3 Biotech Stocks Up on Intellia's CRISPR Therapy Study Data
The gene-editing field crossed a major milestone as Intellia Therapeutics NTLA announced data from the first-ever human study evaluating an in vivo CRISPR-based gene editing therapy candidate. Last weekend, Intellia and its partner, Regeneron REGN announced interim data from a phase I study evaluating their in vivo CRISPR genome editing candidate, NTLA-2001, that supported potential of this singe-dose novel treatment option for patients with transthyretin (ATTR) amyloidosis, a genetic disease.
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. Usually, the standard of care for ATTRv-PN generates TTR reductions of approximately 80%.
Please note that 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.
With the availability of the interim data on NTLA-2001, the scientific community achieved a new feat of precisely editing target cells within the body to treat a genetic disease with a single intravenous infusion of CRISPR. Genomic editing to repair defective genetic material that causes diseases using CRISPR/Cas9 gene editing 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 and may provide cure for these difficult-to-treat diseases.
In fact, companies that have been developing CRISPR gene editing therapies saw their shares soar as Intellia’s NTLA-2001 demonstrated its potential. Apart from Intellia, which surged more than 50% on Jun 28 following the news, CRISPR Therapeutics CRSP was up 6.4%, Editas Medicine’s EDIT shares rose 5% and Beam Therapeutics BEAM was up 16% . This correlated move among biotech happens often as investors view promising news for one company as potentially good news for other companies with similar products, especially novel ones in development.
CRISPR Therapeutics
The company is developing its lead pipeline candidate, CTX001, in collaboration with Vertex Pharmaceuticals VRTX in mid-stage studies as a potential treatment for transfusion-dependent beta thalassemia (TDT) and sickle cell disease (SCD). The gene-editing therapy candidate previously demonstrated a consistent and sustained response to treatment in the given patient population in an ongoing phase I/II study.
Meanwhile, CRISPR Therapeutics is actively seeking collaborations and leveraging its CRISPR/Cas9 gene-editing platform to make therapies for hemoglobinopathies, cancer, diabetes and other diseases.
CRISPR Therapeutics shares have risen 93.9% in the past year against the industry’s decrease of 1.4%.
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CRISPR Therapeutics has a Zacks Rank #3 (Hold).
You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Editas Medicine
The company’s lead pipeline candidate is EDIT-101, which employs CRISPR gene editing to treat LCA10 — a rare genetic illness that causes blindness. Editas is currently enrolling in the first pediatric 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.
It is also designing novel medicines for non-malignant hematologic diseases, such as SCD and beta-thalassemia.
In the past year, shares of Editas have gained 41.6% compared with the industry’s 1.4% increase.
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The company currently carries a Zacks Rank of 4 (Sell).
Beam Therapeutics
The company’s stock has surged 235.2% so far this year against a decrease of 1.4% for the industry.
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The company has two pre-clinical gene editing candidates, BEAM-101 and BEAM-102, in its pipeline that are being developed as potential treatment for SCD. The company plans to file an investigational new drug application to the FDA seeking approval to start a clinical study on BEAM-101 in the second half of 2021.
Beam Therapeutics carries a Zacks Rank #4.
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Regeneron Pharmaceuticals, Inc. (REGN) : Free Stock Analysis Report
Vertex Pharmaceuticals Incorporated (VRTX) : Free Stock Analysis Report
Beam Therapeutics Inc. (BEAM) : Free Stock Analysis Report
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Intellia Therapeutics, Inc. (NTLA) : Free Stock Analysis Report
CRISPR Therapeutics AG (CRSP) : Free Stock Analysis Report
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