Displaying drugs 13876 - 13900 of 14939 in total
ETBX-061
Investigational
GI-6301
Investigational
SynOV1.1
SynOV1.1 is an investigational cancer immunotherapy developed by SyngenTech. It is a recombinant oncolytic adenovirus, controlled by synthetic gene circuit, encoding the human immunostimulating factor cytokine granulocyte-macrophage colony-stimulating factor (GM-CSF). SynOV1.1 is under investigation in clinical trial NCT04612504 (Synov1.1 Intratumoral Injection Study).
Investigational
Ligufalimab
Ligufalimab is a humanized immunoglobulin G4 (IgG4) monoclonal antibody targeting leukocyte surface antigen CD47. It is under investigation in clinical trial NCT04900350 (A Trial of AK117 (Anti-cd47) in Patients With Myelodysplastic Syndrome).
Investigational
IO-202
IO-202 is a first-in-class antibody targeting the Leukocyte Immunoglobulin-Like Receptor subfamily B (LILRB) member 4 (LILRB4). It is being investigated in cancers.
Investigational
PF-04856884
PF-04856884 or CVX-060 is a selective angiopoietin (Ang)-2 inhibitor. It is under investigation in clinical trial NCT01441414 (PF-04856884 (CVX-060) in Combination With Axitinib in Patients With Previously Treated Metastatic Renal Cell Carcinoma).
Investigational
SEA-CD70
SEA-CD70 is an investigational humanized, non-fucosylated monoclonal antibody targeting CD70. SEA-CD70 is under investigation in clinical trial NCT04227847 (A Safety Study of SEA-CD70 in Patients With Myeloid Malignancies).
Investigational
Immunocytokine NHS-IL12
Investigational
ADVM-053
Investigational
OTL-103
Investigational
Ixmyelocel-T
Ixmyelocel-T is an expanded, autologous multicellular therapy cultured from bone-marrow mononuclear cells. Developed by Vericel Corporation, it is being investigated for cardiomyopathy.
Investigational
CDX-6210
CDX-6210 is a modified version of a leucine decarboxylase enzyme from Planctomycetaceae bacterium.
Investigational
Labafenogene marselecobac
Investigational
ARO-APOC3
ARO-APOC3 is an investigational RNAi therapeutic targeting apolipoprotein C-III (APOC3). It received an Orphan Drug designation by the FDA for the treatment of familial chylomicronemia syndrome.
Investigational
Tividenofusp alfa
Tividenofusp alfa, also known as DNL-310, is a brain-penetrant enzyme replacement therapy being investigated for the treatment of Hunter syndrome.
Investigational
RGX-111
RGX-111 is an adeno-associated virus vector AAV9 designed to deliver the α-L-iduronidase (IDUA) gene. Developed by REGENXBIO Inc., it was investigated for the treatment of mucopolysaccharidosis Type I (MPS I).
Investigational
AAV2/8.TBG.hARSB
Investigational
JLK-247
JLK-247 is an adeno-associated viral vector serotype 9 (AAV9)-mediated gene replacement therapy for the treatment of Sanfilippo Syndrome Type C or MPS IIIC.
Investigational
RP-A501
RP-A501 is a gene therapy product consisting of a recombinant adeno-associated virus serotype 9 (rAAV9) capsid containing the human LAMP2B transgene (AAV9:LAMP2B), which encodes lysosome-associated membrane protein 2 (LAMP-2). It is being investigated for Danon disease.
Investigational
EGT-301
EGT-301 is a gene therapy being investigated for Hunter Syndrome. It consists of an adeno-associated viral vector serotype 9 (AAV9) containing the human Iduronate-2-sulfatase (I2S) transgene designed to restore I2S functional deficiency in patients with Hunter syndrome.
Investigational
OTL-201
OTL-201 is an investigational hematopoietic stem cell (HSC) gene therapy being developed by Orchard Therapeutics for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA). It consists of a lentiviral vector to insert a functional copy of the human N-Sulfoglucosamine Sulfohydrolase (SGSH) gene into autologous CD34+ hematopoietic stem cells.
Investigational
Reloxiliase
Reloxiliase is a first-in-class, oral crystalline formulation of the oxalate-specific, microbial enzyme oxalate decarboxylase. It is being investigated for the treatment of hyperoxaluria.
Investigational
MRT5201
Investigational
DCR-PH1
Investigational
SYNB1020
SYNB1020 is an engineered strain of Escherichia coli Nissle 1917 developed by Synlogic Inc. It was initially designed to convert ammonia to L-arginine and granted orphan designation by the FDA for the treatment of urea cycle disorders in August 2016; however, the manufacturer discontinued further development of SYNB1020 to treat...
Investigational
Matched Description: … It was initially designed to convert ammonia to L-arginine [A258838] and granted orphan designation by …
Displaying drugs 13876 - 13900 of 14939 in total