ProQR makes progress with potential treatment for LCA

Posted on: Monday 18 January 2021



Despite the challenges of the pandemic, researchers and pharmaceutical companies are still making progress towards delivering new treatments for inherited sight loss.

Biotechnology company ProQR Therapeutics has announced that it has reached a milestone in its development of a new therapy for Leber Congenital Amaurosis, enrolling all of the participants it needs for the final stage of clinical testing. The innovative treatment is aimed specifically at LCA10, one of the most common forms of LCA, which is caused by mutations in the CEP290 gene.

The trial participants will be followed closely for the next year, with regular testing of their vision; some will receive the treatment via injection into the eye, with others given a placebo (dummy, or sham, injection) for comparison. ProQR hopes to be able to deliver headline results from the trial in the first half of 2022; if the data demonstrates that the treatment is safe and effective, ProQR will press ahead with applications to license the treatment. You can find out more in ProQR’s community blog: www.proqr.com/blogs/milestone-achieved-in-our-leber-congenital-amaurosis-ongoing-research

ProQR is pioneering the use of an innovative technology that uses a special molecular “patch” to cover up faults in the genetic code. Alongside the treatment for LCA10, they are also using this approach in the development of therapies for Usher syndrome, and autosomal dominant retinitis pigmentosa caused by mutations in the RHO gene; these are both at an earlier stage of testing. A Retina UK-funded research team contributed to the early development of this approach, and you can read more about its background here: www.RetinaUK.org.uk/research-news/new-therapy-shows-promise-in-lca-disease-models.

This treatment approach is highly specific to certain genetic errors, meaning that potential recipients will need a genetic test result to confirm whether or not the therapy might be suitable for them. More information about genetic testing is available on our website www.RetinaUK.org.uk/information-support/diagnosis-and-beyond/genetic-testing/ and from our Helpline: 0300 111 4000; Helpline@RetinaUK.org.uk .