Drug Pipeline
34 drugs associated with Sickle cell disease
| Drug | Phase | Sponsor | |
|---|---|---|---|
Anumigilimab Granulocyte colony stimulating factor receptor antagonist | Phase 2 | CSL Behring | ↗ |
BIVV003 | Phase 2 | Sangamo Therapeutics | ↗ |
briquilimab Stem cell growth factor receptor inhibitor | Phase 2 | National Heart, Lung, and Blood Institute (NHLBI) | ↗ |
Busulfan Busulfan is an alkylating agent that contains 2 labile methanesulfonate groups attached to opposite ends of a 4-carbon alkyl chain. Once busulfan is hydrolyzed, the methanesulfonate groups are released and carbonium ions are produced. These carbonium ions alkylate DNA, which results in the interference of DNA replication and RNA transcription, ultimately leading to the disruption of nucleic acid function. Specifically, its mechanism of action through alkylation produces guanine-adenine intrastra | Phase 2 | Sangamo Therapeutics | ↗ |
EDIT-301 | Phase 2 | Editas Medicine, Inc. | ↗ |
Filgrastim (G-CSF) Granulocyte colony stimulating factor receptor agonist | Phase 2 | National Heart, Lung, and Blood Institute (NHLBI) | ↗ |
L-glutamine Supplemental L-glutamine's possible immunomodulatory role may be accounted for in a number of ways. L-glutamine appears to play a major role in protecting the integrity of the gastrointestinal tract and, in particular, the large intestine. During catabolic states, the integrity of the intestinal mucosa may be compromised with consequent increased intestinal permeability and translocation of Gram-negative bacteria from the large intestine into the body. The demand for L-glutamine by the intestine | Phase 2 | Emmaus Medical, Inc. | ↗ |
Plerixafor Plerixafor inhibits the C-X-C chemokine receptor type 4 (CXCR4) on CD34+ cells and reversibly blocks the binding of its ligand, stromal cell-derived factor-1-alpha (SDF-1α). SDF-1α and CXCR4 play a role in the trafficking and homing of human hematopoietic stem cells (HSCs) to the marrow compartment. In the marrow, stem cell CXCR4 can help anchor HSCs to the marrow matrix, either directly via SDF-1α or through the induction of other adhesion molecules. By blocking the interaction between SDF-1α a | Phase 2 | National Heart, Lung, and Blood Institute (NHLBI) | ↗ |
Chloroquine Chloroquine inhibits the action of heme polymerase in malarial trophozoites, preventing the conversion of heme to hemazoin. _Plasmodium_ species continue to accumulate toxic heme, killing the parasite. Chloroquine passively diffuses through cell membranes and into endosomes, lysosomes, and Golgi vesicles; where it becomes protonated, trapping the chloroquine in the organelle and raising the surrounding pH. The raised pH in endosomes, prevent virus particles from utilizing their activity for fus | Phase 3 | London School of Hygiene and Tropical Medicine | ↗ |
Granulocyte colony-stimulating factor Somatic cell supplemental therapy | Phase 3 | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | ↗ |
Hydromorphone Hydromorphone is an opioid agonist that can bind to different types of opioid receptors. Its analgesic effect is suggested to be related to the effect on the mu-opioid receptors. It has been reported to also have a minor affinity for the delta and kappa receptor. On the other hand, it is known to act at the level of the medulla which allows it to depress the respiratory drive and suppress cough. The onset of action of the immediate release form of hydromorphone is achieved in 15-20 minutes and | Phase 3 | Duke University | ↗ |
Morphine Morphine-6-glucuronide is responsible for approximately 85% of the response observed by morphine administration. Morphine and its metabolites act as agonists of the mu and kappa opioid receptors. The mu-opioid receptor is integral to morphine's effects on the ventral tegmental area of the brain. Morphine's activation of the reward pathway is mediated by agonism of the delta-opioid receptor in the nucleus accumbens, while modification of the respiratory system and addiction disorder are mediated | Phase 3 | Duke University | ↗ |
crizanlizumab Crizanlizumab-tmca is a humanized IgG2 kappa monoclonal antibody that binds to P-selectin and blocks interactions with its ligands, including P-selectin glycoprotein ligand 1 (PSGL-1). Crizanlizumab can also dissociate preformed P-selectin/PSGL-1 complex. Binding P-selectin on the surface of the activated endothelium and platelets blocks interactions between endothelial cells, platelets, red blood cells, and leukocytes. | unknown | Novartis Pharmaceuticals | ↗ |
Single blood transfusion | unknown | Carelon Research | ↗ |