CRSP - Intellia Therapeutics Presents Promising In Vivo CRISPR Results

2024-06-05 07:11:15 ET

Summary

• Intellia Therapeutics has released positive data from its Phase I/II study of NTLA-2002, a CRISPR-based gene editing therapy.
• The study shows promising results for in vivo gene editing, offering potential treatments for genetic diseases.
• Robust target engagement and efficacy data pave the way for future Phase II results from Intellia later this year.
• Intellia has the most advanced in vivo gene editing portfolio, which, while speculative, could represent a huge advance for the field in coming years.

Intellia Therapeutics (NTLA) recently reported strong data from the Phase I portion of an ongoing Phase I/II study for NTLA-2002, an in vivo CRISPR-based gene editing therapy for hereditary angioedema ((HAE)). I have previously written about CRISPR Therapeutics (CRSP) and rated the company a strong buy due to its progress in the commercialization of Casgevy, an ex vivo CRISPR-based gene editing therapy. While CRISPR Therapeutics remains a strong buy, certain advantages of Intellia's therapy, particularly its impressive progress toward in vivo treatment, present a distinct competitive advantage. CRISPR/Cas9 represents a transformational technology for the editing of human DNA, the code of life. Companies that can successfully commercialize therapies may transform the landscape for treatment of genetic diseases, a huge advance for human health....

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