Ultragenyx expands into Duchenne gene therapy with Solid Bio deal

Ultragenyx Pharmaceutical, a California-based biotech best known for its rare disease drugs, plans to try its hand at developing a gene therapy for Duchenne muscular dystrophy, announced on Friday 23rd a deal to license a key gene therapy component from Solid Biosciences. Ultragenyx will package the component, a synthetic gene encoding for the crucial protein Duchenne patients lack, into a type of virus that it’s developed to deliver DNA into the body’s cells. Two other gene therapies from Ultragenx that use the virus, a variant of adeno-associated virus 8, are now in clinical trials for other rare diseases. Per deal terms, Ultragenyx will purchase roughly 7.8 million Solid shares for $40 million, valuing each at the equivalent of $5.11 apiece — a nearly 50% premium over the stock’s closing price Thursday. Solid could receive another as much as $255 million more in conditional payments, although those may never materialize. 

For further information, see BIOPHARMA DIVE (https://www.biopharmadive.com/news/ultragenyx-solid-bio-duchenne-gene-therapy/587639/?utm_source=Sailthru&utm_medium=email&utm_campaign=Issue:%202020-10-23%20BioPharma%20Dive%20%5Bissue:30433%5D&utm_term=BioPharma%20Dive)

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