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Gene Editing Licensed for Inherited Disease Treatments

Gene editing illustration

(NIH.gov)

26 October 2015. A Boston biotechnology company is licensing gene-editing technologies from a nearby start-up to design treatments for cystic fibrosis and inherited blood disorders, including sickle cell disease. Vertex Pharmaceuticals, in Boston, received rights to techniques known as CRISPR-Cas9 in a deal that could bring CRISPR Therapeutics, founded in April 2014, as much as $525 million.

CRISPR, short for clustered, regularly interspaced short palindromic repeats, makes it possible to edit genomes of organisms harnessing bacterial defense mechanisms that use RNA to identify and monitor precise locations in DNA.
The actual editing is done by the CRISPR-associated protein 9, or Cas9, enzyme that programs RNA to silence genes and provide immunity against invading genetic material. Cas9 also harnesses RNA to cut DNA at precise points in genomes, making it possible to delete, insert, or correct defects in human genomes.

CRISPR Therapeutics, in Cambridge, Massachusetts, is developing treatments for genetic disorders, where disease-causing mutations are inherited from one or both parents. The company was founded by Emmanuelle Charpentier, a professor at the Helmholtz Centre for Infection Research in Braunschweig, Germany. Charpentier led teams that published pioneering research on gene editing in the journal Science in 2012, and an article in Nature a year earlier. Four other genetics researchers co-founded the company, including Craig Mello, a 2006 Nobel laureate at University of Massachusetts Medical School.

Under the four-year agreement, Vertex receives an exclusive license to develop up to six treatments for cystic fibrosis and inherited blood disorders such as sickle cell disease, as well as other inherited genetic conditions determined later. Cystic fibrosis is an inherited disease of the glands that make mucus, a substance keeping the lungs and airways moist, as well as helping prevent infection. The disease results from mutated cystic fibrosis transmembrane conductance regulator genes passed from each parent to their children. With cystic fibrosis, the mucus becomes thick and sticky, and builds up in the lungs and airways that also affects the pancreas, liver, intestines, and other organs.

Sickle cell disease is a genetic blood disorder affecting hemoglobin that delivers oxygen to cells in the body. People with sickle cell disease have hemoglobin molecules that cause blood cells to form into an atypical crescent or sickle shape. That abnormal shape causes the blood cells to break down, lose flexibility, and accumulate in tiny capillaries, leading to anemia and periodic painful episodes. The disease is prevalent worldwide, and affects 70,000 to 80,000 people in the U.S., including about 1 in 500 people of African descent.

The agreement calls for Vertex and CRISPR Therapeutics to collaborate on these treatments, with CRISPR Therapeutics taking the lead on most discovery efforts and Vertex focusing on development and commercialization. With the exception of inherited blood-related disorders, Vertex will fund discovery and developmental work on the six therapies. For blood-related disorders, the companies will split research and development costs, as well as revenues, with CRISPR Therapeutics leading commercialization of these treatments in the U.S.

Under the deal, Vertex is paying CRISPR Therapeutics an initial $75 million and taking an equity stake in the company valued at $30 million. CRISPR Therapeutics will also be eligible for development, regulatory, and marketing milestones payments of up to $420 million, as well as royalties on sales of products from the collaboration.

A potential brake on the agreement is the ethical concerns being raised about human genome editing, including from scientists working in the field. In April 2015, a research team at Sun Yat-sen University in Guangzhou, China published a report of their attempts to modify a gene in human embryos rejected for in vitro fertilization. The gene in this case causes the inherited blood disorder beta thalassemia that reduces production of hemoglobin, and the research team reported minimal success editing the problem gene. National Academy of Sciences and National Academy of Medicine in the U.S. plan to write guidelines for governing the technology.

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