We are proud to congratulate Prof. Rami Aqeilan on a truly outstanding scientific and clinical achievement. An infant with WOREE syndrome (WWOX-related epileptic encephalopathy) has become the first patient in the world to receive an experimental gene replacement therapy designed to restore WWOX function in the brain, marking a major milestone in precision medicine for rare neurological disorders.
This achievement is the culmination of more than a decade of research led by Prof. Aqeilan and his collaborators. While WWOX was initially studied for its role as a tumor suppressor gene in cancer, Prof. Aqeilan’s work uncovered its essential functions in brain development. Through innovative preclinical studies, his laboratory demonstrated that loss of WWOX causes severe neurological abnormalities, including epilepsy, developmental delay, impaired myelination, and premature death, findings that closely mirror the clinical features of WOREE syndrome. The effort brought together scientists, clinicians, and biotechnology leaders from Israel and the United States.
These discoveries laid the foundation for the development of a gene replacement strategy using an AAV9 vector to deliver a functional copy of WWOX to neurons. The successful translation of these fundamental biological insights into a first-in-human therapeutic intervention represents a remarkable example of how basic research can drive transformative advances in patient care.
Congratulations to Rami and the entire research, clinical, and biotechnology team on this historic accomplishment and on bringing new hope to patients and families affected by WWOX-related disorders worldwide.