Acurex Biosciences Corp. has synthesized phenothiazines acting as ferroptosis inhibitors reported to be useful for the treatment of mitochondrial disease.
Although there are different methods of nuclear gene editing, there are still no effective treatments against mitochondrial disorders due to genetic alterations. Now, a group of researchers at Precision Biosciences Inc. and the University of Miami (UM) has developed a genetic edition platform that targets mitochondrial DNA (mtDNA) to delete its mutations.
“The ARCUS technology that we use is based on an enzyme found in nature called I-CreI. It is an enzyme that recognizes a 22 base pair DNA sequence within a species of green algae. And when it finds that DNA sequence, it will generate double-strand breaks,” first author Wendy Shoop, a scientist at Precision Biosciences, told BioWorld.
Abliva AB has gone above its market capitalization to raise the money it needs to start a phase II/III trial of KL-1333 for the treatment of primary mitochondrial disease.
Luca Science Inc. has raised $30.3 million in an oversubscribed series B financing, enabling it to advance a mitochondria replacement therapy toward the clinic. The company is planning a phase Ib trial in myocardial infarction, in which 8 to 12 patients will receive injections of mitochondria immediately after having a heart attack.
Abliva AB has gone above its market capitalization to raise the money it needs to start a phase II/III trial of KL-1333 for the treatment of primary mitochondrial disease.
Mitorx Therapeutics Ltd. is poised to develop small molecules that reverse impaired sulphide signaling underlying degenerative diseases ranging from Duchenne muscular dystrophy to Alzheimer’s disease. The company was formed some time ago as a spinout from Exeter University, where the founding scientist Matt Whiteman is professor of experimental therapeutics. It is showing its colors for the first time after closing a seed funding round.
Alexion Pharmaceuticals Inc. and Stealth Biotherapeutics Corp. reached a deal for an option to co-develop and commercialize elamipretide for mitochondrial diseases.