Displaying drugs 1326 - 1350 of 1993 in total
PCLX-001
PCLX-001 is a first-in-kind N-Myristoyltransferase (NMT) inhibitor being developed by Pacylex Pharmaceuticals (https://pacylex.com). Current studies have shown that PCLX-001 works differently than other known cancer drugs and has high activity and positive results in breast, lung, bladder and pancreas cancers.
Investigational
GDC-0134
Investigational
WSD-0922
Investigational
ETBX-041
Investigational
STRO-001
Investigational
AL-01211
Investigational
NGGT-002
NGGT-002 is a recombinant adeno-associated virus serotype 8 vector encoding human phenylalanine hydroxylase (PAH).
Investigational
M012
Investigational
M002
Investigational
ADVM-062
ADVM-062 is an adeno-associated virus vector carrying complementary deoxyribonucleic acid encoding the human long-wave-sensitive opsin 1 (AAV.7m8-L-opsin)
Investigational
AVR-RD-04
AVR-RD-04 is an investigational gene therapy for cystinosis. It consists of autologous CD34+ enriched cells transduced with a lentiviral vector containing RNA resulting in codon-optimized cDNA encoding for functional human cystinosin.
Investigational
Marzeptacog alfa (activated)
Marzeptacog alfa (activated) is under investigation in clinical trial NCT01439971 (Phase 1 Safety, Pharmacokinetics And Pharmacodynamics Study Of Recombinant Factor VIIa Variant (813d) In Adult Subjects With Hemophilia).
Investigational
Pegadricase
Pegadricase is under investigation in clinical trial NCT01021241 (Safety and Efficacy Study of Intravenous Uricase-PEG 20).
Investigational
Gavorestat
Gavorestat is under investigation in clinical trial NCT04902781 (Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia).
Investigational
DG041
DG041 is a novel, first-in-class, orally-administered small molecule developmented for the prevention of arterial thrombosis and its complications.
DG041, an anti-platelet compound, has shown to be a selective and potent antagonist of the EP3 receptor for prostaglandins E2. EP3 is a target that associates with increased risk of various vascular...
Investigational
VP025
VP025, a novel drug formulation based on phospholipid microparticles incorporating phosphatidylglycerol, can inhibit neuroinflammation. VP025 may inhibit immune system activation and protect motoneurons from injury. It also shows the ability to reduce inflammation across the blood-brain barrier and improve correlates of memory and learning function. It is being developed to...
Investigational
PRX-08066
Investigational
Sirna-027
Sirna-027 is a chemically modified short interfering RNA(siRNA) targeting Vascular Endothelial Growth Factor Receptor-1(VEGFR-1). VEGFR-1 is a key component of the clinically validated vascular endothelial growth factor (VEGF) pathway. Sirna-027 is developed by Sirna Therapeutics and is under the phase I of clinical trails. It is used to treat Macular...
Investigational
CTCE-0214
CTCE-0214 is a stable peptide agonist of stromal cell-derived factor-1 (SDF-1), a key signaling molecule in the proliferation, homing, engraftment and expansion of hematopoietic stem cells and white blood cells.
Investigational
UK-396,082
UK-396,082 has been used in trials studying the basic science of Safety, Phase 1, Toleration, Multiple Dose, and Pharmacokinetic.
Investigational
NP-G2-044
NP-G2-044 is under investigation in clinical trial NCT03199586 (Phase 1 Clinical Trial of Metastasis Inhibitor NP-G2-044 in Patients With Advanced or Metastatic Solid Tumors (Including Lymphoma)).
Investigational
TRX-E-002-1
Investigational
HL001
Investigational
PBKR03
PBKR03 is a non-replicating recombinant adeno-associated virus serotype hu68 vector, containing a transgene encoding the human galactosylceramidase (GALC) enzyme.
Investigational
Displaying drugs 1326 - 1350 of 1993 in total