The FDA has just approved Alnylam’s patisiran (Onpattro), making it the first medicine specifically cleared to treat a rare and deadly disease called hereditary transthyretin amyloidosis (hATTR). The decision is a scientific milestone too: it marks the first-ever approval for a medicine that uses RNA interference (RNAi), a method cells can use to silence a gene before it makes a harmful protein. The FDA’s approval is specifically for the treatment of the peripheral nerve damage suffered by hATTR patients. The agency’s press release doesn’t mention the heart problems patients also often deal with.
IFM Therapeutics is hiving off its NLRP3 stable into a subsidiary dubbed IFM Tre. The new unit, which starts out with $31 million in series A funding, expects to bring its lead asset into phase 1 in 2019.
In real estate, location is key. The same holds true in the hunt for new drugs. HotSpot Therapeutics is trying to break new ground in drug discovery by hitting locations on a protein that aren’t the conventional targets of most medicines.
Most conventional small molecule drugs engage directly with the engine of a protein’s function, aiming to often turning off an enzymatic activity associated with a disease. These drugs bind and compete against the natural substrate of the protein at what are known as orthosteric sites. But other pockets on a protein can also modulate function, and these sites commonly referred to as allosteric sites.