Vertex Pharmaceuticals Inc (VRTX)

[protagonist12]

New $2.6B deal between Boston-area biotechs a first for gene editing

Cambridge startup CRISPR Therapeutics is getting $105 million up front, and as much as $2.6 billion in coming years, in a new partnership with Vertex Pharmaceuticals that CRISPR CEO Rodger Novak called “the first really big deal” in the burgeoning field of gene editing.

CRISPR, a privately-held company — which, coincidentally, just moved into a building formerly occupied by Vertex(Nasdaq: VRTX) over the summer — said the deal could help develop cures for diseases including sickle cell and cystic fibrosis. While all research in gene editing — treatments designed to fix genes that cause disease — is still very early stage, Nozak said in an interview today it’s “very likely” that a treatment for sickle cell could begin human trials sometime before the end of the four-year research agreement.

If successful, the collaboration could result in a method to correct the underlying gene mutations that cause cystic fibrosis. While the approach is yet unproven, the hope is that a one-time treatment with gene editing would render the drugs now marketed by Vertex for that disease — including Kalydeco and Orkambi — obsolete.

“The main interest of both Vertex and ours is to help patients with cystic fibrosis. Their therapies are helping patients tremendously now ... but there is room for improvement,” said Novak.

Currently, almost all the treatments in development at Vertex are small-molecule, or pill-based, drugs, so the CRISPR deal represents a new type of research for the 30-year-old company. Vertex will have the option to exclusively license up to six potential treatments (including for cystic fibrosis), and for most of them it would pay for all development and commercialization costs — up to $420 million each, plus sales royalties. The only exception would be in sickle cell, where CRISPR will pay half the R&D costs and would lead any commercialization efforts.

The deal appears to be exactly the kind touted as a benefit of Cambridge’s life sciences cluster, where two local companies decide to join forces on a project that would advance human health. Novak said that the physical proximity of the two companies (Vertex is in South Boston; CRISPR is in East Cambridge) played a part, but was also helped by an existing relationship between the top scientists at both companies, Vertex’s David Altshuler and CRISPR’s Bill Lundberg.

The focus on sickle cell makes CRISPR a potential competitor with another big local biotech, bluebird bio (Nasdaq: BLUE). Cambridge-based bluebird has a promising gene therapy treatment (which seeks to replace, rather than edit, a faulty gene) in early-stage clinical development to address the disease. Novak said in this disease, gene editing has the potential to be as effective as gene therapy, but with fewer side effects, though he added that the technology still needs to prove it works.

CRISPR is one of three local biotech firms now pursuing gene editing using a technique known as CRISPR/Cas9, which allows for multiple cuts, or “edits,” to a single gene. Cambridge-based Editas Medicine made a deal in May potentially worth more than $700 million with Seattle-based Juno Therapeutics (Nasdaq: JUNO), which last year held the sector’s biggest initial public offering of of $265 million. Cambridge-based Intellia Therapeutics, which has yet to announce a major deal with a more established company, just got a $70 million investment last month.

http://www.bizjournals.com/boston/blog/bioflash/2015/10/new-2-6b-deal-between-boston-area-biotechs-a.html