Sheila Mikhail, JD, MBA
CEO, President, Co-Founder
Sheila Mikhail co-founded and was the initial CEO of AskBio from 2002 through 2007. Ms. Mikhail left the company to become CEO of NanoCor Therapeutics, Inc. Most recently, Ms. Mikhail co-founded and was CEO of Bamboo Therapeutics, Inc., which in 20 months raised $50 million, advanced a therapeutic for GAN into the clinic, completed Pre-IND studies for the Duchenne Muscular Dystrophy therapeutic, and built a GMP manufacturing facility. Bamboo was acquired by Pfizer in August 2016.
Trained as a lawyer, Ms. Mikhail practiced law for over 15 years, including a law firm that she founded, Life Sciences Law, which serviced clients such as Bayer, Gilead, GSK, Sanofi, and Aventis. Ms. Mikhail also practiced with Ropes and Gray in Boston. Ms. Mikhail earned a JD, with honors, from Northwestern University; a finance MBA, with honors, from the University of Chicago; and a BS, with highest honors, from the University of Illinois at Urbana-Champaign.
Richard Jude Samulski, PhD
Dr. Samulski received his PhD in Medical Microbiology and Immunology from the University of Florida. Dr. Samulski’s graduate work (1978-82) demonstrated the first use of AAV as a viral vector and culminated in the first US patent involving non-AAV genes inserted into AAV. After completing post-doctoral training at Princeton, Dr. Samulski was hired to provide his expertise in AAV biology as a member of the scientific advisory board of Avigen, a new AAV research company. Later, in 1993, Dr. Samulski co-founded an AAV-based gene therapy company called Merlin. This research group was headed by Dr. Xiao and was the first group to demonstrate AAV-mediated long-term gene transduction in muscle (J. Virology, 1996). This finding precipitated a merger of Merlin with Somatix, Inc., which then merged in 1995 with Cell Genesys. In total, Dr. Samulski has worked with AAV for 40 years, and for 25 years, was director of the University of North Carolina Gene Therapy Center. Dr. Samulski was the scientific founder of Bamboo Therapeutics, Inc. and served as the Chief Scientific Officer and Executive Chairman of the company until its acquisition by Pfizer in 2016. Upon its acquisition, Dr. Samulski joined Pfizer, as VP Gene Therapy, to ensure the successful transition of Bamboo’s Duchenne Muscular Dystrophy therapeutic. Dr. Samulski is a former member of the Recombinant DNA Advisory Committee (RAC), a committee tasked with assisting the FDA with approving or disapproving gene therapy clinical trials in the United States. Dr. Samulski also frequently serves as a gene therapy consultant to the FDA. In 2008, Dr. Samulski was recognized by the American Society of Cell and Gene Therapy with the Inaugural Lifetime Achievement Award for his work. Dr. Samulski has been invited by Pope Francis to attend the Vatican as recognition for his work in the treatment of Canavans. Dr. Samulski has advanced into human clinical trials therapeutics for hemophilia, Duchenne Muscular Dystrophy, Giant Axonal Neuropathy, and heart failure. Dr. Samulski is the inventor of over 200 patents related to AAV technology.
Philippe Moullier, MD, PhD
Chief Scientific Officer - Europe
Founder until 2016, the Director of the research INSERM Unit UMR 1089 entitled « Translational Gene Therapy for Retinal and Neuromuscular Diseases » at the University of Nantes, France. Dr. Moullier was also the Scientific Director of GENETHON, a nonprofit research institution, located near Paris, from 2009 through 2011. In 2014, he co-founded HORAMA, a spin-off dedicated to gene therapy for monogenic rare retinal diseases. He established a unique 16,150 ft2 BL2/BL3 confined large animal core facility located at the Nantes veterinary school. He was also key in the establishment of a full GMP-compliant manufacturing facility operated today by the Etablissement Français du Sang (EFS) located in Nantes. From 2006 until 2016, Dr. Moullier was an Adjunct Professor at the Molecular Genetics and Microbiology Department at the University of Florida, USA. Dr. Moullier supervised more than 85 people scattered among the research groups in Nantes, the core facilities and administration, including junior faculty, post-docs, graduate students, undergraduate students, and technicians. His research efforts are focused on: (i) the basic virology of Adeno-Associated Virus with translational developments in recombinant virus assembly; (ii) the preclinical evaluation of recombinant viral vectors in large animal models of rare genetic disorders; (iii) the establishment of Phase I/II human clinical trials in retina and neuromuscular diseases; and (iv) basic viral vector biology (immunology and vector maintenance) in large animal models. Prior to joining the University of Nantes, Dr. Moullier conducted research at the Pasteur Institute in Paris and at the Wistar Institute in Philadelphia where he was engaged in the development of MLV and adenovirus-derived recombinant viral vectors and their in vivo application.
Dr. Moullier was a postdoctoral fellow at Scripps Research Institute in La Jolla, received his PhD degree in Biochemistry from the University of Paris (Mentor Pr Donny Strosberg) and obtained a Master in Immunology at the Pasteur Institute. Dr. Moullier also was an intern in nephrology and internal medicine and received his MD degree from the University of Paris (mentor: Pr Claude Amiel). Dr. Moullier has authored over 120 original publications describing virus biology, viral vector development, gene transfer and genetic diseases. Dr. Moullier served as an expert to the EMA and at the ANSM Gene Therapy Advisory Board and is a world-renowned expert in viral vector development and in gene therapy translational research.
