NC Award Sponsor Nomination Ltr William Hawkins

Dear N.C. Awards Committee,

It is my distinct honor to nominate Sheila Mikhail, J.D., M.B.A., for the prestigious North Carolina Award for Public Service. I have known Sheila for more than 10 years and had the privilege of serving on her Board at AskBio until the sale to Bayer Healthcare, LLC in late 2020. Sheila built her extensive career, which has spanned over 30 years, on her foundational values of integrity, accountability, and relentless perseverance, all underpinned by her passionate commitment to improve the lives of others. A gifted entrepreneur, she possesses a keen ability to analyze the current landscape, and assess the horizon, to capitalize on beneficial differentiation while implementing strategies to mitigate potential risks. A natural collaborator and entity structural expert, Sheila is able to accurately assess synergies and negotiate terms that support successful, long-term partnerships that provide all parties the opportunity for potential positive benefit while mitigating challenges. Her strong foundational values combined with her extraordinary business acumen have produced a series highly successful companies and brought incalculable benefit to the State of North Carolina and to patients facing devastating healthcare prognoses worldwide.

Beginning her career in law, Sheila represented venture companies and large pharmaceutical companies as an attorney with Ropes & Gray LLP and Womble Carlyle PLLC, building an extensive network and fostering long-term relationships that would benefit her future endeavors. Recognizing the need for a legal practice focused on the specific requirements of biotechnology, pharmaceutical, medical devices, bioinformatics and genomics companies in the Research Triangle area, she founded Life Sciences Law and grew this firm into the largest life sciences legal practice in North Carolina, and one of the most profitable in the Southeast, with a client base that included Bayer Pharmaceutical, Aventis, Sanofi, Gilead, Grifols and GlaxoSmithKline. Sheila became an advisor and guest lecturer for a UNC initiative called “Launching the Company” aimed at providing assistance to professors and university researchers in translating their discoveries from the lab to the marketplace to benefit lives. After one such lecture, Sheila was approached by Dr. R. Jude Samulski, a gene therapy pioneer recruited to UNC to build and direct their new Gene Therapy Center, who suggested that they collaborate to form a company to advance his ground-breaking gene therapy technologies. In an unusual pitch, Jude advised that he wouldn’t be able to pay her because he’d probably never be able to raise funds due to the high level of skepticism around gene therapy at that time. However, he predicted, if they were successful, they would change the world. And, so, based on a business model that gauged success not on individual financial gain but on the number of lives beneficially impacted, Sheila Mikhail and Jude Samulski embarked on a journey that would lead them to build a world-renowned, global gene therapy company that has beneficially impacted countless lives.

Asklepios BioPharmaceutical, Inc. (AskBio) was founded in 2001 by Sheila Mikhail, Dr. R. Jude Samulski and Dr. Xiao Xiao (Jude’s first Ph.D. student) as a collaboration with parents of sick children for whom traditional medicine offered no viable treatment. These parents refused to accept a devastating disease progression for their children and turned to gene therapy as a hopeful treatment modality. In a race against time, Sheila employed bootstrap financing strategies to operate the company and fund its important research. During a period when no venture capital was available for gene therapy companies, she defied the odds and creatively secured sufficient financing to keep AskBio moving forward. For the first decade, neither Sheila nor Jude accepted a salary from the company. Sheila reached out to the North Carolina Biotechnology Center and angel investors for early funding support. She landed the first grant ($1.6 million) ever provided by the Muscular Dystrophy Association to finance research into a cure for Duchenne Muscular Dystrophy. The parents organized bake sales and Relay for Life fundraising races to help fund research into therapeutics for their children. She designed a very effective strategy to spin-off Special Purpose Entities (SPEs) which licensed IP from AskBio around a specific therapeutic candidate. Sheila was approached on numerous occasions with financing offers that required relocation of her start-up companies to Boston. Fully committed to building North Carolina into a globally recognized life sciences hub, she declined those offers and continued operations in the State and went on to successfully negotiate the sale of two of the entities to large pharma.

• In 2005, Sheila spearheaded the spin-out of NanoCor Therapeutics, Inc. to pursue the development of AskBio’s technology for the treatment of congestive heart failure and, shortly thereafter, negotiated a significant equity investment (totaling $10 million) with Medtronic, Inc.
• Chatham Therapeutics was spun-out with a focus on AskBio’s rAAV technology for the development of therapeutics for hemophilia and was acquired by Baxter, now Takeda, in 2014, with an initial payment of $70 million.
• Bamboo Therapeutics was spun-out to develop a therapeutic to treat Duchenne Muscular Dystrophy (DMD). With an early stage investment by CureDuchenne Ventures LLC, Sheila negotiated the acquisition of the UNC Vector Core manufacturing facility and oversaw its transition to a cGMP compliant facility to produce AAV for DMD clinical trials. Bamboo Therapeutics was acquired by Pfizer, Inc., in 2016, in a deal valued at over $645 million and Pfizer has recently advanced that treatment into pivotal clinical trials.
• In 2017, Actus Therapeutics was spun-out to pursue development of a treatment for Pompe disease that AskBio has advanced into early stage clinical trials.

