A male patient who harbored a deletion in the intron 7-exon 8 region of the dystrophin gene, DMD, was showing symptoms that matched with Becker muscular dystrophy.

Researchers from Alumis Inc. have presented preclinical data for a novel, potential first-in-class tyrosine kinase 2 (TYK2) inhibitor – A-005.

Semaphorin 3A signaling, through the plexin A1/neuropilin 1 (PLXA1/NRP1) receptor complex, is known to disrupt the differentiation and migration of oligodendrocyte precursor cells and mature remyelinating oligodendrocytes. Both semaphorin 3A and plexin A1 are up-regulated in the central nervous system of patients with multiple sclerosis.

Coave Therapeutics SA’s CTx-TFEB program has received support through a grant from the ALS Association.

San Diego-based Kenai Therapeutics Inc. raised $82 million in a series A round to move its disease-modifying cell therapy for Parkinson’s disease into the clinic. The company, which leverages induced pluripotent stem cell (iPSC) technology, will advance its next-generation allogeneic neuron replacement cell therapies for neurological diseases, specifically completing a clinical proof-of-concept trial for its lead candidate, RNDP-001. The series A was co-led by Alaska Permanent Fund Corp., Cure Ventures and The Column Group, with participation from Euclidean Capital and Saisei Ventures. Proceeds will enable Kenai to submit an IND for RNDP-001 and bring it through the completion of phase I trials, which are expected to begin sometime in 2024.