Gene Editing: Is This "Turning Point in Medicine" a Potential Source of Blockbuster Revenue?
The first treatment based on CRISPR technology is poised to win FDA approval next month, offering hope to patients and opportunities to investors.
"A turning point in medicine"
Scientists have made significant progress in treating thousands of illnesses over the years, but finding cures for rare and deadly diseases remains a significant challenge. While treatments for inherited conditions like sickle cell disease provide some relief, patients still suffer from debilitating symptoms. The gene-editing technology CRISPR could change that.
CRISPR–Cas9 is best known as a laboratory tool for editing DNA, which acts like a molecular pair of scissors that can be used to cut and modify a DNA sequence.
The recent approval of Casgevy, the first CRISPR-based treatment for sickle cell disease and beta-thalassemia patients in the UK, signifies a significant milestone in medicine. The treatment, from $Vertex Pharmaceuticals(VRTX.US$ and $CRISPR Therapeutics(CRSP.US$, could also be approved by the U.S. Food and Drug Administration soon.
In an interview, Jennifer Doudna who has done pioneering work in CRISPR gene editing and won the Nobel Prize in Chemistry three years ago says the approval is "a turning point in medicine because it shows how genome editing can be used as a one-and-done cure for disease."
Wall Street is piling into biotech stocks
Wall Street is excited about the field's long-term potential, even as billions of dollars have been erased from the sector's market value the past two years as rising interest rates hammered small biotech companies.
With some of the top companies such as Crispr Therapeutics and $Intellia Therapeutics(NTLA.US$ trading at more favorable valuations relative to their 2021 highs, that presents investors with opportunities. Cathie Wood, known for her investments in innovative companies, has been increasing her position in CRISPR Therapeutics.
Large pharmaceutical companies have also entered the fray, with $Eli Lilly and Co(LLY.US$ investing up to $600 million in acquiring certain rights from $Beam Therapeutics(BEAM.US$ last month.
Potential for blockbuster revenue?
The road to blockbusters will be challenging, though, with safety and reimbursement concerns top of mind.
From a medical and scientific standpoint, it's incredibly exciting," says Janus Henderson portfolio manager Daniel Lyons. "But you do have to be somewhat cautious when you think about how many patients are going to be willing to go through what essentially is a stem cell transplant procedure to get this kind of curative therapy."
The gene editing market which was valued at USD 7.2 billion in 2022 is expected to witness a CAGR of 18.1%, from 2023 to 2032. It is expected USD 36.4 billion by 2032,according to a report from MarketResearch.
Here are the key takeaways from the report:
● CRISPR/Cas9 technology is currently leading the market accounting for a 43.7% share of revenue.
● The field of genetic engineering which includes stem cell therapies and research has the highest revenue share commanded at 69.2%. This growth can be attributed to the growing demand for biopharmaceuticals.
● North America holds the position in the gene editing market with a share of 40%.
Longer-term, gene editing will have to find ways to get to harder-to-reach tissues and organs if it is to expand its potential for curative therapies. Doudna mentions cancer and Alzheimer's as exciting areas for development in the longer term.
Source: WSJ, Bloomberg, The Motley Fool, Nature
Disclaimer: Moomoo Technologies Inc. is providing this content for information and educational use only.
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