According to a story from globenewswire.com, the drug developer ProQR Therapeutics N.V. has recently announced that the US Food and Drug Administration (FDA) has officially cleared the company’s Investigational New Drug (IND) application in regards to its experimental treatment QR-1123. This therapy is being developed as a treatment for the autosomal dominant form of retinitis pigmentosa. ProQR is committed to the development of life changing RNA medicines for the treatment of rare, genetic diseases.
About Retinitis Pigmentosa
Retinitis pigmentosa is a genetic disorder that affects vision. This disease can progress to total blindness, although this is rare. This disorder can appear on its own or as a complication alongside other disorders or systemic illnesses. Retinitis pigmentosa is usually inherited from the patient’s parents, and a number of genetic mutations can cause it to occur. Symptoms of this disorder include generalized vision loss, difficulty adjusting to lighting conditions, aversion to bright light, night blindness, tunnel vision, and blurred vision. There is a serious lack of treatment options for the condition, and there are no therapies that can definitively delay, halt, or reverse the decline in visual acuity. A retinal prosthesis, or bionic eye, is available in some places and can help improve vision to a limited degree. Gene therapy could be a viable option in the future. To learn more about retinitis pigmentosa, click here.
The Autosomal Dominant Form
While the inheritance pattern of this rare disorder can vary significantly, the autosomal dominant form is usually linked to a mutation called P23H that affects the RHO gene. This is the most frequently associated mutation in autosomal dominant retinitis pigmentosa. This form of the disease is estimated to affect around 2,500 people in the US. It is one of the most severe variants and can lead to blindness by middle age.
QR-1123 is classified as an antisense oligonucleotide. The drug binds to the mutated RNA in order to inhibit the formation of rhodopsin protein. The RHO gene is responsible for the production of this protein and in this disease the mutated protein has toxic effects.
The early phase 1/2 trials of QR-1123 will include 12 patients with the relevant form of the disease. Hopefully these studies will display the potential of this experimental therapy in treating retinitis pigmentosa.