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.
August 10, 2018 | News
Alnylam Gets Landmark FDA OK For First-Ever RNAi Drug
July 19, 2018 | News
IFM Therapeutics’ inflammation-focused subsidiary debuts with $31M
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.
July 17, 2018 | News
Spero nets $54M biodefense R&D contract for its lead antibiotic
July 17, 2018 | News
With $45M, HotSpot Takes Drug Research to New Biological Real Estate
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.
July 17, 2018 | LifeSciVC
Hotspot Therapeutics Turns Up The Heat On Natural Allostery
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.