Chimeric antigen receptor (CAR) T cells are astounding. In B-cell cancers, they have been transformative. Yet engineering-wise, CAR T cells are in the equivalent of the Model T era. CAR T-cell engineering has already evolved, with the addition of costimulatory domains, which affect cell expansion and signaling. But once the cells are injected into a patient, there is really no way to affect their behavior. Read More
Hepcidin deficiency in hereditary hemochromatosis (HH) leads to increased absorption of dietary iron and thus iron overload. Rusfertide is a hepcidin mimetic peptide that has shown efficacy at reducing the need for therapeutic maintenance phlebotomy in patients with HH. Researchers aimed to evaluate the benefits of cotreatment with a hepcidin mimetic peptide plus the rusfertide analogue PN-23114 (Protagonist Therapeutics Inc.) at 7.5 mg/kg t.i.w. and phlebotomy in a murine model of HH. Read More
Type I JAK2 inhibitors improve symptoms and outcomes of patients with myeloproliferative neoplasms (MPNs), but mutant allele JAK2 VF remains unchanged with this therapy. Type II JAK2 inhibitors bind the inactive conformation of the kinase domain and reduce the fraction of JAK2 VF mutant allele in vivo, suggesting an improved approach for JAK2 inhibition. Ajax Pharmaceuticals Inc. presented preclinical data on the type II JAK2 inhibitor AJ1-10502 for the treatment of MPNs. Read More
The histone-lysine N-methyltransferase 2A (KMT2A; also known as mixed-lineage leukemia 1 [MLL1])-fusion proteins require direct interaction with the nuclear scaffolding protein menin in order to form menin-KMT2A complex, which plays a key role in the transcription of multiple leukemogenic target genes. On the basis of this, it is hypothesized that blocking the menin-KMT2A interaction by small-molecule inhibitors could be a promising new strategy for the treatment of KMT2A-altered and NPM1-mutant acute myeloid leukemia (AML). Read More
Investigators from Army Medical University reported the discovery and preclinical characterization of a novel cannabinoid receptor 2 (CB2 receptor) agonist, YX-2102, being developed as a potential therapeutic candidate for alleviating lung fibrosis. Read More
Invectys Inc. and CTMC, a joint venture between MD Anderson Cancer Center and National Resilience Inc., have announced FDA clearance of an IND application for a phase I/IIa study of IVS-3001, Invectys' lead engineered human leukocyte antigen A (HLA-G)-targeting chimeric antigen receptor (CAR) T-cell therapy for the treatment of solid tumors. Read More
Hibercell Inc. has disclosed aminopyrimidine derivatives acting as phosphatidylinositol 5-phosphate 4-kinase type II α (PIP4K2A; PIP5K2A) and type II β (PIP4K2B; PIP5K2B) inhibitors reported to be useful for the treatment of cancer, neurodegeneration, inflammation and metabolic diseases. Read More
To date, there are no current orally available compounds that promote bone formation for treating osteoporosis; most treatments act by inhibiting osteoclastic bone resorption, leading to increased bone mineral density and reduced hip fracture rate in a modest way. Analogues of parathyroid hormone (PTH) are the most frequently used bone anabolic agents, which even though effective, they require daily injections. Read More
Glycomimetics Inc. has identified glycosides acting as galectin-3 (LGALS3) inhibitors reported to be useful for the treatment of cancer, inflammation, thrombosis, cardiovascular disorders, transplant rejection, neurodegeneration, fibrosis and liver diseases, among other disorders. Read More
Neurophth Therapeutics Inc. has received FDA clearance of its IND application for the in vivo gene replacement therapy NFS-02, a novel recombinant adeno-associated viral serotype 2 vector (rAAV2) containing a codon-optimized NADH-dehydrogenase subunit 1 (ND1) gene, for the treatment of Leber hereditary optic neuropathy (LHON) associated with ND1 mutation. Read More
Medshine Discovery Inc. has divulged benzospiroheterocyclic derivatives acting as prostaglandin E receptor 4 (PTGER4; EP4 receptor) antagonists reported to be useful for the treatment of cancer. Read More
Ordaōs Bio Inc. has entered into a joint development agreement with Yatiri Bio Inc. to create new therapeutics for two novel targets in acute myeloid leukemia (AML). Read More
Additional early-stage research and drug discovery news in brief, from: Hong Kong Baptist University, Paratek Pharmaceuticals, Revolution Medicines. Read More
