Displaying drugs 13001 - 13025 of 14386 in total
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
CTx-PDE6b
CTx-PDE6b is an adeno-associated virus (AAV) based gene therapy designed to deliver a full-length non-mutated copy of the functional human PDE6b gene. Being commercialied by Coave Therapeutics, it is being investigated for the treatment of retinitis pigmentosa due to PDE6b gene mutations.
Investigational
MP4CO
Investigational
Skin-derived ABCB5-positive Mesenchymal Stem cells
Skin-derived ABCB5-positive Mesenchymal Stem cells are under investigation in clinical trial NCT04971161 (Allogeneic Abcb5-positive Dermal Mesenchymal Stromal Cells for Treatment of CVU (Phase Iib)).
Investigational
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
GT-AS
GT-AS recombinant adeno-associated virus serotype 9 vector containing the transgene UBE3A encoding for ubiquitin protein ligase E3A/E6-AP.
Investigational
MANF
MANF is a human recombinant mesencephalic, astrocyte-derived neurotrophic factor.
Investigational
Fazirsiran
Investigational
Prademagene zamikeracel
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
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
Bepranemab
Bepranemab is a recombinant humanized Igg4P monoclonal antibody directed against human Tau.
Investigational
Displaying drugs 13001 - 13025 of 14386 in total