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Rare disease breakthrough could unlock new treatments for ageing

Calico's experimental drug targets a key cellular stress pathway

29-Jun-2026

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Calico Life Sciences, the biotechnology company founded by Alphabet to study ageing, has received a major boost after the US Food and Drug Administration (FDA) granted Breakthrough Therapy designation to its experimental drug fosigotifator. The treatment is being developed for Vanishing White Matter disease, a rare inherited neurological disorder with no approved cure, but researchers believe the biological pathway it targets may also provide valuable insights into ageing.

The therapy works by targeting the integrated stress response (ISR), a cellular system that helps cells cope with damage and other forms of stress. While this response is essential in the short term, chronic activation has been linked to neurodegeneration, declining tissue function and other age-related conditions. By restoring normal ISR activity, scientists hope to protect vulnerable cells and slow disease progression.

Although the drug is currently focused on treating a rare disease, the milestone highlights a broader strategy in longevity research: studying uncommon genetic disorders to better understand the biological mechanisms that contribute to ageing. Success in rare disease trials could help pave the way for therapies targeting more common age-related illnesses in the future.

The FDA's Breakthrough Therapy designation is intended to speed up the development of promising treatments for serious conditions, potentially bringing new medicines to patients more quickly. For Calico, the decision marks one of the company's most significant clinical achievements to date and reinforces growing interest in targeting the fundamental biology of ageing rather than treating diseases one at a time.

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Calico

Combating aging and associated diseases. An Alphabet (Google) subsidiary.

Food and Drug Administration (FDA)

Ensuring safety of drugs, medical supplies and food which is used daily.

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Rare disease breakthrough could unlock new treatments for ageing