Sheila Mikhail, Founder and CEO of AskBio
Gene therapy pioneer makes it her mission to save lives around the world.
An unwavering belief that gene therapies can cure disease and change the world has been the driving force behind Sheila Mikhail’s journey from attorney to CEO of a $4b biotech company. Sheila co-founded AskBio in 2001 to focus on developing a gene therapy cure for children with Duchenne muscular dystrophy (DMD), a devastating disease that leads to progressive muscle weakness, loss of motor skills and life-threatening complications.
Sheila’s vision led AskBio to become the first company to treat DMD patients using gene therapy. Currently, this drug is being advanced through pivotal clinical trials in the hope that it will keep these children out of wheelchairs. The company’s adeno-associated virus (AAV) technology — protected by over 850 patents held by the company — allows for treatment of a variety of genetic disorders. This technology has been used in two approved drugs: one enables people with inherited blindness to see, while the other is a life-saving treatment for children suffering from spinal muscular atrophy (SMA). Sheila’s teams are also advancing therapeutics for diseases such as Pompe, Parkinson’s and Huntington’s. Through its Viralgen and TAAV contract manufacturing businesses, AskBio has facilitated the advancement of AAV therapeutics developed by other companies.
This unstoppable entrepreneur attributes her success to the company’s culture and commitment to diversity, equity and inclusion. Women represent the majority of AskBio’s global workforce, operating in five countries, and its most senior leadership positions. Its inclusive culture has attracted the field’s leading scientists to join AskBio, which has grown from 80 employees in 2020 to over 600 today.