Displaying drugs 15226 - 15250 of 15905 in total
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
NeuroCell-HD
Investigational
DYN-101
DYN-101 is an antisense product candidate designed to reduce the expression of dynamin 2 protein.
Investigational
Matched Description: … DYN-101 is an antisense product candidate designed to reduce the expression of dynamin 2 protein. …
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
RTL-1000
RTL-1000 is a recombinant T-Cell receptor ligand being investigated for the treatment of multiple sclerosis.
Investigational
Recombinant human palmitoyl-protein thioesterase-1
Investigational
SPK-1001
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
NX001
Investigational
PLG-0206
PLG-0206 is a peptide antibiotic under investigation for the treatment of periprosthetic joint infections.
Investigational
PHE885
PHE885 is an investigational, autologous B-cell Maturation Antigen (BCMA)-directed chimeric antigen receptor (CAR)-T cell therapy.
Investigational
Matched Description: … PHE885 is an investigational, autologous B-cell Maturation Antigen (BCMA)-directed chimeric antigen receptor …
Anti-CD7 CAR T-cell therapy
Investigational
OTOF-GT
OTOF-GT is a dual vector AAV gene therapy being investigated for the restoration of hearing loss associated with otoferlin deficiency.
Investigational
SBT101
SBT101 is a non-replicating recombinant adeno-associated virus serotype 9 (AAV9) gene therapy vector containing the human adenosine triphosphate (ATP)-binding cassette (ABC) sub-family D member 1 (ABCD1) gene.
Investigational
MTL-CEBPA
MTL-CEBPA is a SMARTICLES liposomal formulation of MTL-501, a small activating RNA (saRNA) oligonucleotide.
Investigational
ION582
Investigational
AK-OTOF
Investigational
Displaying drugs 15226 - 15250 of 15905 in total