Welcome Back From the President
Celebrating Penn People
Welcome back from winter break. I want to begin the New Year by thanking you, Penn’s unsurpassed faculty, students, staff, alumni, and friends, for all the good you do here and in the world. Together, we have propelled our University to unprecedented heights of inclusion, innovation, and impact. Together, we will do even more in the semester to come.
We can gauge Penn’s rise by the world-improving and life-saving work of our faculty, such as the first-ever FDA-approved gene therapy treatment for cancer. We can measure our reach with rankings, with our University being named #4 in Reuters’ list of the top 100 most innovative universities in the world (up from #8 the previous year). Penn also moved up to #3 from #17 in the most recent National Science Foundation Education R&D Survey. We can also be enormously proud of the countless achievements of Penn students, faculty, and staff who, each year, bring great honor and recognition to our University. I want to briefly recognize just a few examples.
Seniors Chris D’Urso and Zikri Jaafar recently earned prestigious Rhodes scholarships for graduate study at Oxford. They both share a passion for community engagement, human rights, and social justice, and their work embodies the finest attributes of Penn scholars who are working to make a profound difference in the world. Chris has focused on consumer protection and advocacy as the founder of Penn CASE (Consumer Assistance, Support & Education), which is providing education and support to local consumers in our community. He also has been actively engaged on the Task Force on a Safe and Responsible Campus Community. Zikri’s undergraduate research has focused on social-impact bonds. He also has done volunteer work with refugees and has been an ambassador for Penn’s Giving What We Can. Chris and Zikri are extraordinarily talented, engaged, and committed individuals who are deeply deserving of this preeminent recognition. Congratulations to our latest Rhodes Scholars!
Penn also boasts seven new faculty inductees to the prestigious National Academy of Medicine (formerly called the Institute of Medicine). Representing SAS, Penn Nursing, and the Perelman School, these exceptional faculty have made seminal contributions to the advancement of the medical sciences, health care, and public health. I invite everyone to join me in congratulating Lewis Chodosh, Christos Coutifaris, Maria Oquendo, Michael Parmacek, Therese Richmond, Dorothy Roberts, and Flaura Winston.
We are enormously proud as well of Penn’s four new faculty fellows of the American Association for the Advancement of Science, the world’s largest general scientific society. Representing SAS, the Perelman School, and Penn Vet, these extraordinary faculty have been honored for their scientifically and socially distinguished efforts. We congratulate Gustavo Aguirre, Daniel José Mindiola, Hongzhe Li, and Anil Rustgi.
As we celebrate these and the many other accomplishments of Penn’s people, I want to remind our seniors and their mentors that the deadline for both the President’s Engagement Prize and the President’s Innovation Prize is January 19. These Prizes not only publicly recognize and reward creative projects that promote the greater good. They also proclaim our most cherished values as a university: to educate and support our students for meaningful leadership and lifelong citizenship.
Each Prize bestows a generous living allowance for one year after graduation and up to $100,000 in project expenses. All full-time undergraduates who will graduate in May, August, or December of this award year are eligible to apply. I encourage seniors with a great idea to submit an application, and I very much look forward to receiving them.
Embarking on the spring semester, I encourage all of us not only to celebrate the wonderfully diverse and creative community we call home, but also to be mindful of our own health and the wellbeing of those around us. We rightfully focus on the wellness of our students, but I want to emphasize that faculty and staff should also reach out if they are in need, take advantage of the resources available to the Penn community, and care for one another. I am grateful for all the students, faculty, and staff who are working so passionately on this important priority, and we will continue to do all that is possible to sustain a campus community in which everyone can thrive.
To the entire Penn community: Welcome back to campus, and welcome to a bright new year at Penn!
FDA Approval: Gene Therapy Developed by Penn and CHOP for Inherited Blindness
In a historic move, the U.S. Food and Drug Administration (FDA) recently approved a gene therapy initially developed by researchers at the University of Pennsylvania and Children’s Hospital of Philadelphia (CHOP) for the treatment of a rare, inherited form of retinal blindness. The decision marks the nation’s first gene therapy approved for the treatment of a genetic disease, and the first in which a new, corrective gene is injected directly into a patient.
