Beam Gets FDA Green Light for First Gene Editing Trial


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Beam technology cuts one of the two twisted strands of DNA, then changes a single letter of genetic code in a way that is less disruptive to the cell.

The time of dreams

Therapeutic bundle

will begin the first clinical trial of its gene editing technology after receiving the green light from US regulators, the biotech said on Monday.

Shares of Beam (ticker: BEAM) closed up 10.6%, at $ 99.06, following the announcement of Food and Drug Administration approval.

Beam describes its “core editing” technology for correcting genetic diseases as the most accurate of today’s genetic medicine toolkit.

In the human trial, the company will use the unique treatment to relieve sickle cell disease. In this inherited blood disorder, the deformed red blood cells can clog a person’s veins and arteries, leading to organ damage and even death.

The FDA decision was included in Beam’s September quarter financial statements. The research-stage company reported less than $ 1 million in revenue and a loss of $ 28 million, or 42 cents per 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 anemia and beta thalassemia, another inherited disease of red blood cells. They understand

organic blue bird


Editas Medicine



(CLLS), as well as the pairings of

Therapeutic Sangamo

(SGMO) and



Therapeutic Crispr

(CRSP) and

Vertex Pharmaceutical

(VRTX), and

Intellia Therapeutics

(NTLA) and



Beam’s approach differs because other companies use gene-editing approaches, such as Crispr-Cas9 or zinc finger nucleases, which cut both strands of a cell’s DNA to trigger mechanisms for repairs that modify a troublesome gene.

Beam technology nicks only one of the twisted strands of DNA, then alters a single letter of genetic code in a way that is less disruptive to the cell.

Many eyes will watch how the basic editing technique in its first human try. A shadow fell on Crispr and other double-stranded cutting approaches last month after the FDA stopped a

Allogenic therapy

(ALLO) clinical trial when DNA sliced ​​in a patient’s cells reconnected unexpectedly.

One company that has compared core editing technology to Crispr is

Therapeutic Verve

(VERV), which is developing unique gene-editing treatments to lower blood cholesterol levels in the long term.

Verve CEO Sekar Kathiresan said Barron that his company spent two years comparing Basic Edition single-stranded breaks with Crispr’s double-stranded breaks – in cells, mice and monkeys.

Verve ended up licensing Beam’s core editing technology for its projects, Kathiresan said, after concluding that core editing was more precise and powerful than Crispr.

Write to Bill Alpert at [email protected]


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