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News|Podcasts|November 11, 2026

The BioPharm Brief: Base Editing Hits Its Mark in Sickle Cell, a Phase 3 Win in Sjögren’s, and Off-the-Shelf CAR-T in Lupus

Beam Therapeutics’ base-edited cell therapy risto-cel meets its primary endpoint in the BEACON sickle cell disease trial, Amgen’s CD40L antagonist dazodalibep reduces systemic disease activity in the phase 3 OASIZ-301 Sjögren’s disease trial, and ImvivaBio’s allogeneic CD19/BCMA CAR-T CTA313 sustains lupus remission through 12 months.

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry. Today: a milestone for base editing, phase 3 results in a disease with no approved therapy, and durable lupus remissions with an off-the-shelf CAR-T.

First, Beam Therapeutics says its base-edited cell therapy risto-cel met the primary endpoint of the BEACON trial in severe sickle cell disease. Risto-cel edits a patient’s own blood stem cells to boost fetal hemoglobin. The endpoint was at least 12 consecutive months free of severe pain crises. Beam has not yet said how many patients met it. The company plans to file a BLA as early as the end of 2026, which could make it the first base-editing therapy submitted to FDA.

Next, Amgen’s dazodalibep met its primary endpoint in the phase 3 OASIZ-301 trial in Sjögren’s disease. Dazodalibep is a fusion protein that blocks CD40 ligand, a signal that drives immune activation. In 648 patients, both dosing regimens reduced systemic disease activity more than placebo at week 48. Earlier antibodies against this target were halted over blood clot risks, but the trial showed no imbalance in thromboembolic events. Sjögren’s has no FDA-approved therapy.

And finally, ImvivaBio reported 12-month phase 1 data for CTA313, an off-the-shelf CAR-T made from donor cells that targets both CD19 and BCMA, in refractory lupus. Adding BCMA is meant to clear the antibody-producing plasma cells that CD19 alone misses. Seven of eight evaluable patients remained in remission at 12 months. The cohorts are small, and the company plans a global phase 1b basket study.

That’s today’s BioPharm Brief. For analysis and expert insights, please visit BioPharmInternational.com.

Key takeaways

  • Gene editing: Risto-cel could become the first base-editing therapy filed with FDA, though detailed efficacy and safety data are still to come.
  • Unmet need: Dazodalibep’s phase 3 results position it as a potential first approved therapy for Sjögren’s disease.
  • Allogeneic cell therapy: CTA313’s early data suggest dual-target, off-the-shelf CAR-T may sustain lupus remissions, pending larger studies.Dual-antigen CAR-T: Prizlon-cel’s designations support US development in patients who relapse after CD19 CAR-T, though it hasn’t been shown that dual targeting beats single targeting.

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