Sheba Medical Center Develops Israel’s First Personalized RNA-Based Drug
A multidisciplinary team at Sheba Medical Center has developed a personalized RNA-based therapy for an 8-year-old girl with GNAO1-neurodevelopmental disorder, a rare and severe genetic condition characterized by developmental delays, involuntary movements, and seizures.
Until now, available treatments have been limited to managing symptoms rather than treating the underlying cause. This newly developed customized genetic therapy is designed specifically for the patient to modulate the expression of the defective gene, aiming to address the root cause of the disorder.
Discovery, Development, and Delivery
The therapy developed at Sheba Medical Center is based on Antisense Oligonucleotide (ASO) technology—short strands of nucleic acids engineered to “silence” the defective and toxic copy of the GNAO1 gene, helping to restore normal cellular function.
To identify the most effective candidate, Sheba’s scientists developed a library of ASO molecules and evaluated their effectiveness in cell models derived from the patient’s own tissue. Through this process, they pinpointed a single ASO molecule with a specific sequence that reduced expression of the defective gene by up to 75% and restored neuronal activity.
Further validation was carried out in laboratory-grown nerve cells generated from the patient’s skin biopsy, showing improvements in both molecular and functional markers. To assess potential clinical benefit, a genetic animal model replicating the patient’s condition was also developed, demonstrating similar improvements in neuronal function.
The therapy was manufactured according to Good Manufacturing Practice (GMP) standards and underwent comprehensive toxicity testing, confirming a strong safety profile. Following review and authorization by Israel’s Ministry of Health under the compassionate use program, the patient received the first dose earlier this week.
The Strength of Multidisciplinary Innovation
The development of this therapy was made possible through close collaboration among Sheba’s leading scientists and clinicians. The core team included Dr. Dan Dominissini, Director of Diagnostics and Vice Chair of Research and Development; Dr. Nofer Mor, Head of the RNA Therapy Research Group; Prof. Gidi Rechavi, Director of the Cancer Research Center; and Prof. Bruria Ben-Zeev, Medical Director of the RNA Therapy Program.
This marks the first time in Israel—and one of the few instances worldwide—where a personalized RNA-based therapy has been created specifically for a patient, designed to modulate the expression of the defective gene. The achievement highlights Sheba’s multidisciplinary approach, enabling rapid innovation and advancement in medical care—from laboratory research to compassionate-use treatment. It also underscores Sheba Medical Center’s growing role in global precision medicine, setting a new standard for personalized therapeutic development.
Dr. Dan Dominissini, Head of Sheba’s Diagnostic Division and Deputy Director of the Research Authority, stated,
“The ability to develop a personalized drug tailored to a patient’s genetic sequence within a world-leading medical center, and in such a short time, reflects the revolution currently transforming medicine. RNA is at the heart of next-generation precision medicine, and Sheba is placing Israel at the forefront of this field.”
Prof. Bruria Ben-Zeev, former Director of the Pediatric Neurology Unit at the Edmond and Lily Safra Children’s Hospital and current Medical Director of the RNA Therapy Program, added,
“For the child and her family, this represents a tangible hope for improved daily functioning and quality of life. We anticipate that in the future, this approach will be expanded to help additional children with rare diseases for which no treatments currently exist.”
A New Chapter in Personalized Treatment
The development of this personalized RNA-based therapy reflects how medicine is gradually moving toward more precise and compassionate approaches to treatment. By combining scientific insight with clinical expertise, Sheba Medical Center continues to work toward translating medical innovation into personalized treatments for patients living with rare and complex conditions—helping to advance approaches that may one day transform care for children worldwide with untreatable rare diseases.
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