ProQR has announced the completion of an in-depth strategic review designed to deliver on our commitment to advance RNA therapies for diseases with high unmet need. We are also sharing additional analyses of the sepofarsen Illuminate trial.
On behalf of all ProQRians, happy Rare Disease Day 2022. Both within ProQR and outside, we join the community in raising awareness and trying to generate change for people worldwide living with a rare disease, their families, and supporters.
In top-line analyses, the Illuminate study did not meet its primary endpoint of Best Corrected Visual Acuity (BCVA) and secondary endpoints. No benefit was observed in either sepofarsen treatment arm versus the sham treated control arm.
We are excited to announce, as of the start of December, the first clinical trial participants have been treated in our new clinical trials Sirius and Celeste of the investigational RNA therapy ultevursen (QR-421a).
Ben Shaberman, Senior Director of Scientific Outreach at the Foundation Fighting Blindness: The diagnosis of a retinal degenerative disease is often overwhelming. But if you or a loved one has been diagnosed with an inherited retinal condition, here are some helpful steps you can take to get on a positive path moving forward.
At ProQR we are getting ready for Usher Syndrome Awareness Day 2021 on September 18th. On this important day and during the month of September we will join the Usher community in raising awareness for this condition. Look to our socials to follow our activities.
Kate Arkell is Research Development Manager at Retina UK. Retina UK is only UK charity dedicated solely to working for people affected by inherited sight loss. At ProQR we are proud of our work with organizations like Retina UK. These patient organizations help us as a company stay connected to the needs of the communities for whom we are trying to develop RNA therapies.
Martin Hills is an active volunteer for Retina UK who lives with retinitis pigmentosa. As a member of ProQR’s Global Patient and Caregiver Steering Committee we asked him his perspective on clinical research and genetic testing.
We are pleased to announce the positive results of our Usher syndrome and retinitis pigmentosa clinical study Stellar, which has met all its stated objectives. This marks a crucial milestone in our ongoing inherited retinal disease research.