ProQR And The Use of RNA Therapies for Rare Genetic Disorders

In a story for Drug Development & Delivery, the CEO of the biotechnology company ProQR, Daniel de Boer, recently sat down for an interview. In this interview, Mr. de Boer provides an overview of the company, its mission and goals in drug development, and its current development pipeline, as well as plans for the future.

A Personal Connection

Daniel has a unique perspective because he is both the CEO of a company that is focused on treating rare genetic disorders and has also experienced the impacts of rare disease in his personal life (his son was born with cystic fibrosis). This experience was a prime motivator for Daniel to decide to pursue a career in rare disease drug development as a way to help others that have been impacted by these diseases, which are so often overlooked and ignored by society and the broader medical community.

ProQR and RNA Therapies

ProQR is currently in the clinical development stage, with no therapies developed by the company being actively used by the public or approved. The company’s primary focus is on rare, genetic diseases and at this juncture the company is primarily focused on the development of RNA-based therapies to treat them. RNA therapies allow for the correction of genetic mutations that cause disease without actually altering a patient’s genetic makeup in the way that gene therapies do. The company plans to develop several RNA-altering methods that can address a diverse array of genetic disorders.

Daniel claims that there are several genetic disorders that can’t be effectively treated with gene therapy, at least with current methods. Daniel says that RNA-based therapies can step in when gene therapy is ineffective. The fact that RNA therapies don’t fundamentally alter a patient’s genes means that any potential side effects are usually transient in nature. They are also easier to deliver compared to gene therapy.

Latest Projects and Updates

The company is currently in the midst of phase 2/3 trials for its experimental treatment sepofarsen, which is being tested as a treatment for Leber’s congenital amaurosis type 10. Another investigational RNA therapy in the ProQR pipeline is called QR-421a; it is being developed as a therapy for Usher syndrome in patients with exon 13 mutations of the USH2A gene. The company expects updates from a phase 1/2 trial of this therapy in early 2020. Another focus of ProQR is its Axiomer system, which will hopefully allow a patient’s body to repair its own RNA.


Follow us