Get the latest tech news
What's going on with gene therapies?
Cell and gene therapies are treating previously untreatable diseases and saving tens of thousands of lives. So why are companies shutting down?
We now find ourselves at an inflection point where the science to perform such miracles, to cure rare and even ultra-rare genetic diseases — like spinal muscular atrophy (SMA), hemophilia A, even restoring vision — is not only advancing rapidly but is demonstrably here. All seven cancer-fighting CAR-T therapies, which include Yescarta for B-cell lymphoma and Kymriah for acute lymphoblastic leukemia (ALL), require similar steps — collect T-cells, modify with vectors, multiply, and reinfuse — and thus potentially have the same scaling drawbacks. Operational complexities, multi-million-dollar price tags, challenging reimbursement landscapes, and a slow and uncertain path to commercial viability represent systemic issues that threaten the broader promise of cell and gene therapies.
Or read this on Hacker News