The reason for the slow development of new drugs in a specific disease can usually be attributed to one of the following two reasons. Low disease awareness or difficulty in developing the drug itself.

The one-shot gene therapy Novatis’s ‘Luxturna (voretigene neparvovec)’ is a drug that overcame both barriers. Luxturna, which is a treatment for IRD (Inherited Retinal Dystrophy) caused by the mutation in both copies of the RPE65 gene, is the first treatment option developed for the difficult rare genetic condition.

IRD is a rare intractable disease caused by a mutation in the gene responsible for the structure and function of retinal visual cells. It includes over 20 types of ophthalmol