Ultra-Rare Cures, Four Shots
Phil Gara@phil
The idea
I believe AI and programmable medicine are about to make drugs for diseases with only a few hundred patients commercially viable, and that regulators letting one editing platform count as the drug across many mutations is the unlock. CHOP's umbrella trial under that framework is the near-term proof point I'm watching, so I built this for conviction, not balance: base and prime editing platforms, an AI drug discovery engine, and a beaten-down RNA editing name, each at a full 25% position with no cash cushion.
What it owns
- These platforms treat many different genetic mutations with a single tool, which regulators are beginning to recognize as one drug across multiple rare diseases rather than requiring separate approvals for each mutation. · Prime MedicinePrime editing can fix many different mutations with one platform, the exact setup that regulators reward when they treat the tool itself as the drug rather than requiring separate approvals per mutation., Beam TherapeuticsBase editing makes single-letter DNA fixes in the liver, where proving that one platform can treat many mutations is most straightforward, and it represents the core mechanism regulators are learning to approve.50%
- If artificial intelligence can make drug discovery faster and cheaper, the economics of treating rare diseases with small patient populations change entirely, making this approach to finding new medicines the take's foundation. · Recursion PharmaceuticalsIt represents the purest bet on whether artificial intelligence can make discovering drugs fast and cheap enough that diseases affecting only hundreds of patients become economically viable to treat.25%
- RNA editing offers a reversible way to correct mutations, providing a second programmable approach with significant upside if the core idea of treating ultra-rare diseases through genetic editing is proven right. · Wave Life SciencesIt edits RNA instead of DNA, offering a reversible way to correct mutations, and its steep decline has priced in market skepticism, making it the position with the largest potential gain if genetic editing for ultra-rare diseases succeeds.25%