RESEARCH


Through Vicky's Eyes has invested so far over £515,000 to advance research on RDH12 LCA.

 

Key progress so far:  

 

  • In 2018 we launched a 3 year research project to identify a possible drug therapy to slow down the progression of the disease. We awarded a total of £440,903. 

The project is a collaboration between the University College of London Institute of Ophthalmology and Moorfield Eye Hospital(Largest Ophthalmological hospital in Europe) and is being led by Professor Mariya Moosajee.

Vicky and 2 further patients have donated both blood and skin cells to create an in-vitro model of the disease using techniques applied to the stem cells.

The cell lines and models created by Professor Mariya Moosajee labare now available to the full scientific community. We already donated them to researchers in Paris to accelerate their work.

3 peer-reviewed articles have been published (see links below) and the work is being extended for another 12-18 months.

  • Gene Therapy offers real hope to Vicky and kids like her. It involves injecting a healthy copy of the missing gene in the eye. Gene Therapy proof of concept has been reached on mice.

There are now 2 companies planning a clinical trial for Vicky’s gene in the next couple of years. 

  • On November 2019, in partnership with the Foundation Fighting Blindness we convened the very first RDH12 Scientific Workshop: a meeting of families, researchers, industry and regulatory representatives to discuss the advancement of therapies for people with Vicky’s LCA.

The scientific workshop is considered one of the best-in-class example of Patient involvement in research and the output has been published on TVST journal. 3 other Patient organizations have now reapplied it.

  • In 2021 we published the second edition of the RDH12 Scientific Landscape. A comprehensive summary of  all Scientific Knowledgeabout Vicky’s condition from an in depth review of global publication and inputs from key stakeholders around the world . The landscape is drivingclarity on research status, gaps and opportunities and has been welcomed by the global research community.

Our key research priorities moving forward include:

Advancing Gene Therapy

Studying potential Small Molecules to slow down the sight loss

Strengthening our Fundamental Disease Understanding and Natural History

PARTNERS & RESEARCH PARTNERS