Intellia's (NTLA) IND for Gene-Editing Therapy Gets FDA Clearance
Intellia Therapeutics, Inc. NTLA announced that the FDA has cleared its investigational new drug (IND) application to move its in-vivo CRISPR-based gene editing candidate NTLA-2001 into clinical studies. The candidate is being developed for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy, a rare and fatal disease, which can lead to heart failure if not treated.
The phase III study evaluating NTLA-2001 for the given indication will begin by the end of 2023.
Per the company, the study will make NTLA-2001 the first in vivo CRISPR-based candidate to enter late-stage clinical development in the United States.
Shares of Intellia have plunged 19.9% compared with the industry’s decrease of 18.5%.
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We note that NTLA-2001 is part of Intellia’s co-development and co-promotion agreement with Regeneron Pharmaceuticals REGN. While NTLA is the lead party in the deal over NTLA-2001, REGN shares some of the development costs and commercial profits.
Intellia’s collaboration with Regeneron has given a boost to the former to develop its pipeline. In 2016, the companies initially entered into a partnership to co-develop and commercialize CRISPR/Cas-based therapies primarily focused on genome editing in the liver. The collaboration was further expanded in 2020 to co-develop therapies for hemophilia A and hemophilia B.
Earlier this month, Intellia once again expanded its existing collaboration with Regeneron to develop additional in vivo CRISPR-based gene-editing therapies focused on neurological and muscular diseases.
Genomic editing, using CRISPR technology to repair a defective genetic material that causes diseases, is probably one of the most promising and exciting healthcare innovations seen in decades.
Several other companies are also actively seeking to develop a potential treatment using CRISPR technology.
Switzerland-based CRISPR Therapeutics CRSP, in partnership with Vertex Pharmaceuticals VRTX, has developed its lead pipeline candidate, exa-cel — an investigational ex-vivo CRISPR gene-edited therapy for treating sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).
CRSP and VRTX’s biologics application seeking approval for exa-cel in SCD and TDT indications is currently under review in the United States. A final decision from the FDA for the SCD and the TDT indications is expected by Dec 8, 2023 and Mar 30, 2024, respectively.
CRISPR, along with Vertex, filed similar regulatory submissions for exa-cel in Europe.
Though the CRISPR technology remains innovative and new competition is stiff in the targeted market, companies are vying to get the first approval for their respective products.
Zacks Rank
Intellia currently has a Zacks Rank #4 (Sell).
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