ProQR是BoA在RNA编辑平台上的新产品

2026-08-20 21:49

Bank of America Securities has initiated coverage of ProQR Therapeutics (PRQR) at buy, saying that the company's Axiomer RNA-editing platform has been validated, supporting its pipeline.
The bank has an $8 price target (~260% return based on August 19 close).
Analyst Alec Stranahan noted that AX-0810, under investigation for cholestatic disorders that are caused by a buildup of bile acids in the liver, has been validated in humans and could help about 20,000 patients worldwide in a market with no disease-modifying therapy.
Ipsen's (IPSEY)(IPSEF) Bylvay (odevixibat) and Mirum Pharmaceuticals' (MIRM) Livmarli (maralixibat) are currently used to treat the condition but use a different mechanism of action.
Stranahan added that some of the company's other candidates—AX-0422 for Hurler syndrome, AX-2911 for genetic liver disease, and AX-2402 for Rett syndrome—"each offer additional multi-billion-dollar market opportunities."
In September 2022, ProQR signed a discovery agreement with Eli Lilly (LLY) for genetic disorders. That partnership includes 10 targets, with a potential of five more, and "adds external validation as well. "

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