The buzz around gene therapy for ocular disorders has been growing louder over the past few years, and dominating much of the conversation in eyecare. Early in 2018 the first ocular gene therapy was approved by the FDA for retinitis pigmentosa (Spark Therapeutics’ voretigene neparvovec-rzyl), and many more are moving into the clinic. However, treatment advancements in ocular disorders are coming from other areas as well.

Several promising treatments on the horizon include:

  • Wet/Dry Macular Degeneration–while gene editing and stem cell therapies are in early phase, advances in other areas such as implantable mini telescopes for late-stage disease have been approved and are showing promise, as are the next-generation anti-VEGFA drugs entering the clinic
  • Cataract–the first adjustable intraocular lens (IOL) was approved over a year ago. For the first time, the estimated focal strength during surgery can be tweaked post-op when the eye has healed, and refraction has stabilized. With more adjustable IOL options in development, less focus will be on surgical accuracy and more on postoperative adjustments that will assure refractive precision, taking cataract surgery to a new level
  • Glaucoma–new advancements in prostaglandin analogs and Rho kinase inhibitors were breakthroughs this past year. While progressive imaging modalities allow early detection to improve outcomes, they will only get better as technology advances
  • Myopia–as the epidemic grows and more patients realize that eyeglasses do not halt progression, many new therapies are in the clinic. Progressive eye-training programs (Treehouse Eyes) and new topical prescription atropine drops (Nevakar, Inc) are already leading the way in 2019, along with other new treatments moving into mid-phase trials in the next year
  • Dry Eye–with the blockbuster Restasis off patent, there are several new therapies in the clinic and at the FDA this year. Newly approved topical solution (Shire’s Xiidra) that blocks a specific antigen has changed the game this past year. Coming soon: intranasal electric stimulation devices and implantable long-lasting eye drops, which are among the top novel dry eye treatments, with more entering mid-phase trials
  • Rare ocular disorders–the pharma industry has been focused on rare disease since the Orphan Drug Act of 1983, and much of its learnings are now being applied to ocular rare disease. While many mutations that drive genetic rare disease have been identified, it will be many years until some of them enter human trials. A few notable gene therapies currently in the clinic that will be seeking approval in the next year include GenSight’s viral vector injection for Leber’s hereditary optic neuropathy and mid-phase gene editing in Stargardt’s disease

The remainder of 2019 will certainly bring more advancements in genetic ophthalmology as well as new improvements to current therapeutic approaches. It’s an exciting time for eyecare professionals and their patients as we look to what next year will bring.

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