Since the development of the base and prime editing technique by David Liu at the Broad Institute, their applications in biomedicine have continued to grow, reaching 17 clinical trials for base editing and one clinical assay for prime editing. The 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) marked a historic milestone this year by presenting the first case of treatment with base editors of a baby with a deadly metabolic disease.
Chengdu Zeling Biomedical Technology Co. Ltd. has described poly(ADP-ribose) polymerase 1 (PARP-1; ARTD1) inhibitors reported to be useful for the treatment cancer.
Scientists at Jubilant Biosys Ltd. and the University of Texas System have divulged fatty acid binding protein (FABP) inhibitors reported to be useful for the treatment of cancer.
Scientists at F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have identified macrocyclic compounds acting as GTPase KRAS (G12D mutant) and/or (G13D mutant) inhibitors reported to be useful for the treatment of cancer.
Bristol Myers Squibb Co. has disclosed proteolysis targeting chimera (PROTAC) compounds comprising a cereblon (CRBN) E3 ubiquitin ligase-binding moiety covalently bonded to a B-cell lymphoma 6 protein (BCL-6)-targeting moiety through a linker. They are reported to be useful for the treatment of cancer and autoimmune diseases.
It is far easier and safer to inject drugs into veins than directly into the brain, yet it is extremely difficult for systemically delivered drugs to cross the blood-brain barrier and achieve therapeutic concentrations there.
Oncolytic viruses trigger immunogenic cell death in tumors, releasing signals that activate innate immune cells and promote tumor-specific T-cell responses. While some oncolytic viruses, including FDA-approved T-VEC, have shown safety and some clinical benefit, their limited effectiveness as standalone treatments underscores the need for combination therapies.
Age-related macular degeneration (AMD) is the primary cause of blindness in people aged 65 and older in developed nations. Recent estimates suggest that, globally, the number of individuals affected by AMD could rise to approximately 288 million by 2040.
Mucosa-associated lymphoid tissue lymphoma translocation protein 1 (MALT1) is an important factor from a complex downstream of Bruton tyrosine kinase (BTK) in the B-cell receptor (BCR) signaling pathway.
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