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Beam Therapeutics
will begin the first clinical trial of its gene-editing technology after getting the go-ahead from U.S. regulators, the biotech announced Monday.
Shares of Beam (ticker: BEAM) closed up 10.7%, at $99.13, on news of the Food and Drug Administration’s signoff.
Beam describes its “base-editing” technology for correcting genetic illnesses as the most precise in today’s genetic medicine tool kit.
In the human trial, the company will use the one-time treatment to alleviate sickle-cell disease. In that inherited blood condition, deformed red-blood cells can clog a person’s veins and arteries, leading to organ damage and even death.
The FDA’s decision was included in Beam’s September-quarter financials. The research-stage company reported less than $1 million in revenue, and a loss of $28 million, or 42 cents a share. The biotech’s balance sheet showed $933 million in cash and investments.
Several companies are developing or testing genetic-medicine treatments for sickle cell and beta thalassemia, another inherited red-cell disorder. They include
bluebird bio
(BLUE),
Editas Medicine
(EDIT),
Cellectis
(CLLS), as well as the pairings of
Sangamo Therapeutics
(SGMO) and
Sanofi
(SNY),
Crispr Therapeutics
(CRSP) and
Vertex Pharmaceuticals
(VRTX), and
Intellia Therapeutics
(NTLA) and
Novartis
(NVS).
Beam’s approach differs because the other companies are using gene-editing approaches—like Crispr-Cas9 or zinc-finger nucleases—that cut through both strands of a cell’s DNA to trigger repair mechanisms that alter a bothersome gene.
The Beam technology nicks just one of DNA’s twisted strands and then changes a single genetic-code letter in a way that’s less disruptive of the cell.
Many eyes will be watching how the base-editing technique in its first human trial. A shadow fell over Crispr and other double-strand cutting approaches last month after the FDA halted an
Allogene Therapeutics
(ALLO) clinical trial when the DNA sliced in a patient’s cells reconnected in an unexpected way.
One company that has compared the base-editing technology against Crispr is
Verve Therapeutics
(VERV), which is developing one-time gene-editing treatments intended to reduce blood cholesterol levels long term.
Verve chief executive Sekar Kathiresan told Barron’s that his company spent two years comparing the single-strand nicks of base-editing with Crispr’s double-strand breaks—in cells, mice and monkeys.
Verve ended up licensing Beam’s base-editing technology for its projects, said Kathiresan, after concluding that base-editing was more precise and more potent than Crispr.
Write to Bill Alpert at william.alpert@barrons.com
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