French Researchers Conduct Gene Therapy to Restore Liver Function Among Patients With Rare Disease

A group of European researchers from a nonprofit biotech firm Genethon in France has arrived with an experimental treatment for a rare liver disease.

The experts managed to restore the living function of some recipients suffering from Crigler-Najjar syndrome, a rare genetic condition.

Gene Therapy to Treat Rare Liver Disease

As Bloomberg spotted on Wednesday, Aug. 17 via the New England Journal of Medicine, Genethon researchers showed that a rare liver disease can be corrected with the help of gene therapy.

For newborns with Crigler-Najjar syndrome, producing an enzyme that gets rid of bilirubin is not possible. When the red blood cells disintegrate, your liver will do its job to clear a toxic chemical passing on it. The said chemical is bilirubin.

Bilirubin is the same chemical that makes people with jaundice have yellowish skin. Crigler-Najjar syndrome is something that is inherited and there's no way to treat it even with the most advanced technologies.

To eliminate the chemical inside the body, the patient will be required to be exposed to fluorescent light for a very long time. That's the usual scenario if the transplant is not accessible.

Trial Yields Good Results For Liver Disease Patients

The trial which started in December 2018 involves five women with Crigler-Najjar syndrome. Their ages are between 21 and 30 during the experiment.

Using gene therapy, the researchers performed a single intravenous infusion to emulate the gene that is inactive because of the disorder.

The research found out that the same treatment has worked well with hemophilia patients. The said condition is an inherited bleeding disorder in which blood doesn't clot normally.

As for the trial, the scientists gave a lower-dose version of gene therapy to two patients.

Meanwhile, the three other patients received a higher dose. According to the researchers, it has significantly decreased the level of bilirubin by 30%.

After 18 months, the researchers discovered that there were no adverse effects left by gene therapy. They did not even require them to restart the treatments. Soon, the team decided to do the same technique, but this time with a larger group of people.

What is Genethon?

Since 1997, Paris-based biotech research firm Genethon has been making meaningful discoveries when it comes to gene therapies.

At the time, the company was working with Sarepta Therapeutics Inc. to conduct a gene therapy for the treatment of Duchenne muscular dystrophy.

According to its LinkedIn profile, Genethon received the Prix Galien France for pharmaceutical research into "Medicines destined for rare diseases - cell therapy and gene therapy" in 2012.

Three years later, it became a recipient of the Global Innovation Competition 2023 under the "Risk Lift" category at the Elysee Palace. The project focuses more on the industrial methods of how to produce gene therapy vectors.