Wills Eye Director of Retina Research led surgery as part of gene therapy clinical trial 

PHILADELPHIA, January 3, 2023 – A collaboration between Wills Eye Hospital and Penn Medicine’s Scheie Eye Institute has led to improvements in night vision for patients with childhood-onset blindness. The clinical trial employs surgically-delivered AAV gene therapy to the retinas of patients with Leber Congenital Amaurosis (LCA) and within days each individual experienced improvements in their low-light sensitivity. Wills Eye Hospital’s Director of Retina Research and Co-Director of the Retina Service Dr. Allen C. Ho served as a key researcher and performed the retinal surgeries.  

LCA is an eye disorder presenting at birth that primarily affects the retina and is found in roughly one in 40,000 newborns, making it one of the most common congenital blindness conditions. The clinical study delivered gene therapy to the retina of one eye per patient in order to gauge the difference compared to the untreated eye. Patients’ eyes became thousands of times more light-sensitive in low light conditions, making a measurable difference in correcting the original issues caused by LCA.   

Dr. Ho worked alongside the clinical study’s lead author Dr. Samuel G. Jacobson and co-author Dr. Arthur V. Cideciyan. Both are professors of ophthalmology at Penn. Wills Eye Hospital’s retina research team has a long history of collaborating with the expert physicians at Scheie Eye Institute on projects and clinical research programs. These efforts have included research with patients afflicted with severe retina degeneration such as retinitis pigmentosa and LCA.  

“At Wills Eye, we have the surgical expertise, procedures, and hardware necessary to deliver gene and cell therapies as well as other devices for patients with severe vision loss,” said Dr. Ho. “Drs. Jacobsen and Cidecyan have followed patients for many years, and it is exciting that we have now advanced to clinical trials for a select few of the retinal degenerations potentially treatable through gene therapy.  Our Wills - Scheie collaborations allow us to leverage our individual strengths and attack vision loss in those afflicted by severe retinal degenerations.  We are energized by preliminary signs of safety and potential efficacy.” 

Full findings of the study can be accessed via iScience: https://www.sciencedirect.com/science/article/pii/S2589004222015462  

About Wills Eye Hospital:
 Wills Eye Hospital is a global leader in ophthalmology, established in 1832 as the nation's first hospital specializing in eye care. U.S. News & World Report consistently ranks Wills Eye as one of America's top ophthalmology Hospitals since the survey began, has the most nationally ranked ophthalmologists in the country and is among the top academic training programs in the U.S. for its residency program as voted by physicians in the nation. Wills Eye is a premier training site for all levels of medical education. Its resident and post-graduate training programs are among the most competitive in the country. The Vickie and Jack Farber Vision Research Center at Wills harnesses the core strengths of Wills, the close connection between innovative research and advanced patient care, to discover therapeutic breakthroughs. Wills provides the full range of primary and subspecialty eye care for improving and preserving sight, including cataract and primary eye care, cornea, emergency care, glaucoma, neuro-ophthalmology, ocular oncology, oculoplastics, pathology, pediatric ophthalmology, refractive surgery, retina, and strabismus. Ocular Services include the Wills Laser Correction Center and Diagnostic Center. Its 24/7 Emergency Service is the only one of its kind in the region. Wills Eye also has a network of six multi-specialty, ambulatory surgery centers throughout the tristate area. 


CONTACT: London Faust