About Us

Mission & History

The goals of the Gene Therapy Program at the University of Pennsylvania (GTP) are to develop and commercialize transformative genetic-based therapeutics. These goals are driven by the unmet needs of patients with inherited genetic diseases. 

Led by Dr. James M. Wilson, GTP focuses on the development of next-generation gene transfer vectors and their application in the treatment of a variety of acquired and inherited diseases. Dr. Wilson began his work in gene therapy during his graduate studies at the University of Michigan more than 40 years ago and has been at the nexus of this emerging therapeutic area from its inception. 

Dr. Wilson created the first and largest academic-based program in gene therapy after being recruited to the University of Pennsylvania in 1993. He initially focused on the clinical translation of existing gene transfer technologies but soon redirected his efforts to the development of second and third-generation gene transfer platforms. Since then, GTP has emerged as the go-to organization for public and private partners who want to participate in the gene therapy space.

The Wilson Lab discovered a family of viruses from primates called adeno-associated viruses (AAV) that could be engineered to be very effective gene transfer vehicles. These vectors have become the technology platform of choice and have set the stage for the recent resurgence of the field of gene therapy.

---

Core Values

The following Core Values have been identified by our leadership and represent the Gene Therapy Program's principles and priorities.

Patient-Focus
Engaging with the patient community and prioritizing their needs, values, and preferences. 

 

Trustworthiness
The quality of being reliable, responsible, and deserving of confidence.

 

Fortitude
The strength to encounter adversity or difficulty with courage.

 

Disruptive Innovation 
An innovation that makes products and services more accessible and affordable, thereby making them available to a larger population. It is driven by enabling technology, an innovative business model, and a coherent value network.

 

Adaptability
The ability to adjust and flourish in a changing environment. 

 

Leadership Team

• 

  

  James M. Wilson, MD, PhD

  Director, Gene Therapy Program
  

  Rose H. Weiss Professor and Director, Orphan Disease Center
  Professor of Medicine and Pediatrics
  Perelman School of Medicine, University of Pennsylvania

  James M. Wilson, MD, PhD, is a Professor in the Perelman School of Medicine at the University of Pennsylvania where he has led an effort to develop the field of gene therapy. Dr. Wilson began his work in gene therapy during his graduate studies at the University of Michigan nearly 40 years ago. He then moved to Boston to do a residency in Internal Medicine at the Massachusetts General Hospital and continued his work in gene therapy at MIT. Dr. Wilson has been at the nexus of this emerging therapeutic area from its inception. He created the first and largest academic-based program in gene therapy after being recruited to Penn in 1993. He initially focused on the clinical translation of existing gene transfer technologies but soon redirected his efforts to the development of second and third-generation gene transfer platforms.

  His laboratory discovered a family of viruses from primates called adeno-associated viruses (AAV) that could be engineered to be very effective gene transfer vehicles. These so-called "vectors" have become the technology platform of choice and have set the stage for the recent resurgence of the field of gene therapy. Dr. Wilson has also been active in facilitating the commercial development of these new gene therapy platforms through the establishment of several biotechnology companies. He is currently leading a national dialogue on the challenges of commercializing these potentially lifesaving treatments due to the disruptive nature they will have on traditional business models. Throughout his career, the focus of Dr. Wilson’s research has been rare inherited diseases, ranging from cystic fibrosis to dyslipidemias to a variety of neurologic disorders.

  Dr. Wilson has published over 600 papers and is named on more than 1200 patents worldwide. He was the 2014 recipient of the William Osler Patient Oriented Research Award of the University of Pennsylvania and received the 2015 Scientific Achievement Award from Pennsylvania Bio. Dr. Wilson was noted by the journal Nature Biotechnology to be the “second most productive bio-entrepreneur in life sciences.” Dr. Wilson was a long-standing Trustee at Albion College where he and his wife established the Lisa and James Wilson Institute for Medicine. He is a founder of a 501(c)3 called Health Through Fitness in Orphan Diseases that supports a bicycle team called Rare Disease Cycling, whose participants compete at a national level and help raise money for rare disease research.

