Gene Therapy for Blood Disorders 2020

Gene Therapy for Blood Disorders is organized by Hanson Wade and will be held from Mar 03 - 05, 2020 at Courtyard by Marriott Boston Downtown, Boston, Massachusetts, United States of America.

Main Conference: March 04 - 05, 2020
Pre-conference Workshop Day Tuesday, March 3

Description:
There is increased investment in gene therapies to treat hemophilia, sickle cell disease and β-thalassemia, however developing these programs pose unique indication-specific challenges. Understanding the specific challenges and opportunities in this space is crucial to ensure the success of your gene therapy program.

This focused agenda brings together key thought leaders across the blood disease space to share their expertise at every stage of clinical and commercial development, including:
• Gene Editing Innovators
• Clinical Developers
• Patient Advocacy Experts
• Commercial Launch Leaders
• Market Access Strategists

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
β-thalassemia.

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.

“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!” 

Pre-conference Workshops:
Workshop A
Date: Tuesday, March 03
Time: 8:30 am - 11:00 am
Enhancing Gene Editing Techniques to Improve Gene Therapy Programs for Blood Disorders
Description: Gene editing is a revolutionary technique which is starting to make its way into clinical trials for a variety of indications. It is evident that more and more companies are realizing the potential of gene editing techniques in the blood disorders space. With real promise to deliver durable, curative and transformative therapies to patients with disorders such as sickle cell disease and β-thalassemia, improving these gene editing technologies and techniques is vital to ensure these gene therapies reach the patients who need it.

Attend this workshop to learn about:
• Improving the precision of gene editing
• Minimizing off-target effects
• Requiring more than one genome edit
• How difficult is it to insert larger genes?
• Which animal models are most appropriate?
• Discussing transferrable insights across gene editing platforms
• Exploring applications of CRISPR in hematopoietic stem cells

Workshop B
Date: Tuesday, March 03
Time: 11:30 am - 2:30 pm
Establishing a Presence in the Blood Disease Space: Understanding Key Clinical & Commercial 
Considerations at the Beginning of Your Gene Therapy Program
Description: There continues to be great interest in creating gene therapies for blood disorders, with one indication approved in this space, and several others in late-stage clinical trials. As more and more companies begin investing in the field, it is crucial that they have an excellent understanding of clinical, commercial and regulatory aspects of the program from the very beginning. Entering the blood disorders space with an excellent grounding will set up for success later in the pipeline.

Attend this workshop to learn about:
• Beginning a program for blood disorders in the gene therapy space
• Exploring key regulatory requirements
• Challenges in translating from pre-clinical studies into the clinic
• Discussing the importance of a commercial outlook from early stages of the program
• How to transition into a more industrialized setting, and understanding the possible limitations
 
Workshop C
Date: Tuesday, March 03
Time: 3:00 am - 6:00 pm
Strategic Gene Therapy Product Platforms and Partnerships: From Codevelopment to Companion Products
Description: With more investments in gene therapies to treat blood diseases than ever before, it is imperative that your product stands out in what promises to be a competitive market. By building an effective codevelopment program, understanding new FDA guidances surrounding codevelopment, and identifying which companion and diagnostics products would best enhance your platform, you can ensure your therapy will have a competitive advantage that enables you to stand out from the crowd.

Attend this workshop to learn about:
• How companies collaborate to form an effective business partnership
• Regulatory strategy in codevelopment
• Using companion products in program development
• Trial design and clinical strategy for codevelopment
• Challenges of having multiple sponsors and concurrent programs
• Companion product marketing and commercialization

Additional details will be posted as soon as information is available.