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Summary
- Intellia stock gained over 45 percent to hit a 52-week high in the morning trade.
- A preliminary clinical study data shows positive results in transthyretin amyloidosis treatment by editing target cells in the body.
- A single 0.3 mg/kg dose of NTLA-2001 can reduce the serum TTR, which causes the disease, by 87 percent.
Intellia Therapeutics, Inc. (NASDAQ:NTLA) stock jumped over 45 percent on Monday morning and rallied to its 52-week high following the positive preliminary data from a clinical trial of genome editing treatment for transthyretin amyloidosis, or ATTR.
Intellia Therapeutics and Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) on June 26 announced the interim data of their first experimental therapy for transthyretin amyloidosis, NTLA-2001 that is based on CRISPR/Cas9 technology.
Intellia Therapeutics share performance
Intellia Therapeutics has a market value of US$8.85 billion. The stock, which rallied up to US$144.40 on Monday, was trading as low as US$16.54 during the 52 weeks period.
The share price gained 143 percent year to date. As of 10:22 am ET, Intellia’s shares were trading at US$129.69, 46 percent.
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What does Intellia do and what is NTLA-2001 treatment?
Massachusetts-based Intellia Therapeutics is a genome editing company. The biotechnology company develops curative therapeutics using CRISPR technology to edit genes.
NTLA-2001 is an experimental therapy based on CRISPR/Cas9 technology. It is co-developed by Intellia and Regeneron Pharmaceuticals. Intellia is the lead development and commercialization party in the program.
The therapy is administered through a vein to edit genes in human tissue. It inactivates the transthyretin (TTR) gene in liver cells and prevents the production of misfolded TTR protein that causes the debilitating and fatal complications of ATTR amyloidosis.
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What does the interim Phase 1 clinical study data say?
The ongoing Phase 1 study evaluates NTLA-2001 in people with hereditary transthyretin amyloidosis with polyneuropathy.
The data on the first six ATTRv-PN patients shows that a single 0.3 mg/kg dose of NTLA-2001 can reduce the serum TTR, which causes the disease, by 87 percent.
Intellia CEO John Leonard said the results support their belief that a single dose of NTLA-2001 can stop and reverse the complications of the genetic nerve disorder.
Please note: The above constitutes a preliminary view, and any interest in stocks/cryptocurrencies should be evaluated further from an investment point of view.
