Leiden-based ProQR Therapeutics NV, a Dutch biotech company specialising in RNA therapies, has secured $8.1M (nearly €7.74M) in additional funding from the Rett Syndrome Research Trust (RSRT). This expands their collaboration to a total of $9.1M, following an initial $1M grant in January 2024.
Funding will support the development of AX-2402, an RNA editing therapy targeting genetic mutations linked to Rett syndrome.
The collaboration focuses on optimising candidates for the Methyl CpG binding protein 2 (MECP2) transcription factor and advancing them into clinical trials.
Advancing transformative RNA therapies
ProQR Therapeutics focuses on developing transformative RNA therapies using its proprietary Axiomer platform.
This next-generation technology leverages the cell’s natural ADAR (Adenosine Deaminase Acting on RNA) editing machinery to make precise single nucleotide edits in RNA, aiming to reverse mutations or modulate protein expression.
Using Editing Oligonucleotides (EONs), Axiomer directs ADAR to convert adenosine (A) to inosine (I), which is interpreted as guanosine (G) during translation.
This technology aims to correct disease-causing RNA mutations, modulate protein expression, or alter protein functions, offering potential treatments for various diseases.
Axiomer claims to form the foundation for ProQR’s expanding therapeutic pipeline.
Rett Syndrome treatment efforts
Rett syndrome, a rare condition affecting nearly 350,000 people worldwide, primarily girls, is caused by mutations in the MECP2 gene. It typically begins with normal development in early infancy, followed by developmental stagnation, regression in motor and language skills, and eventual stabilisation.
Key features include stereotypic hand movements, loss of purposeful hand use, gait ataxia, seizures, breathing irregularities, gastrointestinal issues, scoliosis, anxiety, sleep disturbances, and other neurological and musculoskeletal challenges.
With no cure and limited treatment options, the disorder represents a significant unmet medical need.
ProQR Therapeutics is developing AX-2402, an RNA editing therapy targeting the R270X mutation in the MECP2 gene, using its Axiomer platform.
Axiomer has the potential to address additional mutations impacting a significant portion of the Rett population, with AX-2402 serving as a foundation for broader RNA-based treatments.
Gerard Platenburg, Chief Scientific Officer of ProQR, says, “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.”
“The increased support from RSRT underscores the potential of our Axiomer RNA editing technology platform to address complex genetic CNS conditions. The expanded collaboration enables us to accelerate the development of AX-2402 for Rett syndrome and to advance this program into clinical trials.”
Monica Coenraads, Founder and CEO of the Rett Syndrome Research Trust, adds, “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.”
“We are encouraged by the progress we’ve seen so far and believe that together we are taking critical steps toward achieving our mission of eradicating Rett Syndrome.”
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