CARsgen secures world’s first approval for a CAR-T therapy in a solid tumour
CARsgen has achieved a major industry first after China’s National Medical Products Administration (NMPA) approved satri-cel for patients with Claudin 18.2-positive advanced gastric and gastroesophageal junction cancer who have failed at least two prior lines of therapy. The approval marks the first CAR-T therapy globally to receive regulatory clearance for a solid tumour indication.
Until now, CAR-T therapies have demonstrated their greatest success in haematological malignancies, while solid tumours have remained a persistent challenge due to tumour heterogeneity, hostile tumour microenvironment, and lack of truly selective tumour-associated antigens. CARsgen’s approval provides the first real-world validation that engineered cell therapies can overcome some of the biological barriers that have limited success in solid tumour settings.
The milestone is likely to accelerate investment into next-generation CAR-T platforms, tumour-targeting strategies, and approaches designed to improve persistence and trafficking within solid tumours. While questions remain around scalability, reimbursement, and long-term outcomes, the approval establishes an important proof point for the broader field and could reshape expectations for future solid tumour programmes.
Read more via pharmaphorum
FDA reverse course on Regenxbio’s Hunter syndrome gene therapy
In a significant regulatory turnaround, the US Food and Drug Administration has informed Regenxbio that existing clinical data may support an accelerated approval filing for the company's investigational gene therapy for Hunter syndrome (MPS II). The decision reverses the agency's previous position, which resulted in a Complete Response Letter earlier this year over concerns relating to trial design and evidentiary requirements.
Under the revised pathway, Regenxbio will not be required to enroll additional patients or conduct the previously requested placebo-controlled study. Instead, the company plans to resubmit its Biologics License Application in the third quarter of 2026 using existing and longer-term follow-up data. The move follows a series of recent FDA reconsiderations involving rare disease and advanced therapy programmes, suggesting a potential shift towards greater regulatory flexibility in ultra-rare indications where traditional clinical trial designs are often impractical.
The announcement also follows a similar reversal for uniQure's Huntington's disease gene therapy programme, raising questions about whether a broader regulatory recalibration is underway for advanced therapies targeting severe, unmet medical needs.
Read more via Biopharmadive
Sangamo Therapeutics files for bankruptcy with Lily and Astellas to acquire assets
Sangamo Therapeutics, one of the longest-standing companies in genomic medicine, has filed for Chapter 11 bankruptcy protection and entered into agreements to sell substantial portions of its portfolio and platform technologies. The company has identified Eli Lilly and Astellas as initial bidders in a court-supervised auction process.
Under the proposed transactions, Lilly would acquire several of Sangamo's core technology platforms, including its zinc finger gene regulation platform, capsid delivery technology, modular integrase platform, and prion disease programme. Astellas would acquire the company's Fabry disease gene therapy programme, ST-920. Other assets, including cell therapy and regulatory T-cell programmes, remain available for potential bidders.
Sangamo's restructuring reflects the increasingly difficult funding and commercialisation environment facing many advanced therapy companies. Despite decades of scientific innovation and multiple strategic partnerships, translating platform technologies into sustainable commercial success has remained a challenge.
At the same time, the interest shown by major pharmaceutical companies highlights the continued value of advanced therapy technologies, even when the originating companies struggle financially. The acquisition process may preserve and advance several important scientific programmes while serving as a reminder that breakthrough science alone is not always enough to guarantee long-term business viability.
Read more via FirstWord Pharma and Fierce Biotech
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