Hasil Pencarian (124)
Halaman 1 / 3AAV9/MFSD8 is a gene therapy developed by Neurogene Inc. It is an adeno-associated virus serotype 9 (AAV9) vector with an engineered transgene encoding the human CLN7/MFSD8 gene for expression of active human major facilitator superfamily domain containing 8.…
Abrocitinib is an oral small-molecule inhibitor of Janus kinase 1 (JAK1). Janus kinases are intracellular enzymes involved in transduction pathways that regulate hematopoiesis and immune cell function.[L39774] The Janus kinase (JAK)–signal transducer and acti…
Afamitresgene autoleucel is a melanoma-associated antigen A4 (MAGE-A4)-directed genetically modified autologous T cell immunotherapy product consisting of CD4 and CD8 positive T cells transduced with a self-inactivating lentiviral vector (LV) expressing an af…
Aflibercept is a recombinant protein composed of the binding domains of two human vascular endothelial growth factor (VEGF) receptors, VEGFR1 and VEGFR2, fused with the Fc region of human immunoglobulin gamma 1 (IgG1).[A261130] Structurally, Aflibercept is a …
AMG-553 is an adoptive, anti-FLT3 chimeric antigen receptor T-cell therapy generated through transduction of autologous T-cells with a self-inactivating human immunodeficiency virus-1 based lentiviral vector.
A transdermal gel containing ethinyl estradiol and norelgestromin being investigated by Antares Pharma for use as a female contraceptive.
APR-2020 is a CD34+ cells that have been transduced in vitro with a lentiviral vector carrying a codon-optimized sequence for expressing the wild type RPS19 protein
AT3022, the Altea Therapeutics’s fentanyl citrate transdermal patch designed to provide safe and rapid management of moderate to severe chronic pain.
Atidarsagene autotemcel, sold under the brand name Libmeldy, is a gene therapy comprising genetically modified autologous CD34+ enriched hematopoietic stem and progenitor cells transduced _ex vivo_ using a lentiviral vector encoding the human arylsulfatase A …
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…
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.
AVR-RD-05 is a gene therapy comprising genetically modified autologous stem cells transduced _ex vivo_ with a lentiviral vector encoding the human iduronate-2-sulfatase (IDS) enzyme. It is under investigation for the treatment of Hunter Syndrome (Mucopolysacc…
Axicabtagene ciloleucel is an anti-CD19 chimeric antigen receptor (CAR) T-cell therapy. The drug has a unique mechanism of action, as it utilizes the patient's own T cells, which play a central role in immune response to cancer.[A216163] Once T-cells are coll…
AZD-8330 is a potent, selective, orally active MEK inhibitor that blocks signal transduction pathways implicated in cancer cell proliferation and survival. AZD-8330 has shown tumor suppressive activity in multiple preclinical models of human cancer including …
Azemiglitazone is under investigation in clinical trial NCT01280695 (A Randomized, Double-blind, Comparator- and Placebo-controlled, Multiple-dose Study to Evaluate the Safety, Tolerability and Efficacy of Three Dose Levels of MSDC-0602 in Type 2 Diabetic Pat…
Balovaptan is under investigation in clinical trial NCT01793441 (A Study of RG7314 to Investigate Efficacy and Safety in Individuals With Autism Spectrum Disorders (ASD)).
BEA-17 is a small molecule targeted degrader of the epigenetic enzyme LSD1 and its co-factor CoREST.[L48330] It is under investigation for the treatment of glioblastoma.
Benralizumab is a humanized recombinant monoclonal antibody of the isotype IgG1k immunoglobulin that specifically binds to the alpha chain of the interleukin 5 receptor (IL-5R) expressed on eosinophils and basophils.[A31294] It inhibits the binding of IL-5 as…
Betibeglogene autotemcel is an autologous gene therapy that adds functional copies of the ?-globin gene (?<sup>A-T87Q</sup>-globin) to hematopoietic stem cells in order to treat ?-thalassemia.[L42940] ?-thalassemia is a condition caused by mutations in the ?-…
BNC-210 is under investigation in clinical trial NCT04951076 (A Phase 2b Study of BNC210 Tablet Formulation in Adults With Post-traumatic Stress Disorder (PTSD)).
