Pepgen Inc. has received a no objection letter from Health Canada for its clinical trial application (CTA) to initiate a phase I study of PGN-EDODM1 in patients with myotonic dystrophy type 1 (DM1). Initial data from the study are expected next year.

The FDA has awarded orphan drug designation to Healx Ltd.’s HLX-1502 for the treatment of neurofibromatosis type 1 (NF1). HLX-1502, discovered through the company’s proprietary artificial intelligence (AI) drug discovery platform, has a first-in-class mechanism and supporting data that indicate the potential for a good safety profile.

Bloomsbury Genetic Therapies Limited has disclosed a new pipeline program – BGT-PD...

Tetra Pharm Technologies Aps has completed preclinical testing and announced promising in vivo study results with its candidate compound, TPT-0301, which targets the endocannabinoid system.

A large-scale genetic study found 26 risk loci for epilepsy, a chronic brain disease with multiple forms, not all of them heritable. The work, by more than 300 authors from the International League Against Epilepsy (ILAE), investigated seven different subtypes of this neurological condition. “There are over 100 genes that we know can harbor mutations that cause epilepsy,” the co-corresponding author Gianpiero Cavalleri told BioWorld. These genes have rare forms that cause that epilepsy. However, “this particular GWAS is focused more on common forms of epilepsy,” he said.

Uniqure NV has received FDA clearance of its IND application for AMT-260, the company’s gene therapy candidate that represents a potential one-time administered approach to treating refractory mesial temporal lobe epilepsy (MTLE).

Researchers from Ashvattha Therapeutics LLC recently reported on ASH-41020, a new dendranib compound acting as a CSF-1R inhibitor with anti-inflammatory and immunomodulatory effects.

Monoacylglycerol lipase (MAGL), a member of the serine hydrolase family expressed in the brain and peripheral tissue, is a key enzyme in the hydrolysis of monoglycerides, converting 2-arachidonoyl glycerol (2-AG) into arachidonic acid and glycerol. MAGL inhibition has been previously shown to induce anxiolytic and analgesic phenotypes in animal models. Researchers from Janssen Pharmaceutica NV recently reported the discovery of novel noncovalent MAGL inhibitors.

A Spanish study led by scientists from the Cajal Institute and the National Center of Oncological Research (CNIO) combined the power of artificial neural networks and biological neuronal circuits to identify abnormal brain activity produced by secondary metastases in the CNS and classify these tumors. The work, published online on Aug. 30, 2023, in Cancer Cell, showed how damage in the brain did not depend on the tumor size but on the effect it produced on neuronal circuits, interrupting cell communication.