World-first gene therapy delivers missing gene directly to infant's brain
An eight-month-old infant in Israel has become the first patient in the world to receive an experimental gene replacement therapy delivered directly into the brain to treat WOREE syndrome, a neurological disorder caused by mutations in the WWOX gene. The treatment involved neurosurgical administration of a functional copy of the gene directly into the infant's brain, with the goal of restoring normal neurological function.
One month after treatment, the child reportedly remained seizure-free and clinically stable, offering a first glimpse of what could become a new therapeutic approach for severe genetic neurological disorders.
While gene therapies have transformed treatment options for several inherited diseases, delivery to the central nervous system remains one of the field's greatest challenges. This case demonstrates the growing sophistication of precision gene therapy approaches, where treatments can be tailored to a specific mutation and delivered directly to the affected tissue.
Read more via Business Insider
Cartesian and WestGene partner to advance in vivo CAR-T for autoimmune disease
This week, Cartesian Therapeutics announced a strategic licensing agreement with China's WestGene Biopharma to accelerate development of an in vivo CAR-T platform for autoimmune diseases.
The collaboration will combine Cartesian's clinically validated mRNA CAR-T payloads with WestGene's targeted LNP delivery platform. The partners plan to initiate a Phase I study in patients with generalized myasthenia gravis in the second half of 2026, with initial clinical data expected in 2027. Unlike conventional CAR-T therapies that require cells to be collected, engineered and reinfused, the in vivo approach aims to generate CAR-T cells directly inside the patient through targeted delivery of genetic instructions.
The deal highlights the increasing role of cross-border partnerships in accelerating innovation. If successful, in vivo approaches could significantly expand access to CAR-T beyond oncology and into larger indications such as autoimmune disease, where scalability and cost will be essential for broad adoption.
Read more via The Pharma Letter and Globe Newswire
Sensorion drops hearing loss gene therapy as competition intensifies
French biotech Sensorion announced it will discontinue development of SENS-501, its gene therapy for OTOF-related hearing loss, following a strategic review of the evolving landscape. The decision comes just weeks after Regeneron's gene therapy Otarmeni became the first FDA-approved treatment for genetic hearing loss. Regeneron has also committed to providing the therapy free of charge to eligible patients in the United States, significantly altering the commercial dynamics of the indication.
Reflecting a broader shift occurring across the gene therapy sector, as more products reach commercialisation, scientific differentiation alone is no longer enough. Developers must increasingly assess whether a programme can compete against approved therapies, established clinical data and evolving pricing strategies.
Read more via Biopharma Dive and European Biotechnology
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