ProQR Therapeutics N.V. announced an expansion of its collaboration with the Rett Syndrome Research Trust (RSRT), securing an additional $8.1 million in funding. This new commitment brings the total RSRT funding for ProQR to $9.1 million, building upon an initial $1 million research grant announced in January 2024.
The expanded funding is specifically designated to support the advancement of AX-2402, ProQR's program for Rett syndrome, into clinical trials. AX-2402 is designed to target the R270X mutation in the MECP2 gene, addressing a severe neurodevelopmental disorder that affects approximately 350,000 individuals globally.
This collaboration underscores the potential of ProQR's Axiomer RNA editing technology platform to address complex genetic CNS conditions with high unmet medical needs. The additional capital will accelerate the optimization of therapeutic candidates and their progression toward clinical development.
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