Cell therapy weekly: advocacy groups push to restore Tecartus access
This week: OXB (Oxford, UK) and Bristol Myers Squibb (NY, USA) signed a new multi-year agreement for CAR-T therapy manufacturing and Ultragenyx Pharmaceutical Inc. (CA, USA) provided long-term data on its AAV gene therapy indicated for a fatal neurodegenerative disorder. Plus, patient advocacy groups and healthcare organizations in the UK have expressed concerns following the removal of a CAR-T therapy for relapsed or refractory mantle cell lymphoma from the NHS in England.
The news highlights:
A new multi-year agreement for CAR-T therapy manufacturing
OXB, a contract development and manufacturing organization, and Bristol Myers Squibb have signed a new Commercial Supply Agreement for the manufacture and supply of lentiviral vectors for Bristol Myers Squibb’s CAR-T programs. This new multi-year agreement, which will allow OXB to commence commercial manufacturing, builds on the existing partnership between the companies.
“The transition from clinical to commercial manufacturing with BMS marks an important milestone for both companies,” commented OXB’s Chief Business Officer, Sebastien Ribault. “Having supported these potentially life-saving CAR-T programs through development, we are confident in their commercial success and look forward to continuing our partnership with BMS. This long-term commitment underlines our excellent revenue visibility, reflects our strong track record, and demonstrates continued successful execution of our strategy as a leading cell and gene therapy [contract development and manufacturing organization].”
Positive long-term data on AAV gene therapy for fatal neurodegenerative disorder
Ultragenyx announced promising long‑term data from studies evaluating its investigational AAV9 gene therapy, UX111, for Sanfilippo syndrome Type A (MPS IIIA). MPS IIA is a rare fatal neurodegenerative lysosomal storage disorder. MPS IIIA is characterized by rapid neurodegeneration, progressive cognitive, language and motor decline, behavioral abnormalities and early mortality, with a median life expectancy of 15 years. Currently, there is no approved treatment for this disorder.
UX111 is designed to address the underlying enzyme deficiency in the brain that leads to progressive cell damage and neurodegeneration. The therapy is currently in Phase I/II/III development, and clinical study results have demonstrated several key findings:
- Safety and tolerability: UX111 was well-tolerated and demonstrated a favorable safety profile.
- Biomarker improvements: substantial and durable improvements in biomarkers were observed
- Functional skill retention in later-stage patients: older children and those with advanced disease at the time of treatment showed retention of functional abilities across three areas of assessment
- Meaningful functional benefits compared to natural history: patients demonstrated improvements in receptive and expressive communication, as well as fine and gross motor skills. Notably, eight children reached a 36-month cognitive development milestone – a milestone that none of the natural-history patients were able to achieve.
“These data continue to demonstrate a remarkable and unprecedented separation from the natural history of Sanfilippo syndrome through more than eight years of follow-up, with children in their teens retaining skills at an age when many of their untreated peers have sadly lost their most basic abilities and succumbed to this disease,” Emil D. Kakkis, CEO and President of Ultragenyx. “Our studies consistently show that reductions in heparan sulfate are associated with meaningful clinical benefits across multiple domains, underscoring the urgency to bring forward a treatment for families who currently have no options to stop or delay the heartbreaking and inevitable progression and loss of function associated with this disease.”
Advocacy groups push to restore Tecartus access
Following the removal of Tecartus as a treatment for relapsed or refractory mantle cell lymphoma on January 22, 2026, on the NHS in England, Anthony Nolan (London, UK), Blood Cancer UK (London), and Lymphoma Action (Buckinghamshire, UK) are urging the National Institute for Health and Care Excellence (NICE; Manchester, UK) to reconsider its decision. Tecartus, a CAR-T cell therapy, has been available on the NHS in England and Wales for the past five years and is currently the only CAR-T cell therapy approved for patients with relapsed or refractory mantle cell lymphoma.
The organizations emphasize the critical importance of Tecartus for patients with this aggressive form of blood cancer, advocating for NICE to reassess its decision to ensure continued access to this “last-hope-of-a-cure” therapy.
“For some people with mantle cell lymphoma, whose cancer has come back or hasn’t responded to previous treatment, this CAR T-cell therapy offers a last hope of a cure. We recognize that NICE decisions involve complex clinical and economic considerations, but we are concerned about what this could mean for patients who have very limited alternatives,” commented Rubina Ahmed, director of research, policy and services at Blood Cancer UK. “That’s why we have formally submitted an appeal, raising questions about how this treatment has been assessed and the implications of this decision for patients. It’s vital that advances in blood cancer treatment are reflected in the options available to people in practice.”
