A therapeutic strategy based on alternative splicing of the MECP2 gene could restore protein levels in Rett syndrome, a neurological disorder caused by mutations in that gene. Scientists at Baylor College of Medicine have successfully tested this approach both in vitro in neurons from Rett patients that produce some functional protein, correcting the altered gene expression and improving neuronal functions, and in vivo in mice.

Ono Pharmaceutical Co. Ltd. has entered into an agreement to expand its drug discovery collaboration agreement with Congruence Therapeutics Inc. for the discovery of novel small-molecule modulators against multiple protein targets in the areas of neurology and immunology.

Cure Rare Disease has entered into a multiyear partnership with the LGMD2L Foundation to develop a gene replacement therapy for anoctamin 5 (ANO5)-related disease, a rare genetic disorder.

Montis Biosciences BV has released preclinical data on its lead program, MB-0109, in support of its development to treat neuro-autoimmune indications.

Rgenta Therapeutics Inc. has announced preclinical data on RGT-0474060, an oral PMS1 inhibitor for Huntington’s disease. RGT-0474060 is an orally bioavailable small molecule designed to inhibit PMS1 RNA and protein expression, to modulate splicing of CAG repeat expansion known to cause Huntington’s disease.

The effects of aging pose an additional challenge for people with HIV due to the neurological and psychological consequences that persist despite antiretroviral therapy. At the Conference on Retroviruses and Opportunistic Infections (CROI) held Feb. 22-25, 2026, in Denver, the scientific community examined how the virus affects the brain, how the reservoir is established in the CNS, and which genetic, immunological or treatment-related factors influence cognitive health.

Slate Medicines Inc. has launched with $130 million in series A financing and a focus on advancing next-generation therapeutics for migraine and other headache disorders.