Soufflé Therapeutics

Soufflé Therapeutics is a biotechnology company developing nucleic acid medicines designed to target and enter specific disease-causing cell types. The company engineers cell-specific ligands that deliver siRNA-based therapeutics across the cell membrane to their intended targets, combining proprietary approaches to identifying cell-specific receptors, optimizing ligands, and engineering potent siRNA. The company states its delivery approach is replicable across cell types and intends to address both rare genetic conditions and broader indications.

Initial programs target skeletal muscle and cardiomyocytes, aimed at muscular dystrophies, heart failure, and metabolic disorders. The company's first therapies address facioscapulohumeral muscular dystrophy (FSHD) and genetically driven cardiomyopathies linked to a mutation of the phospholamban (PLN) gene. Alongside these lead programs, Soufflé reports a range of additional pre-clinical programs spanning multiple cell and tissue types.

Founding story

Soufflé was founded and is led by researchers and investors in genetic medicine. CEO Amir Nashat is an Entrepreneur Partner and former Managing Partner at Polaris Partners, where he worked with companies including Alnylam Pharmaceuticals and Acceleron Pharma and founded or led investments in Dewpoint Therapeutics, Fate Therapeutics, Jnana Pharmaceuticals, Morphic Therapeutics, Paratus Sciences, Receptos Pharmaceuticals, and Scholar Rock; he completed his ScD at MIT in the laboratory of Robert Langer. Founding Chief Technology Officer Vadim Dudkin previously founded and led RNA and Targeted Therapeutics at Johnson & Johnson and earlier led siRNA conjugate research at Merck.

Traction

The company reports lead programs in FSHD and PLN-mutation cardiomyopathies, a diversified pre-clinical portfolio across multiple cell and tissue types, and a stated plan to begin clinical trials in 2026. It has assembled a leadership team spanning discovery biology, protein science, preclinical and clinical development, regulatory, quality, legal, finance, and patient advocacy.