Discover the Clinical & Commercial Potential of Gene Therapy & Editing Approaches Revolutionizing Hemophilia, Sickle Cell Disease & β-Thalassemia Treatment
Revolutionary gene therapies are redefining the treatment of blood diseases.
With an approved gene therapy treatment for β-thalassemia in the EU, and hemophilia candidates seeking 2020 approval in the USA and beyond, pioneers in the space must overcome hurdles they have never previously encountered to transform their clinical candidates into a commercial reality. Gene Therapy for Blood Disorders will unite industry leaders across the blood disease space to delve into the specific clinical and commercial challenges faced by companies developing gene therapies for hemophilia, sickle cell disease and
Evaluate the best strategies for a commercial launch in blood disease, optimize your trial design, gain insights on revolutionary novel technologies, and ultimately enhance your current hematology gene therapy program to bring transformative treatments to patients more quickly and efficiently.
Download the full event guide to learn more from companies including bluebird bio, Spark Therapeutics and Editas Medicine.
“This is "the conference" to attend for professionals that are already involved or about to embark in the gene therapy space. All of the relevant experts in their field are represented and available to share their knowledge and experience. A true educational and networking event!”
Gene Therapy for Rare Disorders 2019 Attendee - AveXis
“Best conference of the year for gene therapy companies to understand how our industry is tackling the challenges of pioneering the development of our advanced therapy products.”
Gene Therapy for Rare Disorders 2019 Attendee -Sangamo Therapeutics
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