UniQure – gene therapy pipeline extended to SCA3

November 19, 2018

Lexington (MA, USA) and Amsterdam (Netherlands)

uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the expansion of its research pipeline with novel AAV gene therapy approaches to treating among others Spinocerebellar Ataxia Type 3 (SCA3) at the Company’s Research & Development Day held this morning in New York City.

“We are very proud of the progress the Company has made to deliver extensive preclinical data for these new gene therapy programs that expand our pipeline and further validate uniQure’s potential best-in-class vector delivery platform,” stated Sander van Deventer, M.D., Ph.D., chief scientific officer at uniQure. “The addition of these gene therapy candidates for indications in the liver and CNS brings us yet another step closer towards uniQure’s goal of delivering transformational medicine to patients suffering from genetic diseases. We look forward to advancing these programs closer to the clinic in 2019.”

uniQURE introduced the new gene therapy candidate AMT-150 as a novel treatment for Spinocerebellar Ataxia Type 3 (SCA3), a central nervous system disorder.

AMT-150 is a one-time, intrathecally-administered, AAV gene therapy incorporating the Company’s proprietary miQURE™ silencing technology that is designed to halt ataxia in early manifest SCA3 patients.

In an in-vitro study with human Induced Pluripotent Stem (IPS) derived neurons, AMT-150 has been shown to lower the human ataxin-3 protein by 65 percent, without any off-target effects. The Company also performed a proof-of-concept in-life study in SCA3 mice demonstrating that AMT-150 was able to lower toxic ataxin-3 protein by 65 percent in the brain stem after a single administration. Further studies in non-human primates demonstrate the ability to distribute and express a reporter gene at a clinically relevant level in the most degenerated brain regions in SCA3.

These preclinical studies show that a single administration of AMT-150 results in sustained expression and efficient processing with on-target engagement. They also show that AMT-150 appears to be safe due to the lack of off-target activity.

The Company is currently performing studies in large animals to demonstrate further safety and efficacy.


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