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Advantages of Developing Platform-Agnostic Gene Therapies

The chief executive officer of Rocket Pharmaceuticals, Gaurav Shah, MD, discussed the company’s ex-vivo and in-vivo gene therapy platforms.

Hemophilia A Gene Therapy Placed on Clinical Hold

Pfizer and Sangamo voluntarily paused the trial after participants experienced FVIII activity levels of over 150%.

Targeting the Putamen in AADC Deficiency: Paul Wuh-Liang Hwu, MD, PhD

The professor from National Taiwan University Hospital discussed the benefits of delivering gene therapy directly to the putamen in AADC deficiency.