A clinical-stage biotech developing small-molecule drugs for rare genetic diseases like muscular dystrophy and sickle cell, aiming to correct underlying gene misregulation.
No approved products; revenue came from collaborations (USD 0.08B in 2024) with partners like Acceleron and MyoKardia, but operating losses persist (USD -0.02B in 2024).
Early-stage science and partnerships provide some protection, but no marketed drug or proprietary platform yet—competitors like Sarepta and Vertex could dominate similar targets.
Key events, in time order
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Fulcrum Therapeutics and Slate Medicines announced a merger to advance migraine therapies. The deal is a concurrent oversubscription, indicating strong investor interest.
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