Researchers from Denali Therapeutics Inc. have identified new functional links between progranulin, lysosomal function, and a subtype of frontotemporal dementia caused by progranulin deficiency (FTD-GRN) that suggest progranulin-mediated FTD could be conceptualized as a lysosomal storage disorder (LSD). They also showed that delivery of their experimental therapeutic PTV:PGRN, also known as DNL-593, reduced cell damage and symptoms of FTD in cell and animal models.

Following new positive top-line results from its pivotal phase III study of IPX-203, Amneal Pharmaceuticals Inc. said an NDA submission for the Parkinson’s disease therapy (PD) is now planned for the middle of 2022.

In one of the biggest collaboration deals of the year, Shape Therapeutics Inc. entered a collaboration and license agreement with Roche Holding AG to develop gene therapies for targets in areas that include Alzheimer’s disease, Parkinson’s disease and rare diseases. Seattle-based Shape is eligible to receive an initial payment, development, regulatory and sales milestone payments that could exceed $3 billion in aggregate value.

For most people, neither polyglutamine disorders nor neuromuscular disorders are likely to be among the things they associate with androgen receptor (AR) dysfunction. But the three are indeed linked. And researchers have reported new insights into the nature of those links that could lead to a treatment for spinal and bulbar muscular atrophy, and possibly other disorders linked to AR signaling dysfunction.

LONDON – Persica Ltd. is poised to take a new pharmaceutical approach to curing chronic lower back pain, after delivering positive data in a phase Ib study of an injectable antibiotic. The development rests on research showing that, as with stomach ulcers, chronic lower back pain is caused by localized bacterial infection.

Vigil Neuroscience Inc. CEO Ivana Magovčević-Liebisch said the just-raised $90 million in series B money will let the company reach “a lot of value-generating milestones,” having been “moving extremely fast” in the year since it was founded.

DUBLIN – Beckley Psytech Ltd. raised $80 million in an oversubscribed series B round to move its early stage pipeline of psychedelic therapies into the clinic and to continue research on novel compounds with enhanced properties.

With generic competition on the horizon for its older sleep disorder drug Xyrem, Jazz Pharmaceuticals plc gained an added indication for its GABA B receptor agonist Xywav as the FDA approved it for idiopathic hypersomnia.

Researchers have shown that glucocorticoids, a type of steroid hormones, target both neuroplasticity-related genes and genes related to ciliary function in the brain. However, the effects on the different processes are mediated via different receptors, and in response to different stimuli. A study investigated the specific targets of glucocorticoids, giving new insights into the biological mechanisms of stress adaptations, and how they are linked to neural plasticity.