Intellia Therapeutics ( NTLA Quick Quote NTLA - Free Report) provided an update on its pipeline plans for 2021 wherein it said it will file investigational new drug applications (IND) to begin clinical studies on its two CRISPR-based candidates, NTLA-5001 and NTLA-2002 this year. While an IND or IND-equivalent for NTLA-5001 for the treatment of acute myeloid leukemia (AML) will be filed by mid-year, the same for NTLA-2002 for the treatment of hereditary angioedema (HAE), a swelling disorder, is expected to be filed in the second half. The INDs, if approved, will allow Intellia to begin clinical studies on the candidates.
CRISPR is an acronym for Clustered Regularly Interspaced Short Palindromic Repeats. The technology can selectively delete, modify or correct a disease-causing abnormality in a specific deoxyribonucleic acid segment.
Meanwhile, Intellia Therapeutics’ lead pipeline candidate is NTLA-2001, a potentially curative single-course therapy to treat transthyretin amyloidosis (“ATTR”), a rare inherited disease. In November, Intellia dosed the first patient in a phase I study on NTLA-2001 in hereditary ATTR with polyneuropathy (hATTR-PN), which marked the company’s transition to a clinical stage biotech. Intellia has plans to study NTLA-2001 in a broader ATTR population of both polyneuropathy and cardiomyopathy patients.
In the latest press release, Intellia also announced plans to nominate at least one additional development candidate in 2021.
Shares of Intellia were up 16.1% on Thursday as the update on its gene-editing drugs excited investors. Moreover, the news pushed up stocks of other companies,
Editas Medicine ( EDIT Quick Quote EDIT - Free Report) Fate Therapeutics ( FATE Quick Quote FATE - Free Report) and CRISPR Therapeutics ( CRSP Quick Quote CRSP - Free Report) , which make gene-editing drugs using the CRISPR technology. Shares of CRISPR Therapeutics, Fate Therapeutics and Editas jumped a respective 18.7%, 17.5% and 11.3%
Genomic editing to repair a defective genetic material that causes diseases using CRISPR technology is probably one of the most promising and exciting healthcare innovations seen in decades. The technology has the potential to change how diseases, especially those caused by genetic mutations, are treated.
Editas’ lead pipeline candidate is EDIT-101, which employs CRISPR gene editing to treat LCA10 — a rare genetic illness that causes blindness. Editas completed dosing in the first cohort of the phase I/II BRILLIANCE study, which is evaluating EDIT-101 for LCA10. Editas is also pursuing the development of CRISPR candidates for eye diseases other than LCA10 including Usher Syndrome type 2A and recurrent ocular Herpes Simplex Virus type 1.
CRISPR Therapeutics' lead pipeline candidate CTX001, a CRISPR gene-edited therapy, is being developed in phase I/II studies for treating sickle cell disease and transfusion-dependent beta thalassemia in collaboration with
Vertex ( VRTX Quick Quote VRTX - Free Report) .
In May, Fate Therapeutics got FDA clearance for its IND application to begin clinical studies on FT538, its first CRISPR-edited, iPSC-derived cell therapy. Enrollment is ongoing in phase I studies on FT538 as a monotherapy in AML and in combination with daratumumab, a CD38-directed monoclonal antibody therapy in multiple myeloma.
All these companies have the potential for long-term gains as they are developing drugs using innovative technologies. If their drugs are successfully developed and commercialized, the companies may see astronomical revenues. It is therefore a good idea to add a few such stocks to your portfolio as they may give great returns in the next 2-3 years. Investors, at the same time, should keep in mind that investing in these biotech stocks is risky as either these companies make it or don’t depending on whether the FDA approves their innovative medicines.
While CRISPR Therapeutics, Fate Therapeutics and Editas have a Zacks Rank #3 (Hold), Intellia has a Zacks Rank #4 (Sell).
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