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author：focus 2025-10-01 11:00:22 - browse：79

Colorful broken DNA helix genome. -- health coverage from STAT — Adobe

In 2020, as biotech stocks surged amid the pandemic, a startup called Taysha Gene Therapies raised over $300 million off an audacious promise: It was going to license and develop gene therapies for at least 18 different rare and serious neurological diseases.

The effort brought hope for many rare disease families, some of whom had raised money to fund the UT-Southwestern scientist who designed the treatments after they saw no other options for their children.

Last week, the grand plan took perhaps a final blow. Taysha announced it would discontinue development of a treatment for giant axonal neuropathy, or GAN, a fatal, degenerative disease known to affect fewer than 100 people worldwide. The FDA, Taysha said, had requested an additional clinical trial that would be infeasible.

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