Clinical Research Directory

Investigation of the Treatment of Patients With Mitochondrial Defects by Assisted Reproductive Technology

Mitochondria are important organelles that generate energy (ATP) in human and mammalian cells. Currently, there are more than 50 kinds of diseases known to be related to mitochondrial defects in Taiwan. The incidence of mitochondrial diseases is very rare, about 1/5,000 to 1/10,000. The abundancy of mitochondria riches to 100,000 DNA copies in one oocyte, and the threshold of DNA mutation rate to cause pathogenicity is usually greater than 70%. Since mitochondria are inherited maternally, the pathogenic mitochondrial DNA in oocytes could be removed by modern artificial reproductive technology (ART). By treating with mitochondria replacement therapy (MRT), here we use the spindle transfer, following artificial insemination, the ooplasm from healthy donor oocytes could be transferred into the oocytes of patients to remove defected mitochondria, therefore creating the opportunity to produce healthy offspring. Therefore, in order to further detect the carrying rate of mutated mitochondrial genes in patients with mitochondrial defects after MRT, we aim to detect the mitochondria mutation in germ cells of patients in order to improve the specificity and operation procedures of this therapy.

Evaluate the Safety and Therapeutic Effects of a Single Intravenous Infusion (IV) of Autologous CD34+ Cells Enriched With Allogenic Placenta-derived Mitochondria in Patients With a Diagnosis of Pearson Syndrome (PS)

Primary Mitochondrial diseases are a clinically and genetically heterogeneous group of disorders caused by mutations in genes encoded by nuclear Deoxyribonucleic Acid (DNA) or by mutations and/or deletions in the mitochondrial DNA (mtDNA). While some mitochondrial disorders only affect a single organ (e.g., the eye in Leber hereditary optic neuropathy \[LHON\]), many involve multiple organs. Mitochondrial disorders may present at any age and a frequent feature is the increasing number of organs involved in the course of the disease. Minovia Therapeutics Ltd. ("Minovia") is a biotech company developing novel therapeutics based on its mitochondrial augmentation technology (MAT). MNV-201 is a cell therapy produced by MAT that consists of the participant's autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) enriched with allogeneic placental-derived mitochondria, manufactured in Minovia's GMP facility.

Global Registry and Natural History Study for Mitochondrial Disorders

The main goal of the project is provision of a global registry for mitochondrial disorders to harmonize previous national registries, enable world-wide participation and facilitate natural history studies, definition of outcome measures and conduction of clinical trials.