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On Tuesday, Jones Trading initiated coverage on ProQR Therapeutics N.V. (NASDAQ:PRQR) with a Buy rating and set a price target of $11.00, representing significant upside from the current price of $2.19. The firm’s analyst, Catherine Novack, highlighted ProQR’s extensive experience in RNA therapeutics, now positioning itself as a focused RNA editing biotech company with programs targeting both rare and more common diseases. According to InvestingPro data, the company maintains a strong financial position with more cash than debt and liquid assets exceeding short-term obligations.
ProQR’s pipeline includes treatments for rare diseases such as PSC and Rett syndrome, as well as programs aimed at liver and cardiovascular diseases. Jones Trading expressed a positive outlook on the RNA editing sector, citing its potential safety and versatility advantages over DNA editing technologies, which may lead to broader applications. While InvestingPro analysis shows the company is quickly burning through cash, analysts anticipate sales growth in the current year. Get access to 8 more exclusive ProTips and comprehensive financial analysis with an InvestingPro subscription.
The analyst noted that ProQR is expected to deliver Phase 1 target engagement data for its PSC program in healthy volunteers in the fourth quarter of 2025. This data is anticipated to provide a clear view of the company’s in vivo RNA editing capabilities. For the PSC indication, Jones Trading projects a peak unadjusted revenue of $3.9 billion by 2036, assigning a 15% probability of success to the program. The company’s current market capitalization stands at $224 million, with revenue of $20.47 million in the last twelve months.
Additionally, the company is set to release data on its Rett syndrome program in 2026, with financial support from the Rett Syndrome Research Trust. The collaboration with pharmaceutical giant Eli Lilly (NYSE:LLY), which is currently not rated by Jones Trading, was also mentioned as a testament to ProQR’s platform value. This partnership could potentially lead to future milestone payments for ProQR.
In summary, Jones Trading’s initiation of coverage with a Buy rating and an $11 price target reflects a bullish stance on ProQR Therapeutics’ prospects within the RNA editing industry.
In other recent news, ProQR Therapeutics N.V. has been making significant strides in the realm of RNA editing technology. H.C. Wainwright recently reaffirmed its confidence in the firm, maintaining a Buy rating and a price target of $10.00. This follows ProQR’s investor webcast, which spotlighted its RNA editing-focused pipeline and leading candidate, AX-0810, for the treatment of primary sclerosing cholangitis (PSC).
The company’s RNA editing technology, which modifies the messenger RNA without changing the underlying genetic code, could offer a more precise method for adjusting gene expression. The AX-0810 is currently undergoing Clinical Trial Application enabling activities, aiming to address PSC, a disease with significant unmet medical need.
In other developments, ProQR has secured $9.1 million in funding from the Rett Syndrome Research Trust (RSRT) to advance a potential treatment for Rett Syndrome into clinical trials. The therapy, AX-2402, targets the R270X mutation in the MECP2 gene using ProQR’s proprietary Axiomer RNA editing platform. This additional funding will enable ProQR to accelerate the development of RNA editing therapies for genetic mutations associated with Rett Syndrome.
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