Promising developments in new gene editing trials continue to drive investor interest in the growing biotech sector. Potential advancements include the blocking of virus transmission, as well as single dose infusions combatting heart and brain disease; both of which are major medical breakthroughs.
With gene editing technology on the rise and human trials accelerating, the World Health Organization (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 as a major medical breakthrough.
The technology could transform the way we are currently treating patients with COVID-19, as well as minimizing 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 CRIPSR 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 the first of its kind and 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 CRIPSR gene therapy could be a potential game changer to effectively…
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