The pandemic has heightened interest in COVID-19 therapies, sometimes stealing attention from other pharmaceutical breakthroughs. But the pandemic has also served as a reminder that investment in new drugs can pay dividends while leading to a greater societal appreciation for the industry. A 2019 study published in International Health concluded that pharmaceuticals that have launched…
How ProQR aims to use RNA therapy to target genetic forms of blindness
The rare disease biotech ProQR (NSDQ:PRQR) has a mission of reversing blindness from inherited retinal diseases. The company seems to be on track to realize that ambition, having recently announced promising data in treating a condition known as Leber congenital amaurosis type 10 in clinical studies. One patient with the childhood form of blindness gained…
Investigational drug gives sight to patient with inherited form of blindness
A patient with childhood blindness has gained durable vision improvement after a single injection of sepofarsen, an investigational RNA therapy. The patient had a rare genetic disorder known as Leber congenital amaurosis (LCA) that affects the retina and often causes severe visual impairment. Injection of sepofarsen led to measurable changes in the fovea, a depression…