Long-term brain recordings from four patients with chronic pain have led investigators at the University of California at San Francisco to identify brain signals that could serve as biomarkers for each individual patients’ pain. The study, which was published online in Nature Neuroscience on May 22, 2023, demonstrated that “chronic pain can successfully be tracked, can successfully be predicted, in the real world while patients are ... going about their lives,” lead author Prasad Shirvalkar told reporters at a press conference announcing the findings. Shirvalkar is a neurologist at the University of California at San Francisco. Read More
One of the challenges in designing genetic and cellular strategies is getting the therapy to the right place. This is even more complicated when it comes to the nervous system. The brain is a complex organ that contains the most differentiated and inaccessible cells in human biology. It is an impassable safe, protected by the blood-brain barrier. Read More
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. Read More
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). Read More
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. Read More
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. Read More
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. Read More
An Insilico Medicine IP Ltd. patent describes Egl nine homolog 1 (EGLN1; HPH-2; PHD2) inhibitors reported to be useful for the treatment of ulcerative colitis and Crohn’s disease. Read More
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. Read More
There is a need regarding cardiovascular disease and heart failure for a therapy that reverses the progression of ventricular dysfunction. Previous findings have shown that cardiomyocytes during cardiac dysfunction show an accelerated telomere shortening, thus leading to DNA damage. Read More
Additional early-stage research and drug discovery news in brief, from: Keros Therapeutics, Pieris Pharmaceuticals, Reviva Pharmaceuticals, Revolo Biotherapeutics, Serca Pharmaceuticals, Tune Therapeutics. Read More
