ProQR has a promising pipeline in ophthalmology programs that is built on RNA technology. According to Dr. Peter Adamson, ProQR’s (former) Senior Vice President Ophthalmology Franchise, RNA may reach the ultimate goal: to make blind people see again.
Interview with Dr. Ted Dryja, Scientific Advisory Board member. The pioneer in inherited retinal diseases joined ProQR to help push the organization forward. “I'm excited about what RNA technology may do for ProQR’s ophthalmology pipeline."
When watching Cristian, Silvia and Edoardo (age 6) and Beatrice (age 2) they look like the ideal, happy family. When asked, the parents insist they are, in many ways. The fact that Beatrice has Leber congenital amaurosis, doesn't change that.
2017 was a fruitful year for ProQR, in many ways. The company established proof of concept for the Axiomer® platform, a novel and proprietary RNA editing technology. “This opens up a whole new way of designing medicines for genetic diseases."
2017 may become a very exciting year for Patricia Biasutto, sepofarsen captain. Her work, developing a novel RNA therapy for Leber congenital amaurosis 10, has progressed steadily. The team expects to start a first clinical trial this year.