Harold A. Stein, MD Lecture

Seeing the Light with Retinal Gene Therapy: From Fantasy to Reality

We are pleased to present Jean Bennett, MD, PhD, and Albert M. Maguire, MD
Saturday, October 12, 2019, at 8:00 am in the Continental Ballroom 4 & 5 (Hilton Union Square)

The Harold A. Stein, MD Lecture will be presented by Jean Bennett, MD, PhD, and Albert Maguire, MD, recipients of the Smithsonian Ingenuity Award. Drs. Bennett and Maguire will describe the path they took in moving exciting laboratory research findings into a treatment for individuals with an inherited form of congenital blindness. This path led to development of the first FDA-approved gene therapy product in the US and Europe and the first approved gene therapy for a blinding inherited retinal disease worldwide, that previously had no pharmacologic treatment options. The results have changed medical practice and provide hope to many individuals suffering from inherited forms of blindness. “It serves as a stepping stone to more prevalent diseases,” Maguire said.

Drs. Bennett and Maguire have established a true “from bench to bedside” program involving gene-based treatment strategies. Their team achieved many "firsts" including completion of the first randomized, controlled, multi-center Phase 3 gene therapy trial targeting a genetic disease.

The FDA approval is a culmination of over 25 years of studies on congenital blindness by the married-couple team Bennett and Maguire, who started this pioneering research in mice and dogs. Recommended therapy use by the FDA in patients ages 12 months and older, 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. A patient’s vision can begin to improve within weeks. Today, there are an estimated 1,000-2,000 patients in the United States with RPE65 mutations.

Dr. Jean Bennett is the F.M. Kirby Professor of Ophthalmology and Professor of Cell and Developmental Biology at the Perelman School of Medicine at the University of Pennsylvania and Penn’s Scheie Eye Institute. Dr. Albert Maguire is a Professor of Ophthalmology at the Perelman School of Medicine, University of Pennsylvania and Penn’s Scheie Eye Institute. He is also the Attending Physician in the Division of Pediatric Ophthalmology at Children’s Hospital of Philadelphia (CHOP) and served as the principal investigator of the clinical trials which led to the FDA approval. Both Bennett and Maguire are graduates of Harvard Medical School. Dr. Maguire completed his residency at The John Hopkins Hospital; and Dr. Bennett completed her PhD at the University of California, Berkeley.

We will learn firsthand the ingredients necessary to deliver a gene to cells in the retina and the importance of identifying appropriate tests for assessing efficacy of an intervention. They will discuss the rigorous process of testing a new drug and moving it through the drug approval process, that is critical to patients and the potential of gene therapy for treating retinal disease.

Admission ticket required. If you plan to attend, check the appropriate box on your registration form. 1 GROUP A IJCAHPO CREDIT