L to R: Florence Allouche Ghrenassia, PharmD, President, SparingVision; Frédérique Vidal, French Minister of Higher Education, Research and Innovation; José-Alain Sahel, MD, Co-Founder, SparingVision and Fondation Voir & Entendre; David Brint, Chairman, Foundation Fighting Blindness; and Laure Reinhardt, Deputy CEO, Bpifrance
The development of a vision-saving treatment for people with retinitis pigmentosa (RP) is getting a major boost thanks to the formation of the French biotech SparingVision to move it into a clinical trial and out to the international marketplace.
A spin-off of the Institut de la Vision, SparingVision was established to clinically develop and commercialize a protein known as rod-derived cone-viability factor (RdCVF). The emerging therapy performed well in several previous lab studies funded by the Foundation Fighting Blindness. SparingVision’s goal is to launch a clinical trial for the protein in 2019.
The Foundation Fighting Blindness Clinical Research Institute (FFB-CRI), Bpifrance — Biotechnology Acceleration Fund and Innovative Biotherapies and Rare Diseases Funds created by the French national program (BPI), and the Fondation Voir et Entendre (FVE) have announced a total of €15.5 million in tranche funding for the development of RdCVF. FFB-CRI and BPI are each investing €7 million with FVE providing €1.5 million.
SparingVision also received a €300,000 award known as the Honor Prize from the French Ministry of Research. The award is given to new, innovative companies in France competing in a national contest.
RdCVF is a naturally occurring protein in the retina identified by SparingVision co-founders José Sahel, MD, and Thierry Léveillard, PhD, at the Institut de la Vision. The scientists demonstrated in laboratory studies that RdCVF prevented or slowed the degeneration of cones, the cells in the retina that provide central and color vision and enable people to read, drive, and recognize faces. RdCVF is naturally secreted by rods, the retinal cells that provide night and peripheral vision.
RP is a genetic condition affecting about two million people worldwide. The retinal disease is usually diagnosed in childhood, progressively leading to legal or total blindness in adulthood. RP initially affects rods. The progressive loss of rods leads to loss of cones. There are currently no therapies for RP.
“Saving retinal cone cells is critical for preserving vision for people with genetic retinal diseases,” says Dr. Sahel.
“After several years of investigation, we understand the mechanism of action for RdCVF and have demonstrated its strong efficacy in several lab studies,” says Dr. Léveillard.
“SparingVision’s emerging therapy has the potential to save the vision of millions. I am delighted by our partners’ investment to get RdCVF out to the people who desperately need it,” says Florence Allouche Ghrenassia, PharmD, president at SparingVision. She brings 16 years of experience at Assistance Publique Hôopitaux de Paris as TTO Director in advancing innovative early stage therapies into the clinic to her new role.
FFB-CRI provided much of the earlier research funding to develop RdCVF. “We have been excited about this therapy’s potential for saving vision and therefore committed significant resources to boost its development,” says Patricia Zilliox, PhD, FFB-CRI’s chief drug development officer. “The establishment of SparingVision and the investment by our partners are essential to getting the treatment into the marketplace. We are pleased to be a part of this translational process.”
Steve is highly respected for his expertise and tireless commitment to finding treatments and cures for vision-robbing retinal diseases. 
As the Foundation's Director, Science Communica- tions, Ben writes science and research articles for the Foundation’s website, newsletters and Eye on the Cure blog. 
Dear Sir,s
My son is now 20 years old. He had Degeneration disease since he was 5 years old.
Now his genetic test show he has mutation in ABCA4 gene Cone Rod Dystrophy.
Can you please let me know when Gene and Stemcell therapy will be available or any other treatment coming soon.
Anxiously awaiting for your reply.
Thanks
Akbar
Dear Sir,s
My son is now 20 years old. He had Degeneration disease since he was 5 years old.
Now his genetic test show he has mutation in ABCA4 gene Cone Rod Dystrophy.
Can you please let me know when Gene and Stemcell therapy will be available or any other treatment coming soon.
Anxiously awaiting for your reply.
