Intellia Therapeutics on Friday announced promising preliminary data from clinical studies of two treatments based on CRISPR gene editing. Just weeks after patients received infusions of the experimental therapies, they experienced a dramatic reduction in blood markers linked to their diseases.
Although it could be years before Intellia proves the effectiveness of the treatments in larger studies, the early results bode well for the Cambridge company and CRISPR technology at large — which promises to treat numerous diseases by making subtle, but permanent, alterations to DNA.
“We know increasingly well how this thing is going to behave,” said chief executive John Leonard.
Intellia’s first program, which it is developing with its partner Regeneron Pharmaceuticals, uses CRISPR to reduce a disease-causing protein that accumulates in the nerves or hearts of people with a rare condition called transthyretin amyloidosis.
Last year, the companies published results from the first clinical trial where CRISPR was infused directly into people. A small dose cut in half blood levels of the toxic protein in people with the nerve form of the disease. Earlier this year, the companies said that a larger dose reduced protein levels by about 90 percent.
On Friday, Intellia shared the first results testing the therapy in 12 patients with the heart form of the disease. The one-time treatment reduced the toxic protein by about 93 percent after four weeks.
The firm now needs to test the therapy in larger studies, which may measure how the therapy prevents deaths, hospitalizations, and worsening of disease.
Intellia also announced early results from the first six people treated with its second gene editing therapy: a treatment for the rare disease hereditary angioedema, or HAE, which is characterized by sudden and severe episodes of swelling called HAE attacks. The disfiguring attacks, which can occur several times a month, can be life-threatening if the swelling obstructs breathing.
A protein called kallikrein helps instigate those attacks, so Intellia is using CRISPR to break the gene that makes the protein. Eight weeks after three patients received the smallest dose of the therapy, their kallikrein blood levels dropped by 65 percent. Two of the three patients didn’t experience any attacks in the first 16 weeks after treatment, and the third patient’s attacks were reduced.
“These people live with a fear that a life-threatening event could take place out of the blue,” Leonard said. “What we’re showing here has the prospects to change a patient’s life.”