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Displaying drugs 2276 - 2300 of 3517 in total
Investigational
Investigational
HMI-102 is an investigational gene therapy designed to encode the phenylalanine-4-hydroxylase (PAH) gene, mutated in people with phenylketonuria.
Investigational
OTL-102 consists of autologous CD34+ enriched cells that contain hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human gp91phox gene. It is being investigated for the treatment of X-linked chronic granulomatous disease.
Investigational
Investigational
Tovinontrine is an orally administered, highly selective phosphodiesterase 9 (PDE9) small molecule inhibitor. Tovinontrine is under investigation in clinical trial NCT04474314 (A Study of IMR-687 in Subjects With Sickle Cell Disease).
Investigational
Matched Description: … Tovinontrine is an orally administered, highly selective phosphodiesterase 9 (PDE9) small molecule inhibitor …
Erteberel is an estrogen receptor beta agonist that has been used in trials studying the treatment of Benign Prostatic Hyperplasia.
Experimental
Investigational
Matched Iupac: … (2R,6S,7R)-7-(4-hydroxyphenyl)-8-oxatricyclo[7.4.0.0^{2,6}]trideca-1(9),10,12-trien-12-ol …
Investigational
Matched Iupac: … N-(3-chloro-4-{3,9-diazatricyclo[8.4.0.0^{3,7}]tetradeca-1(10),4,6,11,13-pentaene-9-carbonyl}phenyl)- …
Efonidipine is a calcium channel blocker of the dihydropyridine class, commercialized by Shionogi & Co. (Japan). Initially, it was marketed in 1995 under the trade name, Landel. The drug has been shown to block T-type in addition to L-type calcium channels [A7844, A32001]. It has also been studied in atherosclerosis...
Experimental
PRTX-100 is a highly-purified form of Staphylococcal Protein A that binds directly to monocytes and a subset of B-cells that are involved in the development and progression of various autoimmune diseases. These binding interactions enable the compound to modulate the function of these cells and restore the balance of the...
Investigational
Rivipansel has been used in trials studying the other, basic science, and treatment of Pain Crisis, Renal impairment, Anemia, Sickle Cell, Sickle Cell Disease, and Sickle Cell Disorders, among others.
Investigational
Experimental
Matched Iupac: … ,4,11-tetraen-9-one ... 10-[(2-fluorophenyl)methyl]-4,7-dimethyl-3-thia-7,10,11-triazatricyclo[6.4.0.0^{2,6}]dodeca-1(8),2(6) …
Experimental
Matched Iupac: … (1S,9S)-9-{[(1S)-1-carboxy-3-phenylpropyl]amino}-10-oxo-octahydro-1H-pyridazino[1,2-a][1,2]diazepine- …
AVXS-301 consists of an adeno-associated virus serotype 9 (AAV9) designed to express the superoxide dismutase 1 (SOD1) gene. It is being investigated for the treatment of Amyotrophic Lateral Sclerosis (ALS).
Investigational
Matched Description: … AVXS-301 consists of an adeno-associated virus serotype 9 (AAV9) designed to express the superoxide dismutase …
Experimental
Investigational
IK-930 is an oral, paralog-selective TEAD inhibitor.
Investigational
TAFA93 is a novel prodrug of the mTOR inhibitor rapamycin which has successfully completed Phase 1 clinical development. mTOR inhibitors are currently used in the prevention of organ rejection in transplantation, the treatment of autoimmune and oncological diseases, and as a component of coated stents for the treatment of coronary...
Investigational
OSI-930 is an orally active inhibitor of two clinically validated targets: c-Kit and the vascular endothelial growth factor receptor-2 (VEGFR-2). OSI-930 is designed to target both cancer cell proliferation and blood vessel growth (angiogenesis) in selected tumors. In preclinical studies, OSI-930 shows broad efficacy in tumor models representative of small...
Investigational
R-95845 is an inhibitor of HIV-1 reverse transcriptase.
Experimental
TAK-901 has been used in trials studying the treatment of Lymphoma, Myelofibrosis, Multiple Myeloma, Myeloid Metaplasia, and Advanced Solid Tumors, among others.
Investigational
SR-9009 is a REV-ERB agonist. SR-9011 has been demonstrated that it is specifically lethal to cancer cells and oncogene-induced senescent cells, including melanocytic naevi, and has no effect on the viability of normal cells or tissues .
Experimental
SR-9011 is a REV-ERB agonist. SR-9011 has been demonstrated that it is specifically lethal to cancer cells and oncogene-induced senescent cells, including melanocytic naevi, and has no effect on the viability of normal cells or tissues .
Experimental
GS-9191 is under investigation in clinical trial NCT00499967 (Safety and Effectiveness Study of an Experimental Topical Ointment (GS-9191) for the Treatment of Genital Warts).
Investigational
Matched Iupac: … 2-methylpropyl (2S)-2-{[({2-[2-amino-6-(cyclopropylamino)-9H-purin-9-yl]ethoxy}methyl)({[(2S)-1-(2-methylpropoxy …
Displaying drugs 2276 - 2300 of 3517 in total