Currently, there is no effective treatment for many types of retinal degeneration. Nor are there sufficient treatments for autoimmune uveitis, as current uveitis treatment primarily uses steroidal anti-inflammatory medication, which can produce significant unwanted side effects with long-term use. NIH inventors have discovered that Adeno-associated Virus (AAV) mediated gene therapy or other extended delivery methods of Interleukin (IL)-34 protein to the eyes is a promising strategy for reducing retinal damage caused by ocular inflammation or degeneration and counteracting vision loss.

NIH inventors showed that various retinal degeneration and uveitis mice models with congenital mutations affecting vision have varying degrees of IL-34 deficiency in their intraocular environment. This suggests that IL-34 may be essential in modulating autoimmune uveitis and retinal degeneration. Therefore, AAV8-IL-34-mediated gene therapy or other extended delivery methods of IL-34 protein to the eyes is a promising new therapeutic approach for uveitis and retinal degenerative diseases.

Competitive advantages of this technology include:

• Eliminates the need for chronic steroid use in uveitis, diminishing the risk of side effects from long-term use. 
• AAV8 preferentially infects retinal cells; therefore, it could be a good choice for IL-34 gene therapy of uveitis for improved efficacy.  
• Potential platform approach to the more than 200 inherited retinal dystrophies (IRDs) associated with progressive retinal degeneration. 
  • IRDs are strong candidates for gene therapy when causative mutations have been identified and, to some degree, the eye is an immune-privileged space. 

Additionally: 

• A regulatory path to approval now exists: the first FDA-approved gene therapy, voretigene neparvovec (Luxturna ®; Spark Therapeutics), was already approved by the FDA and the European Medicines Agency (EMA) in November 2018. 
• In clinical trials, no significant immune reactivity or systemic adverse events have been associated with the AAV as a gene delivery vehicle.

For more information or to get in contact with the licensing manager, please view the abstract: Use of Interleukin (IL)-34 to Treat Retinal Inflammation and Neurodegeneration