gene therapy

Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate

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Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate

Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden’s rare, inherited immune deficiency. But it’s not available to him. In 2022, London-based Orchard Therapeutics stopped investing in an experimental treatment for the condition, Wiskott-Aldrich syndrome. And there are no gene therapy studies he can join. “We feel like we are the … Read more

Takeaways from AP’s report on access to gene therapies for rare diseases

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Takeaways from AP’s report on access to gene therapies for rare diseases

The promise of gene therapy looms large for families dealing with rare, genetic disorders. Such treatments offer the possibility of one-time cures. But families and researchers worry such therapies will remain out of reach. Collectively, about 350 million people worldwide suffer from rare diseases, most of which are genetic. But each of the 7,000 individual … Read more

A rural Ugandan community is a hot spot for sickle cell disease. But one patient gives hope

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A rural Ugandan community is a hot spot for sickle cell disease. But one patient gives hope

MBALE, Uganda — Barbara Nabulo was one of three girls in her family. But when a sister died, her mother wailed at the funeral that she was left with just one and a half daughters. The half was the ailing Nabulo, who at age 12 grasped her mother’s meaning. “I hated myself so much,” Nabulo … Read more

6 Deaf Children Can Now Hear After a Single Injection

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6 Deaf Children Can Now Hear After a Single Injection

To get the new genetic material into cells, they engineered harmless viruses to carry it. Doctors carefully injected a tiny amount of liquid containing the viruses into a part of the children’s inner ears called the cochlea, a spiral-shaped chamber that contains hair cells. The first patient in the trial received the gene therapy in … Read more

In a World First, a Patient’s Antibody Cells Were Just Genetically Engineered

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In a World First, a Patient’s Antibody Cells Were Just Genetically Engineered

Researchers at the company collected the patient’s B cells using a machine that removes blood, separates out a particular component, then returns the rest to circulation. There are billions of B cells in the body; Immusoft uses only a portion. “The body is constantly regenerating and producing new B cells,” Ainsworth says. To get the … Read more