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The gene-therapy start-up Encoded Therapeutics has emerged from stealth mode after raising $104 million in series C financing. The company’s lead program is for a type of epilepsy called Dravet syndrome, caused by a deficiency in a sodium channel. The sodium channel gene is too large to be delivered via viral gene therapy, so Encoded scientists devised a creative fix: they made a synthetic transcription factor that boosts expression of the gene, which itself is delivered with gene therapy. They’ve successfully tested the concept in mice and monkeys.
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