There is currently no cure for H-ABC, a disabling and life-threatening genetic disorder caused by a mutation in the TUBB4A gene that affects certain parts of the brain that control movement. In the new collaborative research effort, Dr. Guangping Gao of the University of Massachusetts and Dr. Karel Liem of Yale will team up to develop adeno-associated virus (AAV) vectors to silence or outcompete the mutated TUBB4A gene, according to the foundation.

Avrobio expands Hunter syndrome gene therapy program
Avrobio has signed a research and licensing agreement with the University of Manchester in the U.K. to investigate the use of lentiviral gene therapy...

Diamond Pharma, GenSight submit EMA application for ocular gene therapy
French biotechnology firm GenSight Biologics has submitted a marketing authorization application to the European Medicines Agency (EMA) for its ocular...

Unchained Labs launches new gene therapy tool
Unchained Labs has launched a new gene therapy quantification application on Stunner, its gene therapy platform.

Bluebird Bio gets gene therapy EMA marketing nod accepted
The European Medicines Agency (EMA) accepted Bluebird Bio's marketing authorization application for its investigational elivaldogene autotemcel gene therapy...

BioMarin receives fast track designation for PKU gene therapy
The U.S. Food and Drug Administration has granted BioMarin Pharmaceutical fast track designation for BMN 307, an investigational gene therapy drug for...