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Intellia (NTLA) Up on Positive Gene-Editing Drug Interim Data
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Shares of Intellia Therapeutics, Inc. (NTLA - Free Report) were up 50.21% on Monday after the company, along with its partner Regeneron Pharmaceuticals (REGN - Free Report) , announced positive interim data from the ongoing phase I study evaluating their in vivo CRISPR genome-editing candidate, NTLA-2001, for the treatment of transthyretin (“ATTR”) amyloidosis, on Jun 26.
In fact, the stock has rallied 63.2% so far this year compared with the industry’s rise of 0.5%.
Image Source: Zacks Investment Research
The phase I study, which is being run by Intellia, is evaluating NTLA-2001 in people living with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN).
Interim data from the first six ATTRv-PN patients from the phase I study showed that treatment with a single dose of NTLA-2001 (0.3 mg/kg) led to a mean reduction of 87% in serum TTR levels, with a maximum 96% serum TTR reduction by day 28, with dose-dependent response, including one patient with a 96% reduction.
Usually, the standard of care for ATTRv-PN generates TTR reductions of approximately 80%. Overall, the data showed that a single dose of NTLA-2001 has the potential to halt and reverse the devastating complications of ATTR amyloidosis.
Overall, NTLA-2001 demonstrated an encouraging safety profile with no serious adverse side effect being reported.
The above-mentioned data was published in The New England Journal of Medicine.
Per the company, this is the first-ever clinical data supporting safety and efficacy of in vivo CRISPR genome editing in humans that is precisely editing target cells within the body to treat a genetic disease with a single intravenous infusion of a CRISPR therapy. This data is being viewed as a breakthrough in gene-editing research.
Intellia has several other gene-editing pipeline candidates in pre-clinical stage. The company plans to submit an investigational new drug application for NTLA-5001 and NTLA-2002 as potential gene-editing therapies for acute myeloid leukemia and hereditary angioedema, respectively, later in 2021.
Please note that, other than Intellia, companies such as Editas Medicine (EDIT - Free Report) , CRISPR Therapeutics (CRSP - Free Report) and Beam Therapeutics are also using the CRISPR/Cas9 gene-editing technology to develop their respective candidates for addressing various ailments. Shares of these companies were also up 5%, 6.4% and 16%, respectively, on Monday.
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Image: Shutterstock
Intellia (NTLA) Up on Positive Gene-Editing Drug Interim Data
Shares of Intellia Therapeutics, Inc. (NTLA - Free Report) were up 50.21% on Monday after the company, along with its partner Regeneron Pharmaceuticals (REGN - Free Report) , announced positive interim data from the ongoing phase I study evaluating their in vivo CRISPR genome-editing candidate, NTLA-2001, for the treatment of transthyretin (“ATTR”) amyloidosis, on Jun 26.
In fact, the stock has rallied 63.2% so far this year compared with the industry’s rise of 0.5%.
The phase I study, which is being run by Intellia, is evaluating NTLA-2001 in people living with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN).
Interim data from the first six ATTRv-PN patients from the phase I study showed that treatment with a single dose of NTLA-2001 (0.3 mg/kg) led to a mean reduction of 87% in serum TTR levels, with a maximum 96% serum TTR reduction by day 28, with dose-dependent response, including one patient with a 96% reduction.
Usually, the standard of care for ATTRv-PN generates TTR reductions of approximately 80%. Overall, the data showed that a single dose of NTLA-2001 has the potential to halt and reverse the devastating complications of ATTR amyloidosis.
Overall, NTLA-2001 demonstrated an encouraging safety profile with no serious adverse side effect being reported.
The above-mentioned data was published in The New England Journal of Medicine.
Per the company, this is the first-ever clinical data supporting safety and efficacy of in vivo CRISPR genome editing in humans that is precisely editing target cells within the body to treat a genetic disease with a single intravenous infusion of a CRISPR therapy. This data is being viewed as a breakthrough in gene-editing research.
Intellia has several other gene-editing pipeline candidates in pre-clinical stage. The company plans to submit an investigational new drug application for NTLA-5001 and NTLA-2002 as potential gene-editing therapies for acute myeloid leukemia and hereditary angioedema, respectively, later in 2021.
Please note that, other than Intellia, companies such as Editas Medicine (EDIT - Free Report) , CRISPR Therapeutics (CRSP - Free Report) and Beam Therapeutics are also using the CRISPR/Cas9 gene-editing technology to develop their respective candidates for addressing various ailments. Shares of these companies were also up 5%, 6.4% and 16%, respectively, on Monday.
Zacks Rank
Intellia currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
+1,500% Growth: One of 2021’s Most Exciting Investment Opportunities
In addition to the stocks you read about above, would you like to see Zacks’ top picks to capitalize on the Internet of Things (IoT)? It is one of the fastest-growing technologies in history, with an estimated 77 billion devices to be connected by 2025. That works out to 127 new devices per second.
Zacks has released a special report to help you capitalize on the Internet of Things’s exponential growth. It reveals 4 under-the-radar stocks that could be some of the most profitable holdings in your portfolio in 2021 and beyond.
Click here to download this report FREE >>