Data released from preclinical study into gene therapy for Friedreich’s ataxia – Ataxia

Data released from preclinical study into gene therapy for Friedreich’s ataxia

Data released from preclinical study into gene therapy for Friedreich’s ataxia

Voyager Therapeutics have created a gene therapy vector for Friedreich’s ataxia that has been undergoing pre-clinical testing in mice and non-human primates. They announced their latest findings of their study at the American Society of Gene and Cell Therapy 2018 annual meeting.

In mice, the therapy vector for Friedreich’s ataxia was shown to prevent central and peripheral disease progression up to one year after a single administration. The treatment additionally demonstrated good tolerability in non-human primates, along with widespread gene transfer to the brain and spinal cord after a single intravenous administration.

Voyager plans to continue to advance their preclinical gene therapy programmes and is focussed on developing life-changing treatments for severe neurological diseases, including Friedreich’s ataxia.

You can find and read the full press release here.

Posted on 23/05/2018

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