Minovia Therapeutics Reports First Clinical Data Demonstrating Disease-Modifying Efficacy and Safety of Mitochondrial Augmentation Therapy in Pediatric Patients with Primary Mitochondrial Diseases
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Wednesday, December 21, 2022
Immunodeficiency, Blood, Food, Therapy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Failure, Quality of life, Pearson syndrome, DNA, Mitochondrion, Trial of the century, HSPC, Disease, Sheba Medical Center, KSS, MAT, Patient, Health, Weight gain, Microdeletion syndrome, Tel HaShomer, A-DNA, Safety, Science Translational Medicine, Sideroblastic anemia, Pearson, Hematology, Stem cell, Vaccine, Pharmaceutical industry, Medical imaging
WOBURN, Mass. and HAIFA, Israel, Dec. 21, 2022 (GLOBE NEWSWIRE) -- Minovia Therapeutics, a clinical-stage global biotechnology company, today announced that the findings of the first clinical use of mitochondrial augmentation therapy (MAT) platform to treat pediatric patients with primary mitochondrial diseases have been published in Science Translational Medicine. The study was conducted in collaboration with global leaders in the field of hematology and primary mitochondrial disease at Sheba Medical Center (Tel Hashomer, Israel).
Key Points:
- and HAIFA, Israel, Dec. 21, 2022 (GLOBE NEWSWIRE) -- Minovia Therapeutics, a clinical-stage global biotechnology company, today announced that the findings of the first clinical use of mitochondrial augmentation therapy (MAT) platform to treat pediatric patients with primary mitochondrial diseases have been published in Science Translational Medicine.
- Single, large deletions in mitochondrial DNA (mtDNA) can lead to a variety of devastating diseases, including Pearson Syndrome and Kearns-Sayre Syndrome (KSS).
- Minovia is developing MAT to treat Primary Mitochondrial Diseases such as Pearson syndrome, as well as other mitochondrially-related sideroblastic anemias.
- "We believe these findings lay important groundwork for further development of MAT and future clinical trials to demonstrate the effectiveness and safety of MAT in patients with primary mitochondrial disorders and other diseases.