Will Intellia Make Gene Editing History Saturday?

Intellia_Rafael Henrique/SOPA Images/LightRocket

Rafael Henrique/SOPA Images/LightRocket  via Getty Images

Tomorrow, Intellia Therapeutics may set the biotech world on fire – or not. Shareholders and those suffering from hereditary ATTR amyloidosis will only have to wait one more day for Intellia to present interim phase I data from its lead CRISPR/Cas9 in vivo therapy at the 2021 Peripheral Nerve Society (PNS) Annual Meeting.

In what will be a big moment for CRISPR technology, Intellia and development partner, Regeneron Pharmaceuticals will share the world’s first data from a therapy that edits flawed genes inside the body.   

The presentation will highlight results from the ongoing dose-escalation portion of the study evaluating NTLA-2001 in hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN) and shed light on the therapy’s safety and activity profile. Intellia received the go-ahead for the Phase I trial in October 2020 and dosed the first patient in November.    

Transthyretin amyloidosis, or ATTR, is a rare protein misfolding disorder caused by a specific mutation in the transthyretin (TTR) gene. The protein, which normally dissolves, folds over on itself, building up in the body to form amyloid deposits and causing a whole host of problems.

Patients experience damage to the nerves (polyneuropathy), kidneys, and eyes, and can suffer from cardiomyopathy (ATTR-CM) when the protein mistakenly deposits in the heart.  Hereditary ATTR affects approximately 50, 000 people worldwide, and the prognosis is bleak, with a typical life expectancy of 2-15 years from the onset of symptoms. 

NTLA-2001, which uses Intellia’s proprietary non-viral lipid nanoparticle (LNP) platform, is the first experimental CRISPR therapy to be delivered systematically, or through the vein. The therapy is comprised of guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 protein.

If successful, NTLA-2001 could potentially be curative for ATTR, which currently requires daily, lifetime administration of a variety of drugs. These include Pfizer’s Vyndaqel and Vyndamax, which in 2019 became the first medicines approved by the U.S. Food and Drug Administration (FDA) to treat ATTR-CM.

“By knocking out the disease-causing gene, NTLA-2001 is designed to halt progression and potentially reverse the disease with a single dose, offering the potential of meaningful improvement over the standard of care, which requires chronic, lifelong administration,” said Intellia President and Chief Executive Officer, John Leonard, M.D.

Positive results from the trial would also confirm the gamble of investors who have propelled Intellia’s stock to a 275% increase over the past year. It would also be a boon for Regeneron, whose stock is down 17% since the height of the enthusiasm over its COVID-19 antibody therapies.

“This will be a big catalyst for the whole field of gene editing,” said Luca Issi, an analyst at RBC Capital Markets, who is clearly betting on the results being positive.

In preclinical studies, NTLA-2001 demonstrated long-lasting TTR reduction. Intellia’s next step will be to assess the therapy in a broader range of patients with ATTR amyloidosis, including those with cardiomyopathy.

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