Ionis Pharmaceuticals Inc. has announced its intention to advance four new wholly owned neurology medicines into the clinic by the end of next year.

The broadest view of post-mortem brains in Alzheimer’s disease (AD) has unveiled the genome, transcriptome and epigenome alterations of this neurodegenerative condition. The coordinated research, directed by scientists at the Massachusetts Institute of Technology (MIT), also described new cellular pathways that could help the scientific community design new therapies. Four simultaneous studies published on Sept. 28, 2023, in Cell, presented a brain single-cell atlas of AD, exposed the damage that affects DNA, and described the processes that alter the microglia and dysregulate the epigenome.

Oryzon Genomics SA has announced two grants that will support the exploration of the role of epigenetic targets in the treatment of neuronal pathologies. Funded by the Spanish State Research Agency and the Ministry of Science and Innovation, the grants cover two public-private collaboration projects.

Gro Biosciences Inc. has presented successful proof-of-concept results in two preclinical programs for autoimmune disease and immunogenicity. The company’s genomically recoded organism (GRO) platform enables precise placement of non-standard amino acids (NSAAs) within a protein.

A new gene editing method uses the CRISPR technique to modify the cells of an organ in vivo, creating a mosaic used to identify the effects of each altered gene. Scientists from the Swiss Federal Institute of Technology (ETH) in Zürich developed this technology called AAV-Perturb-seq, based on adeno-associated virus (AAV) to target, edit and analyze single-cell genetic perturbations.