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Vertex Pharmaceuticals expands into Duchenne gene therapy with new deals

(Reuters) – Vertex Pharmaceuticals Inc said here on Thursday it would acquire privately held Exonics Therapeutics for an upfront payment of $245 million and potential milestone payments of up to $750 million.

Exonics Therapeutics develops gene editing therapies to treat patients with severe genetic neuromuscular diseases.

Shareholders of Exonics Therapeutics will also be eligible for milestone payments based on successful achievement of development and regulatory milestones for its Duchenne Muscular Dystrophy (DMD) and another muscle wasting disorder Myotonic Dystrophy Type 1 (DM1) programs.

DMD is a rare, genetic disorder that hampers muscle movement, mainly in men, affecting one in every 3,500 to 5,000 males across the globe.

Vertex also said on Thursday the company is expanding its collaboration with Switzerland-based gene-editing company Crispr Therapeutics for the exclusive worldwide rights to its gene editing products for DMD and DM1.

Crispr is eligible to receive payments of up to $1 billion, which includes a $175 million upfront payment and potential future milestone payments, Vertex said.

Reporting by Manojna Maddipatla in Bengaluru; Editing by James Emmanuel

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