C-O7HAmitochondrial transplantation, mitochondrial augmentation, biomarkers
0-100 chain-logic scale · 15 dimensions · scored on public evidence
Develop a first-in-class mitochondrial therapy for myelodysplastic syndrome.
Mitochondria-based therapeutic approach using Minovia's Mitochondrial Augmentation Technology platform.
FDA Orphan Drug Designation for MNV-201 in myelodysplastic syndrome, adding to Fast Track Designation.
Quantify endogenous mitochondrial DNA copy number for understanding mitochondrial dysfunction and potential clinical diagnostics or point-of-care applications.
Amplification-free solid-state nanopore assay with selective degradation of linear genomic DNA and support-vector-machine classification to directly count and classify mtDNA molecules from biological samples.
ACS Nano publication described validation in plasmids, isolated mtDNA, cancer cells, and blood cells, with comparison to qPCR mtDNA/nuclear DNA ratios.
The assay detected mtDNA at picomolar levels, showed high classification accuracy, required minimal sample preparation, and avoided amplification or purification steps.
Evaluate MNV-201 as a potential therapy for patients with low-risk myelodysplastic syndrome, an aging-associated bone marrow failure condition.
Autologous CD34+ hematopoietic stem and progenitor cell therapy enriched with allogeneic placental-derived mitochondria using Minovia's MAT process.
ClinicalTrials.gov study record published for evaluation of MNV-201 in patients with low-risk MDS.
Evaluate MNV-201 in patients with low-risk myelodysplastic syndromes to reduce disease-associated symptoms, cytopenia-related risk, disease progression, and mortality risk.
Autologous CD34+ hematopoietic stem and progenitor cell therapy enriched with allogeneic placental-derived mitochondria using Minovia's mitochondrial augmentation technology and manufactured in Minovia's GMP facility.
ClinicalTrials.gov study record published for evaluation of MNV-201 in low-risk MDS.
Collect blood and skin samples from patients with primary mitochondrial diseases and healthy volunteers to support MAT development, study baseline heteroplasmy and hematopoietic-cell functionality, identify biomarkers for mitochondrial content and function in liquid biopsies, and study MAT efficacy in different PMDs.
Clinical sample collection from patients and healthy volunteers for cellular, heteroplasmy, biomarker, and MAT-development studies.
ClinicalTrials.gov sample-collection study record published.
Collect blood and skin samples from primary mitochondrial disease patients and healthy volunteers to support MAT development, characterize baseline heteroplasmy and cell functionality, identify liquid-biopsy biomarkers, and study MAT efficacy across PMDs.
Collection of patient and healthy volunteer blood and skin samples for cellular, heteroplasmy, functional, biomarker, and MAT efficacy studies.
ClinicalTrials.gov sample-collection study record published.
Treat Pearson syndrome and related single large-scale mitochondrial DNA deletion disease using mitochondrial augmentation therapy.
Autologous CD34+ hematopoietic cells augmented ex vivo with maternally derived healthy mitochondria using mitochondrial augmentation therapy.
FDA clearance of a second IND application for a Phase II clinical trial of lead product MNV-201 in Pearson syndrome.
In compassionate use treatment of six children with single large-scale mitochondrial DNA deletion syndromes, MAT was reported as well tolerated; heteroplasmy decreased in peripheral blood in four of six patients, mtDNA content increased in all six at 6 to 12 months, and some functional, weight, quality-of-life, and physical exam improvements were observed.
Evaluate the safety and therapeutic effects of a single intravenous infusion of autologous CD34+ cells enriched with allogeneic placenta-derived mitochondria in patients diagnosed with Pearson syndrome.
Single IV infusion of autologous CD34+ HSPCs enriched with allogeneic placental-derived mitochondria using mitochondrial augmentation technology.
ClinicalTrials.gov study record published for Pearson syndrome evaluation.
Treat patients with single large-scale mitochondrial DNA deletion syndromes, including Pearson syndrome and Kearns-Sayre syndrome, where no disease-modifying therapies exist.
