Invitation to Cover
james wilson ODC lab

PHILADELPHIA— In celebration of the 10th Anniversary of the Orphan Disease Center, pioneering gene therapy scientist James Wilson, MD, PhD, a professor and director of the Penn Gene Therapy Program and the Penn Orphan Disease Center at the Perelman School of Medicine (PSOM) at the University of Pennsylvania, will lead a two-day symposium, featuring former Prime Minister of the United Kingdom David Cameron, and National Institutes of Health Director Francis Collins, MD, PhD, among many others. The event will take place on Wednesday, February 24th and Thursday, February 25th.

The ODC marks this anniversary in a phase of unprecedented hope and opportunity for patients and families in the rare disease community. “We’ve gone from an entire area of medicine with no treatments and little science, to a situation where we’re looking at lifesaving therapies — efforts like those of the ODC have transformed what it means to be living with a rare disease,” Wilson said. “When I look at patients, the stunning difference from ten years ago when we started, and now, is hope.”

The ODC is the first center of its kind, dedicated to identifying promising therapies for rare diseases and tackling obstacles to rare disease drug development. In particular, the ODC focuses on the study of diseases with few, or in many cases no, treatment options — and aims to provide access to patients of all populations. The center has awarded nearly $38 million to more than 120 institutions in 25 countries since its founding in 2011.

Wilson’s lab has been pioneering gene therapies for decades. He discovered a new family of adeno-associated viruses and developed them into gene therapy treatments that deliver healthy DNA into the correct cells. Wilson developed this technology largely to treat rare and orphan diseases.

In the biopharma industry, there are 82 preclinical programs and 41 clinical programs that use Wilson’s AAV vectors. Approximately 58% of classic gene therapy trials use Penn–developed technology coming from Wilson’s lab. In 2019, the Wilson lab celebrated the FDA approval of Zolgensma, the first approved drug for the treatment of spinal muscular atrophy, and is now collaborating with Regeneron to study use of AAV vectors to deliver Regeneron’s COVID-19 antibody cocktail for prevention and treatment of COVID-19.

Other prominent speakers at the event will include Janet Woodcock, MD, acting commissioner of the U.S. Food and Drug Administration and director of the FDA’s Center for Drug Evaluation and Research, and Michael S. Brown, MD, PSOM alumnus and a professor at University of Texas Southwestern and recipient of the Nobel Prize in Physiology or Medicine. Topics of discussion will include the ethics of rare disease research, genomics in rare disease diagnostics, patient registries, industry perspectives, and global access.



Virtual Event, hosted on ZOOM













Wednesday, February 24, 2021
10:00 AM EST – 2:30 PM EST


Thursday, February 25, 2021
10:00 AM EST – 3:00 PM EST



Full agenda can be accessed here



  • James Wilson, MD, PhD, director of the Penn Orphan Disease Center and Penn Gene Therapy Program
  • Francis Collins, MD, PhD, director of the National Institutes of Health
  • David Cameron, former Prime Minister of the United Kingdom and parent in the rare disease community
  • Janet Woodcock, MD, acting commissioner of the U.S. Food and Drug Administration and director of the FDA’s Center for Drug Evaluation and Research
  • Michael S. Brown, MD, a professor of Molecular Genetics and Internal Medicine at the University of Texas Southwestern and Nobel Prize winner
  • Lynn Barghout Jafar, co-founder of the Loulou Foundation and parent in the rare disease community
  • Pat Furlong, founding president and chief executive officer of Parent Project Muscular Dystrophy (PPMD)


  • Expert panels on ethics, access, and treatments will include leading scientists, patients, parents, and pharmaceutical researchers


Penn Medicine is one of the world’s leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of the Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nation’s first medical school) and the University of Pennsylvania Health System, which together form a $8.9 billion enterprise.

The Perelman School of Medicine has been ranked among the top medical schools in the United States for more than 20 years, according to U.S. News & World Report's survey of research-oriented medical schools. The School is consistently among the nation's top recipients of funding from the National Institutes of Health, with $496 million awarded in the 2020 fiscal year.

The University of Pennsylvania Health System’s patient care facilities include: the Hospital of the University of Pennsylvania and Penn Presbyterian Medical Center—which are recognized as one of the nation’s top “Honor Roll” hospitals by U.S. News & World Report—Chester County Hospital; Lancaster General Health; Penn Medicine Princeton Health; and Pennsylvania Hospital, the nation’s first hospital, founded in 1751. Additional facilities and enterprises include Good Shepherd Penn Partners, Penn Medicine at Home, Lancaster Behavioral Health Hospital, and Princeton House Behavioral Health, among others.

Penn Medicine is powered by a talented and dedicated workforce of more than 44,000 people. The organization also has alliances with top community health systems across both Southeastern Pennsylvania and Southern New Jersey, creating more options for patients no matter where they live.

Penn Medicine is committed to improving lives and health through a variety of community-based programs and activities. In fiscal year 2020, Penn Medicine provided more than $563 million to benefit our community.

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