Issue 207
It’s a good news week: the FDA has approved the first cell therapy to treat unresectable or metastatic melanoma. Though melanoma accounts for just one percent of all skin cancer cases, it is still responsible for a "significant number" of cancer-related deaths, as the FDA acknowledges. Indeed, the American Cancer Society estimates that in 2024, roughly 100,000 cases of melanoma will be diagnosed and about 8,000 people will die from the disease.
Developed by Iovance Biotherapeutics, Amtagvi (lifileucel) is a tumor-derived autologous T cell immunotherapy that was fast-tracked through the FDA’s accelerated approval pathway – now, the company is investigating the cell therapy in frontline advanced melanoma in a phase III confirmatory trial, as well as additional solid tumor types.
What new approvals are you most interested in this year? My inbox is open to opinions: [email protected]
Until next week,
Jamie Irvine | Associate Editor
Essential Reading
Another FDA approval?
The FDA has advised Sangamo Therapeutics that positive results from a single well-controlled clinical trial, alongside existing evidence, may be sufficient for approving its Fabry disease gene therapy candidate ST-920. During a meeting with the developer, the FDA agreed that the proposed trial would include up to 25 male and female participants with Fabry disease. The agency also agreed that the trial would not require a control arm or a head-to-head comparison with a standard enzyme replacement therapy, which is expected to allow the study to be done more rapidly and at reduced cost.
The Medicine Maker Presents… Industry 4.0
The cell and gene therapy space may not quite be the final frontier, but it is fascinating to learn that, as we find ourselves a quarter of the way through the 21st century, innovators and enablers are still going boldly beyond expectations so that one day we could all live long and prosper. In a recent feature, we spoke with four experts to help make sense of the varying definitions of Industry 4.0 – and what the fourth industrial revolution has in store for the current and future of cell and gene therapy manufacturing.
Worth Your Time...
Research
Researcher suggests the low incidence of secondary T cell lymphomas provides reassurance for the safety of commercial CAR-T cells.
George Buchman, Research Fellow at Catalent Cell and Gene Therapy, discusses how industry can ramp up manufacturing speed with platform processes in gene therapy.
Italian-based scientists assess the long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency.
Business
Astellas and Kelonia Therapeutics enter strategic research collaboration and license agreement to develop in-vivo cell therapies against various oncological targets.
Azzur Group expands service portfolio with the introduction of analytical testing services for advanced therapeutic medicinal products.
MIP Discovery closes £7-million series A financing round to improve speed, safety, and efficiency of manufacturing at scale for advanced therapies.
