Among the companies to provide updates at the J.P. Morgan Healthcare Conference in San Francisco was Vigil Neuroscience Inc., which has intrigued Wall Street more since the deal signed in December by Muna Therapeutics ApS, of Copenhagen, Denmark, with London-based GSK plc.

T-cell leukemia homeobox protein 3 (TLX3) is a key regulator of fate specification of excitatory neurons and found expressed in proliferating granule neuron progenitors (GNPs) of the cerebellum, but its main role is not well known.

Kyorin Pharmaceutical Co. Ltd. has entered into an option agreement with Biodol Therapeutics SAS.

Pheno Therapeutics Ltd. has received clinical trial authorization (CTA) from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for its lead candidate, PTD-802. The program will progress to a first-in-human phase I trial.

Data from two out of three positive studies faced the U.S. FDA’s Anesthetic and Analgesic Drug Products Advisory Committee as members examined the package submitted by Seikagaku Corp., of Tokyo, for SI-6603 (condoliase), a chemonucleolytic drug for radicular leg pain associated with lumbar disc hernia.

Roche AG’s disclosure in December that prasinezumab, partnered with Prothena plc, fell short of its primary phase IIb endpoint put the spotlight on microtubule binding region (MTBR)-targeting therapies in neurological disorders.

2024 saw the completion of several cellular-resolution brain maps, including the entire fly brain and a comprehensive connections map of a cubic centimeter of human brain. 2025 began with the addition of another important map. In the Jan. 1, 2025, issue of Nature, researchers from the Allen Institute presented a map of areas and cell types where aging most affected the mouse brain.

Ips Heart Inc. has been awarded orphan drug designation by the FDA for its GIVI-MPC stem cell therapy for Becker muscular dystrophy. GIVI-MPC has the unique ability to create new muscle with full length dystrophin in Becker muscular dystrophy.

Recent work proposes a promising therapeutic approach for dysferlinopathies.