by Dr. Gearóid Tuohy
Dear EURETINA Members,
A very warm welcome to the May 2nd, 2016 edition of EURETINA’s web-based digital magazine, “EURETINA Brief”. EURETINA are delighted to continue our delivery of up-to-date summary briefs on a range of topics of interest to retinal clinicians, specialists and researchers across Europe. This resource is designed to accommodate the very busy schedules of all our members by providing them with a short overview of some new developments and announcements in our field over recent weeks.
As in previous issues we have incorporated a feedback section where you can comment on any of the news items or articles under discussion and we very much welcome all contributions. Previous articles and issues can be found in the archive section on this website.
The current issue highlights a number of research activities, clinical milestones and business developments in our field, including research reporting on a novel antisense oligonucleotide treatment for up to 25% of Leber’s congenital amaurosis (LCA) patients harbouring mutations in the CEP290 gene; clinical trial follow-up data of a choroideremia gene therapy treatment which continues to show positive results in 2 out of the 6 participants treated with an AAV delivered V Rab escort protein-1 gene and; on the business front, an announcement from Japanese-US gene therapy company Acucela Inc., on the execution of an exclusive licensing deal with the University of Manachester, UK. The deal will provide commercial access for Acucela to a human rhodopsin based optogenetic gene therapy for the treatment of retinal degenerative disease, particularly rhodopsin related retinitis pigmentosa.
Finally, our feature bio-ophthalmology article reports on Science Magazine’s “Breakthrough of the Year”: CRISPR-Cas 9 gene editing technology. The recently developed technology is currently generating significant interest in the biomedical community, not least of all due to its low cost, its incredible ease of use and its versatility across a broad range of applications (see Bayer-CRISPR Therapeutics joint venture announcement in our previous issue). Referred to as “clustered regularly interspaced short palindromic repeats” or “CRISPR/Cas9”, the technology has emerged from research into prokaryotic immune defence systems. The technology has been shown to correct congenital cataract in mammalian models, with many additional ophthalmic applications in the pipeline. Remarkably, this very ancient bacterial technology is being rapidly applied to several areas of 21st century medicine and biotechnology. The impact is additionally been felt beyond university walls with >$150M of venture capital invested to date in 4 start-up companies alone. Such rapid commercial interest in a technology with 10+ years to go to reach market is a clear indicator of the size of CRISPR’s medical potential.
As always, increased interaction by you with the EURETINA web community serves to expand your professional network and keep you up to date with the latest initiatives, activities and research in your field. Our hope is that such cross-fertilisation in an active web-based platform, including our LinkedIn page, will lead to increased collaborative opportunities and ultimately to improved patient care. All readers are invited to submit comments or responses to any of the stories featured and we look forward to hearing from you over the coming month.
Dr. Gearóid Tuohy, EURETINA