Dinakar Singh
👤 SpeakerAppearances Over Time
Podcast Appearances
that had what's called an antisense oligos with the first biologics.
Essentially, the viral vector takes a protein in the body, and it was designed to patch that defect in that backup gene and help that backup gene become a fully functioning regular gene, which would be magic, presto.
That became the first drug, the one the biogen ended up buying in.
Second was good old-fashioned small molecules, syringes and pills.
A bit like Novartis, for example, had found something that worked.
We basically paid a company called PTC Therapeutics lots and lots of money to go and test everything under the sun they could imagine with different screens and ended up finding one that really, really worked.
And so we basically just paid to develop that.
And then I actually got Roshan to go and take it from there.
That's actually the drug my daughter's on now.
In between was gene therapy, which a company called Avexis did, and then Novartis bought in for $8 billion.
And I guess this was really the first gene therapy drug ever approved.
Here, we didn't support it as directly from a financial and other perspective, though our board members were active investors, and we helped them with the network and the library and the clinical trial networks.
Our focus, just being selfish to some degree, was that gene therapy is great, but only works for very little kids.
Essentially, in gene therapy, you're taking viruses, you're bringing protein in your body.
And when you're large, that amount of virus will kill you, basically.
The toxicity becomes an issue.
But we wanted all three shots on goal to be explored.
Look, we got lucky.
Today, we have all three as approved drugs.
So the first was the biogen cells.