C-C Chemokine receptor type 6 (CCR6) antagonists have been reported in a recent Idorsia Pharmaceuticals Ltd. patent as potentially useful for the treatment of cancer, autoimmune disease and inflammatory disorders.
Research at Merck Sharp & Dohme Corp. has led to the development of receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis (ALS) and inflammatory disorders.
Researchers from Applied Genetic Technologies Corp. have reported preclinical data for AGTC-601, a novel AAVrh10-granulin (GRN) gene therapy being developed for the treatment of frontotemporal dementia (FTD) with GRN mutations.
Rabies virus is among the most neurotrophic known virus and it has a fatality rate of almost 100%. There is a need for antibody-induced protection in the central nervous system, since rabies virus can cross the blood-brain barrier but antibodies cannot. Investigators from Auburn University and the Scott-Ritchey Research Center have presented an adeno-associated viral (AAV) vector-based approach for brain encephalitis caused by rabies virus infection.
The U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) and the Gene Therapy Advisory Committee (GTAC) have approved a clinical trial application (CTA) submitted by University College London (UCL) to initiate a phase I/II trial of BGT-OTCD, Bloomsbury Genetic Therapies Ltd.’s liver-targeted AAV-LK03 gene therapy, in pediatric patients with ornithine transcarbamylase deficiency (OTCD).
Researchers from Henan University of Chinese Medicine have reported the discovery and preclinical evaluation of new histone lysine-specific demethylase 1 (LSD1) inhibitors as potential anticancer candidates.
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