Leiden-based ProQR Therapeutics, a biotech company specializing in RNA therapies, has secured €7.7 million ($8.1 million USD) in additional funding from the Rett Syndrome Research Trust (RSRT).
This new funding builds on an earlier €900,000 ($1M USD) grant received in January 2024, bringing their total collaboration funding to €8.5 million.
Developing RNA Editing Therapy for Rett Syndrome
ProQR is developing AX-2402, an RNA editing therapy targeting the MECP2 gene, which is responsible for Rett syndrome, a rare neurological disorder primarily affecting young girls. Using its proprietary Axiomer platform, the firm harnesses the body’s natural ADAR (Adenosine Deaminase Acting on RNA) mechanism to correct mutations by precisely editing single nucleotides in RNA.
How Axiomer works:
- Directs ADAR to convert adenosine (A) into inosine (I), interpreted as guanosine (G) during protein translation.
- Aims to reverse disease-causing RNA mutations, providing a foundation for transformative therapies.
Gerard Platenburg, Chief Scientific Officer, emphasized: “This expanded partnership with RSRT highlights the potential of Axiomer to address complex genetic CNS conditions. We remain committed to accelerating AX-2402 development and advancing it to clinical trials.”
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Addressing a Critical Unmet Need
Rett syndrome impacts approximately 350,000 individuals globally and is characterized by developmental regression, motor skill loss, seizures, and neurological impairments. Currently, no cure or highly effective treatment exists, leaving a significant unmet medical need.
AX-2402 specifically targets the R270X mutation in MECP2 but has the potential to address additional mutations affecting a broader Rett patient population.
Support from Rett Syndrome Research Trust
Monica Coenraads, Founder and CEO of RSRT, stated: “Rett syndrome has a profound daily impact on individuals and families. The innovative approach offers hope for transformative therapies, and we’re encouraged by the progress made so far.”
Next Steps
With this funding, the startup will optimize AX-2402 and prepare for clinical trials. The company’s long-term goal is to expand its RNA editing pipeline to address other diseases with similar genetic causes, leveraging its Axiomer platform as a foundation for breakthrough treatments.
This milestone strengthens the company’s position as a leader in RNA therapies, pushing forward innovative solutions for rare and complex genetic disorders.
