8:50 am Chair’s Opening Remarks

  • Catalina Driscoll Global Head, New Product Planning, Rare Blood Disorders, Sanofi Genzyme

ACHIEVING PRODUCT SUPERIORITY IN A CROWDED THERAPEUTIC LANDSCAPE

9:00 am Highlighting the Importance of Product Differentiation at Early Stages of a Program

  • Catalina Driscoll Global Head, New Product Planning, Rare Blood Disorders, Sanofi Genzyme

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

  • Colleen Dansereau Director, Clinical Operations, Gene Therapy Program, Boston Children’s Hospital

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

  • Angela Johnson Senior Director, Regulatory Affairs, Sigilon Therapeutics

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

  • Julian Down Senior Director, Gene Therapy, Poseida Therapeutics

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

  • Mrudula Donepudi Director, Scientific Communications and Clinical Science, Editas Medicine

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

  • Mrudula Donepudi Director, Scientific Communications and Clinical Science, Editas Medicine
  • Julian Down Senior Director, Gene Therapy, Poseida Therapeutics

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

  • Nicolas Garnier Director, Patient Advocacy, Global Product Development, Pfizer Rare Disease

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’

  • Dawn Rotellini Chief Operating Officer, National Hemophilia Foundation

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

4:00 pm Chair’s Closing Remarks

  • Catalina Driscoll Global Head, New Product Planning, Rare Blood Disorders, Sanofi Genzyme

4:15 pm Close of Conference