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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.

There are currently around 160 people working at ProQR, but that wasn’t always the case. When Hee Lam Chan joined in 2013, she was only employee number six. As a result, she has seen the company grow and develop.

He is one of the founders of ProQR, but even long before that Gerard Platenburg was involved in the development of RNA therapies. Even so, after decades of working on them he still gets enthusiastic when talking about RNA.

Partnerships in our industry are very common as companies look to benefit from knowledge and resource sharing. In this blog we explore why companies, like ProQR, partner with other companies.

Bart Klein was at the forefront of ProQR's Axiomer® RNA editing technology development. This platform technology makes it possible to use the cells own machinery to reverse mutations that cause a genetic disease. Read the interview.

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.