About
GTnetwork
The mission of the GTnetwork is to build knowledge of the evolving gene therapy landscape among healthcare professionals by providing engaging, evidence-based educational content while fostering peer-to-peer interaction.
Objectives: Educational Content
The GTnetwork educational content includes a comprehensive curriculum covering a range of topics from the basic principles of gene therapy to the clinical evidence behind the design, delivery, and use of gene therapy.
The gene therapy landscape is constantly evolving, therefore new content for the GTnetwork will be developed and released over time.
The GTnetwork educational content will:
Review potential disease targets for gene therapy
Provide insight into the clinically relevant aspects of the design and manufacturing of gene therapies
Explain the different therapeutic gene alteration strategies and their clinical uses
Discuss ongoing practical considerations for the use of gene therapy in the clinic
Meet The Steering Committee
All GTnetwork content is overseen and reviewed by a multidisciplinary, internationally renowned steering committee of experts. This group provides leadership and insight into the platform’s content and features, as well as answering questions from the community and discussing “hot topics”.
Chris Jenkins, PhD, MPH
Chairperson
Principal Partner and Chief Gene Therapy Biosafety Officer at Clinical Biosafety Services
Chris Jenkins, PhD, MPH
Principal Partner and Chief Gene Therapy Biosafety Officer at Clinical Biosafety Services
Dr. Chris Jenkins is the Principal Partner and Chief Gene Therapy Biosafety Officer at Clinical Biosafety Services, a regulatory firm specializing in administering Institutional Biosafety Committees for biosafety risk assessment review of gene transfer trials at academic medical centers, hospital systems, and clinics. He has worked 10+ years in the fields of human protections, gene therapy, and biosafety compliance fields in academic, private research, and clinical settings. Dr. Jenkins received his Doctorate of Philosophy in Public Health, with a focus on Biosecurity through the Saint Louis University School for Public Health and Social Justice. He holds academic appointments at the Saint Louis University School for Public Health and Social Justice, and The University of Texas Health Science Center at Houston (UTHealth) School of Public Health in Austin.
Diana Bharucha-Goebel, MD
Director of the Muscular Dystrophy Association Care Center and Assistant Professor in the Division of Neurology
Diana Bharucha-Goebel, MD
Director of the Muscular Dystrophy Association Care Center and Assistant Professor in the Division of Neurology
Dr. Diana Bharucha-Goebel is Director of the Muscular Dystrophy Association Care Center and Assistant Professor in the Division of Neurology at Children’s National Medical Center in Washington, D.C., and the Division of Pediatrics and Neurology at the George Washington University School of Medicine & Health Sciences. Dr. Bharucha-Goebel completed her medical training in the Drexel University College of Medicine in 2007, followed by a General Pediatric Residency in 2009, a Child Neurology Residency in 2012, and a Neuromuscular Fellowship in 2013, all of which were at the Children’s Hospital of Philadelphia. Starting in 2013, she has conducted additional research at the National Institutes of Health (NIH) in Bethesda, Maryland. Her interests include translational research in congenital neuromuscular diseases, with a particular focus on the evaluation and identification of disease-focused outcome measures in rare disease as it pertains to better understanding functional outcomes for clinical trial readiness. Her research focus is in the development of early clinical translational gene replacement studies, as well as in studying the immune consequences and responses following gene transfer. Dr. Bharucha-Goebel is certified with the American Board of Psychiatry and Neurology (ABPN) in Neurology with Special Qualification in Child Neurology.
