Intellia (NTLA) Focuses on Developing Gene-Editing Therapies
Intellia Therapeutics, Inc. NTLA is a clinical-stage genome editing company focused on developing CRISPR/Cas9-based therapeutics.
The company is evaluating its leading in vivo genome-editing candidates — NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis and NTLA-2002 for the treatment of hereditary angioedema (HAE).
Earlier this week, the FDA cleared an investigational new drug (IND) application to begin clinical studies on NTLA-2001.
The phase III study will evaluate NTLA-2001 for the treatment of ATTR amyloidosis with cardiomyopathy, a rare and fatal disease, which can lead to heart failure if not treated. The study is set to begin by 2023-end and is likely to make NTLA-2001 the first in vivo CRISPR-based candidate to enter late-stage clinical development in the United States.
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 related to NTLA-2001, REGN shares some of the development costs and commercial profits.
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.
Intellia’s collaboration with Regeneron has given a boost to the former to develop its pipeline.
Meanwhile, NTLA-2002 is Intellia’s in vivo CRISPR-based investigational therapy candidate, which is currently being evaluated in a phase I/II study for treating HAE.
The FDA has granted Orphan Drug designation and Regenerative Medicine Advanced Therapy designation to NTLA-2002 for the treatment of HAE.
Last week, the European Medicines Agency granted Priority Medicines designation to NTLA-2002 for the treatment of HAE.
NTLA-2002 also received the Innovation Passport from the United Kingdom Medicines and Healthcare products Regulatory Agency.
This apart, Intellia is planning to file an investigational new drug application seeking approval to begin clinical studies on NTLA-3001 for alpha-1 antitrypsin deficiency-associated lung disease by 2023-end.
In the absence of an approved marketed product, successful development of pipeline candidates remains the key focus area for Intellia.
Though CRISPR technology remains innovative and new, competition is stiff in the targeted market, as a few other companies are also vying to get the first approval for their respective products.
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 and Vertex have filed similar regulatory submissions for exa-cel in Europe.
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