Research & Development Intellia Therapeutics scores positive gene therapy results

Intellia Therapeutics scores positive gene therapy results


Intellia Therapeutics had ended on a positive note by revealing positive results from two gene editing experiments. Both are in their early stages, but the business is already preparing to enter what it thinks would be a pivotal study with the transthyretin (ATTR) amyloidosis drug, NTLA-2001.

Earlier this year, NTLA-2001, which Regeneron licensed, showed it could considerably lower a recognized disease biomarker for a protracted period in ATTR amyloidosis patients with polyneuropathy. Data from the study’s other arm, which included ATTR patients with cardiomyopathy, have now revealed a comparable decrease.

28 days following the single intravenous treatment, initial findings indicated reductions in the biomarker transthyretin, or TTR, of 93% and 92% at doses of 0.7 mg/kg and 1.0 mg/kg, respectively.

Lipid nanoparticles are used by NTLA-2001 to deliver a genome editing system in to the liver. The two components to this genome system are:

• The first component is a guide RNA that is unique to the disease-causing gene 

• The second component is a messenger RNA that encodes the Cas9 protein and does precise editing. This technology was made popular by COVID-19 vaccinations.

The latest report by Intellia provides more support for the idea that NTLA-2001 might be used as a single-use therapy to permanently inactivate the TTR gene and lower the disease-causing protein in ATTR patients with cardiomyopathy. The businesses have decided to test the 0.7 mg/kg dose in both research arms, with enrolment expected to be finished by year-end. The company’s main aim is to eradicate all existing and potential attacks.

Intellia CEO John Leonard, M.D. stated “These results strongly indicate that NTLA-2001 might be used as a single-dose treatment regardless of illness manifestation, along with the previously announced data from the polyneuropathy arm of this groundbreaking study, as we get closer to a potential pivotal trial, it is anticipated that the Phase 1 research will involve patients in the U.S.”

People with TTR gene abnormalities at birth are more likely to develop ATTR amyloidosis, a deadly illness in which the liver produces aberrant, frequently misfolded TTR proteins. TTR proteins assist the transport of thyroid hormone and vitamin A in the blood when they are in a healthy state. However, the harmed proteins lead to a wide range of issues in the heart, nervous system, and other organs.

According to Leonard, TTR serum decrease is a recognized illness biomarker that denotes a benefit. After receiving NTLA-2001 treatment, the actual advantages, such as a reduction in disease symptoms, won’t manifest immediately.

In 2020, Regeneron acquired the licensing rights to commercialize NTLA-2001 for $100 million. Another CRISPR candidate from Intellia is NTLA-2002, which is studied for hereditary angioedema in phase 1.

The clinical trial findings indicated that NTLA-2002 decreased levels of a protein linked to the condition by an average of 65% and 92% among six patients given a low (25mg) or high dose (75 mg) respectively. The number of inflammatory attacks suffered by the three patients on the lower dose decreased by 91% during the course of the four-month follow-up period. 

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