Despite a prolonged sell-off in biotech stocks, the development of genomic medicine has continued apace. For just the third time, a potential gene therapy has been approved for in vivo human trials. This treatment, however, marks a first for the process of “base editing” which could be a safer and more precise version of the popular CRISPR gene editing technology. Separate research has shown CRISPR also demonstrates an ability to neutralize genes that predispose humans to mental health disorders and substance abuse.
As large biopharma firms are flush with cash and many valuations across the biotech space remain near multi-year lows, a new wave of M&A may be on the horizon. If that turns out to be the case, genomics firms could be right on the front line of attractive targets.
Related ETF: ARK Genomic Revolution ETF (ARKG)
Most shares in the biotechnology sector have suffered a near continuous downtrend since March 2021. That peak came much earlier than a more recent peak in major indices like the S&P 500 and Nasdaq. Perhaps no subsection of biotech has been hit harder than companies focused on gene editing – particularly those working with clustered regularly interspaced short palindromic repeats (CRISPR) treatments and associated genomic therapies.
Just this year to date, the ARK Genomic Revolution ETF (ARKG) has declined nearly -50%, a steeper decline than the -39% fall in the broader SPDR S&P Biotech ETF (XBI). However, several new developments in genomics research may be set to revitalize interest in battered biotech stocks.
As BioPharmaDive reports, Verve Therapeutics, a gene editing company vying to develop a one-time treatment for cardiovascular disease via base editing (VERVE-101), has received regulatory clearance to begin its first study in humans in New Zealand. This will be significant as just the third inside-the-body gene editing treatment to enter human trials, and a first for base editing.
It was just over a year ago that MRP covered new data from Verve showing its potential treatment for heart disease led to durable reductions in both PCSK9 protein and LDL cholesterol levels in monkeys, which was a significant milestone in its preparations for human testing. With just one infusion, the therapy had 63% frequency at editing the PCSK9 gene in primates. The one-time treatment will hopefully generate significant price savings versus comparable therapies and inhibitors for PCSK9; the latter of which can cost patients as much as $5,850 per year in the US.
Base editing can be described as a new dimension of CRISPR/Cas-mediated precise editing to generate single-nucleotide changes in DNA or RNA without utilizing double-strand breaks and homology-directed repair. Each gene’s code uses the four nucleotide bases of DNA: adenine (A), cytosine (C), guanine (G) and thymine (T). As Labiotech.eu writes, most pathogenic mutations that cause human disease are single nucleotide polymorphisms that only require a single nucleotide change to correct the mutation. The base editor manipulates genes by binding to DNA and replacing one nucleotide with another.
In our early reporting on CRISPR base editing in 2020, we noted base editors were already available to address about 60% of all known genetic diseases – potentially more than 15,000 inherited disorders — caused by a mutation in only one nucleotide. Human trials will…
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