Displaying drugs 626 - 650 of 1496 in total
VY-HTT01
VY-HTT01 is a gene therapy designed to reduce the expression of huntingtin, thereby altering disease progression. VY-HTT01 is comprised of an adeno-associated virus capsid (AAV1) and a proprietary transgene that harnesses the canonical RNA interference pathway to selectively knock down levels of HTT mRNA.
Investigational
BMN-052
Experimental
Withdrawn
Miransertib
Miransertib is under investigation in clinical trial NCT01473095 (Phase 1 Dose Escalation Study of ARQ 092 in Adult Subjects With Advanced Solid Tumors and Recurrent Malignant Lymphoma).
Investigational
Tegoprazan
Tegoprazan (also known as CJ-12420) is a novel therapeutic developed by CJ Healthcare Corp for treating acid-related gastrointestinal diseases.[A234215, A234220] This drug is a potent and high-selective potassium-competitive acid blocker (P-CAB) with a fast onset of action and the ability to control gastric pH for a prolonged period of time.[A234215,...
Investigational
Progerinin
Investigational
Mitoglitazone
Mitoglitazone has been used in trials studying the treatment of Type 2 Diabetes and Alzheimer's Disease.
Investigational
PF-232798
PF-232798 is under investigation in clinical trial NCT01140425 (Study To Evaluate The Effect Of A Multiple Oral Dose Of PF-00232798 On QT Intervals In Healthy Subjects).
Investigational
Sonpiretigene isteparvovec
Sonpiretigene isteparvovec is an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic therapy. Developed by Nanoscope Therapeutics, it is being investigated for vision restoration in advanced retinitis pigmentosa (RP). Sonpiretigene isteparvovec consists of a fusion Adeno-associated virus (AAV) vector with three plasmids encoding opsins with spectrally-separated activation peaks.
Investigational
Apc001PE
Investigational
MORAb-066
MORAb-066 is a humanized antibody directed against tissue factor (TF). It is being investigated in pancreatic cancer.
Investigational
Zanzalintinib
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
VAD-044
VAD-044 is an allosteric AKT inhibitor developed by Vaderis Therapeutics AG. It is being investigated for the treatment of Hereditary Hemorrhagic Telangiectasia.
Investigational
BMN-046
Experimental
Withdrawn
BMN-055
Experimental
Withdrawn
MP-0250
Investigational
Dalotuzumab
Dalotuzumab has been investigated for the treatment of Breast Cancer, Non Small Cell Lung Cancer, and Metastatic Colorectal Cancer.
Investigational
WVE-003
WVE-003 is an allele-selective antisense oligonucleotide that works to selectively reduce the toxic mutant huntingtin (mHTT) protein.
Investigational
Ponezumab
Ponezumab is under investigation in clinical trial NCT00607308 (A Phase I, Single Dose Study Of PF-04360365 In Japanese Patients With Mild To Moderate Alzheimer's Disease).
Investigational
CB-1158
CB-1158 is under investigation in clinical trial NCT03910530 (A Study of INCMGA00012, INCB001158, and the Combination in Japanese Participants With Advanced Solid Tumors).
Investigational
VPO-227
Investigational
Matched Iupac: … -bromofuran-2-yl)-12,14-dimethyl-13,15-dioxo-17-phenyl-8-oxa-1,12,14-triazatetracyclo[8.7.0.0^{2,7}.0^ …
CSIC002
CSIC002 is a small-molecule Sema-3A inhibitor. It is being investigated for the treatment of anterior ischemic optic neuropathy.
Investigational
Adriforant
Adriforant is under investigation in clinical trial NCT00992342 (A Phase 1 Study To Evaluate The Safety And Tolerability Of Different Doses Of PF-03893787 In Healthy Adult Volunteers).
Investigational
Denecimig
Delpacibart is a humanized, bispecific, bivalent monoclonal antibody against coagulation factor 9.
Investigational
Displaying drugs 626 - 650 of 1496 in total