Discover the Clinical & Commercial Potential of Gene Therapy & Editing Approaches Revolutionizing Hemophilia, Sickle Cell Disease & β-Thalassemia Treatment

2020 has been an exciting year for gene therapies targeting blood disorders. Despite FDA’s recent rejection of BioMarin’s hemophilia gene therapy program, the clinical landscape is progressing quickly with Pfizer and Sangamo kicking off their phase 3 and bluebird bio receiving European approval for their beta-thalassemia candidate.

With pipelines advancing and emerging companies joining the blood disorders field, it is essential for gene therapy developers to communicate and share lessons learned to accelerate their programs to market. The 2nd Gene Therapy for Blood Disorders meeting is taking place in March 2021, focusing on the clinical and commercial challenges of developing gene therapies targeting hemophilia, sickle cell disease and beta thalassemia.

Evaluate the best strategies for a commercial launch in blood disease and optimize your trial design to ultimately transform your clinical candidates into a commercial reality.

Download the full event guide to learn more from companies including bluebird bio, Spark Therapeutics and Editas Medicine.

Previous Gene Therapy for Blood Disorders Attendees said:

“The quality of participants, speakers and presentations were all excellent”

Associate Director, Business Insights, Sanofi

“Well balanced conference covering bench to patient perspectives”

Director, Medical Communication & Educational Strategy, CSL Behring

“Very interesting and well organised agenda covering hot topics in the industry”

Director, Strategy & Business Operations, Freeline Therapeutics

2021 Agenda Highlights Include:

John Pasi

Anticipate Commercial Implementation Challenges

Principal investigator for the BioMarin valoctocogene roxaparvovec program John Pasi (Professor, Barts and the London School of Medicine & Dentistry) will be sharing his insights from this experience.


Develop a Long Term Follow Up Strategy 

Consider the specific long terms risks of gene therapies for blood disorders & discuss current FDA and EMA guidelines with Paul Monahan, Hemophilia Clinical Development Lead, Hematology, Spark Therapeutics.

Wendy Pang

Explore Safer Hematopoietic Stem Cell Transplantation

Wendy Pang, Executive Director, Research & Clinical Development, Jasper Therapeutics will be showcasing the potential for next generation conditioning regimes for ex vivo gene therapies.


Optimize Clinical Development Strategies 

Accelerate timing of submission & demonstrate durability with insights from Sanofi’s Pablo Rendo, Global Program Head of The Red Blood Disorders (and previously Executive Director, Global Clinical Lead, Hemophilia at Pfizer).

Other Events in the Gene Therapy Series