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.
Tackling the Pricing Challenges for Advanced Therapies for Rare Diseases
Addressing the Current Limitations of AAV Gene Therapies
Examining the Legislative Landscape for Rare Disease Drug Development
How a Drug Setback Became a Patient Community’s Gain
Helping Regulators and Drug Developers Understand the Challenges of Living with Fabry Disease
Addressing Racial Disparities in a Rare Blood Cancer
How New Sequencing Technology Promises to Alter the Diagnostic Odyssey
Leveraging Technology to Empower Patients and Decentralize Clinical Trials
Equipping The Next-Generation Rare Disease Patient Advocate
How a Family Raced from Diagnosis to Experimental Gene Therapy in Three Years
How One Advocate Went from Losing His Hearing to Being Heard
Building a Pipeline of Therapies to Treat Rare Mineralization Disorders
Advancing an Oral Alternative to Infused and Injected Therapies for HAE
Embracing the Promise of Patient-Centered Biotechnology
Learning to Live with Loss
Using Cryptocurrency to Tap into the Wisdom of Crowds
A Therapy for a Rare Neurodegenerative Disease Moves Towards Regulatory Review
Transforming the Treatment of Neuromuscular Diseases with Next-Gen Oligonucleotides
Teaching the Immune System to Let Medicines Do Their Job
Designing Clinical Trials with the Patient in Mind
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