Genome & Co. Ltd. has reported preclinical findings of its anti-CNTN4 antibody, GENA-104A16, and anti-APP antibody, 5A7 — stressing the contactin-4 (CNTN4) and amyloid precursor protein (APP) axis as a potential target for immuno-oncology. In the latest murine experiments, investigators led by Genome executives and researchers of Gwangju Institute of Science and Technology (GIST) found that blocking the interaction between CNTN4 and APP promoted cancer-destroying responses in mice, suggesting the pathway as a target for immunotherapy.

The Muscular Dystrophy Association (MDA)’s Kickstart program has announced receipt of both U.S. orphan drug and rare pediatric disease designations in support of work for congenital myasthenic syndrome caused by choline acetyltransferase (CHAT) gene deficiency. The FDA awarded the orphan drug designation to AVCHAT-01X (AAV serotype 9 human choline acetyltransferase).

Privately held Dyno Therapeutics Inc. has added another notch to its adeno-associated virus (AAV) vectors development portfolio in a deal with the Roche Group that includes $50 million up front and ultimately could top $1 billion. Dyno will help in developing next-generation AAV vectors, optimized by artificial intelligence, to target neurological diseases.

The serotonin 5-HT2B receptor is widely expressed in the brain, including on serotoninergic, GABAergic and dopaminergic neurons, on astrocytes and microglia, and neuronal fibers of the frontal cortex and cerebellum.

The FDA has granted orphan drug designation to Modalis Therapeutics Corp.’s MDL-101, a novel epigenetic editing therapy being developed for the treatment of congenital muscular dystrophy type 1a (LAMA2-CMD), a severe, early-onset muscular dystrophy caused by the absence of the LAMA2 protein.