8:00 am Coffee & Registration

8:45 am Chair’s Opening Remarks

Advancing Gene Therapies into the Clinic

9:00 am Significance of Neutralizing Antibodies in Gene Therapy in Hemophilia

Synopsis

  • Can a titer that impede transduction be defined
  • Impact of vector on neutralizing antibodies
  • Neutralizing antibodies and clinical trials

9:30 am Emerging Roles of Transgene Mutations in Gene Therapy Products for Hemophilia

Synopsis

  • Protein engineering strategies to increase protein expression and specific activity of Factor VIII and Factor IX proteins
  • Immunogenicity of \variant proteins and clinical concerns
  • How various Factor VIII mutations are moving into clinical applications

10:00 am A Stable & Durable Treatment for Pediatric Patients with Hemophilia

  • Dean Falb Chief Scientific Officer, LogicBio

Synopsis

  • Canonical gene therapy cannot be used to treat pediatric patients due to episomal DNA dilution and lack of therapeutic durability
  • GeneRide provides a safe and durable platform for treating pediatric hemophilia patients through insertion of therapeutic transgenes into the chromosome
  • Preclinical data in Hemophilia B mouse models demonstrates the safety and efficacy of this therapeutic approach

10:30 am Morning Refreshments & Networking

11:00 am Preliminary Data for DB-525 for the Treatment of Severe Hemophilia A

Synopsis

  • Study design
  • Review of safety
  • Review of clinical efficacy

Maximizing Patient Engagement with Novel Technology

11:30 am Improving Patient Education to Build an Informed, Engaged and Empowered Patient Population

  • Tessa Field Patient Advocacy lead , Spark Therapeutics

Synopsis

  • Understanding the questions, concerns, and knowledge gaps of the patient community
  • Bridging communication between industry and the patient community
  • Improving patient assess to balanced scientific information

12:00 pm Patient Advocacy Strategies in World Wide Research & Development of Gene Therapy

  • Rosemarie Sellati Director Development Sciences Patient Advocacy, Hematology,, BioMarin Pharmaceutical

Synopsis

  • Integrating patient voices in research and development
  • Assessing barriers to recruitment and retention across various regions around the world
  • Understanding differences in regulatory bodies, treatment centers and patient needs

12:30 pm Lunch & Networking

Addressing Medical Considerations to Improve Standard of Care

1:30 pm Data from a Prospective Study using FVIIIFc for ITI in Hemophilia Patients with Inhibitors

  • Nisha Jain Executive Medical Director, Bioverativ, a Sanofi Company

Synopsis

  • Interim data from an ongoing studying
  • Predicting responders and non-responders
  • Role of high dose in tolerization success

2:00 pm Women’s Health in Von Willebrand Disease

  • Henry Mead Director, Medical Communication & Education Strategy Hematology Medical Therapeutic Area Coagulation, CSL Behring

Synopsis

  • Relationship between heavy menstrual bleeding and Von Willebrand Disease
  • Underdiagnoses of Von Willebrand Disease and percentage currently require a factor treatment
  • Characterising disparities among different populations and age demongraphics
  • Genetic profiles and bleeding assessment tools

2:30 pm Afternoon Refreshments & Networking

3:00 pm Patient Advocacy and Improving Diagnosis of Von Willebrand Disease

  • Dawn Rotellini Senior Vice President, Program Development , National Hemophilia Foundation

Synopsis

  • Devising New guidelines for the diagnosis and treatment of Von Willebrand Disease
  • Genetic testing for Von Willebrand to determine mutation and carrier status
  • Under-diagnoses of Von Willebrand Disease and percentage currently require a factor treatment
  • Improving awareness and taking concerns seriously

3:30 pm Challenges and considerations in medical affairs for novel hemophilia drug development

Synopsis

  • Understanding the needs for patients
  • Representativeness for minority groups
  • Pharmacoeconomic considerations

4:00 pm Chair’s Closing Remarks

4:15 pm Close of the HDD Summit