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French Gene Therapy Company Advancing Three Programs for Retinal Diseases

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Horama, a French biotech developing gene therapies for rare eye diseases, was established in 2014 as a spin-off of INSERM, France’s public scientific and technology institute. Today, the company has three gene-therapy development programs underway for rare inherited retinal diseases, targeting people with mutations in the genes PDE6B, RPE65, and RLBP1.

“We are pleased with Horama’s growing presence and advancements in gene therapy development for inherited retinal diseases,” says Stephen Rose, PhD, chief scientific officer at FFB. “They are targeting conditions that cause devastating vision loss, and there are currently no approved therapies for PDE6B or RLBP1 mutations, both of which cause retinitis pigmentosa.”

On April 29, at the 2018 meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Honolulu, Horama reported that three people have been treated in its Phase I/II gene therapy clinical trial for people with retinitis pigmentosa (RP) caused by PDE6B mutations. The three‑year trial taking place at University Hospital of Nantes in France will enroll a total of 12 patients.

“Horama is very pleased that, in about one year, we obtained the clinical trial approval for PDE6B gene therapy, set up the study at Nantes University Hospital, and recruited the first three patients,” says Jean-Yves Deslandes, MD, chief medical officer at Horama. “Thanks to all contributors for their efforts to achieve these key milestones. In the coming year, our focus is to complete enrollment in this study.”

Nantes University Hospital, with Pr. Michel Weber as principal investigator (one of the founders of Horama), completed its Phase I/II gene-therapy clinical trial for people with Leber congenital amaurosis caused by RPE65 mutations. Nine people received the treatment, which was safe and showed trends toward improved visual acuity and visual fields, and stabilized nystagmus (uncontrolled eye movements) for patients.

Horama’s gene therapies are injections of healthy copies of a gene underneath the retina to compensate for the defective gene. The healthy 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.

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25 Responses to 'French Gene Therapy Company Advancing Three Programs for Retinal Diseases'

  1. Seyed hojjati says:

    Any gene therapy in PDE6A gene mutation?

  2. Carol Turner says:

    This is such hopeful news. We have to conquer this horrible disorder.

  3. GRETA WAREHAM says:

    This is wonderful news , I have the RLBP1 along with family members and friends
    Can’t wait for the clinical trials to begin . Thank you to all the people who are making this reality..

  4. Vicky Kappelmann says:

    What is the difference between Pr. Weber’s gene therapy for RPE65 LCA and Spark’s Theraputics’ FDA approved treatment for LCA?

    • EyeOnTheCure says:

      Hi Vicky, Pr. Weber’s gene therapy is a unilateral subretinal injection of the adeno-associated vector serotypes 2 and 4 RPE65-RPE65 vector in patients with Leber congenital amaurosis (LCA) associated with RPE65 gene deficiency. Spark’s LUXTURNA (voretigene neparvovec-rzyl) is a prescription gene therapy product used for the treatment of patients with inherited retinal disease due to mutations in both copies of the RPE65 gene.

  5. Denisa says:

    Any gene therapy about FEVR ?Thank you!

  6. Marlow Lim says:

    Thank you gor the research and therapy, much appreciate.
    Would like to follow up on this gene thersphy.


  7. Jane Isgrigg says:

    Please let me know when you start another trail.

  8. Greta Wareham says:

    Hi are you doing a recruitment for RLBP1
    gene ? If so when and where I would love take part..
    Best Regards Greta Wareham

  9. Russ Davis says:

    Any work on USH2A?

  10. Lori says:

    Will this work on RPGR or EYS?

    • EyeOnTheCure says:

      Hi Lori, right now Horama has three gene-therapy development programs underway targeting people with mutations in the genes PDE6B, RPE65, and RLBP1.

  11. Geeta says:

    Any therapy that is available now for RP in the USA?

    • EyeOnTheCure says:

      Hi Geeta, do you know what genetic mutation is causing the RP? That’s an important first step in identifying the correct treatment or trial to enroll in. LUXTURNA is the first FDA-approved gene therapy for the retina that is restoring vision for those with mutations in the gene RPE65. You can contact Spark Therapeutics on their website, or by calling their patient inquiry number at 1 833 SPARK PS (833 772 7577). Their team can help answer further questions on whether you’re eligible for this treatment.

  12. Olga Burke says:

    I have the same question? Are there any RP Therapies in the area of Brooklyn New York or anywhere in the US

    • EyeOnTheCure says:

      Hi Olga, you can search for any clinical trials in your area for RP by visiting The site has a search function to find trials relevant to you. Once you find a trial you’re interested in, view the page and reach out to the contact(s) listed. Best of luck!

  13. Kiwi says:

    Are there any therapies for people with RB-1 ?

    • EyeOnTheCure says:

      Hello, our Foundation strictly focuses on funding research for inherited retinal diseases. The RB1 gene related to cancers, so this is not in our scope of research unfortunately. But if you’d like to learn more about clinical trials for RB1, visit: The site has a search function to find trials relevant to you. We hope this helps!

  14. Sajad Shah says:

    My son affected there any treatmentfor thi mutation.

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