The therapy, known as LUXTURNA™ (voretigene neparvovec-ryzl), significantly improves eyesight in patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Patients with RPE65 mutations suffer from severe visual impairment at infancy or early childhood, and by mid-life become totally blind. They previously had no pharmacologic treatment options.
Spark Therapeutics, a Philadelphia biotechnology company created in 2013 by CHOP in an effort to accelerate the timeline for bringing new gene therapies to market, led the late-stage clinical development of LUXTURNA and built in West Philadelphia the first licensed manufacturing facility in the U.S. for a gene therapy treating an inherited disease. Spark was built on the foundational research conducted over a 10-year period by CHOP’s Center for Cellular and Molecular Therapeutics (CCMT). Those efforts were led by Jean Bennett, the F.M. Kirby Professor of Ophthalmology at the Perelman School of Medicine at the University of Pennsylvania and Penn’s Scheie Eye Institute, and Katherine A. High, who directed the CCMT and now serves as Spark’s president and head of research and development. Albert M. Maguire, a professor of Ophthalmology at the Perelman School of Medicine and an attending physician at CHOP, served as the principal investigator of the clinical trials that led to the FDA approval.
The approval is a culmination of more than 25 years of studies on congenital blindness by married-couple team Drs. Bennett and Maguire at Penn and CHOP, starting with pioneering work in mice and dogs.
“I’ve witnessed the dramatic changes in the vision of patients who would have otherwise lost their sight, and feel exhilarated that this therapy will now make a difference in the lives of more children and adults,” Dr. Bennett said. “I’m hopeful that the path we’ve made with this research, with the help of our collaborators near and far, will be useful to other groups, so that other gene therapies can be developed faster and help more people with other diseases.”
This marks the second FDA approval for a University of Pennsylvania/CHOP-developed therapy within six months. In August, the personalized cellular therapy known as Kymriah™ was approved for the treatment of advanced acute lymphoblastic leukemia in children and young adults (Almanac September 12, 2017).
There are an estimated 1,000 to 2,000 patients in the United States with RPE65 mutations. The newly approved therapy will be available at select treatment centers across the nation.
The one-time therapy corrects the deficits resulting from mutations in the gene RPE65, which is responsible for producing proteins that make light receptors work in the retina and vision possible. To restore production of those proteins, corrected versions of the RPE65 gene are delivered in a single injection, using a genetically engineered, benign adeno-associated virus to carry the genes to the retina. Within weeks, a patient’s vision can begin to improve. The FDA recommends the use of the therapy for patients ages 12 months and older.
“Today’s landmark approval is a great moment for science and the many individuals and families who live with genetic disease,” Dr. High said. “One of the hopes of the Human Genome Project had been that it would be possible to develop gene therapy to expand therapeutic options for people with genetic disease. Now that hope is a reality. We offer our sincere gratitude to the patients and their families as well as the expert investigators who continue to participate in this and other clinical development programs.”
The Penn gene therapy work tightly links animal and human health, having begun in affected mice and dogs during the 1990s. In 2001, Bennett and Maguire and Penn colleagues Greg Acland, Tomas Aleman, Samuel Jacobson, and Artur Cideciyan reported they had successfully restored the sight of three blind dogs with a canine form of LCA, in collaboration with Cornell University’s Gus Aguirre (now a professor of Medical Genetics and Ophthalmology at the University of Pennsylvania School of Veterinary Medicine) and the University of Florida’s Bill Hauswirth. After the therapy, the dogs freely navigated through a dimly lit obstacle course, whereas before they would bump into objects.
The first Penn/CHOP-led Phase 1/2 clinical studies began in late 2007, with a total of 12 patients ranging from eight to 46 years old, most of whom experienced vision improvements within weeks of receiving the therapy. Half of the patients improved enough to no longer be classified as legally blind. Those initial clinical trials brought together patients and scientists from Penn and the Second University of Naples in Italy and Ghent University Hospital in Belgium. The first three patients to receive the therapy in 2007 are all now in their 20s and 30s, and continue to enjoy their improved vision.
