Boston's Vertex Pharmaceuticals is collaborating with CRISPR Therapeutics, a Cambridge startup in developing a new technology for gene editing. This new technique sheds light in the future treatments for cystic fibrosis (CF), sickle cell anemia and other genetic diseases.

The company said on Oct. 26 that it would give $105 million up front cash and equity deal with CRISPR along with $2.6 billion in the coming years. The agreement included a 4-year partnership in which they will determine if CRISPR's flagship platform, CRISPR-Cas9, can edit and repair faulty genes that cause or predispose a person to genetic diseases.

Genome or gene editing has been revolutionized through the discovery of CRISPR-Cas9 system from Streptococcus pyogenes. CRISPR or otherwise known as clustered regularly interspaced short palindromic repeat, is a platform that uses bacterially-derived protein (Cas9) and a synthetic guide RNA to introduce a double strand break in the faulty genome.

The process involves a kind of molecular scissors to cut and repair defective genes that causes many diseases like cystic fibrosis and sickle cell anemia. However, this collaboration will initially target the genetic mutations and alterations that occurs in cases of cystic fibrosis.

Cystic fibrosis is a life-threatening obstructive lung disease caused by the presence of a gene that creates a thick build-up of mucus in the lungs, pancreas and other organs. This results to the occurrence of persistent lung infections that will lead to difficulty of breathing and eventually, respiratory failure.

"This puts Vertex at the forefront of what we believe may be a fundamental change in the future treatment of disease - using gene-editing technologies to address the underlying genetic causes of many diseases," Dr. David Altshuler, Vertex's chief scientific officer said.

Around the globe, an estimated 70,000 people are living with cystic fibrosis and in the United States, there are about 30,000 people with this genetic condition. Every year, there are 1,000 new cases of CF diagnosed.

CRISPR, a privately-owned company, said the deal could open doors to the development of potent treatments for one of the most difficult-to-treat conditions and the collaboration is dubbed as 'the first really big deal' when it comes to genetic editing.

"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," CRISPR CEO Rodger Novak said.

As part of their deal, the two companies agreed to share costs of research and profit for treatments using genetic editing. Vertex will have the opportunity to exclusively license up to six potential treatment options.

Thus, they plan to shoulder expenses for development and commercialization of other treatments. For each of the six treatments, Vertex will pay $420 million based on their sales targets.

"We're looking for potential partners who had capabilities we don't have. With this partnership, we have access to the cash we need to support our research programs. Our financing position has changed overnight so we have a lot of flexibility," Novak added.

Photo: Greg Emmerich | Flickr  

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