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AVR-RD-01
go.drugbank.com/drugs/DB17651InvestigationalAVR-RD-01 is an investigational _ex vivo_ lentiviral gene therapy developed by AVROBIO Inc. … It consists of autologous CD34+ cells that transduced with Lentiviral Vector/alpha-galactosidase A (AGA) encoding for the human AGA complementary deoxyribonucleic acid (cDNA) sequence.
Devafidugene civaparvovec
go.drugbank.com/drugs/DB16790InvestigationalDevafidugene civaparvovec is an investigational _in vivo_ genome editing product. … It uses proprietary zinc finger nucleases (ZFN) that insert a functional copy of the alpha-L-iduronidase (IDUA) enzyme.
NGN-401
go.drugbank.com/drugs/DB18594InvestigationalDeveloped by Neurogene Inc., it is being investigated for the treatment of Rett syndrome. … NGN-401 is a recombinant serotype 9 adeno-associated virus (AAV) encoding an intron encoding a miRNA generating sequence, complementary miRNA binding sites and a full-length human methyl cytosine binding
Synonyms: Recombinant serotype 9 adeno-associated virus (AAV) encoding an intron encoding a miRNA generating sequence, complementary miRNA binding sites and a full-length human methyl cytosine binding protein 2CY-1503
go.drugbank.com/drugs/DB17605InvestigationalIt was investigated in cardiopulmonary bypass during surgical repair of congenital heart lesions.
WNY0824
go.drugbank.com/drugs/DB23606Experimental[A274253] It is a dual inhibitor of PLK-1 and BRET-4, which have both been found to be involved in CRPC via suppression of AR/MYC-mediated transcription. [A274258]
Synonyms: PLK1/BRD4-IN-1CSIC002
go.drugbank.com/drugs/DB18584ExperimentalIt is being investigated for the treatment of anterior ischemic optic neuropathy.
Squalamine
go.drugbank.com/drugs/DB06461InvestigationalCategories: Compounds used in a research, industrial, or household settingCeramide NP
go.drugbank.com/drugs/DB14111InvestigationalCeramide 3 consists of a phytosphingosine backbone N-acylated with a saturated fatty acid (stearic acid) [A27233]. It is widely used as a moisturizer in various cosmetic and personal products. … Ceramide NP is a lipid molecule included in a group of lipid molecules called ceramides. Ceramides are major lipid components in the stratum corneum of the human skin.
Synonyms: Cer(t18:0/18:1n-9), N-oleoylphytosphingosineMivebresib
go.drugbank.com/drugs/DB21300InvestigationalThe usage of the INN stem '-bresib' in the name indicates that Mivebresib is a inhibitor of the bromodomain and extra-terminal motif (BET) family of bromodomain (BRD) protein, antineoplastic. … Mivebresib is under investigation in clinical trial NCT04480086 (Safety and Tolerability Study of Mivebresib Tablet Alone or in Combination With Ruxolitinib Tablet or Navitoclax Tablet in Adult Participants
NE-180
go.drugbank.com/drugs/DB05619ExperimentalNE-180 was under investigation in a clinical trial conducted in the European Union.