Displaying drugs 15676 - 15700 of 16205 in total
ETBX-021
Investigational
ETBX-061
Investigational
GI-6301
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
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
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
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
AVR-RD-01
AVR-RD-01 is an investigational ex vivo lentiviral gene therapy developed by AVROBIO Inc. It consists of autologous CD34+ cells that transduced with Lentiviral Vector/alpha-galactosidase A (AGA) encoding for the human AGA complementary deoxyribonucleic acid (cDNA) sequence. AVR-RD-01 is being investigated for Fabry Disease.[L45963, L45968]
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
Recombinant human arylsulfatase A
Investigational
Porphobilinogen deaminase, human recombinant
Investigational
Oxabact
Investigational
PRX-ASL
PRX-ASL is an intracellular enzyme replacement therapy designed to replace the missing or defective enzyme in patients with argininosuccinate lyase deficiency.
Investigational
LB-001
LB-001 is a gene editing therapy designed to incorporate a functioning version of the faulty methylmalonyl-COA mutase (MUT) gene into the genome of patients with methylmalonic acidemia.
Investigational
CB-010
CB-010 is a CRISPR-edited allogeneic CAR-T cell therapy targeting CD19. It is being investigated for the treatment of follicular lymphoma.
Investigational
Displaying drugs 15676 - 15700 of 16205 in total