Neurogene has hired an industry veteran to take the reins as it looks to make the leap from clinical biotechnology to commercial biopharmaceuticals with leading gene therapies.
Christie Schaefer is the company’s new chief commercial officer, most recently serving as senior vice president and general manager for North America at Avidity Biosciences, which was later acquired by Novartis.
She also served as Chief Commercial Officer of Marinus Pharmaceuticals and previously held several positions at Alexion Pharmaceuticals.
Neurogene founder and CEO Dr. Rachel McMinn said in a statement that she joined specifically to “guide our commercial strategy and prepare for the launch of NGN-401, our gene therapy product for Rett syndrome.”
It’s going to be a lot of work. It’s a difficult transition for any small-scale biotech with no prior commercial experience to come to market, but Neurogene is tackling an area of gene therapy that has been marked by commercial struggles, while its own therapy, NGN-401, faces serious safety concerns. This includes the death of a patient who received a high dose of NGN-401 about 18 months ago, which the biotech company disclosed last May.
As a result of this incident, Neurogene discontinued the high-dose arm of the NGN-401 study and continued the lower-dose 1E15-vg arm with approval from the U.S. Food and Drug Administration.
Things seem to be improving for the company now. The biotech company secured Breakthrough Therapy designation for the treatment in February, and it is currently being tested in the Enbolden registration study, with dosing expected to be completed in the second quarter.
“We believe NGN-401 is increasingly de-risked as we move toward the BLA filing and potential launch of NGN-401,” McMinn said in the company’s full-year financial report last month. The hiring of Christy Schaefer will be a key element of that potential launch.
NGN-401 is designed to use an AAV vector to deliver a functional copy of the MECP2 gene to patients with Rett syndrome. Rett syndrome is a rare genetic disorder that usually develops within the first few years of life.
Most cases of Rett syndrome involve mutations in MECP2, and symptoms include growth retardation, motor, coordination, and communication problems, and intellectual disability.
