Vertex bolsters gene editing capabilities with Exonics Therapeutics buyout, expanded CRISPR Therapeutics partnership

Vertex Pharmaceuticals announced Thursday that it entered into a definitive agreement to acquire Exonics Therapeutics, and has also broadened its existing collaboration with CRISPR Therapeutics, in a move it says "[enhances] its gene editing capabilities" to develop novel therapies for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). 

As part of the Exonics deal, Vertex will acquire all outstanding shares of the company, which will become a separate wholly-owned subsidiary of Vertex. Exonics equity holders are eligible to receive payments of about $1 billion, including $245 million upfront plus potential future payments based on the achievement of certain milestones for the DMD and DM1 programmes. 

Exonics focuses on gene editing therapies to repair mutations that cause DMD and other severe neuromuscular diseases, with Vertex noting that "in multiple small and large animal DMD preclinical models, Exonics has used SingleCut CRISPR to genetically repair and restore dystrophin, the key protein missing in children with DMD." The acquisition is slated to close in the third quarter. 

Meanwhile, Vertex said it will pay $175 million upfront for the exclusive worldwide rights to CRISPR Therapeutics' existing and future intellectual property, including foundational CRISPR/Cas9 technology, novel endonucleases, single and double cut guide RNAs, and adeno-associated virus vectors for DMD and DM1 gene editing products. 

CRISPR Therapeutics is eligible to receive up to $1 billion, which includes the upfront payment, as well as potential future milestones for the DMD and DM1 programmes. Vertex will also pay tiered sales royalties on products stemming from the collaboration. Further, upon the filing of an investigational new drug application, CRISPR Therapeutics will be able to choose whether to forego the DM1 milestones and royalties in favour of co-developing and co-commercialising all DM1 products globally. 

Vertex will be responsible for all R&D, manufacturing and marketing activities and related costs tied to the DMD programme. For the DM1 programme, both companies will share research costs for specified guide RNA research to be conducted by CRISPR Therapeutics, while Vertex will assume  all other costs associated with R&D, manufacturing and commercialisation. This transaction is also expected to be finalised in the third quarter. 

Vertex CEO Jeffrey Leiden remarked that through both deals, "we are bringing together the intellectual property, technologies and scientific expertise needed to establish a leading gene-editing platform for DMD and DM1." He added that Vertex is "continuing to build a toolbox of small molecule and nucleic acid technologies and capabilities that will allow us to drive scientific innovation to produce transformative medicines for a broad portfolio of diseases." 

Last month, Vertex and CRISPR Therapeutics said the FDA had fast-tracked their experimental gene-edited haematopoietic stem-cell therapy, CTX001, for patients with severe haemoglobinopathies. 

Meanwhile, the Institute for Clinical and Economic Review (ICER) recently released its first-ever draft evidence report of treatments for DMD, including Sarepta Therapeutics' Exondys 51 eteplirsen) and the company’s other DMD drug golodirsen, which is under FDA review, as well as PTC Therapeutics' Emflaza (deflazacort). ICER suggested that evidence that the medicines work was so sparse that the group could not even say how much they should cost. Sarepta has criticised ICER's approach as "fatally flawed" and that "ICER's model is unfit to evaluate rare disease populations in a manner that would encourage innovation." 

For more on what’s happening in the gene therapy space, see ViewPoints: ASGCT 19 Preview- what to watch in gene and cell therapy. See also, ViewPoints: CRISPR Therapeutics finally makes it official with first patient treatment.    

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