Displaying drugs 15001 - 15025 of 15902 in total
Recombinant botulinum A/B vaccine
Investigational
Ultevursen
Ultevursen is a single-stranded RNA-based oligonucleotide targeting exon 13 of the USH2A gene that encodes usherin. It was developed by ProQR Therapeutics and is being investigated for the treatment of retinitis pigmentosa.
Investigational
Matched Description: … It was developed by ProQR Therapeutics and is being investigated for the treatment of retinitis pigmentosa …
AGA2115
AGA2115 is a humanized bispecific antibody neutralizing both sclerostin and Dickkopf-1. Developed by Angitia Biopharmaceuticals, AGA2115 is being investigated for the treatment of osteogenesis imperfect.
Investigational
Matched Description: … AGA2115 is a humanized bispecific antibody neutralizing both sclerostin and Dickkopf-1. …
OAV201
OAV201 is a non-replicating, recombinant adeno-associated virus serotype 9 (AAV9) containing the human Methyl CpG Binding Protein 2B (MECP2B) cDNA under the control of a segment of the murine MECP2 promoter. It was first developed by Novartis and investigated for the treatment of Rett syndrome, but further investigation in clinical...
Investigational
Matched Description: … It was first developed by Novartis and investigated for the treatment of Rett syndrome, but further investigation …
AMT-080
AMT-080 is an investigational gene therapy being investigated for the treatment of Duchenne muscular dystrophy.
Investigational
tgAAVCF
tgAAVCF is an adeno-associated virus (AAV) vector encoding the human cystic fibrosis transmembrane conductance regulator protein (CFTR) cDNA. It is being investigated for the treatment of cystic fibrosis.[A259821, A259826]
Investigational
TAK-754
TAK-754 is an adeno-associated virus serotype 8 vector (AAV8) expressing B domain deleted codon optimized factor VIII. Developed by Takeda, it is being investigated for the treatment of hemophilia A.
Investigational
Baliforsen
Investigational
Bifidobacterium Longum Infantis 35624
Bifidobacterium Longum Infantis 35624 is a probiotic being investigated for the treatment of gastrointestinal disorders, such as Crohn's disease and irritable bowel syndrome.[A259872, A259877]
Investigational
Matched Description: … probiotic being investigated for the treatment of gastrointestinal disorders, such as Crohn's disease and …
Bacteroides thetaiotaomicron
Bacteroides thetaiotaomicron is a live biotherapeutic. Developed by 4D Pharma, Thetanix, an oral formulation comprising lyophilized Bacteroides thetaiotaomicron, is being investigated for the treatment of Crohn’s disease.
Investigational
MANF
MANF is a human recombinant mesencephalic, astrocyte-derived neurotrophic factor.
Investigational
Exon 45 specific phosphorothioate oligonucleotide
Investigational
Exon 52 specific phosphorothioate oligonucleotide
Investigational
SAR422459
SAR422459 is a Lentiviral Vector Containing The Human Abca4 Gene that is currently being investigated to treat Stargardt disease.
Investigational
SAR421869
SAR421869 is a Lentiviral Vector Containing The Human My07A Gene currently being investigated for the treatment of retinitis pigmentosa associated with Usher syndrome 1B gene defect.
Investigational
Sorghum
Investigational
NeuroCell-HD
Investigational
AMT-162
AMT-162 is an adeno-associated virus serotype rh10 encoding artificial anti-SOD1 miRNA.
Investigational
TTAX01
TTAX01 is a cryopreserved human umbilical cord product being developed by TissueTech.
Investigational
ALMB-0166
ALMB-0166 is a umanized connexin 43 (Cx43) monoclonal antibody being investigated for the treatment of acute spinal cord injury. It was granted FDA orphan designation in November 2018.
Investigational
Suvodirsen
Suvodirsen is an antisense oligonucleotide that has been investigated for the treatment of Duchenne muscular dystrophy.
Investigational
Recombinant human palmitoyl-protein thioesterase-1
Investigational
PLX-PAD
Investigational
AMB-301
AMB-301 is an AAV gene therapy delivering E-selectin. It is under investigation for the treatment of Buerger disease.
Investigational
Displaying drugs 15001 - 15025 of 15902 in total