Thanks
Akbar
Dear Muhammad, You will be pleased to know that the Foundation Fighting Blindness is partnering with Sanofi Pharmaceuticals on a gene therapy clinical trial for Cone-Rod and Stargardt disease caused by mutations in the ABCA4 gene. For more information on this trial, see the following link:
http://clinicaltrials.gov/ct2/show/NCT01367444?term=stargardt&recr=Open&no_unk=Y&rank=2 In order to participate in this or any other gene therapy trial, you must first obtain a molecular (genetic) diagnosis. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf
You should also consider enrolling in “My Retina Tracker”, a free registry that helps link people with retinal disease to appropriate clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/
Below is a list of companies that are developing treatments for ABCA4-mediated Disease:
Alkeus, (http://alkeus.com/) Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical trials.gov recruitment page: https://www.clinicaltrials.gov/ct2/show/NCT02402660
Vision Medicines (http://visionmedicines.com/index.html) is developing VM200, an oral small molecule drug candidate for the treatment of Stargardt disease. Stargardt disease is caused by mutations in a key transporter called ABCA4, which lead to the buildup of toxic vitamin A aldehydes and derivatives in the retina to cause blindness. VM200 neutralizes the toxic vitamin A aldehyde and has the potential to be the first treatment for the disease. For a more detailed overview of VM200, please see the following weblink: http://visionmedicines.com/pdf/VM200-Overview-Presentation.pdf
To read more about the partnership between Vision Medicine and FFB, see the following web link: http://www.blindness.org/foundation-news/foundation-fighting-blindness-partners-vision-medicines-develop-stargardt-disease
Astellas Pharma (https://www.astellas.us/ ) acquired Ocata Therapeutics in May of 2016. Headquartered in Marlborough, Massachusetts and supported by a research team in Tsukuba, Japan, Astellas serves as a global hub for regenerative medicine and cell therapy research in ophthalmology and other therapeutic areas that have few or no available treatment. At the present time, the RPE transplantation studies have been completed and the Phase 3 trial has yet to be announced. http://www.clinicaltrials.gov/ct2/show/NCT01469832?term=advanced+cell+technology&rank=2
My daughter-in-law has rp. She is 31 and still has some vision.
I am wondering how I found out more and if you have a treatment program. We can come to France. Thank you.
Hi Lynda,
There are several clinical trials underway (or will soon begin) for RP. Visit: http://www.blindness.org/blog/ — then search on “retinitis pigmentosa”. Some of therapies are designed to slow vision loss — others are for restoring vision. You can also visit http://www.clinicaltrials.gov for clinical trials.
Dear Sir,
We have four generations in our family who have Retinitis Pigmentosa, my husband, his mother and grandfather and now our daughter. If there is anything that possibly may help us please let us know.
Thank you
Hi Richard,
Have you been genetically tested? That is an important first step toward getting into a clinical trial. You can reach out to http://www.idyourird.com and see if you qualify for their no-cost genetic testing.
Also, you might want to learn more about clinical trials at http://www.clinicaltrials.gov.
If this is successful when is this likely to be available to clients.
With Corp.
Hi Neville,
A clinical trial for the typical emerging therapy could take anywhere from 5 to 10 years. The clinical trial for the Sparing Vision therapy is planned to begin in 2018.
My sister has to and needs to be treated for her type of RP. Do you have some treatment or suggestions to slow down the progression of this disease.
The following emerging therapies are cross-cutting and may provide benefits to people with many forms of RP:
http://www.blindness.org/blog/index.php/jcyte-reports-results-for-phase-12a-clinical-trial-for-retinal-cell-treatment/
http://www.blindness.org/blog/index.php/sparingvision-formed-to-advance-sight-saving-protein-for-rp/
http://www.blindness.org/blog/index.php/foundation-investing-in-drug-to-slow-many-forms-of-rp/
We also suggest you register at http://www.myretinatracker.org to qualify for free genetic testing (coming soon) and clinical trials for which you may be eligible.
My 3 sister and I have RP.
Will this treatment be available to us.
Sparing Vision’s clinical trial is planned for 2018, so stay tuned. Here is another approach already in a clinical trial:
http://www.blindness.org/blog/index.php/jcyte-reports-results-for-phase-12a-clinical-trial-for-retinal-cell-treatment/