As gene therapies began to transform medicine, Sheila had the foresight to address the impending demand escalation for adeno-associated virus (AAV) vector manufacturing and, in 2017, initiated AskBio’s Contract Development and Manufacturing Organization (CDMO) business unit, forming a joint venture with Columbus Venture Partners to establish Viralgen Vector Core, SL, an AAV manufacturing facility in San Sebastián, Spain supported, in part, by a public-private relationship Sheila negotiated with the Basque government. She led an expansion into commercial grade vector manufacturing with a second facility in San Sebastián that will be operational in late 2021. Additionally, she established a manufacturing facility in Research Triangle Park, North Carolina with plans to expand this facility in coming years. Both the Viralgen and RTP facilities manufacture AAV based on AskBio’s Pro10® system that produces one of highest yields in the industry. As the number of gene therapy clinical trials has dramatically increased over the past five years, exceeding 4,000 as of February 2020, AAV manufacturing companies are struggling to meet demand. As a result of Sheila’s proactive strategies, AskBio has secured AAV vector for its own clinical trials and sells excess capacity to the industry, generating additional revenue for the company. In their effort to address the industry’s growing production demand, in 2019, Pfizer announced a $500 million expansion to the gene therapy manufacturing facility purchased in their acquisition of Bamboo Therapeutics which has added 300 high-skilled jobs in Sanford, NC.

To further maximize revenue sources to support AskBio’s operations, Sheila has negotiated out-licensing agreements providing rights to the use of AskBio’s IP in fields where the company is not developing drugs. One such transaction is the non-exclusive, worldwide license that Sheila negotiated with AveXis (now Novartis) in 2015 for the use of AskBio’s self-complementary vector (scAAV) technology in the development of their SMA drug, Zolgensma®, which is one of only two AAV gene therapy drugs currently approved by the FDA for commercialization.

After 18 years of self-funding initiatives, Sheila negotiated and closed AskBio’s first capital raise ($235 million), in 2019, which was the largest-ever Series A funding, not only in gene therapy, but throughout the booming life sciences industry in North Carolina. With this capital infusion, Sheila closed on three acquisitions to add key technologies to AskBio’s technology platform and preclinical programs to its portfolio.

• Synpromics Ltd, based in Edinburgh, Scotland, which specializes in the development of synthetic and inducible promoters (August 2019)
• BrainVectis SAS, based in Paris, France, which develops gene therapy treatments for a pipeline of CNS therapeutics, including Huntington’s disease, based on the work of Dr. Nathalie Cartier (April 2020)
• Brain Neurotherapy Bio, Inc., a company Sheila co-founded in 2018, to develop gene therapy treatments for a pipeline of CNS therapeutics, including an active clinical trial for Parkinson’s disease, based on the work of Dr. Krystof Bankiewicz (October 2020)

Additionally, Sheila negotiated collaborations with leading biopharmaceutical companies to explore cutting-edge scientific discoveries to address challenges limiting the potential further development of, and patient access to, gene therapeutics.

• Editas Medicine for the development of a pipeline of CNS indications using their gene editing technology
• Selecta Biosciences for the use of their ImmTOR technology for repeat administration for a pipeline of gene therapy drugs delivered to the liver and muscle

Having built AskBio into a global, industry-leading gene therapy company, Sheila negotiated and closed its acquisition by her former Life Sciences Law client, Bayer Healthcare, LLC, in a deal valued at up to $4 billion. Throughout the four month due diligence process and transaction negotiation, which closed in late 2020, Sheila successfully managed AskBio’s global operations while addressing daily challenges resulting from the Covid-19 pandemic and significant economic and political uncertainty. She held daily web meetings with senior management throughout the organization to check on employee welfare and the implementation and effectiveness of protocols to ensure employee safety while shoring up supply chains and advancing operational objectives.

