The BioPharm Brief: Immunotherapy Wins, Next-Gen CAR-T, and Hope for Duchenne Patients

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry.

Today, we’re looking at positive late-stage oncology data from AstraZeneca, fresh investment in in vivo CAR-T technology, and encouraging pivotal Duchenne muscular dystrophy gene therapy results from REGENXBIO.

First, AstraZeneca reported positive interim results from its Phase III VOLGA trial evaluating an investigational perioperative immunotherapy combination in patients with muscle-invasive bladder cancer who are ineligible for or declined cisplatin chemotherapy. According to the company, the regimen demonstrated statistically significant improvements in both event-free survival and overall survival compared with surgery alone. AstraZeneca also said the safety profile was consistent with previously known data for the therapies involved. The findings add to growing industry interest in perioperative immunotherapy approaches for earlier-stage cancer treatment.

Meanwhile, Create Medicines announced it secured 122 million dollars in financing to support development of its in vivo CAR-T pipeline. The company is developing technologies designed to engineer CAR-T cells directly inside the body, rather than relying on traditional ex vivo manufacturing processes. Interest in in vivo approaches continues growing as developers look for ways to simplify production, reduce manufacturing complexity, and potentially expand patient access to cell therapies.

And finally, REGENXBIO announced positive topline Phase III results from its AFFINITY DUCHENNE study evaluating investigational gene therapy RGX-202 for Duchenne muscular dystrophy. The company reported that the trial met its primary endpoint, with 93 percent of participants achieving targeted microdystrophin expression levels at Week 12. Interim findings also showed a statistically significant correlation between microdystrophin expression and functional improvement in evaluated patients. REGENXBIO said the therapy was generally well tolerated and plans to pursue a potential accelerated approval pathway beginning in 2027.

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Summary of key developments

• AstraZeneca reported positive Phase 3 VOLGA trial results in muscle-invasive bladder cancer using perioperative Imfinzi plus enfortumab vedotin.
  
• Create Medicines raised $122 million to advance in vivo CAR-T therapy development and platform technologies.
  
• Both developments highlight continued momentum in oncology innovation and next-generation therapeutic platforms.