The genomic-editing space has come under the spotlight with the release of encouraging data from the first-ever human study assessing an in vivo CRISPR-based gene editing therapy candidate, NTLA-2001. On Jun 26, Intellia Therapeutics, Inc. (
NTLA Quick Quote NTLA - Free Report) and Regeneron Pharmaceuticals, Inc. ( REGN Quick Quote REGN - Free Report) released positive interim data from an ongoing Phase 1 clinical study of NTLA-2001, which is being developed as a single-dose treatment for transthyretin (ATTR) amyloidosis.
Notably, the Phase 1 study is assessing NTLA-2001 in people living with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2001 will stand out as the first curative treatment for ATTR amyloidosis. It is also the first investigational CRISPR therapy candidate to be administered systemically, via intravenous infusion, for precision editing of a gene in a target tissue in a human body.
Interim data from the first six ATTRv-PN patients across two single-ascending dose cohorts of the Phase 1 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%.
It is worth noting here that the ongoing Phase 1 clinical study of NTLA-2001 is being conducted in the U.K. and New Zealand. Going on, NTLA-2001 is presently being studied only in the polyneuropathy subtype of the disease, per a Fierce Biotech article. However, per the same article, Intellia aims to cover the two other subtypes, namely, the wild type and the cardiomyopathy manifestation.
Latest Data Highlights Gene-Editing Potential
According to the sources, genome editing is a technique to alter or modify the DNA of a cell or organism. It uses an enzyme to cut the DNA at a particular sequence and then it is repaired by the cell, making a change to the sequence. As a result, the characteristics of a cell or organism are changed.
Intellia’s gene editing study data is being considered a major milestone in the field as it highlighted for the first time that gene editing can work in a human. Notably, the results “decisively exceeded” analyst expectations, per a Fierce Biotech article. Going by the same article, Chardan Research commented that the data highlight that NTLA-2001 has the possibility of becoming a “functional cure” for ATTR amyloidosis. It is also expected to create some pressure on the FDA to relax some resistance to hosting gene editing trials in the United States, per the same article.
Genomic ETFs That Are Gaining
The release of positive data was widely cheered by investors. Apart from Intellia, which surged more than 55% on Jun 28 following the news, CRISPR Therapeutics (
CRSP Quick Quote CRSP - Free Report) was up 6.4%, Editas Medicine’s ( EDIT Quick Quote EDIT - Free Report) shares rose 5% and Beam Therapeutics ( BEAM Quick Quote BEAM - Free Report) was up 16%.
According to analysts, growing demand for personalized medicine, solid investments and higher R&D activities will soon make genomics the next big thing in the investing space. In fact, going by a MarketsandMarkets report, the $18.9-billion global genomics market is expected to reach $35.7 billion by 2024, at a CAGR of 13.5%.
Here we highlight a few ETFs that investors can keep a tab on:
ARK Genomic Revolution ETF ( ARKG Quick Quote ARKG - Free Report) — up 3.3% on Jun 28
This is an actively-managed ETF focusing on companies likely to benefit from the extension and enhancement of the quality of human and other life by incorporating technological and scientific developments plus improvements and advancements in genomics into their business. The fund typically holds 30-50 stocks in its basket. The fund charges 0.75% in expense ratio. It has accumulated $9.26 billion in its asset base (read:
Top-Performing Biotech ETFs of Last Week). Global X Genomics & Biotechnology ETF ( GNOM Quick Quote GNOM - Free Report) — up 3%
This is a new entrant in the space, having accumulated $248.5 million since its inception on Apr 5, 2019. It seeks to invest in companies that stand to benefit from advancements in the field of genomic science, such as companies involved in gene editing, genomic sequencing, genetic medicine/therapy, computational genomics and biotechnology. The product follows the Solactive Genomics Index, charging 50 bps in annual fees. It holds 40 stocks in its basket (read:
4 ETF Areas Surged Last Week). iShares Genomics Immunology and Healthcare ETF ( IDNA Quick Quote IDNA - Free Report) — up 1.9%
This is another new entrant, which was launched in June 2019. Tracking the NYSE FactSet Global Genomics and Immuno Biopharma Index, the fund provides exposure to developed and emerging market companies that could gain from long-term growth and innovation in genomics, immunology and bioengineering. It holds a basket of 49 securities. The fund has AUM of $313 million and charges a fee of 47 basis points (read:
ETFs to Win as Moderna Seeks COVID-19 Vaccine Nod for Teens). Want key ETF info delivered straight to your inbox?
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