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For the First Time Ever, Sheba Delivers PR001 Gene Therapy to Child with Type 2 Gaucher

Type 2 Gaucher
February 2020 – Doctors at the Edmond and Lily Safra Children’s Hospital at Sheba Medical Center pioneered the administration of PR001, an investigational novel gene therapy drug, to treat a child with Type 2 Gaucher disease. After the Israel Ministry of Health approved the experimental administration of PR001, physicians at Sheba submitted a compassionate use request to Prevail Therapeutics Inc., the US-based biotechnology company that developed the gene therapy – and permission was granted.

Dr. Zion Zibly, Senior Physician at the Department of Neurosurgery, carried out the procedure at Sheba, injecting PR001 into the young patient’s cisterna magna. Still in an experimental phase, PR001 is an AAV9-based gene therapy delivering the GBA1 gene.

Gaucher disease is a rare neuronopathic disease that presents in infancy. Caused by mutations in GBA1, the glucocerebrosidase gene, it is a lysosomal storage disorder that leads to multi-organ pathology. When patients suffer a severe mutation in the GBA1 gene, they can present with Type 2 Gaucher disease, the more severe form of the disease. Typically, neurodegeneration progresses rapidly and leads to death in infancy or early childhood. At present, there are no FDA-approved therapies for the treatment of neuronopathic Gaucher disease.

Sheba is proud to be the first hospital in the world to administer this potentially disease-modifying gene therapy, which is a great example of the work we’re doing to help advance potential new therapeutic modalities for inborn errors of metabolism,” said Prof. Yair Anikster, Director of the Metabolic Disease Unit.

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