The recent funding boost of $8.1 million from the Rett Syndrome Research Trust highlights ProQR Therapeutics' commitment to advancing RNA editing therapies for Rett syndrome.
ProQR’s Axiomer platform represents a significant innovation in RNA therapy development by using the cell's ADAR editing machinery to make precise edits that can potentially reverse harmful mutations.
Rett syndrome, affecting around 350,000 individuals, presents serious developmental challenges and currently lacks effective treatments, making funding for research and development critical.
The collaboration between ProQR and the RSRT underscores a joint effort to optimize RNA therapy candidates targeting the MECP2 gene, aiming for clinical trials.
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