Displaying drugs 14851 - 14875 of 15574 in total
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
DYN-101
DYN-101 is an antisense product candidate designed to reduce the expression of dynamin 2 protein.
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
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
BZ371A
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
OTOF-GT
OTOF-GT is a dual vector AAV gene therapy being investigated for the restoration of hearing loss associated with otoferlin deficiency.
Investigational
DYNE-251
DYNE-251 is a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1). It is being investigated for the treatment of Duchenne muscular dystrophy (DMD) in patients who are amenable to exon 51 skipping.
Investigational
DS-2325a
DS-2325a is an inhibitor of kallikrein 5 under development for the treatment of Netherton Syndrome.
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
Displaying drugs 14851 - 14875 of 15574 in total