Mavrix Bio has received IND clearance from the FDA for MVX-220, an investigational AAV gene therapy for the treatment of Angelman syndrome. The company expects to initiate its first-in-human study, ASCEND-AS, in the second half of this year.

Capsida Biotherapeutics Inc. has gained IND clearance from the FDA for CAP-002, its first-in-class, intravenously administered gene therapy for syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE). Dosing in the phase I/IIa SYNRGY trial will begin in the third quarter of this year.

The U.K. National Institute for Health and Care Excellence (NICE) endorsed the use of the Orbit system by Mindtech Ltd. as a treatment for tics and Tourette syndrome.

Individuals with cystic fibrosis show a notably higher rate of attention deficit hyperactivity disorder (ADHD) symptoms than the general population. This association points to a possible role of the cystic fibrosis transmembrane conductance regulator (CFTR) gene, whose mutations cause cystic fibrosis, in the development of ADHD.

Ahead Therapeutics SL has received positive feedback from the EMA on its way toward initiating regulatory toxicology studies for its lead program in myasthenia gravis. The feedback supports the company’s scientific approach.

The recent 510(k) clearance by the U.S. FDA for a core part of Precision Neuroscience Corp. brain-computer interface technology is certainly a boon for the company, and others developing the devices. For the millions of people suffering from health disorders, such as motor neuron disease, spinal cord injury or severe stroke, its sign that a solution which could transform their lives could be just a few years away.