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Displaying drugs 14976 - 15000 of 15881 in total
Timdarpacept
IMM01 is a recombinant human signal regulatory protein α (SIRPα) IgG 1 fusion protein. IMM01 is under investigation in clinical trial NCT05140811 (A Study of IMM01 Combined With Azacitidine in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndrome).
Investigational
Matched Description: … trial NCT05140811 (A Study of IMM01 Combined With Azacitidine in Patients With Acute Myeloid Leukemia and …
PEG-interleukin-2
Investigational
OCE-205
Investigational
TAK-611
TAK-611, formerly known as SHP611, is a recombinant human arylsulfatase A. First developed by Takeda, it is being investigated for the treatment of metachromatic leukodystrophy.
Investigational
ABX1100
ABX1100 is an investigational Centyrin-siRNA conjugate that targets the Gys1 gene, which encodes glycogen synthase 1 (Gys1), an enzyme that is responsible for glycogen synthesis in the muscle. It was first developed by Aro Biotherapeutics and is being investigated for the treatment of Pompe disease.
Investigational
Matched Description: … It was first developed by Aro Biotherapeutics and is being investigated for the treatment of Pompe disease …
PEG-Glucocerebrosidase
Investigational
rAAV9-rsATP7A
A recombinant adeno-associated viral vector gene therapy under investigation for the treatment of Menkes disease.
Investigational
mRNA-3705
mRNA-3705 is an investigational mRNA encoding methylmalonic CoA mutase (MUT) enzyme. Developed by Moderna TX Inc., it is being investigated for the treatment of methylmalonic academia.
Investigational
Recombinant human alkaline phosphatase
Investigational
LV-FAH
Investigational
BBM-H901
BBM-H901 is a liver-tropic adeno-associated viral (AAV) vector carrying cassette coding hyperactive Padua factor IX (FIX) protein. It is being investigated for the treatment of hemophilia B.
Investigational
Dalcinonacog alfa
Dalcinonacog alfa is a recombinant human factor IX (FIX) variant. It is being investigated for the treatment of hemophilia B.
Investigational
mRNA-3351
mRNA-3351 is a modified mRNA encoding the UGT1A1 gene. Developed by Moderna, it is currently being investigated as the treatment for Crigler-Najjar syndrome
Experimental
OMS906
OMS906 is a humanized Igg4 monoclonal antibody against mannan-binding lectin-associated serine protease-3 (MASP-3) currently being developed by Omeros.
Investigational
MCMV3068A
MCMV3068A is an anticytomegalovirus monoclonal IgG1 antibody used in combination with MCMV5322A to form RG7667.
Investigational
VAC-52150
Investigational
SIG-001
SIG-001 is a cultured human retinal pigment epithelial cell line (ARPE-19) genetically modified with a non-viral vector to express B domain deleted human factor VIII protein, encapsulated within two-layer modified alginate spheres.
Investigational
RST-001
RST-001 is a non-replicating recombinant adeno-associated virus vector containing a fragment of the gene encoding channel rhodopsin-2 protein.
Investigational
GS030-DP
GS030-DP is an optogenetic gene therapy being investigated for the treatment of retinitis pigmentosa. It targets retinal ganglion cells that encodes a light-sensitive channelrhodopsin, ChrimsonR-tdTomato (ChR-tdT), delivered by an AAV2.7m8 vector. It is a component of GS030, an investigational optogenetic treatment, in combination with a medical device GS030-MD.[A259816, L46701]
Investigational
WVE-N531
Investigational
SPL23-2
SPL23-2 is an antisense oligonucleotide (ASO) targeting the W1282X nonsense mutation associated with a severe form of cystic fibrosis.
Investigational
QR-1123
QR-1123 is a first-in-class investigational RNA-based oligonucleotide designed to treat autosomal dominant retinitis pigmentosa due to the P23H mutation in the rhodopsin (RHO) gene.
Investigational
AAV-dXIAP
AAV-dXIAP is a recombinant adeno-associated virus-encoded gene for X-linked mammalian inhibitor of apoptosis protein (XIAP) developed by Neurologix.
Investigational
Exon 55 specific phosphorothioate oligonucleotide
Investigational
Recombinant human galactocerebrosidase
Investigational
Displaying drugs 14976 - 15000 of 15881 in total