Author Archives: Margarida Azevedo, MSc

CohBar recently announced the discovery of a series of new biologically active peptides in the mitochondrial genome — mutations in which can cause mitochondrial disease — and the filing of 29 additional related provisional patent applications. The new applications apply not only to the new peptides but also to analogs that are built…

Moumita Das, a theoretical physicist and assistant professor at Rochester Institute of Technology’s School of Physics and Astronomy, has been awarded funding totaling $112,000 for her groundbreaking work on mitochondrial DNA and on the movement of vesicles and substances between cells (intercellular cargo transport).

Stealth Bio, a company that develops drugs to treat mitochondrial dysfunction and genetic mitochondrial diseases, recently launched Phase 2 of the clinical trial IDDEA-HF assessing elamipretide in patients hospitalized with congestive heart failure. Heart failure affects about 21 million people in the U.S. and Europe with cases expected to rise as populations continue to age. Heart…

The Howard Hughes Medical Institute (HHMI), the Simons Foundation and the Bill & Melinda Gates Foundation have given Cole Haynes, Ph.D., an associate professor of molecular, cell and cancer biology, an award for his work on mitochondria and aging. Cole HaynesPhoto: UMass…

Courtagen Life Sciences and Astellas Pharma announced a collaboration to use Courtagen’s molecular information platform to assist in the development of a predictive model to support early stage therapeutic development for mitochondrial disease. Under the terms of the agreement, Courtagen will use its ZiPhyr bioinformatics…

Stealth BioTherapeutics has initiated an extension trial of its Phase 2 study evaluating elamipretide in patients with primary mitochondrial disease. The study, MMPOWER-2, will evaluate patients who completed the initial clinical trial. “Patients with rare primary mitochondrial diseases have no FDA-approved treatment options to address their needs,” Stealth CEO Reenie McCarthy…

A federal spending bill in 2015 banned the human testing of gene-editing techniques that could lead to the “production” of genetically modified babies. But it also included two critical sentences that may have had unwanted consequences. The legislation, which is up for renewal this year, includes a technique with promising features…

The National Organization for Rare Disorders (NORD), a nonprofit organization representing all patients and families affected by rare diseases, published the Physician Guide to Mitochondrial Myopathies, available free online to medical professionals worldwide. The guide, written by Drs. Michelangelo Mancuso and Michio Hirano, can be…

GenSight Biologics released new preliminary findings from a Phase 1/2 clinical trial assessing the safety and tolerability of the company’s lead investigational gene therapy, GS010, in patients with Leber’s hereditary optic neuropathy (LHON), a maternally inherited mitochondrial disease causing irreversible vision loss. The data show significant improvement for those in earlier stages of the disease. GS010…

Stealth BioTherapeutics recently reported positive data from its Phase 2 MMPOWER clinical trial, a double-blind, placebo-controlled study that evaluated the safety, tolerability, and efficacy of elamipretide (Bendavia) in patients with mitochondrial myopathy (MM). The MMPOWER study investigated Bendavia for the treatment of myopathy (muscle weakness) in patients with genetic mitochondrial diseases. These diseases…