8:50 am Chair’s Opening Remarks
ACHIEVING PRODUCT SUPERIORITY IN A CROWDED THERAPEUTIC LANDSCAPE
9:00 am Highlighting the Importance of Product Differentiation at Early Stages of a Program
Synopsis
- Developing strategies to showcase unique properties of your product
- Determining how to frame your product in a potentially crowded market
- Emphasizing the importance of product differentiation in research stages as well as clinical stages
9:30 am Evaluating the Unique Opportunities in the Blood Disease Space
Synopsis
- Utilizing real-world data to assess unmet need in the gene therapy landscape
- Developing novel economic models to understand product value and opportunity
10:00 am Morning Refreshments & Networking
CONSIDERING KEY ASPECTS OF CLINICAL TRIAL DESIGN FOR NON-MALIGNANT BLOOD DISORDERS
11:00 am The Keys to a Successful Clinical Trial for Sickle Cell Disease/ β-Thalassemia
Synopsis
- Understanding the operational considerations in conducting these trials – a site perspective
- Optimizing site selection for a successful short- and long-term partnership
- Understanding the co-morbidities in the sickle cell and thalassemia patients – considerations for study design and treatment planning
11:30 am Gaining Insights into a Successful Hemophilia Gene Therapy Trial
Synopsis
- Overview standard outcomes measures and recent surrogate endpoints, safety measures, and patient important outcomes (PIOs) and patient reported outcomes (PROs)
- Understand logistical considerations for early trial clinical development and scalable clinical product management
- Examine patient selection and retention in hemophilia advanced therapy trials
12:00 pm Lunch & Networking
REALIZING GENE EDITING POTENTIAL IN REVOLUTIONIZING THE TREATMENT OF SICKLE CELL DISEASE & Β-THALASSEMIA
1:00 pm Application of Ex Vivo Non-Viral Gene Therapy and Facilitating Engraftment of Gene-Corrected Hematopoietic Stem Cells
Synopsis
- Delivery of Poseida’s platform technology in gene addition (piggyBac transposons) and gene-editing (Cas-Clover CRISPR)
- Production of pure CAR-T and HSC products
- HSC homing and expansion
- Selective targeting of HSCs for safer transplant conditioning
1:30 pm Designing a Clinical Trial for a Gene-Edited Gene Therapy Treatment
Synopsis
- Translating CRISPR-Cas9 technology into the clinic: key considerations
- Addressing potential quality and compliance challenges specific to gene editing trials
2:00 pm Panel Discussion – Discussing Challenges of Gene Editing Approaches in the Gene Therapy Space
Synopsis
- Translating from pre-clinical studies into the clinic
- How to decide which gene editing approach is best for each indication
- Navigating the regulatory landscape
- Ensuring patients understand what treatment involves compared to current options
- Possible applications of gene editing technology across blood disease indications
2:30 pm Afternoon Refreshments & Networking
IMPROVING THE PATIENT EXPERIENCE – IN CLINICAL TRIALS & POST-APPROVAL
3:00 pm Running Patient-Centric Gene Therapy Programs to Optimize Success
Synopsis
- Utilizing patient advocacy connections to gain full understanding into patient needs
- Informing drug development decisions through insights into clinical unmet need
- Incorporating patient engagement initiatives in internal development strategies for truly valuable pipeline development
3:30 pm Do Patients Really Want These Therapies? Alleviating Patient Concerns to Bring Curative Therapies into the ‘Real World’
Synopsis
- Understanding patient perspectives from the hemophilia, sickle cell and beta-thalassemia community in taking the gene therapy step
- Why will some patients choose to wait to try gene therapy, even if they are eligible? Why will some be the first to “sign up”?
- Identifying the best ways to engage patients after treatment has been administered: what is the follow-up protocol, for how long, and how to do that if the patient does not live near the center that administered the Gene Therapy
- Bringing gene therapy into the public sphere