By Eric Craypo
In a press release, Novartis announced that "the technologies acquired include light-sensing proteins that can be delivered to cells in the retina and unique, adeno-associated virus (AAV) delivery vectors that enable treatment via intravitreal injection. Together, these assets have the potential to vastly expand the number of patients who could be treated for vision loss due to photoreceptor death, including all inherited retinal dystrophies.
Inherited retinal dystrophies (IRDs) include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss. The conditions, which impact more than 2 million people globally, often result in complete blindness. Yet existing treatments target only one of the more than 250 genes that can cause IRDs, limiting the population of patients who can benefit.
The optogenetics approach acquired from Vedere Bio acts directly on surviving cells in the retina, altering their behavior and making it possible for anyone suffering from vision loss due to photoreceptor cell death to potentially benefit. This approach also holds promise for treating other conditions that involve photoreceptor loss, including a 'dry form' of age-related macular degeneration (AMD) called geographic atrophy, which affects more than 5 million people globally."
Congratulations to the Flannery lab for contributions that will make optogenetics and gene therapy technologies more accessible and more mainstream!
Dr. Flannery is a Professor of Optometry and Vision Science and Neurobiology.