For over four decades, the aspiration of gene therapy has been to develop novel therapies with the potential to enhance human health. Recombinant AAV (rAAV), a type of gene therapy,…
Astellas Pharma has seen their ASPIRO trial put on clinical hold for the second time after the fourth patient passed away. The Phase I/II trial is evaluating AT132, a potential…
Written by: Ashley Walker Like many couples, my husband Johnny and I felt that the birth of our twin sons would mark the beginning of our family’s story. We certainly…
According to MedCity News, a third patient dosed with AT132 during the ASPIRO trial has died. The Phase 1/2 clinical trial tested the efficacy, safety, and tolerability of AT132 for…
At the end of June, the National Hemophilia Foundation (NHF) released a warning to patients with hemophilia regarding AT132, an investigational gene therapy candidate. Developed by Audentes Therapeutics, AT132 uses…
According to a story from sciencemag.org, an experimental gene therapy for the rare genetic disorder X-linked myotubular myopathy is starting to produce significant improvements for patients. A devastating disease that…
The investigational drug AT132 has been awarded Regenerative Medicine Advanced Therapy designation for the treatment of X-linked Myotubular Myopathy by the United States FDA. You can read the source press…
According to a story from medicalxpress.com, early tests of a gene therapy for X-linked myotubular myopathy are showing promising results. This could represent a major breakthrough for this disease, which…
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