Allergan, Editas Medicine partner to develop CRISPR genome-editing medicines for eye diseases

Allergan and Editas Medicine on Tuesday announced that the companies entered into a strategic R&D alliance under which Allergan will receive exclusive access and the option to license up to five of the latter's early-stage genome-editing ocular programmes, including its preclinical LCA10 programme for Leber's congenital amaurosis. Editas' CRISPR genome-editing platform, which includes CRISPR/Cas9 and CRISPR/Cpf1, "holds the potential to transform the treatment of many genetic and non-genetically derived diseases, including diseases and conditions of the eye," commented Allergan's chief R&D officer David Nicholson. He added that the LCA10 programme "is highly complementary to our ongoing eye-care development programmes." 

Under the terms of the agreement, Allergan will pay Editas $90 million upfront for the development of five candidate programmes, with Editas also eligible to earn unspecified near-term milestones specifically tied to LCA10. Allergan will be responsible for development and commercialisation of the optioned products, subject to Editas' option right to co-develop and co-promote up to two optioned products in the US. The deal also calls for Editas to receive development and commercial milestones, as well as royalty payments on a per-programme basis. 

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Editas has stated that it plans to file papers with the FDA by the end of the year to initiate the first clinical trials of the LCA10 treatment. The company's pipeline also includes investigational therapies for a form of retinitis pigmentosa called Usher syndrome, and herpes simplex virus type 1, which can affect the eye. Meanwhile, Allergan generated $660 million in revenue for eye therapies in 2016, mainly from treatments for glaucoma and dry-eye disease. 

In 2015, Editas Medicine signed a deal with Juno Therapeutics to develop chimaeric antigen receptor and high-affinity T cell receptor therapies for cancer using Editas' genome-editing technologies, including CRISPR/Cas9. Intellia Therapeutics and CRISPR Therapeutics are also advancing CRISPR-Cas9 treatments toward clinical testing, either on their own or through partnerships. Intellia has deals in place to develop treatments using its CRISPR technology with Novartis and Regeneron Pharmaceuticals. Meanwhile, CRISPR Therapeutics has set up a joint venture with Bayer aimed at developing treatments for blood disorders, blindness and congenital heart disease using the latter's gene-editing technology. CRISPR Therapeutics is also partnered with Vertex Pharmaceuticals to use its CRISPR-Cas9 gene-editing platform to develop therapies targeting the underlying genetic causes of disease. 

Earlier this year, the US Patent and Trademark Office issued a favourable ruling to the Broad Institute of MIT and Harvard University regarding certain CRISPR-Cas9 patents, which Editas exclusively licenses from the institute, in an intellectual property dispute with the University of California. For related analysis, see ViewPoints: Editas wins first round of gene editing patent dispute but rivals still think they can prevail.

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