The FDA is now investigating 22 cases of blood cancer developing after CAR-T cell therapy treatment, said Marks, the director of the FDA’s Center for Biologics Evaluation and Research, on Monday at an Alliance for Regenerative Medicine event. “Although sequencing is not available for all of them, in at least a few of the cases we know that the CAR construct is in the malignant clone, which really suggests that there was probably an association there,” Marks said.
“And the onset of these was soon enough after the administration, in some cases, that it does look like there’s a causal relationship,” he continued.
The FDA announced in November that it was reviewing the safety of CAR-T therapies after reports of some cancer patients developing a second blood cancer following treatment. In December, the agency added a warning label on secondary cancers to Carvykti, a CAR-T cell therapy developed by Legend Biotech and Johnson & Johnson for multiple myeloma.
But as clinicians and cell therapy experts have said previously, Marks was keen to emphasize that the regulatory agency still believes the benefits of these treatments outweigh the safety risks.
That includes not only approved indications in blood cancers, but clinical trials in autoimmune diseases as well, where impressive early results have jolted the field’s interest.
“Where we are settling out is that, in general, the risk benefit is still going to be favorable to move forward in those development plans,” Marks said. “And that’s in part because autoimmune diseases are not benign diseases. Anyone who’s ever known somebody who’s had lupus cerebritis or lupus nephritis will know that those are potentially lethal diseases.
“Those development programs can and should continue. We may ask for some additional safety monitoring, more to come on that,” Marks continued.
![GMP mRNA](https://static.packgene.com/wp-content/uploads/2023/07/13.jpg)
Check out our AAV CDMO service to expedite your gene therapy research
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
Related News
Skyline Therapeutics Receives FDA Orphan Drug Designation for Gene Therapy for Retinitis Pigmentosa
BOSTON & SHANGHAI, September 2, 2024 – Skyline Therapeutics, a gene therapy company focused on innovative treatments for rare and severe diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for SKG1108....
Novavax Receives Emergency Use Authorization for Updated Covid-19 Vaccine
Novavax has received emergency use authorization (EUA) from the U.S. Food and Drug Administration (FDA) for its updated Covid-19 vaccine, NVX-CoV2705, joining other vaccine manufacturers in addressing evolving virus variants. The EUA permits the use of Novavax's...
Turn Biotechnologies Reports Historic Skin Cell Rejuvenation Breakthroughs at ESDR this Week
Company will present data from its successful rejuvenation of fibroblasts and keratinocytes to influential gathering of international dermatologists MOUNTAIN VIEW, Calif., Sept. 4, 2024 /PRNewswire/ -- Turn Biotechnologies, a developer of novel mRNA medicines and...
H5 influenza vaccines: What needs to be done to reduce the risk of a pandemic
As the global threat of H5N1 influenza looms with outbreaks across species and continents including the U.S., three international vaccine and public health experts say it is time to fully resource and support a robust strategy to address this and future potential...
Related Services
![](https://static.packgene.com/wp-content/uploads/2023/01/9-1.png)
Plasmids GMP Services
Multiple scales & grade of solutions of various kind of plasmids suitable for multiple treatments in a fast and cost effective way.
READ MORE
![](https://static.packgene.com/wp-content/uploads/2023/01/10-2.png)
AAV GMP Services
Ranging from small-scale AAV production, to large-scale AAV cGMP manufacturing for animal studies.
READ MORE
![aav icon](https://static.packgene.com/wp-content/uploads/2023/01/9-1.png)
Technology Platforms
PackGene’s proprietary π-Alpha™ 293 AAV High-yield Platform increases AAV production by 3 to 8 times that of traditional platforms.
READ MORE