AskBio’s Sheila Mikhail Wins National Entrepreneurial Award

Sheila Mikhail, co-founder and chief executive officer of Asklepios BioPharmaceutical (AskBio), a gene therapy pioneer that was acquired by Bayer AG last year for $4 billion, has been named national Entrepreneur of the Year by Ernst & Young, the accounting and business services firm.

An independent panel of judges selected Mikhail from 222 regional winners representing 185 companies in diverse industries across the United States.

“Sheila has proven herself to be a savvy entrepreneur, and her creativity, grit and agility has led AskBio on an incredible journey,” said Andrew Jordan, EY Americas Entrepreneur of The Year director. “Her dedication to advancing diversity and inclusion, both within her business and respective industry, along with her unwavering commitment to advancing lifesaving cures across the globe, makes Sheila an outstanding addition to our Entrepreneur of the Year community.” 

Candidates for the award were evaluated for their entrepreneurial leadership, talent management, financial performance, societal impact, degree of difficulty in navigating and overcoming obstacles to make their companies successful, and originality, among other contributions and attributes.

Mikhail, previously a life sciences attorney, was responsible for increasing AskBio’s employee base from three to more than 350 employees operating globally, significantly growing the company’s revenues, and positioning the company for a blockbuster acquisition by Bayer AG, the Germany-based healthcare and agriculture conglomerate, for $2 billion up front and up to $2 billion more in potential milestone payments.  

As a Mexican American, Mikhail has emphasized making diversity and inclusion a priority at AskBio. Today, women comprise 41 percent of the leadership roles in the company’s therapeutics business, which is significantly higher than their counterparts in the biotechnology industry. 

Along with AskBio’s other founders, she has worked to provide equitable and affordable access to gene therapy for children with ultra-rare genetic diseases, funding the nonprofit Columbus Children’s Foundation to help achieve that goal. 

‘A very hard road’

Founded in 2001, AskBio is a clinical-stage company headquartered in Research Triangle Park. It is developing gene therapies for rare genetic childhood and adult-onset diseases such as Parkinson’s disease, multiple system atrophy, Pompe disease, heart failure and Duchenne muscular dystrophy. 

AskBio was an early developer of adeno-associated virus (AAV) technology for delivering therapeutic genetic material into living tissues, potentially curing disease. 

“It was a very hard road,” Mikhail said in her acceptance remarks at a Nov. 13 awards gala during EY’s Strategic Growth Forum in Palm Springs, Calif. “It took us over 15 years to get our first round of funding, and literally for many years people thought gene therapy was not a reality, so it was just incredibly difficult to bring therapeutics to patients with devastating disease.”

She and her colleagues pressed onward “because we knew that there were no options for many patients, many of them who were children, so we were just singularly motivated to break down the barriers and bring life-changing drugs to patients desperately in need.” 

Mikhail credited Jude Samulski, Ph.D., one of AskBio’s two other co-founders and the first scientist to clone AAVs for therapeutic use, for his vision and perseverance.

“Almost 20 years ago when we started on this road together, he told me that he couldn’t pay me,” she said. “And indeed he didn’t for 15 years, but he said we would change the world, and I think we have done that.”

Mikhail also thanked AskBio’s employees, whose “dedication to patients and to changing the boundaries of science has really made transformative discoveries,” and the patients and families who supported the company in its early years.

“When Wall Street didn’t believe in gene therapy, they were willing to take the risk,” she said. “They put their own money into our company so we could advance drugs. They even had things like bake sales. It’s because of the bravery of the patients and the families that the field has advanced, and I hope that we can continue to bring life-changing drugs to them.”

Biotech Center board member

Mikhail serves on the board of directors of the North Carolina Biotechnology Center, which previously supported early work by Samulski and AskBio with grants and loans totaling about $1 million. 

The support included a $250,000 faculty recruitment grant that helped attract Samulski to the University of North Carolina at Chapel Hill from the University of Pittsburgh in 1993. In addition to serving as chief scientific officer at AskBio, Samulski is a professor of pharmacology at UNC where he also directed the Gene Therapy Center for many years.

Mikhail said the Biotech Center’s financial support of AskBio in its earliest days was instrumental to the company’s development of drugs for a wide variety of diseases.

Doug Edgeton, president and chief executive officer of the Biotech Center, said the state’s bioscience community is “immensely proud” of Mikhail for her national recognition.

“It’s a richly deserved honor for her hard work, tenacity and entrepreneurial brilliance at AskBio,” Edgeton said. “AskBio’s home-grown success has helped establish North Carolina as a major hub for gene therapy innovation and commercial development.”

A search of the Biotech Center’s directory of North Carolina bioscience companies for “gene and cell-based therapies” returns a list of 47 companies involved in the sector, including some big brands such as Novartis and Pfizer.