March 22, 2016 12:27pm

 

Publication in Molecular Therapy-Nucleic Acids demonstrates silencing of mutated Huntington’s protein using microRNA delivered with QURE's AAV5 Vector

 

 

QURE’s publication of preclinical data supports its proprietary Huntington's disease gene therapy program, AMT-130.

 

Findings published in the current issue of the peer-reviewed journal Molecular Therapy-Nucleic Acids (www.nature.com/mtna/journal/v5/n3/index.html ) provide preclinical proof of concept for QURE's AMT-130 program and demonstrate the potential of a one-time administration of AAV5-delivered gene therapy into the central nervous system (CNS) to silence the Huntingtin gene (HTT).

The data published today were in part presented at the 11th Annual CHDI Huntington's Disease Therapeutics Conference on February 24, 2016 by Dr. Konstantinova.

 

The paper, titled "Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease", was authored by a research team led by Pavlina Konstantinova, Ph.D., Director of Emerging Technologies at uniQure under the direction of Chief Scientific Officer Harald Petry, Ph.D.

  • The publication describes multiple approaches to silencing HTT using expression cassette-optimized artificial microRNAs (miHTTs).
  • Several miHTT scaffolds were incorporated in an AAV5 vector using uniQure's established baculovirus-based manufacturing platform and administered to a humanized mouse model.

 

 

The Bottom Line: An inherited, mutated form of HTT causes Huntington's disease, a rare, fatal, neurodegenerative disorder that leads to severe physical and cognitive deterioration. The data demonstrate strong silencing of mutant HTT and total HTT silencing in vitro and in vivo. Furthermore, it was shown that HTT knock-down efficiency could be increased to 80% by using optimized miHTT scaffolds.

Based on these results, QURE has initiated further studies of AMT-130 to support the filing of an investigative new drug application with the FDA.

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