Keyword: AveXis

João Siffert, M.D., has been given the permanent CEO job at Abeona Therapeutics after serving as interim chief.

Gene therapy specialist RegenXBio has added another program to its pipeline, going after Batten disease variant CLN2.

The Swiss pharma is walking away from olesoxime after running into “many difficulties in developing” the oral mitochondria-boosting drug. 

Neuromuscular scores improved as expected in the first months of the trial, raising hopes the Novartis-bound gene therapy will deliver positive data.

Biogen is paying a $375 million fee and splurging $625 million on Ionis’ stock for a chance to pick up a series of neurological antisense drugs.

Newly minted CEO Vas Narasimhan is paying a hefty premium to establish Novartis as a frontrunner in the emerging gene therapy sector.

AveXis is expanding trials of its gene therapy for children with spinal muscular atrophy (SMA) as it plays catch-up with Biogen and Ionis.

The data show an uptick in the proportion of patients able to sit unassisted and talk, fueling AveXis’ hopes of challenging Biogen’s Spinraza.

AveXis has posted top-line data from a phase 1 trial of its gene therapy against spinal muscular atrophy (SMA). All 15 patients were event-free at 13.6 months of age, a significant improvement over the natural history of the disease.

AveXis was down 6% today as Biogen and partner Ionis got an early present in the form of the first-ever approval for a drug that helps children with spinal muscular atrophy.