Results presented in 2015 from a pivotal phase III trial of 29 patients aged four to 44, some of whom were treated at the University of Iowa, showed the therapy had significantly improved their ability to navigate an obstacle course designed to mimic daily activities in low light. The therapy dramatically restored most patients’ ability to see, increased their sensitivity to light, and improved their side vision, the researchers reported.
In the trials, the gene therapy safety profile was consistent with vitrectomy and subretinal injection procedures. Side effects included cataracts, changes in intraocular pressure, changes in macular structure (macular thinning, macular pucker, macular hole), intraocular infection in one patient, and a reduction in visual acuity in one patient.
To date, a total of 41 patients have been treated with the therapy at CHOP and Iowa.
Many of them are now reading the chalkboard, grocery shopping, taking driver’s license tests, having more job opportunities, and recognizing people’s faces, among other activities that seemed impossible before. “It has been amazing watching them grow up,” Dr. Bennett said. “It’s like they are an extended part of our family.” Drs. Bennett and Maguire have also adopted two dogs, Mercury and Venus, that were part of early trials of the new approach.
“The approval of the gene therapy approach for LCA opens up the door to develop therapies that target other mutations behind hereditary blindness and retinal diseases, and emphasizes the importance of genetic testing so that people living with inherited diseases can potentially benefit from gene treatments as they emerge,” Dr. Maguire said. “It also serves as a stepping stone to more prevalent diseases.”
A gene therapy clinical trial that delivers a corrected gene in patients with wet macular degeneration, for instance, is already underway at centers including the Scheie Eye Institute. Today, patients with this disorder must receive injections of a much-needed protein once a month. Using gene therapy could bring that down to just a single injection. Similar types of therapy may also help treat other diseases, such as hearing-related problems and muscular dystrophy, for example.
Additional contributors to the research include Jeannette Bennicelli, J. Fraser Wright, Jennifer Wellman, Federico Mingozzi, Manzar Ashtari, Junwei Sun, Kathleen Marshall, Nadine Dejneka, Vibha Anand, Arkady Lyubarsky, Valder Arruda, Ken Shindler, Dan Chung, Sarah McCague, Dominique Cross, Julie DiStefano-Pappas, T. Michael Redmond, Kristina Narfstrom, Julia Haller, Alberto Auricchio, Enrico Surace, Tim Hopkins, Tonia Rex, Eric Pierce, Michael Ward, Ali Zaidi, Jason Ruggiero, Dina Gewaily, Edwin Stone, Francesca Simonelli, Bart LeRoy, and Stephen Russell.
Consultative Committee for the Selection of a Vice Provost and Director of Libraries
Provost Wendell Pritchett announces the formation of an ad hoc consultative committee to advise him on the selection of the University’s next Vice Provost and Director of Libraries. H. Carton Rogers, who has served as Vice Provost and Director of Libraries since 2004 and worked in the Penn Libraries since 1975, will retire from Penn at the end of this academic year.
“Over more than four decades at Penn,” said Provost Pritchett, “Carton Rogers has made our Libraries into vibrant hubs of activity, brilliantly combining respect for the past with vision for the future. He has been a particular leader in charting the future of research libraries, both nationally and here on campus, and in reimagining the Van Pelt-Dietrich Library Center, in the heart of campus, as a wide-ranging center for collaborative learning and new ideas, while sustaining its essential role as a catalyst for study and scholarship. He has built an extraordinary legacy of innovation and imagination for his successor—and for the benefit of all of us in the Penn community.”
The committee welcomes nominations and input from all members of the Penn community, which can be sent to firstname.lastname@example.org by February 28, 2018.
