Vertex signs $100m CRISPR deal to use sickle cell treatment

Biotech firm agrees licence deal days after its gene editing treatment gets landmark FDA approval | Patents owned by the Broad Institute protect technology sold via Editas Medicines.

Vertex Pharmaceuticals has signed a licence agreement for Cas9 technology owned by the Broad Institute group of CRISPR inventors, the largest deal involving the gene-editing innovation to date.

The agreement sees Vertex, which focuses on serious diseases, license patents from Editas Medicine, a clinical-stage genome editing company and exclusive licensee of certain CRISPR patent estates for making human medicines—including Cas9 patents owned and co-owned by Harvard University, the Broad Institute, the Massachusetts Institute of Technology (MIT), and The Rockefeller University.

Under the terms, Vertex will obtain a non-exclusive license for Editas Medicine’s Cas9 gene editing technology for ex vivo gene editing medicines targeting the BCL11A gene in the fields of sickle cell disease and beta thalassemia, including Casgevy (exagamglogene autotemcel).

The deal secures Editas Medicine’s “cash runway” until 2026, the company said in a statement.

Vertex is paying up to $100 million, plus potential licensing fees, for rights to the technology, according to  Fierce Pharma.

Editas will receive $50 million in upfront cash and is eligible to receive another $50 million through a contingent payment, said the report.

The biotech may also receive yearly licensing fees ranging from $10 to $40 million per year through the patent's expiry in 2034, an Editas spokesperson told Fierce Biotech via email.

First CRISPR/Cas9 treatment brings relief for sickle cell sufferers

Under the new deal, Vertex secures non-exclusive licensing rights to Editas’ Cas9 tech for ex vivo gene-editing medicines, targeting the BCL11A gene in sickle cell disorder (SCD) and beta thalassemia, including CRISPR Therapeutics-partnered Casgevy (exagamglogene autotemcel).

SCD is a group of inherited blood disorders most common among people with an African or Caribbean family background.

Casgevy achieved landmark approval at the US Food and Drug Administration (FDA) on December 8, becoming the first approved treatment to use CRISPR technology.

Under the treatment, patients’ hematopoietic (blood) stem cells are modified, with CRISPR/Cas9 technology directed to cut DNA in targeted areas, to accurately edit (remove, add, or replace) DNA where it was cut, said the FDA.

The modified blood stem cells are transplanted back into the patient where they engraft (attach and multiply) within the bone marrow and increase the production of fetal hemoglobin (HbF), a type of hemoglobin that facilitates oxygen delivery.

In patients with sickle cell disease, increased levels of HbF prevent the sickling of red blood cells.

The one-time treatment will be among the most expensive ever sold, with a price tag of $2.2 million, reported MIT Technology Review.

In statement issued via email to LSIPR, Editas said: “As the exclusive licensee of Broad Institute’s Cas12a patent estate and Broad Institute and Harvard University’s Cas9 patent estates for human medicines, we are uniquely positioned to issue exclusive and non-exclusive sub-licences for Cas9 to any company seeking to use this enzyme to make human medicines—including in vivo and ex vivo uses.

“We regularly engage in licensing discussions regarding this technology with other companies, and we can grant exclusive and non-exclusive licences to our Cas9 and Cas12a patent estates so that other companies can leverage this technology to benefit patients."

It added that it "entered into a Cas9 licence agreement with Vertex Pharmaceuticals to ensure this game-changing technology can reach more patients in need of transformative medicines".

Litigation with Charpentier group

CRISPR/Cas9 technology has been the subject of intense and ongoing litigation over ownership.

In February last year, the US Patent and Trademark Office ruled in favour of the Broad Institute of MIT and Harvard against Nobel Prize-winning researcher Emmanuel Charpentier, together with the Regents of the University of California and University of Vienna. The Charpentier group said it will appeal.

This week saw licensor of the Charpentier group’s patents, ERS Genomics, survive an invalidation attempt in Japan.

This article was updated on December 18 to incorporate a statement from Editas Medicine.

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