The FDA has granted orphan drug designation to Modalis Therapeutics Corp.’s MDL-101, a novel epigenetic editing therapy being developed for the treatment of congenital muscular dystrophy type 1a (LAMA2-CMD), a severe, early-onset muscular dystrophy caused by the absence of the LAMA2 protein.

Scientists from different laboratories around the world have presented the latest advances in research into malignant brain tumors at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), which is being held Oct. 22 to 25 in Rome.

Oblique Therapeutics AB has achieved a milestone within its research project on the Nav1.8 ion channel with the goal of developing innovative therapeutics for patients with chronic pain.

At the ongoing European Society of Gene & Cell Therapy meeting in Rome, Aviadobio Ltd. presented preclinical data for a novel ATXN2-targeting miRNA-containing vector, AVB-205, developed based on previous research that has shown the potential of ATXN2 silencing as a promising therapeutic strategy for amyotrophic lateral sclerosis (ALS) and tau-negative frontotemporal dementia (FTD).

Glutamate oxaloacetate transaminase (GOT) is a glutamate scavenger that has been proposed to be used to counteract the excitotoxicity secondary to stroke or traumatic brain injury, among other pathologies. Furthermore, recent research suggests that mitochondrial GOT protects against energy failure after ischemia.

Spastic paraplegia type 47 (SPG47) is a rare congenital and neurological disorder characterized by dysfunction of the long axons in the corticospinal tract neurons that lead to progressive limb weakness and spasticity.