1. Santen Innovation Labs
  2. Gene therapies: A visionary modality

Gene therapies: A visionary modality

In this feature, global experts from Santen, Oxford Biomedica and the RIKEN Center discuss the potential impact of regenerative medicine on rare inherited retinal diseases (IRDs). They provide insight into the LentiVector® technology – a platform for delivering large genes into the eye – and retinal organoids models used to assess new, cutting-edge treatments. 

The article includes expert opinion from: 

  • Abu Abraham (Vice President, Vitreous & Retina TA Strategy, Santen, US)
  • Kenji Imoto (Global Development Lead, Gene Therapy, Santen, Japan)
  • Yatish Lad (Head of Early Development, Oxford Biomedica, UK)
  • Jason Slingsby (Chief Business Officer, Oxford Biomedica, UK)
  • Kyriacos Mitrophanous (Chief Scientific Officer, Oxford Biomedica, UK)
  • Akishi Onishi (Senior Scientist, Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology, Japan)

LentiVector® is a trademark of Oxford Biomedica



“Gene-based correction for life-long conditions, such as IRDs, requires a technology platform capable of mediating long-term efficacy (stable gene expression) with acceptable safety. To protect sight in these patients, and to avoid the huge impact of vision loss on them and their families, fundamentally new therapeutic approaches are required.”

Jason Slingsby, Chief Business Officer, Oxford Biomedica, UK

“[Gene therapy is] a new way to treat diseases that were previously untreatable – and it’s applicable to a broad range of IRDs. [However] gene therapy development involves a broad range of challenges, from vector construction and titer optimisation to efficient transduction of target cells. Even after you have a finished product, you have to pay attention to factors such as administration. The efficiency of subretinal delivery is related to the precision of the surgeon.”

Abu Abraham, Vice President, Vitreous & Retina TA Strategy, Santen, US

“Retinal organoids allow us to obtain retinal neurons that are comparable to those of native retinas. I predict that clinicians will be able to provide a more accurate treatment by the use of these models… as well as estimate the optimal dosage of the drug. Retinal organoids are a beautiful experimental system, but there are barriers that must be overcome before this model is adopted for wider use.”

Akishi Onishi, Senior Scientist, Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology, Japan