body myositis.[L46742, L46747, L46752]
Soraprazan
go.drugbank.com/drugs/DB17856ExperimentalSynonyms: (7r,8r,9r)-7-(2-methoxyethoxy)-2,3-dimethyl-9-phenyl-7,8,9,10-tetrahydroimidazo(1,2-h)(1,7)naphtyridin-8-olCategories: Heterocyclic Compounds, 1-Ring, Heterocyclic Compounds, 2-RingSAR-439459
go.drugbank.com/drugs/DB17864InvestigationalSAR-439459 is a pan-transforming Growth Factor-beta (TGFβ) neutralizing antibody.[A259862] Developed by Sanofi, it is being investigated for the treatment of cancers and Osteogenesis Imperfecta.
QRX-411
go.drugbank.com/drugs/DB17868ExperimentalIt addresses the underlying cause of Usher syndrome due to the c.7595-2144A>G mutation in the _USH2A_ gene.[L46777] … QRX-411 is a first-in-class RNA-based oligonucleotide designed to restore wild-type USH2A mRNA, leading to the production of the functional USH2A protein.
MANF
go.drugbank.com/drugs/DB17885ExperimentalMANF is a human recombinant mesencephalic, astrocyte-derived neurotrophic factor.
GIVI-MPC
go.drugbank.com/drugs/DB17905ExperimentalGIVI-MPC is a therapy that uses human-induced pluripotent stem cell-derived myogenic progenitor cells.
DYN-101
go.drugbank.com/drugs/DB17921InvestigationalDYN-101 is an antisense product candidate designed to reduce the expression of dynamin 2 protein.
Synonyms: 5’-cEtG-sp-cEt5MeU-sp-cEt5MeU-sp-dT-sp-dA-sp-dT-sp-dT-spdA-sp-dT-sp-dA-sp-dG-sp-dG-sp-dG-sp-cEt5MeC-sp-cEt5MeU-sp-cEt5MeU-3’AMT-162
go.drugbank.com/drugs/DB17922InvestigationalAMT-162 is an adeno-associated virus serotype rh10 encoding artificial anti-SOD1 miRNA.
Debamestrocel
go.drugbank.com/drugs/DB17934InvestigationalDebamestrocel is under clinical development by BrainStorm Cell Therapeutics and is being investigated for the treatment of Amyotrophic Lateral Sclerosis (ALS).[L47266]