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RegMed Investors (RMi) Research Note: Intellia Therapeutics (NTLA +$2.30 or +5.32% TO $45.54) pops on news of its CRISPR-based gene-editing drug almost completely eliminated attacks in a rare swelling disease

June 12, 2023 11:16am

NTLA tested its drug in 10 patients with hereditary angioedema, or HAE. Patients with this disorder experience severe, recurring and unpredictable inflammatory attacks in various organs and tissues across the body

After a single dose of NTLA's gene-editing drug, NTLA-2002, patients experienced an average 95% reduction in monthly swelling attacks. Intellia has followed the patients for 5.6 months to 14.1 months. All but one patient have been attack-free for 16 weeks following treatment.

NTLA's journey could be telling for the future of CRISPR drugs. Intellia is the first with an in vivo treatment that's also systemic. That means the actual gene-editing takes place inside the body. Further, the drug is infused and then must find its way to the right organ. In this case, that's the liver.

NTLA-2002 focuses on lowering the level of an inflammatory molecule called plasma kallikrein. Due to a genetic defect, patients with hereditary angioedema make too much plasma kallikrein.

Just one patient who received the low dose didn't reach the 60% reduction in plasma kallikrein. That patient experienced a swelling attack after 12.3 months free of episodes. The event followed a sports injury to the hand, and was mild enough not to require any treatment.

The Bottom Line: NTLA-2002 demonstrating a very favorable safety profile," Intellia Chief Executive John Leonard said in a written statement. "These remarkable attack rate reductions have been consistent, even in patients with the most severe symptoms." <Source: IBD>

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