ProQR Therapeutics NV, a biotechnology firm based in Leiden, Netherlands, has expanded its collaboration with the Rett Syndrome Research Trust (RSRT) by securing an additional $8.1 million, totaling $9.1 million in funding. This new investment follows an initial grant of $1 million received in January 2024. The funding is earmarked to advance AX-2402, an RNA editing therapy aimed at addressing genetic mutations associated with Rett syndrome. This financial boost underscores ProQR’s commitment to advancing RNA-based solutions for genetic disorders.
Earlier announcements from ProQR have highlighted the company’s commitment to leveraging RNA therapies using innovative technologies. While past developments have paved the way for the progression of RNA-based treatments, this funding intensifies efforts to bring AX-2402 closer to clinical trials. The company’s Axiomer platform, central to these efforts, continues to be pivotal in developing therapies that target specific genetic mutations. Historically, ProQR has focused on diseases with significant unmet medical needs.
How Does ProQR’s Axiomer Technology Work?
ProQR’s Axiomer platform utilizes the cell’s innate RNA editing machinery, ADAR, to make precise edits in RNA. This proprietary technology directs ADAR to convert adenosine to inosine, which during translation is read as guanosine. This process aims to correct mutations, adjust protein expression, or alter protein functions to potentially treat a range of diseases. Axiomer serves as the foundation of ProQR’s expanding pipeline, offering hope for advancements in RNA therapies.
Can Axiomer Address Rett Syndrome Effectively?
Rett syndrome, characterized by mutations in the MECP2 gene, affects approximately 350,000 individuals globally, primarily girls. Symptoms manifest after normal early development, leading to developmental regression and various neurological challenges. Current treatment options are limited, marking a significant unmet need. ProQR’s AX-2402 targets the R270X mutation within the MECP2 gene, representing a critical step in addressing this condition. By focusing on RNA editing, Axiomer offers potential solutions for a broader spectrum of Rett syndrome mutations.
Gerard Platenburg, Chief Scientific Officer of ProQR, emphasized the expanded partnership with RSRT, noting the potential of the Axiomer platform to address complex genetic conditions affecting the central nervous system.
“We are deeply committed to bringing innovative solutions to patients with high unmet needs, such as those living with Rett Syndrome, and are grateful for the expanded partnership with the Rett Syndrome Research Trust,”
he stated.
This collaboration is seen as a significant step forward in the mission to develop transformative therapies for Rett syndrome. Monica Coenraads, Founder and CEO of the Rett Syndrome Research Trust, conveyed optimism about the partnership, stating,
“Individuals with Rett Syndrome live with the profound effects of this condition every day, and they deserve transformative therapies. This partnership with ProQR is exciting and ambitious.”
The joint efforts aim to accelerate the development of AX-2402 into clinical trials.
The advancements made by ProQR Therapeutics and their collaboration with RSRT highlight a focus on addressing complex genetic disorders through RNA-based therapies. As research progresses, the potential of RNA editing technology in treating genetic conditions becomes increasingly evident. Continued funding and partnerships will be crucial in accelerating the transition from research to clinical application, offering hope for individuals affected by Rett syndrome and similar genetic conditions.
