Cell And Gene Therapies Target Rare Inherited Retinal Diseases

Inherited retinal diseases (IRDs) can cause vision loss that ranges from mild to severe, and can even cause blindness. These diseases affect people of all ages, and different IRDs progress at different rates. They are degenerative and get worse over time. Common IRDs include:

• Leber congenital amaurosis (LCA)
• Retinitis pigmentosa
• Choroideremia
• Stargardt disease
• Achromatopsia

IRDs are caused by a gene that is not functioning properly. Thus far, more than 260 retinal disease genes have been identified. Gene therapy, which corrects and compensates for faulty genes, can help slow disease progression. IRDs are good candidates for this type of therapy due to the location and composition of the retina. Human eyes are small and easily accessible by physicians for treatment.

The overall process of FDA approval and arrival on the market can take several years, which helps ensure the therapy’s safety and effectiveness. Finding a partner with experience can help you navigate PK, immunogenicity, biodistribution, viral clearance, and ADME properties of these novel modalities in a rapidly expanding field.