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


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