U.S. FDA approved the first clinical trial of a gene therapy for HIV patients Lee Woo-sang Global Watch

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A human immunodeficiency virus (HIV) drug candidate being developed by Excision Biotherapeutics, a US gene therapy development company, has received clinical approval from the US Food and Drug Administration (FDA). This is the first time the FDA has approved an IND for a gene therapy product for the treatment of HIV.

Excision Biotherapeutics announced on the 15th (local time) that it had received approval from the FDA for phase 1 and 2 clinical trials for ‘EBT-101’, a gene therapy using CRISPR gene scissors.

“Existing antiviral treatment can manage HIV infection, but it requires lifelong treatment and has side effects,” said Daniel Donbusch, CEO of Exigene Biotherapeutics. I hope to be able to,” he said.

The reason HIV could not be treated with existing treatments is because HIV fuses its genetic information into the host cell. In particular, because it uses immune cells as hosts, the natural immune system is useless to fight the virus. Even with the use of drugs, there was no way to select or remove only the gene of the virus that had penetrated into the host cell.

EBT-101 is designed to be administered to a patient as a non-pathogenic viral vector (AAV9) with CRISPR gene scissors and a guide RNA that can target the genetic material of HIV. The company explained that the combination of EBT-101 and antiretroviral therapy has succeeded in eliminating HIV in animal experiments on mice and primates.

EBT-101 is a one-time gene therapy. The goal is to show a therapeutic effect on HIV with a single inoculation without repeated administration.

The problem is safety. Recently, in a gene therapy clinical trial of Astellas, a Japanese gene therapy developer, a case of patient death due to hepatotoxicity was reported. When a fourth patient died, the FDA stopped the trial. Astellas also used the AAV9 vector, like Excigene Biotherapeutics.

Excigene Therapeutics said it will continue clinical trials with the lower doses that have been shown to work well in animal studies.

Reporter Lee Woo-sang [email protected]

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