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Gene Editing May Terminate Virus Transmission, Combat Heart and Brain Disease With a Single Shot
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 McAlinden Research Partners McAlinden Research Partners explores the acceleration of gene-editing technology and notes the market is poised to expand as the World Health Organization (WHO) creates new standards for companies to follow.

With gene-editing technology on the rise and human trials accelerating, the WHO recently announced new guidelines for genomic editing firms that could pave the way for treatments to move from a mostly experimental healthcare tool with only a few marketable products to mainstream adoption.

Related ETF & Stocks: ARK Genomic Revolution ETF (ARKG), Intellia Therapeutics Inc (NTLA), Crispr Therapeutics AG (CRSP)

Excitement Builds in Gene-Editing Industry as Technology Advances

Advancements in gene-editing technology have begun to accelerate, creating a great deal of excitement and promise in this rising corner of the biotech sector.

One of those developments, highlighted by Interesting Engineering, is the ability to stop COVID-19 from replicating in humans using CRISPR gene-editing technology. Scientists have successfully used CRISPR to block the virus from spreading between infected human cells, a development widely considered a major medical breakthrough.

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The technology could transform the way we are currently treating patients with COVID-19, as well as help minimize the spread of future pandemics. Researchers involved in the trial believe that patients who test positive could use CRISPR gene editing with an antiviral approach, mitigating the risk of severe complications from the virus. The treatment is not unique to just COVID-19, as CRISPR can potentially be used to remedy any future virus, but the process needs to develop further.

Similarly, a clinical trial from University College London and Intellia Therapeutics has delivered promising results for a simpler way to deliver CRISPR treatment. The treatment is aimed at people with a heart condition called hereditary transthyretin amyloidosis, which causes a build-up of malformed protein in the heart and nerves that eventually turns fatal.

Per Singularity Hub, patients who used the treatment received a single infusion of a CRISPR-based therapy into their bloodstream that turned off the mutated gene and minimized production of the errant protein. Infusing a gene-editing treatment into the bloodstream is a novel treatment and represents a significant step for the development of gene therapy.

The Phase 1 trial only tested six individuals, but the results are strong enough compared to other treatment options to generate enthusiasm in the gene-editing industry. Bloomberg writes that within weeks, levels of proteins that cause disease dropped by nearly 96%, with no adverse effects noted after four weeks. Additionally, most gene therapies can be intense and expensive, which is why researchers believe that promising results from the single-infusion therapy are a major milestone for patients.

Since the trial has a relatively small sample size, the technology requires additional testing ahead. However, the early-stage results of single-dose CRISPR gene therapy could be a potential game changer to effectively eliminate genetic diseases as the industry continues to grow.

WHO Raises Regulations, Market Poised to Grow as Earnings Season Approaches

Potential applications of gene-editing technology are virtually endless, boosting interest in what was once considered a relatively niche market.

Recent developments are even more impressive when you consider just how new gene-editing technology is. Advanced Science News writes that CRISPR tools were developed no more than a decade ago, and the growth of the market over that period has been tremendous.

According to a report from Markets and Markets, the global gene-editing market is poised to rise from $5.1 billion in 2020 to 11.2 billion by 2025, a CAGR of 17%. Similarly, Market Screener writes that the potential of gene editing is still largely unrealized, noting that the technology could spur a flurry of activity leading to the next biotech investment boom.

One of the key factors that will contribute to growth is an increase in government funding, but in order for that to occur, gene-editing technology needs improved regulation and stricter guidelines to allow the public to effectively access the treatments.

The Wall Street Journal recently reported that the World Health Organization (WHO) has announced new standards for gene-editing technologies, aimed at avoiding potentially dangerous usage of the tools. The announcement noted the exceptional growth of gene editing since 2018, a sign that WHO officials realize the potential of the technology, and that the continued growth of gene editing requires additional regulation. Gene-editing companies would be held by the same standards for trial development and testing, which could accelerate the timetable of when these technologies become widely available to the public.

Having successfully tested the aforementioned CRISPR infusion strategy, Intellia Therapeutics told CNBC they expect their gene-editing treatment to reach patients "very, very soon."

Further, a handful of gene-editing companies are getting ready to report earnings in August, some of which could receive a boost from the slew of positive results seen in the second quarter. Forbes notes that the CRISPR therapy results from Intellia should give the overall gene-editing sector a lift. Intellia's results caused the stock to jump nearly 80%, and it's now up roughly 170% year to date. However, other gene therapy stocks have largely underperformed the market recently, but recent advancements are overall bullish news for the sector.

Gene-editing tech still has a long road ahead before it becomes widely available to the public, but initial results of recent human trials have been nothing short of remarkable. The success of Intellia's single-dose CRISPR therapy has paved the way for additional gene-editing companies to follow suit, proving the new infusion breakthrough has the potential to more effectively treat a wide variety of genetic diseases. The market is poised to grow further as the WHO creates new standards for companies to follow, making it easier for patients to access CRISPR therapies in the future while biotech companies continue to develop gene-editing technology.

Investors can gain exposure to gene editing via the ARK Genomic Revolution ETF (ARKG).

Originally published July 22, 2021.


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 McAlinden Research Partners McAlinden Research Partners (MRP) provides independent investment strategy research to investors worldwide. The firm's mission is to identify alpha-generating investment themes early in their unfolding and bring them to its clients' attention. MRP's research process reflects founder Joe McAlinden's 50 years of experience on Wall Street. The methodologies he developed as chief investment officer of Morgan Stanley Investment Management, where he oversaw more than $400 billion in assets, provide the foundation for the strategy research MRP now brings to hedge funds, pension funds, sovereign wealth funds and other asset managers around the globe.

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