Exicure to launch gene therapy programme in Friedreich’s ataxia – Ataxia

Exicure to launch gene therapy programme in Friedreich’s ataxia

Exicure to launch gene therapy programme in Friedreich’s ataxia

Exicure is a biotechnology company that specializes in the development of therapeutics based on a unique approach to gene therapy, which they call ‘spherical nucleic acid’ (SNA™) technology. SNAs consist of nucleic acid sequences attached to a spherical nano particle. This three dimensional arrangement gives the SNAs unique properties that could be useful for delivering gene therapy to people with Friedreich’s ataxia (FA). For example, they can enter into virtually all cell types, including those in the brain and nervous system, without causing the harmful side effects sometimes associated with conventional forms of gene therapy. Once inside the cell, they are able to deliver therapies which alter gene expression.

In December last year, Exicure announced FA as the therapeutic indication for the company’s first neurology development programme utilizing the SNA approach. The FA programme hopes to use the SNA platform as a means of increasing the expression of frataxin protein in the central nervous system tissues to change the course of the disease, and will be designed and developed in collaboration with the Friedreich’s Ataxia Research Alliance (FARA). Previous experiments in animal models have demonstrated that the technology can successfully reach the parts the parts of the brain affected in FA when administered via injection into the spinal canal.

David Giljohann, PhD, Exicure’s chief executive officer, said: “Over the past year Exicure has developed extensive preclinical data supporting the development of our SNAs for neurological disorders. We’re eager to evaluate our technology for this important unmet medical need.”

The full press release from Exicure can be viewed on their website.

Posted on 21/02/2020

SUBSCRIBE TO OUR NEWSLETTER

Something went wrong. Please check your entries and try again.
fundraise image

FUNDRAISE FOR US

Take part in a challenge or create your fundraiser. Every penny you raise will help those affected by ataxia.

Donate Image

DONATE

To make either a one off or recurring donation which will help fund research into treatments and cures and supports those affected ataxia

Volunteer Image

VOLUNTEER WITH US

Support the ataxia community and volunteer with Ataxia UK. From social media to telephone befriending, there are loads of ways you can make a difference to someone's life.

Donate Now
Welcome to our new site! We hope you like it.
As it's brand new, there may be a few temporary glitches such as broken links - we are working on getting these all fixed ASAP. If you'd like to give us any feedback or let us know if something isn't quite right, please email website@ataxia.org.uk
+
Scroll to Top