Therapeutics Manchester: treatments for inherited retinal disease

"This is an exciting area because it gives patients hope that there may be something that can be done to stop them going blind or even to restore their vision"


Delegates to Therapeutics Manchester will have the chance to learn about the latest developments in surgical therapeutics for inherited diseases of the retina.

University of Oxford surgical research fellow, Dr Harry Orlans, will detail strategies aimed at slowing disease progression and restoring vision in his presentation, New and emerging treatments for inherited diseases of the retina.

Speaking with OT in advance of the event, Dr Orlans highlighted that historically the treatment of inherited retinal conditions has proved difficult.

“Traditionally, patients who have been given a diagnosis of inherited retinal degeneration have been told that there is nothing that can be done and they are going to lose their sight,” he shared.

“There are really few areas of ophthalmology now where that is true. That used to be the case in neovascular age-related macular degeneration, for example, but with the advent of anti-VEGF, it’s become a treatable disease,” Dr Orlans elaborated.

In recent years, there have been developments in the treatment of inherited retinal diseases, including the first US Food and Drug Administration-approved gene therapy, Luxturna for Leber congenital amaurosis caused by RPE65 mutations.

In the UK, clinical trials are underway examining the use of gene therapy for the treatment of choroideremia, X-linked retinitis pigmentosa caused by RPGR mutations and achromatopsia.

Although widespread NHS funding is not yet available for the Argus II electronic implant, the treatment is CE-marked and available in Europe.

Dr Orlans told OT that he enjoys being at the “frontier” of research that is creating new treatments for patients where nothing was available previously.

“It is an exciting area because it gives patients some hope that there may be something that can be done to stop them going blind or even to restore their vision,” he shared.

“As a clinician scientist, it’s nice to be able to tell patients that although there is no treatment at the moment, we’re working on it,” Dr Orlans added.

During his presentation, Dr Orlans will discuss a variety of treatment approaches including gene replacement therapy, optogenetics, retinal prostheses and using light filters to protect the retina from the degenerative process.

When asked for his view on the most promising treatment approaches coming through for inherited retinal conditions, Dr Orlans responded that this varies depending on the condition.

“It really depends on each individual disease and even individual mutations. As we move into the age of personalised medicine, we will be in a situation where different modalities of treatment are deemed appropriate for different patients depending on their molecular diagnosis,” he explained.

Therapeutics Manchester takes place at the Hilton Manchester Airport Hotel on 24 June. Bookings can be made through the AOP website.