The members of the committee are:
Wendell Pritchett, Provost and Presidential Professor of Law and Education
Jeffrey Kallberg, Associate Dean for Arts and Letters and William R. Kenan Jr. Professor of Music, School of Arts and Sciences
Jonathan Moreno, David and Lyn Silfen University Professor in the Perelman School of Medicine and the School of Arts and Sciences
Jennifer Pinto-Martin, Viola MacInnes/Independence Professor of Nursing; Chair-Elect, Faculty Senate
Bethany Wiggin, Associate Professor of German, School of Arts and Sciences
Hannah Bennett, Director, Fine Arts and Museum Libraries
Paul George, Director, Biddle Law Library
Thomas Murphy, Vice President for Information Technology and Chief Information Officer
Julian Siggers, Williams Director, Penn Museum of Archaeology and Anthropology
Miles Owen, President, Graduate and Professional Student Assembly; School of Design and Fels Institute of Government
Shawn Srolovitz, SEAS ’18, Past Chair External (2017), Student Committee on Undergraduate Education
Judith Bollinger, WG ’81
Erik Gershwind, W ‘93
Staff to the Committee
Lynne Hunter, Associate Provost for Administration
Consultants to the Committee
Jonathan Fortescue, Park Square Executive Search
Anne Coyle, Park Square Executive Search
MLK Day: At Penn, A Day of Service and Beyond
The University of Pennsylvania is closed on Monday, January 15, in observance of Martin Luther King, Jr. Day. The Dr. Martin Luther King, Jr. Commemorative Symposium on Social Change begins with the January 15 Day of Service and continues with events taking place through January 31.
For the latest information on the numerous events, visit www.upenn.edu/aarc/mlk
After Martin Luther King, Jr.’s assassination on April 4, 1968, some members of Congress proposed that his birthday should be a national holiday. However, it was not until the Ronald Reagan administration that a bill passed and his birthday was designated a national holiday to be celebrated on the third Monday in January starting in 1986 (Almanac April 25, 2000). Penn focused its energy and attention on community service to realize Dr. King’s vision of a “beloved community.”
During the administration of Penn President Judith Rodin, the University then made MLK Day an official holiday at Penn as of January 2001, making it “a day on not a day off.” It was then that the annual Day of Service was launched as part of the extensive Commemorative Symposium (Almanac December 12, 2000). Penn’s Commitment to the Legacy: 2018 Dr. Martin Luther King Jr. Commemorative Symposium on Social Change includes opportunities for service, lectures, performances, an interfaith program and awards ceremony. The Symposium is coordinated by the African-American Resource Center, (215) 898-0104. Events are free and open to the community.
Student Diplomat/Junior Model UN Program for Middle School Students
A Cultural Diplomacy Conference for Middle School Students presented by the World Affairs Council of Philadelphia and the Penn Museum is the kick-off event of a rigorous Student Diplomat/Jr. Model United Nations Program, a five-month series of programs introducing students from around the Greater Philadelphia area to the world of international relations and cultural diplomacy. With an emphasis on developing students’ research, writing, and communication skills, the Student Diplomat Program meets a growing demand for international affairs curricula in area middle schools, preparing students for successful futures in an increasingly interconnected world.
Students come to the University of Pennsylvania Museum of Archaeology and Anthropology on one of three days (250 students per day) in early January. At the Penn Museum, students will be introduced to a wide and culturally diverse world beyond their classroom walls, via docent tours of the renowned international collections and conversations with Museum International Classroom speakers from China, Greece, and Kenya, and archaeologists who have spent extensive time in Egypt and Italy. The conference program includes a keynote welcome and closing from World Affairs Council speakers, setting the stage for intercultural learning and framing the lessons that will follow in the spring semester.
The World Affairs Council, a non-profit organization whose mission is to create global citizens, devotes much of its energy to education. The Cultural Diplomacy Conference is for middle school students from all across the region in an effort to educate and prepare them to contribute to and succeed in a 21st century learning environment. The World Affairs Council brings students from public, private and charter schools around the Greater Philadelphia area together to debate and discuss issues of global importance.
Over the course of five months, 750 middle school students will gather to examine global conflicts and trends while working together to create promising solutions to some of the world’s greatest challenges. The program emphasizes building cultural competency and diplomatic skills; expanding knowledge of world geography, cultures and political structures; and increasing understanding of how national and international institutions tackle key global issues. This year’s focus: “Ending Food Insecurity” and “Protecting Women’s and Girls’ Access to Education.”