Arthur Burghes, PhD
Professor at Wexner Medical Center College of Medicine in the Department of Biological Chemistry and Pharmacology
Arthur Burghes, PhD
Professor at Wexner Medical Center College of Medicine in the Department of Biological Chemistry and Pharmacology
Professor Arthur Burghes completed his BSc in cellular and microbial biology in 1978 at the University of Calgary, his PhD in biochemistry at the University of London, and completed postdoctoral work at the University of Toronto where he was involved in cloning the dystrophin gene. During his graduate work at the Hammersmith Hospital, Dr Burghes received extensive training in the study of neuromuscular disorders and first became familiar with Spinal Muscular Atrophy. After graduating from the University of London, Arthur did his postdoctoral fellowship under Dr. Worton at the Hospital for Sick Children in Toronto, where he was involved in cloning and charterization of the Duchenne Muscular Dystrophy gene. He then started as an Assistant Professor at the Ohio State University in August of 1988 while he continued working on Duchenne. He also, together with Dr. Mendel, started working on spinal muscular atrophy and collected families for linkage mapping of the SMA gene. The main focus of the Burghes laboratory is the molecular understanding of genetic neuromuscular disorders. In particular, the laboratory focuses on unraveling the molecular biology of spinal muscular atrophy (SMA). In this regard Dr. Burghes is currently a Professor at Wexner Medical Center College of Medicine in the Department of Biological Chemistry and Pharmacology. The laboratory has undertaken many studies in spinal muscular atrophy from the early studies characterizing the SMN genes, developing carrier and SMN2 copy number testing to developing SMA mice models which are used worldwide for SMA studies. Dr. Burghes has been involved in the development of therapies for SMA, in particular, in collaboration with the Kaspar Laboratory, they have developed gene therapy and used it to correct both SMA mice and the SMA pig a large animal model of SMA that the Burghes laboratory developed. Dr Burghes consults for Novartis.
James Dowling, MD, PhD, FANA
Professor of Pediatrics and Molecular Genetics at the University of Toronto
Uta Griesenbach, DIPL-BIOL, PhD
Professor in Molecular Medicine at the National Heart and Lung Institute at Imperial College London
James Dowling, MD, PhD, FANA
Professor of Pediatrics and Molecular Genetics at the University of Toronto
Dr. James J Dowling is a Professor of Pediatrics and Molecular Genetics at the University of Toronto, and a neurologist and senior scientist at the Hospital for Sick Children, also in Toronto. He obtained his PhD in molecular genetics and cell biology in 1998 and MD in 2001 from the University of Chicago. He is board certified in Neurology with a Special Qualification in Child Neurology and in Neuromuscular Medicine.
Dr. Dowling leads a translational research program focusing on the diagnosis and treatment of pediatric neuromuscular disorders, with an emphasis on congenital myopathies and muscular dystrophies. He uses a combinatorial approach that includes new gene discovery (using next-generation sequencing technology), model organism development, and drug discovery screening, complementing this work with cutting-edge rare disease clinical research related to natural history, outcome measure development, and interventional trials.
Dr. Dowling has published more than 100 peer-reviewed manuscripts on topics related to neuromuscular disorders, and he has won several international and national research awards. He is Chair of the executive committee for the neuromuscular network TREAT-NMD and of the Canadian Pediatric Neuromuscular Group (CPNG), a collaboration of Canadian pediatric neuromuscular specialists aimed at facilitating research, clinical care, and education for patients’ families with neuromuscular disorders. He is also on the editorial board of Journal of Neuromuscular Diseases, Neuromuscular Disorders, and Disease Models & Mechanisms, and an executive board member of the World Muscle Society.
John Petrich, MS, RPh
Director Investigational Drug Services at the Cleveland Clinic
John Petrich, MS, RPh
Director Investigational Drug Services at the Cleveland Clinic
John Petrich MS RPh has directed Investigational Drug Services at the Cleveland Clinic for the past 22 years. During that time, his team has supported a wide variety of clinical trials across an international landscape. John routinely assists investigators in protocol development, while his day-to-day focus is on clinical excellence, regulatory compliance, quality improvement, and biosafety awareness. The recent emergence of gene therapies has motivated John to enrich the guidance on gene therapy, particularly those involving safe handling, site preparedness, and risk evaluation. Accordingly, recent work by John has sought to provide much needed direction to site pharmacies on gene therapy. In 2019, John authored a review in The Journal of Pharmacy Practice titled Gene Replacement Therapy: A Primer for the Health-system Pharmacist and he has presented nationally and internationally on the topic of gene therapy. John’s current interests include site education and assessment of facilities for gene therapies. John received his pharmacy degree from the University of Toledo and his Masters from Cleveland State University. He is a long-standing contributor to the American Society of Health-System Pharmacists (ASHP), and past president of the Society of Clinical Research Associates (SOCRA).
