Catarina Silva, MSc,  —

NeuroVive Pharmaceutical AB has completed recruitment of healthy subjects for the second part of an ongoing Phase 1a/b study testing its oral treatment candidate KL1333 for primary mitochondrial disease. The company also announced that it will soon begin the third part of this three-part, double blind, randomized, placebo-controlled,…

Researchers in Spain have made a discovery that has important implications for treatment strategies to prevent mitochondrial diseases from being passed from mother to child. Specifically, they found that the mechanisms involved in the transfer of mitochondrial genetic material from a mother to her offspring is controlled at two distinct…

A retrospective study of nucleoside therapy for treating the very rare mitochondrial disorder thymidine kinase 2 deficiency (TK2d) has shown positive results, including improved survival rates among children and adults with this genetic disorder, for which there are no approved therapies. Those findings were presented in a poster titled “…

The U.S. Department of Defense (DoD) has awarded a $4 million, three-year grant to the Children’s Hospital of Philadelphia (CHOP) to advance NeuroVive Pharmaceutical AB‘s NVP015 program for genetic mitochondrial diseases. The donation was made by DoD’s Office of the Congressionally Directed Medical Research Programs.

BridgeBio Pharma has entered into an exclusive licensing agreement with NeuroVive Pharmaceutical AB for the development of a subset of chemical compounds under NeuroVive’s NVP015 program. The goal is to further develop therapeutic solutions for mitochondrial diseases. BridgeBio also launched Fortify Therapeutics, a subsidiary that will work on the…

The cell’s powerhouse and garbage disposal — two distinct cell structures known as mitochondria and lysosome, respectively — regulate their function by communicating directly with each other within the cell, a new study says. The discovery was made at the Northwestern University Feinberg School of Medicine in Chicago, and…

Stealth BioTherapeutics‘ investigational medicine elamipretide, formerly known as Bendavia, has been granted orphan drug status by the U.S. Food and Drug Administration’s (FDA) for the treatment of primary mitochondrial myopathy (PMM). Stealth specializes in the development of drug candidates for mitochondrial disease treatment. The FDA’s Office of Orphan…