Garraway will take over as Roche's next CMO—Chutes & Ladders by Kyle Blankenship Friday, August 23, 2019 Lilly’s Garraway pegged as newest CMO at Roche; Ousted AveXis CSO Kaspar denies wrongdoing in data scandal; Longtime NICE chief Dillon set to exit.
Once interim, now full-time, Siffert becomes new Abeona chief by Ben Adams Tuesday, February 12, 2019 João Siffert, M.D., has been given the permanent CEO job at Abeona Therapeutics after serving as interim chief.
RegenXBio adds another CNS program to gene therapy plans by Phil Taylor Thursday, August 30, 2018 Gene therapy specialist RegenXBio has added another program to its pipeline, going after Batten disease variant CLN2.
Roche scraps €120M SMA drug after hitting ‘many difficulties’ by Nick Paul Taylor Friday, June 1, 2018 The Swiss pharma is walking away from olesoxime after running into “many difficulties in developing” the oral mitochondria-boosting drug.
AveXis posts look at data from pivotal SMA gene therapy trial by Nick Paul Taylor Wednesday, April 25, 2018 Neuromuscular scores improved as expected in the first months of the trial, raising hopes the Novartis-bound gene therapy will deliver positive data.
Biogen pays Ionis $1B to form discovery-stage pact by Nick Paul Taylor Friday, April 20, 2018 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.
Novartis inks $8.7B AveXis buyout to build gene therapy unit by Nick Paul Taylor Monday, April 9, 2018 Newly minted CEO Vas Narasimhan is paying a hefty premium to establish Novartis as a frontrunner in the emerging gene therapy sector.
AveXis steps up gene therapy pursuit of Biogen’s SMA pathfinder by Phil Taylor Tuesday, January 16, 2018 AveXis is expanding trials of its gene therapy for children with spinal muscular atrophy (SMA) as it plays catch-up with Biogen and Ionis.
Chasing Biogen, AveXis posts improved gene therapy data by Nick Paul Taylor Thursday, November 2, 2017 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 SMA gene therapy advances toward pivotal trial by Nick Paul Taylor Friday, March 17, 2017 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.