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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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Research Update: Investigational Gene Therapy for Cystinosis

Data from the AVROBIO Phase 1/2 investigational gene therapy for cystinosis was released today at WORLDSymposium in San Diego....

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AVROBIO Cystinosis Focus Group

AVROBIO is looking for perspectives from the cystinosis community. They will be conducting a small focus group to help...

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AVROBIO reports: U.S. Food and Drug Administration granted Fast Track Designation to AVR-RD-04 for cystinosis

In a quarterly update, AVROBIO announced:   “Data from the patients dosed to date across three indications continue to...

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New Cystinosis Gene Therapy Data from AVROBIO

AVROBIO Announces 100% Kidney Substrate Reduction at 12 Months Post-Gene Therapy in...

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AVROBIO: Cystinosis Gene Therapy Update Functional & Clinical Improvements Reported

November 17, 2020 – AVROBIO announced that data suggests functional and clinical improvements for the first cystinosis patient at...

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Program to Advance Cystinosis Treatment Options

Partnering with RDMD, this program aims to accelerate cystinosis research to ultimately improve treatment options. We welcome you to...

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Second Patient Dosed in Gene Therapy Trial

AVROBIO announces the second patient with cystinosis has been dosed in the company’s AVR-RD-04 investigational gene therapy Phase 1/2...

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Update: AVROBIO & COVID-19

A letter from Fernanda Copeland, Executive Director, Head Patient Advocacy & Engagement at AVROBIO March 24, 2020 We hope...

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