RTTNews | Jun 26, 2021 11:41AM EDT

11:41 Saturday, June 26, 2021 (RTTNews.com) - Intellia Therapeutics Inc. (NTLA) and Regeneron Pharmaceuticals Inc. (REGN) announced positive interim data from an ongoing phase 1 clinical study of their lead in vivo genome editing candidate, NTLA-2001. The clinical data showed deep reduction in disease-causing protein after single infusion of NTLA-2001, an investigational CRISPR Therapy for Transthyretin (ATTR) Amyloidosis.

The Phase 1 study, run by Intellia as the program's development and commercialization lead, is evaluating NTLA-2001 in people living with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN).

The Interim readout in ongoing Phase 1 trial found that single 0.3 mg/kg dose of NTLA-2001 led to 87% mean reduction in serum TTR, with a maximum 96% serum TTR reduction by day 28, with dose-dependent response.

Intellia said the clinical data suggested that the companies could precisely edit target cells within the body to treat genetic disease with a single intravenous infusion of CRISPR. The interim results supported their belief that NTLA-2001 has the potential to halt and reverse the devastating complications of ATTR amyloidosis with a single dose.

By contrast, the standard of care for ATTRv-PN, which requires chronic treatment, typically yields TTR reductions of approximately 80%.

Intellia expects to begin a single-dose expansion cohort in Part 2 of the Phase 1 trial later this year. After completion of the Phase 1 trial, the company plans to move to pivotal studies for both polyneuropathy and cardiomyopathy manifestations of ATTR amyloidosis.

Intellia intends to present additional data from the study at a medical or scientific meeting later this year.

Transthyretin amyloidosis, or ATTR amyloidosis, is a rare, progressive and fatal disease. Hereditary ATTR (ATTRv) amyloidosis occurs when a person is born with mutations in the TTR gene, which causes the liver to produce structurally abnormal transthyretin (TTR) protein with a propensity to misfold. These damaged proteins build up as amyloid in the body, causing serious complications in multiple tissues, including the heart, nerves and digestive system.

ATTRv amyloidosis predominantly manifests as polyneuropathy (ATTRv-PN), which can lead to nerve damage, or cardiomyopathy (ATTRv-CM), which can lead to heart failure. Some individuals without the genetic mutation produce non-mutated, or wild-type TTR proteins that become unstable over time, misfolding and aggregating in disease-causing amyloid deposits. This condition, called wild-type ATTR (ATTRwt) amyloidosis, primarily affects the heart.

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