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THE EVOLVING GENE THERAPY FOR BLOOD DISORDERS LANDSCAPE

8:00 am Chair’s Opening Remarks

8:20 am Discussing Gene Therapy in Hemophilia– What Does the Past, Present and Future Look Like?

  • Ian Winburn Vice President, Global Medical Lead, Hemophilia, Endocrine & IEM, Rare Diseases, Pfizer

Synopsis

  • Discussing the history, recent advances and potential future developments of gene therapy in hemophilia
  • How recent developments in the approval space may influence the future
  • The critical importance of solving “Known Unknowns”

8:40 am Optimizing Regulatory Strategy for Gene Therapies: Current Trends, Guidance & Industry Milestones

  • Angela Johnson Senior Director, Regulatory Affairs , Sigilon Therapeutics

Synopsis

  • Get the latest updates on the dynamic regulatory landscape
  • Explore how new and upcoming guidance has reshaped strategy
  • Apply lessons learned from key regulatory milestones across the industry

9:00 am Q&A Panel Discussion

  • Ian Winburn Vice President, Global Medical Lead, Hemophilia, Endocrine & IEM, Rare Diseases, Pfizer
  • Angela Johnson Senior Director, Regulatory Affairs , Sigilon Therapeutics

9:20 am Speed Networking

Synopsis

Grab a quick cup of tea or coffee from the comfort of your own home or office and jump straight into your opportunity to connect with new contacts from active companies in the field and exchange digital business cards. Network and form lasting connections through this exclusive virtual speed networking!

10:00 am Morning Break

ADVANCING GENETIC THERAPIES FOR BLOOD DISORDERS TOWARDS THE CLINIC

10:40 am Session Reserved for Editas Medicine

11:00 am Progress with Non-Viral Gene Therapy Technologies

  • Julian Down Senior Director, Gene Therapy, Poseida Therapeutics

Synopsis

  • Utilizing Poseida’s proprietary platform technologies for gene addition
    (piggyBac® transposons) and high-fidelity gene-editing (Cas-CLOVER™ CRISPR)
  • Ex-vivo application for gene-correction of hematopoietic stem cells and in manufacture of CAR-T cells
  • In-vivo delivery with nanoparticles for permanent liver-directed treatment of
    genetic diseases

11:20 am Gene Editing with Investigational Zinc Finger Nucleases for the Potential Treatment of Sickle Cell Diseas

  • Alexandra Hicks Head of Dupixent Research, Immunology & Inflammation Research Therapeutic Area, Sanofi

Synopsis

An overview of the supporting preclinical data of a novel ex vivo gene editing approach to increase fetal haemoglobin in sickle cell disease will be presented

Details of the technology and detailed characterization of the gene editing will be reviewed

Clinical trial design and updates will also be presented

11:40 am Q&A Panel Discussion

  • Kate Zhang Vice President, Editas Medicine
  • Julian Down Senior Director, Gene Therapy, Poseida Therapeutics
  • Alexandra Hicks Head of Dupixent Research, Immunology & Inflammation Research Therapeutic Area, Sanofi

12:00 pm Lunch & Networking

OPTIMIZING CLINICAL TRIAL DESIGN & COLLABORATION

12:40 pm Optimizing the Clinical Development Strategies to Accelerate the Timing of the Submission & Demonstrate Treatment Effect Durability for AAV Platform

  • Pablo Rendo Global Project Head, Red Blood Disorders & Rare Disease Gene Therapy, Sanofi

Synopsis

• How the recent FDA feedback impacts the clinical development with regards
to surrogate endpoints and clinically meaningful endpoints
• What is the right patient follow up for the accelerated submission?
• How long do we have to follow up with the patients from the scientific,
patients community, and payer points of view?

1:00 pm Anticipating the Commercial Implementation Challenges of Gene Therapies for Hemophilia

  • John Pasi Professor, Haemostasis & Thrombosis; Barts & the London School of Medicine, Principal Investigator, BioMarin Valoctocogene Roxaparvovec Program

Synopsis

  •  Considering emerging gene therapy for hemophilia with impending transition to commercialisation, discussing the evolving issues and questions that treaters and patients will face with such a new approach to treatment
  • Based on the challenges experienced with clinical trials, what strategies and preparations are required to successfully implement gene therapy treatments for hemophilia?

1:20 pm Real World Evidence & Managing Data from Heterogeneous Data Sources

Synopsis

  • Considering the heterogeneity of blood disorders patient populations – which indication represents which evidence generation challenges?
  • Highlighting data collection strategies for optimal data quality
  • 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?

1:40 pm Q&A Panel Discussion

  • John Pasi Professor, Haemostasis & Thrombosis; Barts & the London School of Medicine, Principal Investigator, BioMarin Valoctocogene Roxaparvovec Program
  • Marcus Droege Vice President & Head Global RWE , bluebird bio
  • Pablo Rendo Global Project Head, Red Blood Disorders & Rare Disease Gene Therapy, Sanofi

2:10 pm Afternoon Break

OVERCOMING CLINICAL ENROLMENT CHALLENGES TO INCREASE DATA QUALITY & ADHERE TO SHORTER TIMELINES

2:30 pm Improving Awareness, Education & Accessibility for Patients to Improve Enrolment

  • Allison Intondi Senior Director, Head of Clinical Operations, Graphite Bio

Synopsis

  •  Increasing the speed of recruitment for clinical trials when other treatments are available, particularly with shorter timelines, and educating patients on a novel therapeutic
  • Addressing the lack of ethnic diversity and social-economic diversity to be more representative of the target patient population
  • Improving access to and quality of clinical trial information and access to the clinical trial themselves (diversity of sites, travel reimbursement, appointment timings etc.)

2:50 pm Pediatric Gene Therapy & Medical Ethics: Informed Consent

  • Lesha Shah Assistant Professor, Medical Director, Child, Adolescent & Family Services, Icahn School of Medicine at Mount Sinai, Mount Sinai Health System

Synopsis

  •  Informed consent and pediatric assent for trial participation
  • Novel considerations in operationalizing consent in gene therapy
  • Emerging ethical dilemmas in pediatric gene therapy research

3:10 pm Jumping into Icy Water: A Patient Perspective on Enrolment Challenges for Gene Therapy Trials

  • David Page National Director of Health Policy, Canadian Hemophilia Society

Synopsis

  •  Informing the patient community through the patient associations
  • Emphasizing “informed” in the informed consent process
  • Making clinical trials “patient friendly”

3:30 pm Q&A Panel Discussion

  • Nicolas Garnier Director, Patient Advocacy, Pfizer
  • Lesha Shah Assistant Professor, Medical Director, Child, Adolescent & Family Services, Icahn School of Medicine at Mount Sinai, Mount Sinai Health System
  • David Page National Director of Health Policy, Canadian Hemophilia Society
  • Beverley Francis-Gibson President & Chief Executive Officer, Sickle Cell Disease Association of America
  • Allison Intondi Senior Director, Head of Clinical Operations, Graphite Bio

4:00 pm Biotech Quick Fire Round: Introducing the New Preclinical Players

  • Gabriela Denning Vice President, Research & Development , Expression Therapeutics

4:30 pm Chair’s Closing Remarks

5:00 pm Scientific Poster Session