Phase I
Under the terms of the agreement, OPKO will accept 60% of the development costs, while Entera will shoulder 40%.
Dyne is eyeing an accelerated approval filing for DYNE-251 in early 2026 that would pit the asset against Sarepta’s Exondys 51 in a patient population amenable to exon 51 skipping.
Having established success in cancer, biopharma is now looking to leverage CAR T therapies against a new target, autoimmune disorders, with several early- to mid-stage readouts expected this year.
Pfizer was studying PF-07820435, an orally available agonist of the STING protein, for solid tumors.
Gilead plans to go straight to Phase III studies for once-yearly lenacapavir, while GSK and ViiV will push forward with their long-acting antivirals after touting positive early-stage results.
BEAM-302 “has set the bar for efficacy in this space,” William Blair analysts wrote in an investor note on Monday.
The vaccine space has been battered by strong headwinds in recent weeks, including high-level disruptions to FDA and CDC advisory committee meetings.
The treatment, called DB-OTO, is one of several early-stage gene therapies being developed to treat relatively straight-forward causes of genetic deafness.
With the modality now in early clinical trials, experts say more efficiency, broader editing capabilities and delivery breakthroughs are needed to propel RNA editing to the next stage.
Exelixis’ next-generation tyrosine kinase inhibitor zanzalintinib is being tested for colorectal cancer, renal cell carcinoma and head-and-neck cancer, with several readouts slated for the second half of 2025.
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