First Mitochondrial Cell Therapy Trial at Israel’s Sheba Medical Center, Tel HaShomer, Offers Hope for Pearson Syndrome Patients

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In collaboration with Minovia Therapeutics, Sheba Medical Center has taken part in the first ever mitochondrial cell therapy trial aimed at changing the course of the devastating mitochondrial disease Pearson syndrome. The first trial patient was dosed at Sheba Medical Center, Tel HaShomer in Israel, and soon clinical operations will expand to the U.S. operations of Minovia Therapeutics Inc.

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The clinical and lab improvement evidenced in the first three patients treated by Dr. Jacoby and team under compassionate use encourages us to continue with the current clinical study and in the future hopefully to implement this technique in the treatment of other mitochondrial diseases.

A global medical collaboration between Sheba Medical Center, Tel HaShomer in Israel and Minovia Therapeutics has made history as the first ever mitochondrial cell therapy trial undertaken to treat Pearson Syndrome, a mitochondrial disease. Pearson Syndrome affects approximately 100 children globally and is caused by a deletion in mitochondrial DNA that leads to poor energy production by the cells in the body, often resulting in problems with the bone marrow and the pancreas.

Three children with Pearson syndrome have already been treated under compassionate use, and one patient was dosed under a Phase I/II trial that recently started. In total, the trial will include seven pediatric patients. The patients will receive single doses to determine the safety and therapeutic effects of transplantation of cells enriched with maternal blood-derived mitochondria. After one year, treatment related adverse events, improvement in the quality of life as measured by the IPDMS questionnaire, and changes in cognitive status and muscle function will be used to determine the outcome of the trial.

“Although our main expertise is treating children with cancer, our vast clinical and lab experience with stem cell transplantations and cellular therapies enabled a smooth and uneventful entrance to the project,” said Professor Amos Torren M.D., Ph.D., MHA, head of Sheba Medical Center’s Division of Pediatric Hemato-oncology and principal investigator of the study, and Sheba’s Elad Jacoby, M.D., head of pediatric immunotherapy center co-PL. “The clinical and lab improvement evidenced in the first three patients treated by Dr. Jacoby and team under compassionate use encourages us to continue with the current clinical study and in the future hopefully to implement this technique in the treatment of other mitochondrial diseases.”

Minovia, based in Haifa, has also recently opened its U.S. operations in Cambridge, Massachusetts to facilitate clinical and research collaborations with medical and academic institutions in addition to biotech and pharmaceutical companies across North America.

“The trial of MAT in Pearson Syndrome is an important step toward addressing mutations and deletions of mitochondrial DNA, because there are many other mitochondrial diseases with no available therapies,” said Natalie Yivigi Ohana, Ph.D., Co-Founder and Chief Executive Officer of Minovia. “If it is successful, it will not only provide improved quality of life for those living with Pearson syndrome; it could pave the way for the development of treatments for all mitochondrial diseases.”

About Sheba Medical Center, Tel HaShomer
Born together with Israel in 1948, Sheba Medical Center, Tel HaShomer is the largest and most
comprehensive medical center in the Middle East. Sheba is the only medical center in Israel that combines an acute care hospital and a rehabilitation hospital on one campus, and it is at the forefront of medical treatments, patient care, research and education. As a university teaching hospital affiliated with the Sackler School of Medicine at Tel-Aviv University, it welcomes people from all over the world indiscriminately. In 2019, Newsweek magazine named Sheba one of the top ten hospitals in the world. To learn more, visit: eng.sheba.co.il.

About Minovia
Minovia Therapeutics, a clinical-stage international biotechnology research company, is the first company to use mitochondrial cell therapy to treat mitochondrial diseases through our Mitochondrial Augmentation Therapy (MAT) platform. Our initial clinical focus is on rare mitochondrial diseases for which there are no approved treatments, and we are committed to exploring the full potential of our MAT platform to address a range of disorders associated with mitochondrial dysfunction. Minovia is headquartered in Haifa, Israel, with operations in Cambridge, Massachusetts. For more information, visit http://minovia.com/

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