Since March, investors have quietly accumulated shares of Regeneron (NASDAQ:REGN) for many reasons. First, its antiviral cocktail to treat severe, hospitalized COVID-19 patients will bring in billions in revenue. Second, the Intellia/Regeneron CRISPR therapy is a breakthrough.
On June 26, Intellia and Regeneron announced their clinical results for treating Transthyretin (ATTR) Amyloidosis. The first-ever clinical data support the safety and efficacy of in vivo CRISPR genome editing in humans. The companies reported an incredibly good data readout.
In the ongoing Phase 1 trial, a single 0.3 mg/kg dose of NTLA-2001 resulted in an 87% mean reduction in serum TTR. After 28 days, the maximum serum TTR reduction was 96%.
Importantly, the safety data showed no adverse events in the first six patients by 28 days.
The precision editing of a gene in the target tissue is the future of advanced therapies. Intellia’s CEO, John Leonard, said “we can precisely edit target cells within the body to treat genetic disease with a single intravenous infusion of CRISPR.” Targeting the delivery of CRISPR/Cas9 to the liver will open up the addressable market in genetic disease treatments.
Intellia has a modular platform. After these results, the company may explore and expand its pipeline.
REGN stock is too cheap at these levels. The forward price-to-earnings is around 11.5 times. Investors who missed out on buying the bottom should consider accumulating the stock on its way up.