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Funding raised for AAV9-based gene therapy research for HSP50 by end of 2024
2
Resolution / Starting Odds
Less than $1 million • 33%
$1-5 million • 33%
More than $5 million • 33%
Press releases, crowdfunding platforms, and official funding announcements
Story
Gene Therapy Halts Progression of Rare Genetic Condition in 4-yr Old Toronto Boy Michael
Jun 28, 2024, 11:33 PM
A groundbreaking gene therapy has halted the progression of a rare genetic condition in a young boy from Toronto. The therapy, conducted at SickKids Hospital and published in Nature Medicine, involved a personalized AAV9-based gene therapy for hereditary spastic paraplegia type 50. The phase 1 trial, which included only one patient, showed that the therapy was well tolerated and led to preliminary disease stabilization within three years from diagnosis. The patient, Michael, a 4-yr old, is the son of Terry Pirovolakis, who played a significant role in making this treatment possible.
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NIH • 33%
Gates Foundation • 33%
Wellcome Trust • 34%
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Government grants • 25%
Private investment • 25%
Pharmaceutical companies • 25%
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Private donations • 25%
Pharmaceutical companies • 25%
International organizations • 25%
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