LEIDEN, Netherlands & CAMBRIDGE, Mass. - ProQR (NASDAQ:PRQR) Therapeutics NV (NASDAQ:PRQR), a biopharmaceutical company focused on RNA therapies, has announced an expanded collaboration with the Rett Syndrome Research Trust (RSRT) to advance a potential treatment for Rett Syndrome into clinical trials. The collaboration, initially supported by a $1 million grant in January 2024, has been bolstered with an additional $8.1 million from RSRT, bringing the total funding to $9.1 million. This funding news comes as ProQR's stock has shown remarkable momentum, with InvestingPro reporting an impressive 80.87% return over the past six months. Get access to 10+ additional ProTips and comprehensive financial analysis with InvestingPro.
Rett Syndrome is a severe neurodevelopmental disorder that predominantly affects girls, with an estimated 350,000 cases worldwide. Currently, there are no cures and limited treatment options for this condition, which is caused by mutations in the MECP2 gene. ProQR's therapy, AX-2402, targets the R270X mutation in the MECP2 gene using the company's proprietary Axiomer RNA editing platform.
The Axiomer platform allows for the editing of RNA molecules in a highly specific manner, potentially correcting disease-causing mutations or modulating protein expression. The technology employs the cell's own editing machinery, ADAR, to make precise nucleotide changes in RNA.
ProQR’s Chief Scientific Officer, Gerard Platenburg, expressed gratitude for the partnership with RSRT and emphasized the potential of the Axiomer technology to address complex genetic conditions of the central nervous system. Monica Coenraads, Founder and CEO of RSRT, also highlighted the partnership's ambition and the progress made towards eradicating Rett Syndrome.
The additional funding from RSRT will enable ProQR to accelerate the development of RNA editing therapies for genetic mutations associated with Rett Syndrome. The focus of the collaboration will be to optimize therapeutic candidates targeting the MECP2 transcription factor and to advance them towards clinical development.
This news is based on a press release statement from ProQR Therapeutics. Looking ahead, InvestingPro analysts anticipate significant sales growth for the current year, though the company is not expected to be profitable. Discover the complete financial health analysis and access the detailed Pro Research Report, available for ProQR and 1,400+ other US stocks on InvestingPro.
In other recent news, ProQR Therapeutics N.V. has announced a public offering of its ordinary shares. This move is aimed at funding the company's research and development efforts, specifically to advance product candidates and expand R&D programs. The remaining funds will be allocated to support working capital and other corporate expenses. Evercore ISI, Cantor, Raymond (NS:RYMD) James, and Oppenheimer & Co. are serving as the joint lead bookrunning managers for the offering. The company's Axiomer® RNA editing platform, which is central to its efforts to develop new treatments for diseases with unmet needs, could potentially offer a new class of medicines for both rare and common diseases. While the company has provided forward-looking projections about the offering and its intended use of proceeds, it has noted that these are subject to market conditions, customary closing conditions, and other risks. These recent developments highlight ProQR's ongoing commitment to advancing its RNA therapy research.
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