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Vision Improvements Reported in ProQR’s Clinical Trial for LCA10 Treatment

ProQR, a biotech company in the Netherlands, has reported vision improvements for patients in a Phase 1/2 clinical trial for QR-110, a therapy for people with Leber congenital amaurosis 10 (LCA10), which is caused by the p.Cys998X mutation in the CEP290 gene. The mutation is estimated to affect about 2,000 people in the Western world.

The company reported that 60 percent of subjects in the trial demonstrated improvements in visual acuity and their ability to navigate a mobility course. The treatment was also safe for patients.

As a result of the encouraging interim results, ProQR has concluded the Phase 1/2 trial and plans to move the treatment into a Phase 2/3 clinical trial. Ten people were treated in the Phase 1/2 study.

Results from the interim analysis were presented on September 5, 2018, at the Retinal Degeneration 2018 meeting in Killarney, Ireland, by principal investigator Artur Cideciyan, PhD, research professor of ophthalmology at the Scheie Eye Institute, University of Pennsylvania.

The Foundation Fighting Blindness has entered into a partnership with ProQR to develop a retinal therapy for people with Usher syndrome type 2A (USH2A) caused by mutations in exon 13 of the USH2A gene. FFB will be investing up to $7.5 million in milestone-based funding to advance the treatment, known as QR-421a.

“The results from ProQR’s trial are great news for people with LCA10. Furthermore, the safety and vision improvements observed in the study provide strong evidence that antisense oligonucleotides, ProQR’s treatment approach, are an effective way to save and restore the vision of people with retinal diseases,” says Ben Yerxa, PhD, chief executive officer at FFB. “ProQR’s results for QR-110 are groundbreaking, because it is the first time this treatment approach has improved vision in humans with retinal disease.”

ProQR’s LCA10 therapy is an antisense oligonucleotide (AON), which works like “genetic tape” to repair the mutation. Unlike gene replacement therapies in which copies of whole genes are delivered to replace defective copies, AONs correct the mutation in the patient’s messenger RNA, which conveys genetic information for protein production. AONs can be advantageous when large retinal-disease genes — such as CEP290 or USH2A — exceed the capacity of viral gene-replacement delivery systems.

Patients in the LCA10 Phase 1/2 trial are receiving four intravitreal injections of QR-110 into one eye—one injection every three months. The trial is being conducted at three centers: the University of Iowa, Iowa City, Iowa; the Scheie Eye Institute at the University of Pennsylvania, Philadelphia; and the Ghent University Hospital, Ghent, Belgium.

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12 Responses to 'Vision Improvements Reported in ProQR’s Clinical Trial for LCA10 Treatment'

  1. Don Rivers says:

    my son just hot diagnosed with lebors today

  2. Raghvendra Sharma says:

    We are from india and my daughter is aaradhya sharma diagnosed with usher syndrome type 1c.she is 4.5 years.what i can do to slow down vision loss.what research is going in this field

    • EyeOnTheCure says:

      Hi Raghvendra, we are aware of the following clinics in India, which may be helpful:
      Shankara Netralaya, Chennai
      L. V. Prasad Eye Institute, Hyderabad – They have branches in different states
      Aravind Eye Hospital, Madhurai
      All India Institute of Medical Sciences- New Delhi

      If you are interested in a clinic in a different location, contact one of these clinics and ask them for a suggested clinic in your region. Hope this helps and best of luck!

  3. Marlane Barker says:

    I was diagnosed with Stargardt’s at 20, now had retinal degeneration also, not sure if same one. Also maternal grandpa sand mat. Aunt. They all claim different diseases, is that possible? I am searching for genetic testing program, also drug or gene therapy research programs to participate in if possible. Any suggestions? I live in GA between Savannah and Jacksonville, FL, about 2 hrs either way, but will be able to travel. I feel like there must be something available, but no one seems to know, or I keep asking and the ball gets dropped somewhere along the line. I thank your organization for the wonderful support and progress you are making.

  4. Sidiksha says:


    We are from India.My baby is 15 months old.He has been diagnosed with LCA.We have done genetic testing and it came as inconclusive with defect in NMNAT gene(LCA9).

    Are there any clinical trials going on for this gene?we are not sure how much vision he is having.can you please guide us what procedure we need to do from our end?

    • EyeOnTheCure says:

      Hi Sidiksha, it’s great that you’ve gotten him genetic testing, that is the first step to finding treatments. At the moment, we don’t see any trials for NMNAT1, however we recommend checking frequently. The site has a search function to find trials relevant to a specific disease and location. We hope this helps!

  5. Ronel Taylor says:

    My nephew who is 11 has LCA10 CEP290 . We live in South Africa so would love to be kept up to date with these medical trials!

  6. Renee says:

    Will this same therapy be available for Ush1d cdh23 in the future?

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