Neurogene is looking to make the leap from a clinical biotech to a commercial biopharma with its lead gene therapy and has hired an industry veteran to take the reins.
Christy Shafer becomes the company’s new chief commercial officer, having most recently served as senior vice president and general manager, North America, at Avidity Biosciences, which was later snapped up by Novartis.
She also served as chief commercial officer of Marinus Pharmaceuticals and held several roles at Alexion Pharmaceuticals before that.
She comes on board specifically “to guide our commercial strategy and launch readiness for NGN-401, our gene therapy product for Rett syndrome,” said Rachel McMinn, Ph.D., Neurogene’s founder and CEO, in a statement.
That will be a tough job. Any launch for a smaller biotech with no previous commercial experience is a difficult transition, but Neurogene is working in gene therapy, an area marked by commercial struggles, while its own treatment, NGN-401, has faced serious safety concerns. That includes the death of a patient who received a high dose of NGN-401, which the biotech disclosed in November 2024.
Following that event, Neurogene dropped the high-dose arm of the NGN-401 trial and continued with the lower-dose 1E15-vg arm with permission from the U.S. Food and Drug Administration.
Things now seem to have turned around for the company. In February, the biotech secured a Breakthrough Therapy designation for the treatment, which is currently being tested in the “Embolden” registrational study, with dosing expected to be completed in the second quarter.
McMinn said in the company’s full-year financial report last month that “we believe NGN-401 is increasingly de-risked as we advance toward a BLA submission and potential launch of NGN-401.” The hiring of Christy Shafer will be a key component of that potential launch.
NGN-401 is designed to use an AAV vector to deliver functional copies of the MECP2 gene to patients with Rett syndrome, a rare genetic disease that typically emerges within the first few years of life.
Most Rett syndrome cases involve mutations in MECP2, with symptoms including slowed growth, problems with movement, coordination and communication, and intellectual disabilities.