  Close modal dialog

• 

  

  Antony Appleyard, PhD

  Executive Director, Regulatory Affairs
  

  Antony Appleyard serves as the Executive Director of Regulatory Affairs at the University of Pennsylvania’s Gene Therapy Program (GTP). In this role, Antony leads the regulatory affairs department responsible for regulatory strategy and global health authority interactions. Antony has more than 20 years of experience in research, clinical development, and manufacturing. Prior to joining the GTP team, Antony was a consultant at Diamond Pharma Services for nine years where he led a multidisciplinary group providing regulatory support to clients developing cell and gene therapy products. Earlier in his career, Antony was engaged in antibacterial development at Novacta Biosystems, and he was responsible for the technical transfer of antibody manufacturing processes at a subsidiary of Abbott Laboratories. Antony holds a bachelor’s degree in Chemistry and a PhD in Biochemistry from the University of Leeds, and he carried out postdoctoral research in the Departments of Chemistry and Biochemistry at the University of Cambridge. He is a Fellow of the Royal Society of Chemistry, a Chartered Chemist, and a member of The Organisation for Professionals in Regulatory Affairs (TOPRA).

  Close modal dialog

• 

  

  Jenny Greig, PhD

  Executive Director, Research
  

  Jenny Greig has a Bachelor of Science (Hons) in Pharmacology and a PhD in Gene Therapeutics from the University of Glasgow in Scotland. Jenny joined the University of Pennsylvania’s Gene Therapy Program (GTP) in 2010 as a postdoctoral researcher investigating muscle and liver transduction with gene therapy vectors. In her current role at GTP, she leads a team of scientists developing preclinical approaches for the treatment of liver disease from proof-of-concept through to Investigational New Drug (IND)-enabling studies to support submission of applications to regulatory agencies. Jenny is a member of the American Society for Gene and Cell Therapy (ASGCT) and is part of the ASGCT New Investigator Committee. She is the author of 13 peer-reviewed scientific papers and is a co-inventor on five patent applications. 

  Close modal dialog

• 

  

  Juliette Hordeaux, DVM, PhD, DECVP

  Executive Director, Research
  

  Juliette Hordeaux has 13 years of experience in AAV-mediated gene therapy and translational research. She has a French Veterinary Medical Degree, has been European board certified in Veterinary Pathology since 2011, and received her PhD from the University of Nantes. She has been working at the University of Pennsylvania’s Gene Therapy Program (GTP) for seven years where she has led or co-led five programs through successful Investigational New Drug (IND) applications. Juliette currently oversees the neurology and cardiology portfolios, as well as investigative toxicology and histopathology. She leads a team of research directors, investigators, and specialists developing cutting-edge AAV gene therapies and working with internal core facilities and external industry partners to collect quality and focused preclinical data. Her team also investigates AAV-mediated toxicity and ways to develop safer gene therapy modalities. Juliette leads the GTP Histopathology Core that supports research and discovery activities as well as Good Laboratory Practice (GLP)-compliant toxicology studies. She has authored or co-authored more than 20 peer-reviewed articles, is a co-inventor on several patent applications, and is regularly invited to speak at events discussing AAV-mediated toxicity.

  Close modal dialog

• 

  

  Lori D. Kellam, PhD

  Executive Director, Portfolio Management
  

  Lori Kellam is the Executive Director of Portfolio Management for the University of Pennsylvania’s Gene Therapy Program (GTP). Lori has a PhD in Molecular Genetics from the University of Florida and conducted postdoctoral research at Temple University before spending nine years in the lab of Dr. John Gearhart at the University of Pennsylvania where she developed innovative strategies for cell-based therapies of infertility. Lori is the author of four peer-reviewed scientific publications. In 2017, Lori joined GTP as a project manager, coordinating and supporting the internal scientific team to execute translational research projects while maintaining strong and transparent relationships with GTP’s alliance partners. Since then, her role has matured and she now runs the Translational Project Management and Good Laboratory Practice (GLP) Program Management teams who are responsible for executing GTP’s research portfolio to deliver transformational gene therapy products to the clinic in tight coordination with Dr. James Wilson and other senior leaders.

