Day One

Wednesday, March 30 | 08.50 AM - 5.10 PM

ADDRESSING SAFETY & EFFICACY OF GENE THERAPY PRODUCTS

8:50 am Chair’s Opening Remarks

9:00 am Autologous HSPC-targeted LV Gene Therapy for Fanconi Anemia & LAD-I: An Update From Rocket Pharmaceuticals

Synopsis

  • Specific challenges for cell- and gene-based therapy in life-threatening hematopoietic disorders.
  • Fanconi anemia gene therapy program: updated results
  • Leukocyte adhesion deficiency gene therapy program: updated results

9:30 am CRISPR/Cas9 Gene Editing in Hemophilia B

Synopsis

  • A landmark phase 1 clinical trial demonstrated successful gene editing for transthyretin amyloidosis, establishing proof of concept for CRISPR-Cas9 treatment in humans (Intellia Therapeutics and Regeneron Pharmaceuticals)
  • Intellia Therapeutics and Regeneron Pharmaceuticals are developing a targeted insertion platform for the expression of Factor IX from the Albumin locus in liver for the treatment of Hemophilia B, with pre clinical data showing robust and durable FIX expression in mouse and non-human primates
  • The Factor IX gene insertion program for the treatment of Hemophilia B is currently advancing toward IND-enabling studies

10:00 am Evolution of Patient Concerns Regarding Safety of Gene Therapy Products in the Hemophilia Community

  • Glenn Mones Advocacy Leader , The Coalition Haemophilia B

Synopsis

  • The tainted blood crisis had a long-term effect on patient concerns about product safety
  • This effect carries over into concerns regarding gene transfer and other newer technologies
  • Adequately addressing these concerns is critical to patient acceptance and adoption of these therapies

10:30 am Morning Break & Structured Networking

11:30 am Hepatological Impact of Current Gene Therapy Products – Where Do We Stand & What Lies Ahead?

  • Graham Foster Professor of Hepatology , Queen Mary University of London

Synopsis

  • Gene therapy related hepatic inflammation – what causes it and what to do
  • Gene therapy and liver cancer, is there a real risk and how should it be managed
  • How should we assess liver function before and after gene therapy

12:00 pm Development of an Anti-CD117 Antibody Drug Conjugate for Hematopoietic Stem Cell Based Gene Therapy

Synopsis

  • Overview of targeting CD117 for conditioning
  • Pre-clinical validation of ADC approach using experimental tool anti-CD117 ADCs in NHP gene therapy models
  • Development of MGTA-117: an amanitin-based ADC under clinical evaluation

12:30 pm Joint Q&A Session

Synopsis

  • Have the speakers answer your questions

1:00 pm Lunch

BUILDING A PRODUCT TO DELIVER MAXIMUM BENEFITS FOR THE PATIENT

2:00 pm LV-HSC Platelet-Derived Factor VIII “Pleightlet™” for Severe Hemophilia A with Inhibitors

  • David Wilcox VP Research & Translational Medicine, Medical College of Wisconsin

Synopsis

  • Summary of HSC gene therapy strategy for using platelet FVIII for hemophilia A
  • Seminal pre-clinical and validation studies – in support of clinical efficacy
  • Current status of the FDA approved phase I clinical trial

2:30 pm Understanding the Patient Perspective in Europe

Synopsis

  • Paying for gene therapy – The European Model
  • Opportunities and challenges for haemophilia patients

3:00 pm Dissecting the Current & Future Drug Development Landscape for Blood Disorders & What This Means for the Patients

  • Guy Young Director Hemostasis & Thrombosis Center, Children’s Hospital LA

Synopsis

  • Present a brief history of hemophilia treatment and current treatment options.
  • Discuss the current unmet needs for patients with hemophilia
  • Review the novel therapies currently being developed for hemophilia

3:30 pm Afternoon Break

4:00 pm Joint Q&A Session

  • David Wilcox VP Research & Translational Medicine, Medical College of Wisconsin
  • Brian OMahoney CEO, Irish Haemophilia Society
  • Guy Young Director Hemostasis & Thrombosis Center, Children’s Hospital LA

Synopsis

  • Have the speakers answer your questions

4:30 pm Pediatric Gene Therapy & Medical Ethics: Informed Consent

  • Lesha Shah Co-Chair , Pediatric Gene Therapy & Medical Ethics Working Group

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

5:00 pm Joint Q&A Session

  • Lesha Shah Co-Chair , Pediatric Gene Therapy & Medical Ethics Working Group

Synopsis

  • Have the speakers answer your questions

5:30 pm Chair’s Closing Remarks

5:40 pm End of Conference Day One