Brexpiprazole is an atypical antipsychotic and a novel D2 dopamine and serotonin 1A partial agonist called serotonin-dopamine activity modulator (SDAM). It has a high affinity for serotonin, dopamine and alpha (?)-adrenergic receptors.[L46417] Although it is …
Mantle cell lymphoma is a heterogeneous sub-category of non-Hodgkin's lymphoma that can be classified as either an aggressive nodal or an indolent leukemic non-nodal variant. Despite the introduction of Bruton's tyrosine kinase (BTK) inhibitors such as [ibrut…
Calcium plays a vital role in the anatomy, physiology and biochemistry of organisms and of the cell, particularly in signal transduction pathways. The skeleton acts as a major mineral storage site for the element and releases Ca2+ ions into the bloodstream un…
Carindacillin or Carbenicillin isdanyl was an oral penicillin prodrug of [carbenicillin] marketed by Pfizer as Geocillin. It is no longer marketed in the United States.
Clozapine is a tricyclic dibenzodiazepine, classified as an atypical antipsychotic agent.[A256708] Clozapine displays affinity to various neuroreceptors with a particularly low affinity to the dopamine receptors, thus breaking the mold of first-generation ant…
Cobimetinib is an orally active, potent and highly selective small molecule inhibiting mitogen-activated protein kinase kinase 1 (MAP2K1 or MEK1), and central components of the RAS/RAF/MEK/ERK signal transduction pathway. It has been approved in Switzerland a…
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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.
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Dabocemagene autoficel is an _ex vivo_, autologous cell-based gene therapy. It comprises autologous dermal fibroblasts genetically modified to express functional type VII collagen. Developed by Castle Creek Biosciences Inc., dabocemagene autoficel is being in…
Dental pulp is a soft, connective tissue present in the dental crown of both third molar and first premolar teeth. Dental pulp stem cells (DPSCs) exhibit fibro-blast like plasticity and adherent properties. They are harvested through surgical extraction of wi…
Deuruxolitinib is a deuterated form of [ruxolitinib] that selectively inhibits Janus kinases (JAK) JAK1 and JAK2. Deuteration allows the drug to circumvent extensive oxidative metabolism around the cyclopentyl ring, which increases the duration of the pharmac…
In 2016, the global burden of dementia was estimated to be 43.8 million, demonstrating a significant increase from a global prevalence of 20.2 million in 1990.[A182411] Donepezil, also known as Aricept, is a piperidine derivative acetylcholinesterase inhibito…
Efmarodocokin alfa is under investigation in clinical trial NCT04386616 (A Study to Evaluate the Safety and Efficacy of MSTT1041A (Astegolimab) or UTTR1147A in Patients With Severe COVID-19 Pneumonia).
Estradiol is a naturally occurring hormone circulating endogenously in females. It is commercially available in several hormone therapy products for managing conditions associated with reduced estrogen, such as vulvovaginal atrophy and hot flashes. Some avail…
Hemophilia B - also called factor IX deficiency or Christmas disease - is an X-linked genetic disorder resulting in an absence or deficiency of clotting factor IX.[L44176] Clotting factors, including factor IX, are necessary components of the signaling cascad…
Evexomostat is under investigation in clinical trial NCT05570253 (A Study of SDX-7320 in Combination With Eribulin for People With Breast Cancer).
Fasinumab is a human IgG1k monoclonal antibody targeted against nerve growth factor (NGF). It is currently under investigation for the treatment osteoarthritic pain[A241045] and has demonstrated significantly greater pain relief and functional improvement as …
Flibanserin is the first drug to be approved for hypoactive sexual desire disorder (HSDD) in premenopausal women by the FDA in August 2015. It was originally developed as an antidepressant medication by Boehringer Ingelheim, but showed lack of efficacy in tri…
Formebolone, a derivative of androstane, is an anabolic androgenic steroid. It is on the list of substances prohibited by the Word Anti-Doping Agency, and is regularly screened for in athletes.[A14416] It is also classified by the US Drug Enforcement Administ…
Fosdagrocorat has been used in trials studying the treatment and basic science of Rheumatoid Arthritis.
Molybdenum cofactor deficiency (MoCD) is an exceptionally rare autosomal recessive disorder resulting in a deficiency of three molybdenum-dependent enzymes: sulfite oxidase (SOX), xanthine dehydrogenase, and aldehyde oxidase.[A230088] Signs and symptoms begin…
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