We're back with the 🎀 2025 Consumer Biotech Gift Guide 🎀:
Our top product picks this year include biodesigned palm wine, fasting in pill form, edible candles made from lab-grown quail fat, oral biomimetic GLP-1, probiotic clothing, cultivated salmon omakase, and much more...
Political extremism on both sides is bad, but the reason I tend to zero in on the far left is because it’s hijacked everything I’ve ever loved—literature, art, music—and has convinced almost the entire world that the value of art lies in its relation to social justice.
Your preferences in movies clothes books music sports food drink art doesn’t make you special. How you act in the airport and on the airplane makes you special
The cell and gene therapy field has already proven itself: personalized therapies for rare diseases and what look like actual cures in blood cancers are now a reality.
But the field is being gutted by manufacturing requirements. New for my blog:
https://t.co/7MexHbJk7R
1. A company is dying now because of manufacturing requirements. Ultra-rare disease company Grace Therapeutics may be forced to shut down after the FDA demanded a second manufacturing run it cannot afford.
2. Grace Therapeutics is far from alone. The launch of Carvytki, one of the most impressive cell therapies that leads to what are
3. Around 30% of batches of Carvytki were being thrown away for no good reason, due to overly stringent manufacturing requirements. The FDA tightened its potency assay thresholds between the trial and commercial approval. This led to an artificial spike in batches labelled as "manufacturing failures" or "out-of-specification".
4. This is absurd, because the potency assay isn't even a safety test. More importantly, the two parameters that most frequently triggered out-of-specification failures were traced not to genuine defects in the product but to minor technical artefacts in the assay itself, so noise in the measurement, as opposed to evidence of a non-functional therapy.
5. The human cost of this could have been significant. Imagine a 67-year-old with relapsed myeloma who has already been through five prior lines of treatment. While waiting for Carvykti, she is placed on bridging chemotherapy, a holding pattern designed to stop her disease from advancing while her cells are being processed. And, after weeks of deterioration and waiting, she is told that her batch has failed the potency assay by a few percentage points and sent home.
6. Rather than discard the failing batches, J&J routed them through expanded access, a pathway that allows patients to receive products that haven't cleared formal approval requirements. The catch is that companies cannot charge for therapies given this way, so every out-of-specification batch was administered for free. In exchange, J&J collected outcomes data. When they compared patients who received passing versus failing product, they found no meaningful difference between the two groups. This proved the FDA was wrong all along.
7. This is the rule, not the exception. 43% of cell and gene therapy programmes entering Phase III experience major disruptions, and 60% of those trace back to manufacturing and regulatory issues.
8. Although in the case of Carvytki patients ultimately got their treatment on time, this came at a great cost to the company. Such events have an important negative effect on further investment. Venture funding for cell and gene therapy declined significantly in the last years due to such fears. Until manufacturing risks become manageable, the next generation of cancer cures or personalized gene therapies may never get built.
quit my job today and a sales guy out of Germany sent me an incredibly heartfelt slack thanking me for teaching him the phrase “does a bear shit in the woods” when responding to something that is an obvious yes 😭