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MeiraGTx Treats First Patient in XLRP Gene-Therapy Trial

MeiraGTx, a gene-therapy company in London and New York City, has treated its first patient in a gene-therapy clinical trial for people with X-linked retinitis pigmentosa (XLRP) caused by mutations in the gene RPGR. The Phase I/II study is taking place at Moorfields Eye Hospital in London. The safety-oriented trial will enroll 36 participants. Three dose levels of the therapy will be evaluated.

XLRP is a leading cause of inherited, progressive retinal degeneration and vision loss. The condition usually affects males, but is also diagnosed occasionally in females. Mutations in the gene RPGR cause about 70 percent of XLRP cases. RPGR mutations affect about 15,000 people in the United States and tens of thousands more around the world.

The MeiraGTx gene therapy involves injection of healthy copies of RPGR underneath the retina. The RPGR copies are contained in a human-engineered virus — known as an adeno-associated virus or AAV — which is designed to readily penetrate retinal cells to deliver the therapeutic genetic cargo.

In March 2017, Nightstar, a biopharmaceutical company in the U.K., announced treatment of the first patient in its XLRP (RPGR) gene-therapy clinical trial at Oxford University.

Applied Genetics Technology Corporation (AGTC), a U.S. gene-therapy company, is planning to launch an XLRP (RPGR) gene therapy clinical trial in the U.S. AGTC recently submitted an Investigational New Drug (IND) Application to the U.S. Food and Drug Administration to gain authorization to launch the study.

“We are very pleased that these XLRP gene-therapy trials are getting off the ground. The condition is a relatively common inherited retinal condition and causes significant vision loss,” says Stephen Rose, PhD, chief research officer, Foundation Fighting Blindness. “We are encouraged by sustained vision improvement provided by an XLRP gene therapy in FFB-funded canine studies at the University of Pennsylvania.”

MeiraGTx also reports that it has treated the first person in the second group of patents in its Phase I/II gene-therapy clinical trial at Moorfields for achromatopsia caused by mutations in CNGB3. A low dose of the treatment was safe for the three patients in the study’s first group. Achromatopsia is an inherited retinal disease that causes blindness in lighted conditions as well as reduced visual acuity and color perception.

MeiraGTx has treated seven patients in its Phase I/II gene therapy clinical trial at Moorfields for people with Leber congenital amaurosis caused by mutations in RPE65.

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10 Responses to 'MeiraGTx Treats First Patient in XLRP Gene-Therapy Trial'

  1. Hossein says:

    Dear Sir Madam

    It is very exciting to see this much progress
    I am suffering from RP since i was 6 years old
    I am male 44 years old
    I hardly see much from my lefteye
    however my right eye still can see line 3 and 4 but it is foggy

    I was wondering when would be the estimate time for availability of treatment for public

    what are the required conditions that one patient souldhave

    and some indication of the treatment cost

    Looking forward to hear from you



    • EyeOnTheCure says:

      Each clinical trial has its own parameters (genetic profile, visual ability, etc.). Genetic testing and identifying your disease causing gene is an important step if you want to get into clinical trials – many will want to know the gene causing your condition. Visit to learn more about trials that may apply to you.

  2. Nasrin Beeler says:

    My sister has RP usher cendurem , she is also deaf.
    Now she is blind about four years.
    She could see before she became blind.
    We want to know oh there is a cure for her.

  3. Dyllis Everett-Wells says:

    I have AMD in both eyes. I had a miniature telescope implanted at theondon Eye Hispital 2 years ago, it doesn’t help. Will this telescope exclude me from have gene therapy in the future when it is approved

    • EyeOnTheCure says:

      Hi Dyllis,

      It should not exclude you from future FDA – or EMA-approved therapies. It may exclude you from some clinical trials.

  4. Sita Reddy says:

    I m sita suffering from rp , I will do anything for d treatment, I hv two daughter and facing lots of problems in life …plse help

  5. Louise Z says:

    Hello, we learnt of our 11 year olds RP condition a year ago. He is our second son and has inherited this condition from me, the carrier, who it was passed to by my father.
    We had no idea it was hereditary and are obviously devastated. He has had genetic testing which confirmed he had the most common form of RP.
    Is there anything proven to help slow down the deteriation of his vision loss? Vitamins ? That we can be giving him?
    Are you hopeful there be a succesful treatment before he loses his sight?
    We also havent told him. How do you tell your child with all their hopes and dreams ahead of them, that they will go blind by 30.

    I very much appreciate any guidance, advise and help
    thank you

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