AVROBIO plans to initiate a global phase 2/3 trial of AVR-RD-02 in GD3 in the second half of 2023.
Lysosomal Disorders
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Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The chief medical officer of Forge Biologics discussed the potential advantages of the investigational combination therapy approach.
Review top news and interview highlights from the week ending December 2, 2022.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Lysogene’s MPS IIIA gene therapy program recently failed its primary endpoint in the phase 2/3 AAVANCE trial.
The chief medical officer of Forge Biologics discussed clinical data presented at ESGCT.
Review top news and interview highlights from the week ending November 25, 2022.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Statistically significant improvements in cognitive development were only seen in the cohort of patients younger than 30 months.
The gene therapy was approved in the European Union earlier this year.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The first 3 patients dosed have been attack-free for up to 10.5 months.
The Genomics and Epigenetic Guided Safe Harbor mapper will aid in the future design of gene-editing therapies.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Review top news and interview highlights from the week ending November 4, 2022.
Abeona has announced topline results from its phase 3 VIITAL study of EB-101.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The pediatrician from Royal Manchester Children’s Hospital discussed how further research in Gaucher disease can benefit the field of rare lysosomal diseases as a whole.
Review top news and interview highlights from the week ending October 28, 2022.
CGTLive highlights the PROVIDE trial of PR001 for Gaucher Awareness Month.
AVR-RD-02 also received ILAP designation in the UK earlier this month.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The pediatrician from Royal Manchester Children’s Hospital discussed progress with the PROVIDE trial of PR001.
The trial is being conducted at the University of California – San Diego under the leadership of Stephanie Cherqui, PhD.
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