The lack of animal models that mimic human disease impedes the study of many pathologies that still lack treatment beyond symptom relief. This is what has happened so far with PURA syndrome, a rare disorder affecting brain development for which a mouse model has finally been developed. Other times, small and large models exist, but an effective treatment remains elusive, as is the case with Krabbe disease, a fatal disease in children that could be prevented with the advances in gene therapy.

Sanofi SA made good on its plan to bear down on M&A by agreeing to buy Vigil Neuroscience Inc. for $8 per share (NASDAQ:VIGL). Included in the transaction is a non-tradeable contingent value right entitling the holder to potentially collect $2 per share more in cash, payable following the first commercial sale of the phase II-ready VG-3927, a small-molecule triggering receptor expressed on myeloid cells 2 (TREM2) antagonist for Alzheimer’s disease, if achieved within a specific period. Watertown, Mass.-based Vigil’s stock closed May 22 at $7.88, up $5.57, or 241%.

A collaboration of scientists from the NIH Brain Initiative consortium has published eight simultaneous studies in Neuron, Cell, Cell Genomics, Cell Reports and Cell Reports Methods, with the results of the Armamentarium project, a new set of gene therapy tools for the research and treatment of human brain disorders. The methodology, based on genetic techniques, RNA detection, genomic enhancers and viral vectors, is designed to access different CNS cell types, neuronal and non-neuronal cells, with common and reproducible protocols now available for any laboratory.

Orionis Biosciences Inc. is sticking with Genentech Inc. in a second deal to discover small-molecule monovalent glue therapies for treating cancer. Privately held Orionis is getting $105 million up front and could earn more than $2 billion in R&D, development, commercial and net sales milestones, plus royalties. The multiyear collaboration calls for Orionis to handle discovery and optimization of molecular glues, with Genentech in charge of later-stage preclinical and clinical development, regulatory filing and commercialization of any small molecules the partnership produces.

Glioblastoma (GBM) is an aggressive cancer from the CNS usually characterized by a very bad prognosis. It is known that around 30%-35% of patients with GBM develop epilepsy as a comorbidity of the disease.

Being able to detect and monitor the aggregation of α-synuclein in situ could lead to more objective, earlier diagnosis of Parkinson’s disease as well as allow real-time monitoring of whether patients are responding to treatment.