Neuroblastoma is a pediatric extracranial solid tumor arising from the sympathetic nervous system. Disialoganglioside GD2-based therapies, including CAR T cells and other immunotherapies, have shown some success. However, GD2 is also expressed on pain fibers and other neurons, raising safety concerns, and relapses after anti-GD2 therapy are frequent.
Glioblastomas (GBMs) are the most prevalent and highly lethal primary brain tumors and currently rely on limited treatment options. Chimeric antigen receptor (CAR) T cells targeting cell-surface antigens have shown promise in GBM patients, inducing transient tumor regression. However, the lack of known tumor-restricted antigens in GBM limits further improvement of their therapeutic efficacy.
Lynk Pharmaceuticals Co. Ltd. has received IND clearance from the FDA for LNK-01006, an allosteric TYK2 inhibitor with potential to treat neurodegenerative diseases.
In a study published in Molecular Therapy, researchers from H. Lee Moffitt Cancer Center and Research Institute and the University of South Florida developed an E6-targeting proteolysis targeting chimera (PROTAC) that inhibits the growth of human papillomavirus (HPV)-positive tumors. HPV is the most prevalent sexually transmitted infection worldwide, with persistent infections causing up to six different types of cancer.
Vectory Therapeutics BV has obtained IND clearance from the FDA for a phase I/II trial (PIONEER-ALS) of VTX-002, a first-in-class vectorized antibody targeting TDP-43 pathology in amyotrophic lateral sclerosis (ALS). VTX-002 delivers an engineered antibody designed to selectively target toxic species of TDP-43.
Latus Bio Inc. has reported IND clearance by the FDA for LTS-101, a gene therapy candidate to treat the CNS manifestations of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease. The FDA has also granted orphan drug, rare pediatric disease and fast track designations to LTS-101.
Abion Inc. has presented data for ABN-202, a hyperglycosylated interferon (IFN) β mutein fused to a monoclonal antibody targeting tumor-associated calcium signal transducer 2 (TROP2) that retains activity under acidic conditions.
CCNK is frequently upregulated in diverse tumor types, where its increased expression promotes proliferation and its depletion triggers apoptosis. Researchers from Guangdong Pharmaceutical University reported the discovery and preclinical characterization of a novel series of CCNK molecular glue degraders with antitumor activity.
Australian researchers have found a drug combination that can bypass the cellular defenses in neuroblastoma that lead to relapse, and the discovery could lead to better treatment strategies for children whose cancers have stopped responding to standard chemotherapy.
Alebund Pharmaceuticals (Hong Kong) Ltd. has described complement factor B (CFB) inhibitors reported to be useful for the treatment of glomerular disorders and rheumatoid arthritis.
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