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Tag: investigational

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Novartis Statement: Cystinosis Stem Cell Gene Therapy Program

In May 2023, Novartis acquired the investigational autologous hematopoietic stem cell (HSC) gene therapy program for the treatment of...

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AVROBIO Reports Positive Data From Phase 1/2 Clinical Trial of Investigational Gene Therapy for Cystinosis, Including New Interim Data on Neurocognitive Measures

Press release originally published May 17, 2022 on Avrobio.com. New early data show key visual motor integration, visual perception...

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Brochure Available: Details Gene Therapy Used in Cystinosis Trial

AVROBIO has released a brochure providing detail on investigational gene therapies for lysosomal storage disorders. Earlier this year, AVROBIO’s...

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