rAAV2/8.hRKp.AIPL1 (gene therapy)

History

2025: Product Announcement

A team of doctors from Great Ormond Street Hospital in London have successfully applied gene therapy to treat congenital blindness in four children with a rare hereditary condition. The results of the world's first such intervention were published in February 2025.

According to The Lancet, experimental rAAV2/8.hRKp.AIPL1 therapy, developed by University College London in conjunction with the biotechnology company MeiraGTx, was used by doctors to restore vision function. The method is based on the introduction into the eyes of healthy copies of the AIPL1 gene, which is responsible for the work of retinal photoreceptors.

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Фото: Unsplash

All patients suffered from Leber's congenital amaurosis, a disease in which babies are born virtually blind, preserving only the ability to distinguish between light and darkness. During the first years of life, children lose this function as well.

Surgery of 60 minutes was performed on only one eye of each patient to minimize possible risks. The children were monitored for five years after surgery.

As a result of treatment, patients gained the ability to distinguish the outlines of objects and recognize the faces of their parents. If before therapy they could not even see bright objects at close range, then after it they were able to serve themselves in everyday life, and some mastered reading and writing.

The British Office for the Control of Medicines and Medical Devices (MHRA) has not yet approved this treatment method for widespread use, but allows its use in special cases. By February 2025, 11 children aged 1 to 4 years had received therapy.

Regulatory authorities in the European Union and the United States have designated the drug as an orphan drug, which should accelerate its further development and implementation in clinical practice.^[1]

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