gene-silencing
(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.
Flashpoint Therapeutics
Flashpoint Therapeutics is a clinical-stage biotech spun out of Chad Mirkin's nanotechnology lab at Northwestern University, developing 'structural nanomedicines' - drugs whose potency comes not from new molecules but from how existing therapeutic components are arranged at the nanoscale. By co-delivering RNA, DNA, peptide and CRISPR payloads to individual cells with precise stoichiometry and synchronized activation, the company reports it can turn conventionally ineffective drug components into curative therapies, with early data showing dramatically stronger immune activation and tumor killing. Founded by Adam Margolin and backed by a $10M seed round led by Riyadh-based Beta Lab plus a $50M KAIMRC partnership, Flashpoint is advancing its lead immuno-oncology candidate FLASH-001 through the clinic.
Atalanta Therapeutics
Atalanta Therapeutics is a Boston-based biotechnology company pioneering RNA interference (RNAi) therapies for diseases of the brain and spinal cord. Its proprietary divalent small interfering RNA (di-siRNA) platform is engineered to silence disease-causing genes durably and broadly across the central nervous system - long the hardest place in the body to reach with RNAi. Founded out of the RNA Therapeutics Institute at UMass Chan Medical School by leaders in the RNAi field, including Nobel laureate Craig Mello, the company is advancing wholly-owned programs in KCNT1-related epilepsy and Huntington's disease toward the clinic, alongside collaborations with Biogen and Genentech.
Joanne Kotz
Joanne Kotz is a chemist-turned-biotech-CEO who runs Atalanta Therapeutics, a Boston company using divalent siRNA to silence disease-causing genes throughout the brain and spinal cord. She took the top job in June 2025 after co-founding and leading Jnana Therapeutics from a blank slate to a roughly $1 billion acquisition by Otsuka. Before founding companies she helped build them from the science up, as a director at the Broad Institute, a leader of F-Prime's neurodegeneration initiative, and the founding editor of Nature Chemical Biology. Her career runs an unusual loop: bench scientist, science journal editor, partnership architect, founder, and now CEO of a company chasing some of the hardest diseases in the central nervous system.