Mila's Miracle Foundation has put together a world-renowned team of scientists and doctors to work on our gene therapy trial for Mila's type of Batten Disease. We have been fortunate to have attracted the world’s leaders in gene therapy to run our trial work. Scientists believe that our trial will not only help children with Mila's type of Batten, but will serve as the blueprint for ending hundreds of rare diseases forever.
Steven Gray, Ph.D.
Dr. Steven Gray is the Chief Researcher and Scientific Advisor for the Batten CLN7 Trial. He earned his Ph.D. in molecular biology from Vanderbilt University in 2006, after receiving a B.S. degree with honors from Auburn University. He performed a postdoctoral fellowship focusing on gene therapy in the laboratory of Jude Samulski at UNC. He is currently an assistant professor at UNC in the Department of Ophthalmology. He is also a member of the Gene Therapy Center and the Carolina Institute for Developmental Disabilities (CIDD).
Dr. Gray’s core expertise is in AAV gene therapy vector engineering, followed by optimizing approaches to deliver a gene to the nervous system or eye. His major focus is in AAV vector development to develop vectors tailored to serve specific clinical and research applications involving the nervous system or eye. His research focus includes preclinical studies for Rett Syndrome,Tay-Sachs, Krabbe, AGU, and Batten Disease. He has also expanded his preclinical work into human gene therapy trials for Giant Axonal Neuropathy (GAN).
Dr. Gray has published over 50 peer-reviewed papers in journals and has 3 pending patents. His research is funded by the National Institute for Neurological Disorders and Stroke, as well as numerous large and small research foundations. Dr. Gray was recently recognized with the 2016 Healthcare Hero award by the Triangle Business Journal, and his work on GAN was featured in a story by the CBS National Evening News in 2015.
Myriam Mirza, PH.D.
Dr. Myriam Mirza is the Principal Scientist at Mila’s Miracle Foundation, Inc., and has been working with Batten Disease since 2010, including academic research as well as supporting other Batten Disease Foundations.
She joins the foundation with the purpose of coordinating different therapeutic projects, developing investment strategies as well as collaboratively working with clinicians, researchers and industry partners to find a viable treatment for CLN7 Batten Disease.
She received her Ph.D. in Neurobiology from the University of Regensburg, Germany in 2013, after receiving a M.Sc. and B.Sc. in Biochemistry from the University of McGill, Canada. During her under-graduate and early graduate work, she focused on identifying therapies for cystic fibrosis as well as other rare disease with similar mechanisms. Her Ph.D. consisted of studying the immune response in brain and retinal cells of CLN6 and CLN3 Batten disease animal models, which was funded by NCL-Stiftung, a German Batten Disease Foundation. She currently works as a freelancing healthcare and strategy consultant for large medical device and pharmaceutical companies as well as non-profit organizations.
Jeffrey Hung, Ph.D
Dr. Jeffrey Hung is Chief Commercial Officer of Vigene Biosciences, a leading gene delivery company whose mission is to make gene therapy affordable. Dr. Hung is also the General Manager who oversees the cGMP business of Vigene Biosciences which is producing the AAV9 vector for the Batten CLN7 trial.
Dr. Hung is instrumental in catapulting Vigene Biosciences into a leading position for gene therapy manufacturing by orchestrating the acquisition and integration of Omnia Biologics in 2016, a boutique CMO specializing on GMP manufacturing of viruses.
Dr. Hung served as the Vice President of GenScript and was instrumental in growing the company and bringing the company to IPO in December 2015. Dr. Hung was the Chief Marketing Officer at ATCC before joining GenScript. Prior to that role, Dr. Hung was key in growing SABiosciences’ sales for 4 consecutive years which led to QIAGEN’s acquisition of the company in 2011. Dr. Hung is the author of multiple patents, per reviewed publications and book chapters. Dr. Hung earned his Ph.D. in Genetics from Cornell University, MBA from UC Berkeley, and BS from Peking University.
An innovative leader in growing life science and biomedical companies, Dr. Hung is passionate about bringing breakthrough innovations to benefit scientists and consumers.