  Close modal dialog

• 

  

  Mike LaBruto, MS

  Executive Director, Quality Assurance
  

  In his current role at the University of Pennsylvania’s Gene Therapy Program (GTP), Mike LaBruto leads a team responsible for managing quality assurance activities that support the development and commercialization of novel genetic-based therapeutics. Mike has more than 25 years of experience in the development and implementation of cGMP Quality Systems, supporting both small molecule and biologic manufacturing and testing facilities. Prior to joining GTP, Mike spent 15 years at GSK where he held a variety of senior leadership roles within quality assurance. Most recently, Mike served as the Executive Director of Quality for the One Pharma Supply Chain, including R&D Operations. Mike has presented quality systems topics at numerous pharmaceutical conferences and currently serves on the PDA/FDA planning committee supporting the annual joint regulatory conference. He holds a bachelor's degree in Biology with a minor in Chemistry from Liberty University and a master's degree in Pharmaceutical QA/RA from Temple University. Mike is also an adjunct professor at Temple University's School of Pharmacy where he teaches graduate courses in pharmaceutical management.

  Close modal dialog

• 

  

  Thomas Logan, PhD

  Chief Strategy and Business Officer
  

  In his current role at the University of Pennsylvania’s Gene Therapy Program (GTP), Thomas (Tom) Logan leads a team responsible for managing industry and nonprofit foundation alliance programs as well as pursuing new business development opportunities in support of GTP’s mission. Tom brings extensive biopharmaceutical industry experience to GTP. Prior to joining, he spent 17 years at BTG, a global healthcare company, where he held a variety of senior leadership roles in the areas of business development, alliance management, strategy, and research program management, most recently serving as Vice President of R&D Strategy and Operations. Tom has a PhD in Molecular Biology and Biochemistry from Thomas Jefferson University. He currently serves as a board member for several nonprofit organizations in the Philadelphia area.

  Close modal dialog

• 

  

  Monique Molloy

  Chief Administrative Officer
  

  Monique Molloy serves as the Chief Administrative Officer for the University of Pennsylvania’s Gene Therapy Program (GTP). In this role, she oversees general administration, human resources, communications, publications, laboratory operations, and information technology. Monique has a bachelor’s degree in Biology from Villanova University. She has worked within GTP since 1994, serving in various administrative capacities throughout the years. In addition, Monique has served as the Executive Director of the Orphan Disease Center (ODC) since 2015.

  Close modal dialog

• 

  

  William Singleton, DVM, DACLAM

  Executive Director, Program for Comparative Medicine
  

  William Singleton received his Doctor of Veterinary Medicine from Tuskegee University and is boarded in Laboratory Animal Medicine. He was the Associate Director for Clinical Care and Training at the University of Pennsylvania prior to his founding of Animal Care Training Services, a training and advisory consultancy for the biomedical research community. Starting his company has allowed him to bring together his personal purpose with the global need of helping people and improving scientific outcomes through better animal welfare. William has worked with many of the Fortune 500 pharmaceutical companies, large academic institutions, and fast-growing biotech companies providing leadership training and advice on best practices to grow a successful business. He has spent more than 30 years speaking and training on effective leadership, culture, and technical competence as a catalyst to create more productive and engaged workplaces.

  Close modal dialog

• 

  

  Simin Zaidi

  Chief Technology Officer
  

  Simin Zaidi has a degree in Bioprocess Engineering from the University of New South Wales in Kensington, Australia. As Chief Technology Officer (CTO) for the University of Pennsylvania’s Gene Therapy Program (GTP), Simin oversees AAV manufacturing and analysis in addition to supporting teams who work in CMC project management, technical, and business operations. Simin has more than 21 years of experience in the biopharmaceutical industry, therapeutic protein and AAV process development, manufacturing, and taking candidates through the increasing regulatory stringencies of early phase clinical through commercial use. Prior to her role as CTO, Simin served as the Executive Director of GTP’s Manufacturing Sciences and Technology (MS&T) team. Before joining GTP in 2019, she served as Director of Manufacturing Sciences at Cook Pharmica. Previously, Simin ran process development lab services, consulting businesses, and worked for a protein oncology therapeutic company in Southern California as head of Process Development for a decade. Simin has a graduate certificate in Business Management from the Kelley School of Business at Indiana University. She is an active member of the BioPhorum Operations Group (BPOG) with publications in the Parenteral Drug Association (PDA) and has both presented and published work on the topic of biologics process development.

  Close modal dialog