Alice Melão,  —

A gene therapy designed to correct the underlying defect that causes mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) could potentially improve survival in patients with the disease, a mouse study suggests. The potential therapeutic strategy was described in the study, “Long-Term Sustained Effect of Liver-Targeted Adeno-Associated Virus Gene Therapy for Mitochondrial…

Phase 1/2 trial long-term clinical data on GS010 showed that it is safe and can improve visual acuity in patients with Leber hereditary optic neuropathy (LHON), a mitochondrial disease that affects the eyes. Although the trial (NCT02064569) had not been designed or powered to assess the effectiveness…

Researchers are suggesting that genetic variants of the PARS2 gene are responsible for an entirely new mitochondrial disease, one with distinctive clinical and molecular patterns. Their findings were reported in  “Clinical and molecular characteristics of newly reported mitochondrial disease entity caused by biallelic PARS2 mutations,” published in the…

NeuroVive Pharmaceutical AB’s investigative compound NV556 holds therapeutic potential for the treatment of mitochondrial myopathy, according to recent data of a preclinical study. Conducted in collaboration with researchers at Karolinska Instutet in Stockholm, Sweden, the study showed that treatment with NV556 could…

Side alternating vibration training (SAVT), which uses a vibrating platform to simulate exercise, can improve muscle power in patients with mitochondrial diseases, according to a study published in Archives of Physiotherapy. Mitochondrial diseases comprise a group of inherited disorders that changes the way cells in the body produce energy.

Mitochondria genetics may be more complex than previously thought, with new research reporting that DNA content can vary significantly among different mitochondria within a single cell, and between mice and people — a finding of potential importance to research that relies on mouse models of disease. The work may also…

NeuroVive Pharmaceutical has made significant advances in its NVP015 program for mitochondrial diseases with the identification of a lead compound to develop in preclinical studies. Preliminary analyses in experimental models of genetic mitochondrial disease revealed one specific compound that was well tolerated and able to reach the brain, while…

Defective cellular energy production by mitochondria can result from abnormal expression of enzymes involved in the synthesis of the essential co-factor coenzyme Q, a study with mice revealed. This finding resulted from the collaborative work between researchers at the Max Planck Institute for Biology of Ageing, in Germany, and at…

The U.S. Patent and Trade Mark Office (USPTO) sent a notice of allowance — meaning it intends to issue a patent — to Mitochon Pharmaceuticals regarding the clinical development of its investigative drug MP101 for the treatment of several mitochondrial-related diseases, including Huntington’s disease and Duchenne muscular dystrophy, among others.

Mitobridge’s investigative PPARδ modulator MA-0217 has been shown to improve mitochondria activity and kidney function in animal models of acute kidney injury. The findings were the subject of two poster presentations titled, “Modulation of PPARδ with MTB-2 Post-Reperfusion Attenuates IR-Induced AKI Injury Biomarkers and Histopathology in…