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Gene therapy has the potential to cure rare diseases, yet pharmaceutical companies lack sufficient motivation, leaving families in dire need

Robin Alderman and Camden Alderman (Via Quentin White/Shutterstock)

Robin Alderman is facing a difficult situation: Gene therapy could potentially cure her son Camden’s rare inherited immune disorder. However, this treatment option is not accessible to him.

In 2022, Orchard Therapeutics, based in London, halted its investment in an experimental treatment for Camden’s condition, Wiskott-Aldrich syndrome. Currently, there are no available gene therapy trials that Camden can participate in.

“We feel like we have been forgotten,” said Alderman, who has been advocating for her 21-year-old son since he was an infant.

Worldwide, approximately 350 million people suffer from rare diseases, many of which are genetic. However, each of the 7,000 different disorders affects only a few people per million or less.

There is little financial motivation for companies to develop and bring to market these one-time therapies that aim to repair faulty genes or replace them with healthy ones. As a result, families like the Aldermans are struggling to find assistance, with some attempting to raise funds themselves for treatments that may never become available.

Robin Alderman and Camden Alderman (Via Quentin White/Shutterstock)

“These children have been unlucky twice: first, because they have a genetic disease, and second because the disease is so rare that it does not attract attention,” said Dr. Giulio Cossu, a professor of regenerative medicine at the University of Manchester in England. “Companies are driven by profit.”

Scientists warn that this situation could hinder progress in the emerging field of gene therapy, despite promising research indicating potential treatments for various disorders.

Researchers are actively seeking solutions, often turning to charitable organizations, patient groups, and governments for support.

In February, a major Italian charity announced its takeover of the Wiskott-Aldrich treatment program previously pursued by Orchard Therapeutics.

Additionally, an arm of the charitable Foundation Fighting Blindness collaborated to establish Opus Genetics, a company focused on advancing gene therapy research led by Dr. Jean Bennett from the University of Pennsylvania and her colleague.

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