ProQR develops RNA-based therapies for severe rare genetic diseases such as dystrophic epidermolysis bullosa (DEB), cystic fibrosis, and Leber’s congenital amaurosis 10 (LCA 10), which is the most frequent genetic cause of blindness in children.
Rare Disease Day is an annual global campaign held every year on the last day of February. It was originally created by EURORDIS-Rare Diseases Europe and its Council of National Alliances.
Its main purpose is to bring together patients, family members, patient organizations, and healthcare providers to raise awareness of rare diseases.
This year’s theme is “Research, continuing on from Rare Disease Day 2017.”
ProQR will host a luncheon at the company’s headquarters, where a family in which all three children are affected by LCA 10 will be special guests. The family will share its experiences and the impact of this disease on their lives.
“We fully support Rare Disease Day 2018, which will raise public awareness about rare diseases and bring attention to the importance of research to develop therapies for these patients who have no available treatment options,” Daniel A. de Boer, CEO of ProQR, said in a press release.
“With an estimated 7,000 rare diseases known today and with less than 400 available therapies available, there is a dire need to quickly develop and bring to patients the medicines they need,” de Boer said.
The company is also co-organizing a commemoration of the legacy of the late Henri A. Termeer. The event will be held in Boston today, Feb. 28, which is also Termeer’s birthday.
Termeer was ProQR’s co-founder in 2012 and vice chairman of its supervisory board until his death in May 2017. He was a former CEO of Genzyme and a board member of several other pharmaceutical companies.
Termeer’s friends and associates will celebrate the renaming of the “North” plaza to Henri A. Termeer Square in Cambridge, Massachusetts, across from Genzyme Center, which was built under his leadership. A life-size sculpture of Termeer will be installed on the plaze in 2019.
Termeer was a leading contributor in the biotech and healthcare fields, and was also active in humanitarian assistance, policy issues, and innovative strategies to increase access to healthcare.
“On this day, it is a privilege to pay tribute to Henri, a good friend and mentor and pioneer in rare diseases. We plan to honor his legacy through completing the mission that we set out on together,” de Boer said.
In its DEB program, ProQR is developing QR-313 for patients with a specific mutation in the COL7A1 gene. The treatment candidate received orphan drug status from both the U.S. Food and Drug Administration and the European Medicines Agency. The company expects to start the first clinical trial on QR-313 this year.
For more information about Rare Disease Day 2018’s events, please visit this link.