In a report released on August 8, Ananda Ghosh from H.C. Wainwright maintained a Buy rating on ProQR, with a price target of $12.00.
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Ananda Ghosh has given his Buy rating due to a combination of factors including ProQR’s strategic advancements and promising clinical prospects. The submission of the Clinical Trial Application (CTA) for AX-0810 marks a significant transition for ProQR from preclinical research to clinical execution, which could validate the translational potential of their Axiomer platform. Additionally, the progression of AX-2402 for Rett syndrome and other candidates towards clinical selection in 2025 highlights the company’s expanding scope beyond hepatic targets, introducing high-value central nervous system indications.
Furthermore, the potential of the EON RNA editing platform is underscored by the anticipated reduction in bile acid levels, which could validate the mechanism of action in primary sclerosing cholangitis (PSC). The platform’s efficacy is supported by preclinical studies and human genetics data, showing promising results in reducing bile acid uptake and confirming target engagement. The next-generation AX-0810’s increased potency and favorable safety profile further strengthen the investment case, making ProQR a compelling buy opportunity according to Ghosh.