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Clinical trial of new gene therapy for serious eye disease

4 april 2019

In partnership with St. Erik Eye Hospital, the pharmaceutical company Novartis is currently conducting a first-in-human clinical trial of the gene therapy CPK850. The aim is to assess CPK850’s safety and whether it can improve visual function in patients with the inherited eye disease retinitis pigmentosa, caused by mutations in the RLBP1 gene.

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To the left: A normal fundus of the eye. To the right: A fundus of a patient with retinitis pigmentosa, where large pale areas with degeneration of the retina and choroid are visible.

Retinitis pigmentosa destroys the photoreceptor cells in the retina, leading to impaired vision. The cause is the absence of a specific protein in the eye, and there is currently no effective treatment. However, a number of gene therapy studies are under way globally, and now for the first time the gene therapy CPK850 is being tested in patients.

The study, which began in autumn 2018, has 15 subjects, who are being treated with the study medication at St. Erik Eye Hospital. Each subject receives a single subretinal injection in one eye, and all subjects will be followed for five years after receiving the injection.

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Anders Kvanta. Photo: Eva Tov
“It is fantastic to now be testing a potential treatment for a disease that otherwise inevitably leads to blindness. First, however, we have to verify the efficacy and safety of this experimental gene therapy,” says Anders Kvanta, an eye surgeon at St. Erik Eye Hospital and a professor at Karolinska Institutet, who is leading the study.

The majority of patients come from Västerbotten and the trial will be conducted in cooperation with the ophthalmology clinic at University Hospital of Umeå. 

“That St. Erik Eye Hospital has been given responsibility for such a unique and demanding trial is a great recognition of our expertise. It provides us with invaluable knowledge about conducting advanced early phase trials and puts us on the front line within clinical ophthalmic research,” says Anders Kvanta.

Text: Helena Mayer

Facts about retinitis pigmentosa
  • Retinitis pigmentosa (RP) is a collective name given to around 100 inherited eye diseases that affect the retina. 
  • In cases of RP, there is metabolic dysfunction in the retina caused by gene mutations. There are more than 500 known proteins in the retina and if just one protein is defective, a person will often gradually lose their sight. 
  • The disease and the severity of visual impairment varies widely, and it is believed that there are between 3,000 and 4,000 people in Sweden living with the disease.

Read more about the study at Novartis webpage

More on Anders Kvanta's research

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15 januari 2020