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Welcome to Phacilitate’s weekly cell and gene therapy (CGT) news round-up, which will bring you the latest advanced therapies developments each Friday in the lead up to Advanced Therapies Week 2025 in Dallas.
This week’s round-up has a particularly heavy UK focus with news about a B cell lymphoma who is now cancer free after CAR-T cell therapy, along with the formation of the London Advanced Therapy Center and a deep dive into the UK’s CAR-T capabilities.
If you have any news you’d like to share, or if you’d like to add additional commentary, please get in touch.
Read on to find out what else has been happening this week.
Headlines
The first news story is a good reminder of the transformative power of cell therapy, and why we’re all working so hard in this industry. After being given only two weeks to live after four rounds of chemotherapy for B cell lymphoma, 58-year-old Lisa Noble is now cancer free after CAR-T cell therapy. She became one of the first patients at Addenbrooke’s Hospital in Cambridge, England to be given CAR-T cell therapy.
The BBC also reports that The NHS and University of Cambridge have joined forces to create a new cellular therapy laboratory, which they hope will unlock new and potentially “kinder” treatments for people with cancer.
The second headline this week also touches upon the UK’s CAR-T capabilities. Pharmacy Times reports that while the UK is one of the few health systems in the world that offers patients access to so many commercial CAR-T products, there may be some bumps in the road without sufficient investment.
With this in mind, UK stem cell charity Anthony Nolan has outlined the actions the UK Government can take to future-proof the country’s thriving cell therapy system.
With Advanced Therapies Week moving to Dallas for 2025, we’re always excited to cover Texas biotech news. Temperature-controlled supply chain solutions firm Cryoport has partnered with VGXI, a subsidiary of GeneOne Life Science, to support the development of cell and gene therapies and mRNA-based treatments. Cryoport will provide biostorage and bioservices for plasmid DNA products manufactured at VGXI’s facilities in the Woodlands, Texas, and its headquarters and manufacturing location, also in Texas.
Industry Analysis
The FDA approved two gene therapies for sickle cell disease in late 2023, but they are not being used as much as expected. In an interview with AJMC, Kevin Niehoff, PharmD, BCMAS, associate director, Market and Financial Insights, IPD Analytics, puts this down to a few factors that make these gene therapies different from others approved before them.
Fierce Biotech has covered the news that results from a MeiraGTx clinical trial indicated that its gene therapy improves motor function and quality of life in phase 2 Parkinson’s. The company is now discussing the possibility of initiating a phase 3 trial with regulators in the U.S., Europe and Japan.
Clinical Trials and Therapeutic Development
A systematic review and meta-analysis of 16 prospective clinical trials and real-world studies showed that CAR T-cell therapy demonstrated efficacy and manageable adverse effects in patients with relapsed/refractory mantle cell lymphoma.
King’s College London, Guy’s and St Thomas’ NHS Foundation Trust and King’s College Hospital NHS Foundation Trust have been awarded funding to form the London Advanced Therapy Treatment Centre, alongside partners across the city. The new center will be focused on accelerating the delivery of cell and gene therapy clinical trials.
The FDA approved a new treatment for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients 12 years of age and older with hemophilia A or B without inhibitors. Pfizer’s Hympavzi is a new type of drug that, rather than replacing a clotting factor, works by reducing the amount, and therefore, the activity of, the naturally occurring anticoagulation protein called tissue factor pathway inhibitor.
Manufacturing and Supply Chain News
A new partnership will see Fresenius Kabi’s suite of cell therapy processing technologies digitally and physically integrated within Cellular Origins’ robotic system, Constellation. The aim is to help cell therapy developers to manufacture their therapies at scale using their preferred processing tools.
CGT Catapult, Pharmaron Biologics (UK), and Complement Therapeutics have been awarded a £1.4m Innovate UK Transforming Medicines Manufacturing (TMM) Programme grant to develop a perfusion process that will improve gene therapy production.
If you want to continue the discussion, why not register for Advanced Therapies Week 2025 in Dallas, the must-attend event for the biotech ecosystem.
