Congruence Therapeutics Inc. has received a research grant of $5 million from The Michael J. Fox Foundation for Parkinson's Research (MJFF) to advance its GCase-targeting small molecules for GBA1 Parkinson’s disease. Mutations of the GBA1 gene, encoding the enzyme GCase, represent the single largest genetic risk factor for Parkinson’s disease.

Current treatments for Alzheimer’s disease have limited effects. While they can slow cognitive decline or alleviate symptoms, they do not reverse this complex neurodegenerative condition caused by multiple factors. Researchers from the Gladstone Institutes and the University of California, San Francisco (UCSF) have screened FDA-approved drugs in search of agents that could potentially modify the disease.

Brain-derived neurotrophic factor (BDNF) is a neurotrophin that supports neuronal growth and survival and plays a critical role in regulating synaptic plasticity and cognitive function. Reduced BDNF levels have been observed in Alzheimer’s disease, Parkinson’s disease and mood disorders, suggesting that agents capable of enhancing BDNF expression may represent promising therapeutic strategies for both neurodegenerative diseases and depression.

The U.S. FDA has granted Precision Biosciences Inc.’s PBGENE-DMD orphan drug designation for the treatment of Duchenne muscular dystrophy (DMD). PBGENE-DMD uses two complementary Arcus nucleases delivered via a one-time administration in a single AAV to excise exons 45-55 of the dystrophin gene in order to restore near full-length dystrophin protein within the body to improve functional outcomes.

Researchers at the University of Minnesota and collaborating institutions have developed a promising stem cell-based therapy for the treatment of muscular dystrophies. The team has successfully created a novel myogenic progenitor cell product called Myopaxon, derived from human-induced pluripotent stem cells (iPSCs).

Parkinson’s disease (PD), the second most prevalent neurodegenerative disorder worldwide, is characterized by the progressive degeneration of dopaminergic neurons in the substantia nigra, leading to a decline in both motor and cognitive functions. Incretin-based therapies, initially designed for the treatment of type 2 diabetes, have demonstrated relevant neuroprotective effects in preclinical models of PD.