Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-201 in Pediatric Patients With Pearson Syndrome

Overview

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.

Eligibility

Inclusion Criteria:

1. Male or female participants aged from 1 to 18 years old.
2. Diagnosis of Pearson Syndrome
3. Body weight ≥ 10 kg.
4. Participant has anemia and/or thrombocytopenia, and/or leukopenia and/or blood transfusion dependent (receives blood transfusions every 6 weeks or less).
5. Participant is medically able to undergo the study interventions, as determined by the investigator.
6. Participant's living parent(s) and/or legal guardian(s) able to understand and provide voluntary written informed consent.

Exclusion Criteria:

1. History of infection with HIV-1, HIV-2, and HTLV I/II.
2. Current active infection with HBV (including HBcore and HBsAg positive), HCV, HTLV I/II, Treponema Pallidum and HIV I-II
3. Participant has been diagnosed with Myelodysplastic Syndrome, by FISH and/or karyotype.
4. Participant is unable to undergo leukapheresis.
5. Total number of CD34+ cells collected is lower than 20x106 cells
6. Participant has known hypersensitivity to murine proteins or iron-dextran.
7. Participant has chronic severe infection.
8. Participant has disease or condition that may risk the participant or interfere with the ability to interpret the study results.
9. History of malignancy
10. History of treatment with gene therapy, bone marrow or allogeneic cord blood transplantation.
11. Currently participating in another clinical trial, or participation in another clinical trial within 1 year prior to study enrollment.
12. In the opinion of the Investigator, the participant is unsuitable for participating in the study for any reason.