California Institute for Regenerative Medicine (CIRM) has awarded a $5.8 million translational research grant to a scientist at the University of California, Los Angeles (UCLA) to further support the development of a hematopoietic stem cell (HSC) gene therapy to treat Angelman syndrome, including preparation of a pre-IND package submission to the FDA.

Researchers from Columbia University Irving Medical Center published data from a study that assessed GiGA1, a selective G protein-gated inwardly rectifying potassium channel (GIRK) activator, in physiologically and clinically relevant ex vivo mouse models of tumor-associated and provoked seizures.

Harness Therapeutics Ltd. has raised fresh financing to further develop its technology for upregulating the translation of mRNA into proteins, and in particular to take on a previously undruggable target in Huntington’s disease.

Bioage Labs Inc. has nominated BGE-102 as a development candidate. The orally available, small-molecule NLRP3 inhibitor has high potency and high brain penetration.

Atalanta Therapeutics Inc. has completed a $97 million series B financing to support its investigational RNA interference (RNAi) therapies for KCNT1-related epilepsy and Huntington’s disease toward IND filings this year and through clinical proof of concept.

The way the brain ages is not the same in women and men. A study in mice has observed differences in the expression of the maternal and paternal X chromosomes that could explain variation in brain aging between the sexes and a faster deterioration in some women. Another study has discovered different survival strategies in the microglial cells of females and males. Both studies highlight sex differences that could have implications for several age-related neurological disorders, such as Alzheimer’s or Parkinson’s.

Lifearc has announced a collaboration with Neuropeutics Inc. to develop a new small molecule for the treatment of motor neuron disease (MND), or amyotrophic lateral sclerosis (ALS). Neuropeutics and Lifearc intend to develop a lead candidate therapeutic to prevent and reverse TDP-43 protein aggregation in MND/ALS.