Compassionate-use treatment using autologous CD34+ hematopoietic cells augmented with maternally derived healthy mitochondria.
Six patients with SLSMDs were treated through a compassionate-use program reported in Science Translational Medicine.
Procedure was well tolerated; study-related severe adverse events were leukapheresis-related or baseline-disorder-related. Peripheral blood heteroplasmy decreased in four of six patients; mtDNA content increased in all six at 6 to 12 months; some aerobic-function, weight, quality-of-life, and physical-exam improvements were reported.
Develop mitochondria-based therapies for diseases linked to mitochondrial dysfunction by augmenting patient cells with healthy functional mitochondria and measuring mitochondrial health.
Transplantation or ex vivo augmentation of hematopoietic stem and progenitor cells with exogenous healthy mitochondria, supported by analytical assays and biomarkers.
Clinical-stage platform described in supplied company analysis, with human compassionate-use and clinical-trial applications referenced in publications and public records.
Preclinical work showed dose-dependent improvement in mitochondrial content and oxygen consumption in healthy and diseased HSPCs, superior engraftment of augmented patient HSPCs in a mouse model, and durable engraftment with mitochondrial transfer in a Polg mouse model.
Quantify endogenous mitochondrial DNA copy number without amplification for improved assessment of mitochondrial dysfunction and potential clinical diagnostic or point-of-care use.
Solid-state nanopore sensing combined with exonuclease V treatment and support vector machine classification to directly count and distinguish mtDNA molecules from biological samples.
2025 ACS Nano publication reporting validation in spiked samples, cancer cells, and blood cells with comparison to qPCR mtDNA/nuclear DNA ratios.
The assay detected and quantified mtDNA at picomolar levels, showed high classification accuracy, and avoided amplification or purification steps.
Support harmonized, shareable clinical research data standards for primary mitochondrial disease to improve natural-history understanding and therapeutic development.
Community consensus effort addressing FAIR data standards, metadata sharing, harmonized data deposition, and clinical research data-resource interoperability.
2022 publication describing community consensus guidelines and the PMD clinical research data-sharing landscape.
The work identifies obstacles and opportunities for structured data sharing intended to improve PMD drug development efficiency and natural-history understanding.
Collect bone marrow aspirate and peripheral blood samples from patients with myelodysplastic syndrome for non-clinical research into mitochondrial function, sequence, and effects relevant to MDS.
Collection of bone marrow aspirates and peripheral blood from MDS patients for non-clinical research.
Clinical-trial public record listed for MDS sample collection.
Collect bone marrow aspirate and peripheral blood samples from patients with myelodysplastic syndrome for non-clinical research into mitochondrial function, sequence, and effects relevant to MDS.
Sample collection study using bone marrow aspirates and peripheral blood from MDS patients for non-clinical mitochondrial research.
Clinical trial record listed in supplied public records for MDS sample collection.
Enable mitochondria-based therapies for patients with mitochondrial dysfunction by increasing functional mitochondrial content in target cells.
Ex vivo augmentation or transplantation of healthy functional mitochondria into hematopoietic stem and progenitor cells, supported by analytical assays and biomarkers for mitochondrial health.
Clinical-stage platform with preclinical and compassionate-use evidence supporting MAT as a potential disease-modifying therapy for mtDNA disorders.
Preclinical work showed MAT was feasible and dose dependent, improved mitochondrial content and oxygen consumption in healthy and diseased HSPCs, improved engraftment in a patient-derived mtDNA disorder model, and enabled in vivo mitochondrial transfer in a Polg mouse model.
Measure mitochondrial DNA copy number for assessing mitochondrial dysfunction and potential clinical diagnostics or point-of-care applications.
Amplification-free solid-state nanopore sensing combined with exonuclease V treatment and machine-learning classification to directly quantify endogenous mtDNA.
Publication of a nanopore-based mtDNA quantification method validated against spiked samples, cancer cell lines, blood cells, and qPCR comparison.
The assay classified and quantified mtDNA directly from biological samples, detected mtDNA at picomolar levels, and avoided amplification or purification steps.