Past Steering Committee Members
Jahannaz Dastgir, DO
Medical Director of the Pediatric Neuromuscular Medicine Program at Goryeb Children’s Hospital
Jahannaz Dastgir, DO
Medical Director of the Pediatric Neuromuscular Medicine Program at Goryeb Children’s Hospital
Dr. Jahannaz Dastgir is the Medical Director of the Pediatric Neuromuscular Medicine Program at Goryeb Children’s Hospital in Morristown, New Jersey. She is affiliated with Morristown Medical Center and Overlook Medical Center. Dr. Dastgir completed her medical training in the Philadelphia College of Osteopathic Medicine in 2004, pediatric training at the University of Connecticut, a child neurology fellowship at Boston Children’s Hospital, and neuromuscular subspecialty training at Massachusetts General Hospital and Brigham Women’s Hospital. Her special areas of interest include pediatric neuroscience, neurodegenerative diseases, movement disorders, and neurogenetics. Dr. Dastgir is certified with the American Board of Psychiatry and Neurology in both Neuromuscular Medicine and Neurology with Special Qualification in Child Neurology.
Alex MacKenzie, MD, PhD
Principal Investigator at the
Children’s Hospital of Eastern Ontario
Alex MacKenzie, MD, PhD
Alex MacKenzie Principal Investigator at the
Children’s Hospital of Eastern Ontario
Dr. Alex MacKenzie is a principal investigator at the Children’s Hospital of Eastern Ontario (CHEO) Research Institute and a professor at the University of Ottawa where he holds a Chair in Rare Neurologic Disease Therapeutics. He received his MD in 1983 and his PhD in medical biophysics in 1986, both from the University of Toronto. Dr. MacKenzie works on the molecular genetics of pediatric disease with a current research focus on the molecular genetics of spinal muscular atrophy (SMA) and has published extensively in the areas of molecular genetics of pediatric disease. He was co-lead of the ‘Enhanced CARE for RARE Genetic Diseases in Canada’ initiative focusing on the repositioning of drugs with the hope of therapeutically modulating genes that impact rare disease. He has also served as the CEO and Science Director of the CHEO Research Institute as well as Vice President of Research for both CHEO and Canada’s federal genomic research funding agency Genome Canada in addition to being founding scientist of the AeGera biotech company.
Petra Kaufmann, MD, MSc
Director of the Division of Clinical Innovation at the National Center for Advancing Translational Sciences
Petra Kaufmann, MD, MSc
Director of the Division of Clinical Innovation at the National Center for Advancing Translational Sciences
Dr Petra Kaufmann was formerly the Vice President of R&D Translational Medicine at Novartis. Prior to joining Novartis, Dr Kaufmann was Director of the National Center for Advancing Translational Sciences (NCATS) Office of Rare Diseases Research where her work included overseeing the Rare Diseases Clinical Research Network programme, Genetic and Rare Diseases Information Center and the Toolkit for Patient-Focused Therapy Development. Dr Kaufmann received her medical degree from the University of Bonn, Germany, and a Master of Science degree in biostatistics from Columbia’s Mailman School of Public Health. She completed a postdoctoral fellowship in the molecular biology of mitochondrial diseases at Columbia’s H. Houston Merritt Center for Muscular Dystrophy and Related Diseases. Dr Kaufmann worked with investigators in her role as Director of the Office of Clinical Research at the National Institute of Neurological Disorders and Stroke to develop a large portfolio of clinical research studies in neurological disorders, many of which were in rare diseases. This is where she launched NeuroNEXT, a trial network to support scientifically sound, biomarker-informed Phase 2 trials for neurological diseases in partnership with academia, industry and patient groups. Her research focus is on the clinical investigation of spinal muscular atrophy, amyotrophic lateral sclerosis and mitochondrial diseases.
Loren del Mar Peña, MD, PhD
Associate Professor of Pediatrics
at the University of Cincinnati, Ohio
Loren del Mar Peña, MD, PhD
Associate Professor of Pediatrics
at the University of Cincinnati, Ohio
Dr. Loren del Mar Peña is Associate Professor of Pediatrics at the University of Cincinnati, Ohio. She completed her PhD at Northwestern University, Evanston, Illinois in 2002, received her MD at Northwestern University, the Feinberg School of Medicine, Chicago in 2004 with residencies in pediatrics and clinical genetics at the University of Chicago. She specializes in the diagnosis, treatment, and management of hereditary diseases. She is a clinical geneticist and trialist in the orphan disease arena, with expertise in implementation of studies for rare and ultra-rare disorders. Dr. Peña has served as principal investigator in multiple clinical trials focused on rare disorders and spanning different treatment approaches that include gene therapy. She has also described several new genes involved in rare disorders.
