A new gene therapy using patients’ own stem cells has safely cured ADA-SCID — the “bubble boy” disease — in 95% of children, restoring full immune function.
A new gene therapy using patients’ own stem cells has safely cured ADA-SCID — the “bubble boy” disease — in 95% of children, restoring full immune function.
This is so amazing. Lentiviral therapies are exploding, although I’m used to seeing them referred to as “Cell Therapies” to distinguish them from AAV gene therapy that doesn’t use the patients cells to produce the target viral vector.
The trick now is being able to get a commercial manufacturer to take the risk of developing this into an approved product, knowing that a. It’s a one time treatment so your customer base erodes down to only new babies born with the condition after a few years, and b. Insurance companies will fight not to pay since there are existing treatments that will likely be cheaper.
Best of luck to whatever startup decides to take on that challenge.
This is why drug research should be nationalized; society should pay for and benefit from the research taxes pay for. Right now, we pay for research and only get the ‘profitable’ outputs.
Some ghoul company will charge about $2M for this therapy, which a few parents can pay, but most cannot and hence, fuck those kids. That is the American way.
This has been happening with SMA disease for years. Novartis gives away doses by lottery. You win, yay, you lose, your baby dies.
Vasant Narassimhan sleeps very well in a mansion in Boston. He makes $13,000,000 a year.
Why do we need a commercial interest?
NIH should do this. Too bad they were shit-canned by the Fascists.
He’s got immunities!!!
Seriously, good news.