Researchers at the University of California at San Diego have used a gene therapy approach to treat pain by specifically suppressing the Nav 1.7 ion channel in the spinal cord, both preventing and reversing pain in several animal models with distinct underlying reasons for pain.

Now that Johnson & Johnson (J&J), through its Janssen Pharmaceuticals unit, has won the FDA’s nod for Ponvory (ponesimod), market factors will decide how the once-daily oral selective sphingosine-1-phosphate receptor 1 modulator fares against several others already approved in the class.

Small-molecule mRNA translation expert Anima Biotech Inc. has landed a significant new preclinical research deal with Takeda Pharmaceutical Co. Ltd., covering as many as six programs for genetically defined neurological diseases. The deal starts with $120 million in up-front and preclinical research milestone payments for Anima, but altogether the two-part collaboration could hold as much as $2.3 billion in clinical and commercial milestone payouts for the company, providing substantial support for its internal pipeline, Anima co-founder and CEO Yochi Slonim told BioWorld.

With the prevalence of central nervous system disorders such as Alzheimer’s disease, multiple sclerosis, epilepsy, Parkinson’s disease and stroke increasing annually, the need for novel therapeutics to treat neurologic and psychiatric disorders has never been greater. Unfortunately, even though there is a significant unmet medical need, because of the high risk and low approval rates of drugs targeting those devastating diseases, in the past decade big pharma companies have been dramatically reducing their R&D spending on CNS disorders, noted Naheed Kurji, president and CEO of Toronto-based Cyclica Inc.

LONDON – The National Health Service (NHS) in the U.K. has agreed to reimburse Zolgensma, the one-off gene therapy treatment for spinal muscular atrophy billed as the most expensive drug in the world, after a confidential deal was struck with Novartis Gene Therapies.

DUBLIN – A novel real-world study methodology that marries digital technology with a citizen science or do-it-yourself approach to “drug” procurement, formulation and administration has found – yet again – that the placebo remains one of the great wonders of the medical world.

Bionaut Labs emerged from five years in stealth mode raising $20 million to develop Bionauts, microrobots designed to deliver therapies to treat brain disorders. The financing will support the company’s therapeutic program in glioma through preclinical development and further research and development in Huntington’s disease.