ProQR Therapeutics, a company committed to transforming lives through innovative RNA therapies, has shared its financial results for 2024 along with key business updates.

The company is entering a significant phase of growth as it advances its Axiomer RNA editing platform. With up to four clinical trial data readouts expected in 2025 and 2026, including topline results from its NTCP program later this year, ProQR is preparing to demonstrate the broad potential of RNA editing in treating disease. Its expanding pipeline, now including applications for central nervous system disorders, along with strategic partnerships—such as its $3.9 billion collaboration with Eli Lilly and a partnership with the Rett Syndrome Research Trust—reinforces its leadership in the field.

A successful financing round in late 2024 has strengthened the company’s financial position, securing funding into mid-2027. This stability enables ProQR to advance key milestones, including the submission of its first RNA editing therapy, AX-0810, for regulatory approval in the second quarter of 2025 and its first clinical data readout later in the year.

Recent developments include the expansion of ProQR’s collaboration with the Rett Syndrome Research Trust, securing additional funding to advance an RNA therapy for Rett Syndrome into clinical trials. The company has also appointed Dr. Peter A. Beal, a renowned expert in RNA biology, as Chief ADAR Scientist to help refine and optimize its Axiomer platform.

In October 2024, ProQR strengthened its financial foundation through a successful public offering, raising over $82 million, including an investment from Eli Lilly. These developments position the company to continue pioneering RNA editing therapies for patients in need.

Source: https://www.quiverquant.com/news/ProQR+Therapeutics+Advances+Axiomer+RNA+Editing+Pipeline+with+Key+Milestones+Set+for+2025