rnai-therapeutics
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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.
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.