Astellas Pharma took a big step into gene therapies when it announced an agreement at the end of 2019 to acquire Audentes Therapeutics for $3 billion. The company continues to build on that acquisition and has just completed construction on a 154,000 square foot, state-of the art cell and gene therapy facility to bring together teams dispersed across 20 locations. We spoke Ha Tran, medical head of cell and gene therapy for Astellas Pharma, about the company’s vision for cell and gene therapies, its efforts to restart trials for its X-linked myotubular myopathy gene therapy, and how it is looking to other programs beyond that.
Charging into the Storm
A Gene Therapy Developer that Embraces Different Models for Reaching Patients
Using Directed Evolution to Develop New Vectors for Genetic Medicines
Addressing a Shortage of Genetic Counselors with AI
A Small Molecule Therapy to Regenerate Muscle in People with DMD
Forging a Faster Path for Gene Therapies
Determining the Value of Rare Disease Therapies
A Clinical Trial Failure Derails a Promising Technology
Helping People with Undiagnosed Rare Diseases Find Answers
Speeding and Scaling the Development of Genome Editing Therapies
Getting a Next-Generation Genome Editing Therapy for Sickle Cell Disease Back on Track
Trying to Break a Leg in a Wheelchair
A Drug Developer that Makes Pediatric Cancers a Priority
Harnessing the Body’s Natural RNA Machinery to Treat Diseases
Targeting the Leading Cause of Death in Friedreich’s Ataxia with a Gene Therapy
How Inhaled mRNA May Help Rare Disease Patients Breathe Easier
Changing What’s Possible with Cell and Gene Therapies
Why Flying with a Wheelchair Is a Civil Rights Issue
Mapping Spheres of Influence
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