Berge A. Minassian, MD CM, FRCP(C)
Berge A. Minassian, MD CM, FRCP(C) is a Scientific Advisor for the Batten CLN7 trial. He went to medical school at McGill University, did his residency in adult neurology at UCLA, a fellowship in pediatric epilepsy and molecular genetics at the University of Toronto, and then was a clinician-scientist at the Hospital for Sick Children in Toronto until his move in 2017 to head Pediatric Neurology and Pediatric Neurosciences at the University of Texas Southwestern.
Dr. Minassian was deeply affected by the suffering of children and families with neurodegenerative diseases including Lafora disease and neuronal ceroid lipofuscinosis (NCL). He has been involved in the discovery of many a disease-causing gene in this area including Lafora, CLN6 and CLN7 forms of NCL. His two decades of work at the Hospital for Sick Children in Toronto have brought Lafora disease to the brink of a cure - a clinical trial is now being organized. He moved to Dallas to pursue an opportunity to build a gene-based therapies program for a wide-spectrum of these diseases, including NCL's. His greatest wish is for a day where the pediatrician having diagnosed one of these fatal diseases will be able to smile to the family and say: "give your child this; all will be well."
Timothy Yu, MD, Ph.D
Dr. Yu is a Scientific Advisor for the Batten CLN7 trial. He is a neurogeneticist at Boston Children’s Hospital with expertise in genomics, neurobiology, informatics, and personalized medicine. His research group focuses on applying advanced genomic methods to understand the genetic underpinnings of neurodevelopmental disorders, and has identified over a dozen novel disease genes for intellectual disability and autism. His group is also at the forefront of efforts to integrate advanced genomics into clinical practice, through projects like Babyseq (evaluating the utility of genome sequencing in newborns), and Rapidseq (rapid turnaround sequencing in the NICU).
Dr. Yu received his A.B. (Biochemistry and Molecular Biology) from Harvard College and M.D. and Ph.D. (Neuroscience) degrees from the University of California at San Francisco. He completed neurology residency training at Massachusetts General Hospital and Brigham and Women’s Hospital (where he served as Chief Resident), and a neurodevelopmental disorders fellowship at Boston Children’s Hospital and the MGH Lurie Center. His laboratory is based in the Division of Genetics and Genomics at Boston Children's Hospital. He also holds academic appointments at Harvard Medical School and the Broad Institute of MIT and Harvard, and is co-founder of Claritas Genomics, a subsidiary of BCH devoted to practicing genomic medicine for children with rare disease.
Mimoun Azzouz, PH.D.
Dr. Mimoun Azzouz is a Scientific Advisor for the Batten CLN7 trial. He obtained a Master in Neuroscience with 1st Class Honours from the University of Marseille in 1994. In 1997 he was awarded a PhD in Neuropharmacology at the University Louis Pasteur in Strasbourg. He then worked as postdoctoral scientist at the Gene Therapy Center in Lausanne, Switzerland. He was then recruited by Oxford BioMedica plc as Senior Scientist then appointed as Director of Neurobiology in 2003. In 2006, he was invited to join the University of Sheffield and was appointed to the Chair of Translational Neuroscience.
Professor Azzouz is currently Deputy Head of Neurology and Director of Research & Innovation. One of his major achievements is his involvement in a gene therapy approach designed to achieve dopamine replacement in models of Parkinson’s disease. This strategy has yielded significant translational impact having entered into phase I/II human clinical trials since 2008. His pioneering work, which has already produced major breakthroughs in animal models, has short and medium term potential for real translation into major therapeutic advances for human neurodegenerative disease.
He recently won the prestigious ERC Advanced Investigator Award (2011). This award is a top-level EU ad hominem award acknowledging his pre-eminence in European biomedical research. He is/was a member of scientific Panels/Boards for various funding bodies such as the Medical Research Council (Translational MRC Panel, UK), the French Muscular Dystrophy Association (AFM), The Research Council of Norway, the Health Research Board (HRB) of Ireland and the Neuroscience Board, Germany. He was elected as Board member of the British Society of Gene and Cell Therapy Society in 2016.