Josh Grieger, PhD
Chief Technology Officer
Josh Grieger was a co-founder and Vice President of Process Development and Manufacturing with Bamboo Therapeutics, Inc. Dr. Grieger led and managed the GMP manufacturing and quality release of the rAAV drug product for the GAN Phase I clinical trial and supported the Pre-IND for Duchenne Muscular Dystrophy AAV gene therapy program. Bamboo Therapeutics, Inc was acquired by Pfizer in 2016. As part of Pfizer, Dr. Grieger served as a Senior Director of Gene Therapy focused on process development and optimization of scalable rAAV vector manufacturing processes for early and late phase gene therapy clinical trials.
From 2008 through 2015, Dr. Grieger was employed by the UNC-Chapel Hill Gene Therapy Center functioning as a post-doctoral research scientist, Research Associate Professor and Research Assistant Professor (Director of UNC Vector Core facility). Dr. Grieger’s research focused on the development of scalable transfection-based manufacturing process for rAAV vectors. The Pro10 cell line was established through this work and led to the transition from adherent cell-based manufacturing of rAAV vectors to animal-derived component-free suspension cell manufacturing in WAVE and Stir tank bioreactors with associated scalable purification technology. Dr. Grieger also consulted for AskBio over this time and was key in the Pro10 rAAV vector manufacturing process technology transfer to Baxter to support the Hemophilia gene therapy pre-clinical and clinical programs.
Dr. Grieger received his Doctorate Degree from UNC-Chapel Hill in the Molecular Biology and Genetics Program in 2005. Dr. Grieger carried out his Doctoral dissertation work in Dr. Richard Jude Samulski’s laboratory focused on various aspects of the AAV life-cycle.
Martin K. (Casey) Childers, DO, PhD
Chief Medical Officer
Dr. Childers was a professor in the Department of Rehabilitation Medicine, and a co-director of the Institute for Stem Cell and Regenerative Medicine at the University of Washington. He is a graduate of Seattle Pacific University (BA, music performance), Western University (DO, medicine, osteopathic), and the University of Missouri (PhD, physiology and pharmacology; residency, rehabilitation medicine). Dr. Childer’s research focused on two areas of investigation: in preclinical studies, they addressed issues related to systemic gene replacement delivery for patients with rare neuromuscular diseases, such as X-Linked Myotubular Myopathy and, in other studies, they used a “disease in a dish” approach with induced pluripotent stem cells to study heart disease in patients with Duchenne muscular dystrophy. Previously, Dr. Childers’ clinical medicine practice at the University of Washington Medical Center was dedicated to serve patients with neuromuscular diseases.
Dr. Childers studied music as an undergraduate at Seattle Pacific University. Dr. Childers’ graduate studies were completed at Western University, College of Osteopathic Medicine (DO) and at the University of Missouri, Department of Physical Medicine & Rehabilitation, and Department of Physiology (PhD).
Diane Balderson, PhD
VP Regulatory Affairs
Dr. Balderson has more than 30 years of pharmaceutical experience having worked at Pfizer, University of Ulster, GSK and Editas Medicine. Dr. Balderson started her career as a pharmacologist, moving into clinical pharmacology and then regulatory affairs. Dr. Balderson has led clinical teams from first-in-human studies through proof-of-concept and regulatory teams through all drug development phases (pre and post-approval) in a variety of areas and indications including gene editing, gene and cell therapy, antiviral/infectious disease, ophthalmology, GI, osteoporosis, osteoarthritis, urology, women’s health, diabetes, obesity and cardiovascular disease. Dr. Balderson’s interests include rare disease and pediatrics.
Diane earned her PhD from Queen’s University, Belfast, Northern Ireland.
Candace Summerford, PhD
Chief Patent Officer
Dr. Candace Summerford combines her scientific expertise with over 17 years of experience in Patent Law and serves as Chief Patent Officer of AskBio. Prior to joining AskBio, she spent 15 years at Nixon Peabody LLP practicing in Intellectual Property Counseling and Transactions, and prior to that she was at Palmer and Dodge LLP. Her intellectual property practice focuses on strategic patent portfolio management, performing due diligence reviews and freedom-to-operate analyses for venture capital financing, and on the preparation and prosecution of patent applications.
Examples of extensive worldwide portfolios prosecuted and managed by Dr. Summerford include portfolios in the fields of tissue engineering, stem cells, biomaterial scaffolds, and gene therapy. Dr. Summerford’s experience further includes performing due diligence reviews and freedom-to-operate analyses, for example in the areas of RNA interference, gene therapy, orphan disease therapeutics, stem cell therapies, and gene delivery including e.g., lentivirus vectors, AAV vectors, and HSV vectors. She has also prepared and prosecuted patent applications in a variety of disciplines, including applications directed to regenerative medicine, silk biomaterials, angiogenesis, genomic and protein diagnostic biomarkers, gene delivery vectors, next-generation DNA sequencing, RNA interference, and induced pluripotent stem cells.
Dr. Summerford’s dissertation work was carried out in the laboratory of Dr. R. Jude Samulski where she made seminal contributions in the field of adeno-associated virus (AAV) biology and AAV gene therapy vectors, including the identification of heparan sulfate as a cellular receptor for AAV. She continued as a Postdoctoral fellow in Dr. Samulski’s laboratory focusing on AAV biology, and was a Postdoctoral fellow in the laboratory of Dr. Richard Mulligan at Harvard Medical school. She earned her Doctorate degree at the University of North Carolina, Chapel Hill, from the department of Pharmacology where she graduated with honors. Dr. Summerford is registered to practice before the United States Patent and Trademark Office.