ProQR Therapeutics N.V. (PRQR), a biotechnology company focused on RNA therapies, announced on Wednesday the expansion of its collaboration with the Rett Syndrome Research Trust, or RSRT, through an additional $8.1 million in funding.
This follows the initial $1 million grant awarded in January 2024, bringing the total partnership funding to $9.1 million.
The funding will support the advancement of AX-2402, a treatment under development for individuals with Rett Syndrome who carry the R270X mutation in the MECP2 gene.
Rett Syndrome is a rare neurodevelopmental disorder that primarily affects girls and currently has no cure.
AX-2402 is based on ProQR's proprietary Axiomer RNA editing platform, which aims to correct genetic mutations at the RNA level. This compound is under discovery phase.
The new funding will help accelerate ProQR's efforts to optimize therapeutic candidates targeting MECP2, advancing AX-2402 into clinical trials.
"We are deeply committed to bringing innovative solutions to patients with high unmet needs, such as those living with Rett Syndrome," said Gerard Platenburg, Chief Scientific Officer of ProQR.
"The expanded partnership with RSRT enables us to fast-track the development of AX-2402 and continue our work to address complex genetic CNS conditions."
Monica Coenraads, Founder and CEO of RSRT, added, "This partnership is a critical step toward achieving our mission of eradicating Rett Syndrome, and we are encouraged by the progress made so far."
ProQR's stock closed Wednesday (Dec. 11, 2024) trading at $3.38, up 2.11%.
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