Post-acquisition, Sheila has been retained to continue serving as AskBio’s Chief Executive Officer, leading the company’s work to fulfill milestones negotiated in the transaction. These milestones include the expansion of AskBio’s CDMO business and the company is currently constructing Viralgen Commercial Therapeutic Vector Core, S.L., which will be Europe’s largest commercial AAV manufacturing facility when completed in mid-2021, bringing AskBio’s manufacturing infrastructure to over 350,000 sq. ft. Additionally, operations will expand within Touchlight AAV (TAAV), a joint venture to produce doggie bone DNA, an innovative technology offering an alternative to plasmid DNA - the starting material for AAV production. TAAV is a joint venture that Sheila negotiated with Touchlight Genetics in 2018. This disruptive technology has a faster cycle time (two weeks versus months), lower costs, and is potentially safer because it does not use e-coli and fermentation processes, thereby avoiding some bacterial contaminants. This cutting-edge technology will provide AskBio a significant competitive advantage while improving the safety profile of gene therapeutics.

Under Sheila’s leadership, AskBio has advanced four therapeutic programs - Pompe disease, congestive heart failure, Parkinson’s disease and multiple systems atrophy - into the clinic and is planning clinical trials for Limb-Girdle 2i, MMA, and Huntington’s disease. Additionally, AskBio’s therapeutic pipeline includes several programs, such Pain Management and Myotonic Dystrophy, which are in earlier stages of development.

Widely recognized as an unwavering, passionate champion of gene therapy, Sheila is a highly regarded and sought-after speaker at industry and advocacy conferences throughout the world. Her’s is a voice the industry listens to, and learns from, as the field advances.

As a prolific entrepreneur and successful businessperson, she serves, and has served, as a board director and advisor to many gene therapy companies, such as Verve Therapeutics, a gene editing company developing treatments for cardiovascular disease, and Chameleon Biosciences, Inc., which is advancing a technology to potentially overcome existing gene therapy treatment barriers. Sheila is actively involved in community associations, serving on the Boards of the NC Biotech Center, the NC Biosciences Organization, as well as the Local Advisory Board for BB&T-Truist (Durham). She serves as an advisor to academic organizations such as the UNC & NC State Joint Biomedical Engineering Program. Additionally, she is an active mentor to young women in the biotech space through the Phacilitate program and is an advisor within SpringBoard Enterprises, which provides training and a support network for women entrepreneurs seeking to obtain venture capital financing for their enterprises. A SpringBoard alumna, Sheila was recognized as its 2019 Northstar Honoree, a national award that recognizes outstanding entrepreneurial women leading high-growth companies in transforming their industries.

As a result of her remarkable and far-reaching success in advancing Dr. Jude Samulski’s gene therapy technologies, Sheila has brought worldwide attention to North Carolina and has been a catalyst in the booming gene therapy industry in our State. Since forming AskBio in 2001, 43 gene therapy companies have chosen to locate in North Carolina, creating thousands of fulfilling and financially rewarding jobs in our communities.

Sheila’s track record of success in advancing the scientific discoveries and patient access to curative technologies developed by Jude Samulski is well known throughout the life sciences industry. Consequently, she is often approached by scientists requesting her assistance in advancing their own break-through technologies. In 2019, Sheila co-founded Jurata Thin Film, Inc., headquartered in Chapel Hill, with Dr. Maria Croyle to advance a disruptive technology that will dramatically improve distribution and storage of medicinal products throughout the world. This technology stabilizes vaccines and biologics within a film matrix to facilitate economical transport and long-term storage at ambient temperature, a cost-saving alternative to current methods which require specialized storage containers and -80°C freezers. The drug is extracted from the film and reconstituted on-site for delivery to patients. A single wafer-thin film, weighing 1/100 of a pound, can be used to store up to 500 doses of a vaccine allowing efficient distribution of drugs to patients anywhere in the world at a fraction of the current cost.

Sheila’s commitment to accelerate equitable access to curative gene therapeutics for patients in need across the globe led her to co-found, and serve as a Trustee for, Columbus Children’s Foundation (CCF), a non-profit that develops AAV-based treatments for ultra-rare diseases not commercially viable for investment by the pharma and biotech industries. Within the last two years, CCF has treated over 30 children suffering from amino acid decarboxylase (AADC), at no cost to their families, and has invested in the advancement of therapeutic programs that would have otherwise lagged, giving hope to families facing devastating diagnoses.

Through her boundless ingenuity, steadfast perseverance in the face of often overwhelming adversity, and tireless drive, Sheila Mikhail answered the challenge in Jude Samulski’s initial pitch. She has successfully funded his research and translated his technologies from the university lab to improve the health of patients across the globe. In so doing, she has brought honor, significant economic growth, and opportunity to the State of North Carolina. Together, she and Jude have beneficially improved the lives of millions of people and they have indeed changed the world. For all of these reasons, Sheila Mikhail is most deserving of the prestigious North Carolina Award for Public Service.

Sincerely,

William A. Hawkins III
Former Chairman and CEO, Medtronic
Vice Chair, Duke University Board of Trustees
Senior Advisor to EW Healthcare Partners
Corporate Director