sirna
(5)
Alnylam Pharmaceuticals
Alnylam spent 16 years teaching a fragile strand of RNA to reach the right cells. Now its gene-silencing medicines are a multibillion-dollar business - and the next test is whether the same platform can move from rare diseases into the medical mainstream.
City Therapeutics
City Therapeutics is a Cambridge, Massachusetts biotechnology company engineering the next generation of RNA interference (RNAi) medicines. Founded by pioneers of the RNAi field, including former Alnylam CEO John Maraganore and Nobel-adjacent academic scientists, the company designs novel siRNA trigger molecules and targeting ligands intended to make RNAi drugs more potent, more specific, and able to reach tissues beyond the liver. Launched in October 2024 with a $135M Series A and backed by a further $99.5M Series B in 2026, City is advancing a pipeline led by CITY-FXI for thromboembolic disease and CITY-RBP4 for Stargardt disease, alongside research collaborations with Biogen and Bausch + Lomb.
Creyon Bio
Creyon Bio is a San Diego biotech reinventing how RNA-targeted medicines are designed. Founded in 2019 by former Ionis Pharmaceuticals scientists, the company pairs machine learning with quantum-level molecular modeling in its NucleIQ platform - an AI-Powered Oligo Engineering Engine - to predict the safety, efficacy, and delivery of oligonucleotide-based medicines before they ever reach a lab bench. The goal: replace slow, expensive trial-and-error with computational design rules that make precision therapeutics engineerable 'on demand,' for both rare and common diseases across neuromuscular, CNS, and immunologic areas.
Portal Biotechnologies
Portal Biotechnologies is a Watertown, Massachusetts cell-engineering platform company building tools that push complex cargo - mRNA, siRNA, CRISPR complexes, proteins - into living cells by squeezing them through microscopic holes in a silicon surface, rather than relying on viruses or chemistry. Founded in 2023 by Armon Sharei, the MIT-trained scientist behind SQZ Biotechnologies, Portal sells hardware and consumables to drug discovery and cell therapy labs instead of developing its own drugs. Its instruments are used across more than 100 customer sites, including many of the world's largest pharmaceutical companies, and the company has raised seed and follow-on funding plus a DARPA contract for portable, point-of-care cell manufacturing.
Switch Therapeutics
Switch Therapeutics is a South San Francisco preclinical biotech building conditionally activated siRNA (CASi) therapies that only switch on inside the cell types where they're needed. The platform - born from research at Caltech, Harvard and City of Hope - aims to make RNA interference safe enough for the brain, with a lead program targeting APOE for Alzheimer's disease.