By Victoria Johnson – CGT Live =
The FDA has granted Regenerative Medicine Advanced Therapeutic (RMAT) Designation to uniQure’s AMT-130 gene therapy for the potential treatment of Huntington disease (HD).1
“We’re thrilled to receive the first ever RMAT designation for an investigational therapy for HD,” Matt Kapusta, chief executive officer, uniQure, said in a statement.1 “This achievement is a significant milestone for the program and supports the potential for AMT-130 to address the high unmet medical need of those suffering from this devastating disease.”
AMT-130 is an adeno-associated virus (AAV5) vector gene therapy that delivers an artificial micro-RNA designed to silence the huntingtin gene and therefore inhibit the production of mutant huntington protein. The therapy was developed with the use of uniQure’s proprietary miQURE™ silencing technology.
