- Intellia Therapeutics, an early entrant in the race to develop medicines using CRISPR gene editing, has won a grant from the Bill & Melinda Gates Foundation to fund research of an "in vivo" treatment for sickle cell disease using the Nobel Prize-winning technology.
- The Cambridge, Massachusetts-based biotech has already advanced an ex vivo sickle cell treatment, now being developed by Novartis, that involves editing the DNA of cells outside the body before infusing them into a patient. The Gates-funded research would take the technology a step further by delivering intravenously the machinery for CRISPR gene editing.
- The Nov. 11 announcement comes two days after Intellia confirmed the first patient with a rare disease called transthyretin amyloidosis was treated in a study of its NTLA-2001 therapy, which also uses CRISPR gene editing in vivo. The study is the first to test a CRISPR medicine infused directly into the blood.
The Gates Foundation grant is yet another signal of the broad potential of CRISPR/Cas9 "genetic scissors," which was the basis of this year's award of the Nobel Prize in Chemistry to Jennifer Doudna and Emmanuelle Charpentier.
Doudna was a co-founder of Intellia, one of three high-profile biotechs launched several years ago to turn the revolutionary science into medicines. The two others, CRISPR Therapeutics and Editas Medicine, have both started clinical trials of treatments using CRISPR/Cas9 gene editing.
CRISPR Therapeutics and partner Vertex are studying a CRISPR therapy for both sickle cell and the blood disease beta-thalassemia, while Editas and partner Allergan are testing a retinal injection to treat a rare form of genetic blindness.
With its work in transthyretin amyloidosis, Intellia is taking the technology another step further, testing a direct infusion into the blood of a CRISPR-based medicine. The company's goal is to cure the disease by knocking out a mutated gene that causes a buildup of misfolded protein in the body, which can lead to nerve and heart damage.
Intravenous therapy has obvious benefits. There's no need for a multi-step process and the potential complications that come with extracting cells from a patient's body. That could make it more accessible in developing countries, a major goal for the Gates Foundation.
Sickle cell disease is most common among people with ancestral roots in sub-Saharan Africa. Nigeria has the highest incidence, with about 150,000 babies born each year with sickle cell anemia, Intellia said.
The company didn't release the value of the Gates Foundation grant.