July 1, 2026 | 9 a.m. | HNO Lecture Hall

We are vey pleased to announce the talk and visit at the IOR of Stephen H. Tsang from the Columbia University. 
Our guest, invited by Sven Schnichels and Pietro De Angeli will give a talk on “Precision Genome Surgery for Imprecision Medicine”

Abstract:
CRISPR genome editing has rapidly progressed from a futuristic concept to a clinically relevant therapeutic platform. The personalized treatment of Baby KJ, a child with CPS1 deficiency, illustrates the potential of mutation-specific base editing to deliver precise, potentially life-saving interventions. Recent regulatory approvals of CRISPR-edited hematopoietic stem cells for sickle cell disease and β-thalassemia further demonstrate the field’s transition from experimental promise to approved medicine, establishing important standards for safety, efficacy, and long-term monitoring. As the field advances toward in vivo genome editing, the eye has emerged as an especially attractive target because of its accessibility, low dosing requirements, and capacity for direct structural and functional monitoring. Together, these developments highlight both the urgency and transformative potential of CRISPR-based therapies, particularly for inherited diseases where durable, precision treatments are urgently needed.

SHORT INTRODUCTION:
Stephen H. Tsang, M.D, Ph.D. is an acclaimed clinical geneticist in the care of individuals with retinal degenerations. He has been culturing stem cells since 1992 and created the first mouse model for a recessive form of retinitis pigmentosa (RP) by applying genome engineering to stem cell technology in 1995. He successfully treated preclinical models of Pde6a, Pde6b, Mfrp, Rho, Cngb1 and autosomal recessive bestrophin retinopathies.

He has expertise in designing and testing genome engineering strategies in pre-clinical models, developing patient-specific knock-in models, generating of patient cell lines and providing care to patients with a precision medicine approach. He is also leading efforts in FDA trials for gene therapies, including PDE6A, RAB geranylgeranyl transferase, RPGR, CNGB3, CNGA3 and ABCA4 retinopathies.