Gene Therapy for Blood Disorders: BioMarin Insights, FDA & EMA Guidelines, Demonstrating Durability and More
Exciting Times:
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.
Featuring insights from the above programs and more, the digital 2nd Gene Therapy for Blood Disorders Summit is returning to discuss the clinical and commercial challenges of developing gene therapies targeting hemophilia, sickle cell disease and beta thalassemia.
Insights-Driven Agenda:
The meeting’s agenda has been specifically designed to leverage the insights from this year’s industry news to accelerate the development of gene therapies for blood disorders.
Agenda highlights include:
- Principal investigator for the BioMarinvaloctocogene roxaparvovec program, John Pasi (Professor, Barts and the London School of Medicine & Dentistry), will be sharing his insights from this experience on day one of the meeting, and highlighting exactly what strategies and preparations are required to successfully implement gene therapy treatments for hemophilia.
- Sanofi’s Pablo Rendo, Global Program Head of The Red Blood Disorders (and previously Executive Director, Global Clinical Lead, Hemophilia at Pfizer) will be exclusively discussing how to optimize clinical development strategies and demonstrate treatment effect durability using insights from the recent FDA feedback.
- Meanwhile day two of the summit will feature a session dedicated to developing a long term follow up strategy for genetic therapies for blood disorders and the current FDA and EMA guidelines for long-term follow up with Spark Therapeutics’ Clinical Development Lead, Hematology, Paul Monahan.
- And how do you design real-world evidence based data generation activities in a way that is feasible both for the hospital and for industry whilst meeting the regulatory requirements? bluebird bio’s Vice President & Head Global RWE, Marcus Droege, will be highlighting the data collection strategies for optimal quality.
Other expert speakers include:
- Beverly Francis-Gibson, President & Chief Executive Officer, Sickle Cell Disease Association of America
- Sandeep Soni, Executive Director, Clinical Development, CRISPR Therapeutics Inc.
- Kate Zhang, Vice President, Editas Medicine
- Steven Arkin, Executive Director, Clinical Research, Rare Diseases Research Unit, Pfizer
- Wendy Pang, Executive Director, Research & Clinical Development, Jasper Therapeutics
- And many more…
For full agenda details, view the event guide here.
Uniting the Industry:
Experts from all stages of clinical and commercial development will unite to share their learnings and developments on all the hottest topics in this space through discussions, workshops and live Q&As.
As well as the expert speaker faculty, the meeting boasts a stellar attendee line-up with attending companies in 2020 including Bayer, Novartis, novo nordisk, Pfizer, Roche, Sanofi, Takeda, UniQure and many more.
Through the bespoke digital platform, delegates will have the opportunity to directly message all other attendees and speakers to spark discussions around their specific challenges and meet like-minded professionals or start new collaborations through the dedicated networking function.
Despite the ongoing crisis, the industry still needs to learn and network. The digital Gene Therapy for Blood Disorders Summit will allow delegates to access all of the above from the comfort of their home or office, whilst still providing all the benefits of a physical conference.
Find Out More:
If you would like to learn more about this conference, view the full event guide here.
Alternatively, contact the team using the details below.
And listen out for more updates on the 2nd Gene Therapy for Blood Disorders!
Contact
General Enquiries & Registration Assistance
Matty Slingsby
Delegate Acquisition Manager
Hanson Wade
(+1) 617 455 4188
info@hansonwade.com
Partnership Opportunities
Nicholas Ramovic
Partnerships Director
Hanson Wade
(+1) 617 455 4188
sponsor@hansonwade.com