Day One

Wednesday, March 10

Day Two

Thursday, March 11

*All times are shown in EST

COMMERCIALIZATION OF GENE THERAPIES FOR BLOOD DISORDERS

9:00 am Standardization to Create Stable Relationships Between Industry Developers & Hospitals

Synopsis

  •  Anticipating a surge in hospital demand and preparing to manage upcoming gene therapies which require certain degrees of resources and unique requests
  • Standardization of blood collection processes, labelling, traceability systems, formatting for data printing, contracting processes and real-world evidence generation
  • Encouraging conversation between standardization bodies and industry partners, and bringing in regulators to negotiate with hospitals to implement standardization where possible

9:20 am Differentiating Your Development Strategy for Gene Therapies

Synopsis

  • Encouraging industry collaboration to share lessons learned
  • Considering the impact of gene therapy landscape for your development strategy
  • Collaboration to ensure support of clinicians and patients
  • Differentiating against competitors

9:40 am Q&A Panel Discussion

  • Richard McFarland President , Standards Coordinating Body (SCB)
  • Alexandria Petrusich Head of Translational Medicine , Graphite Bio
  • John Pasi Professor, Haemostasis & Thrombosis; Barts & the London School of Medicine, Principal Investigator, BioMarin Valoctocogene Roxaparvovec Program

10:10 am Morning Break

PRICING & REIMBURSEMENT: SETTING UP FOR THE PUSH TO MARKET

10:30 am Developing a Gene Therapy Payment Model & Pricing Strategy for Hemophilia when Alternative Treatments are Available

  • Nick Li Senior Director, Health Economics & Outcomes Research, UniQure

Synopsis

  • Changing a system to allow for onetime payment that captures the value of the product
  • Exploring creative payment models with payers and supplying compelling data to payers to prove long term benefit
  • Discussing the commercial implications of having other treatments available

10:50 am Evaluating Different Payment Models for Different Blood Indications

  • Mark Trusheim Strategic Director, NEWDIGS & Visiting Scientist, MIT Sloan

Synopsis

  •  Explaining a payer’s perspective on pricing and reimbursement for gene therapies for blood disorders
  • Discussing their requirements and views on innovative payment models
  • Predicting state changes and national policy implications
  •  Highlighting the differences between sickle cell disease, beta-thalassemia and hemophilia

11:10 am Gene Therapy Precision Financing Models for Blood Conditions

  • Clark Paramore Head of Value Demonstration, Vice President HEOR, bluebird bio

Synopsis

  • Addressing Value Demonstration Challenges for Gene Therapy for Blood Disorders
  • Bringing a one-time potentially curative therapy to a chronic disease with history of limited treatment options has inherent value demonstration challenges
  • Proper investment in real-world evidence to best characterize disease unmet needs is critical to ensure patient access and commercial success
  • Novel payment models that are linked to credible estimates of product value can help overcome access hurdles

11:30 am Q&A Panel Discussion

  • Nick Li Senior Director, Health Economics & Outcomes Research, UniQure
  • Mark Trusheim Strategic Director, NEWDIGS & Visiting Scientist, MIT Sloan
  • Clark Paramore Head of Value Demonstration, Vice President HEOR, bluebird bio

12:00 pm Lunch Break

ADDRESSING RISK CONCERNS & LONG TERM SAFETY STRATEGIES

1:00 pm Antibody-Based Conditioning for Safer Hematopoietic Stem Cell Transplantation

  • Wendy Pang Executive Director, Research & Clinical Development, Jasper Therapeutics

Synopsis

  • Exploring the current challenges of patient conditioning for ex vivo products
  • Highlighting new research in antibody-based conditioning regimens for hematopoietic stem cell transplantation, including those for gene therapies

1:20 pm Developing a Long Term Follow Up Strategy for Genetic Therapies for Blood Disorders

  • Paul Monahan Clinical Development Lead, Hematology, Spark Therapeutics

Synopsis

  • Considering the long term risks of gene therapies for blood disorders
  • How do the risks differ by patient population and specific approach?
  • Discussing the current FDA and EMA guidelines for long term follow up

1:40 pm Q&A Panel Discussion

  • Wendy Pang Executive Director, Research & Clinical Development, Jasper Therapeutics
  • Paul Monahan Clinical Development Lead, Hematology, Spark Therapeutics

2:00 pm Afternoon Break

NAVIGATING LOGISTICAL & MANUFACTURING CHALLENGES FOR DIFFERENT THERAPEUTICS

2:30 pm Navigating Early Phase CMC & Regulatory Challenges with CRISPR/ CAS9 Technology

  • Sandeep Soni Executive Director, Clinical Development , CRISPR Therapeutics, Inc.

Synopsis

  • Harnessing what can be learned from previous successes and failures in blood disorders trials
  • Anticipating process changes and forming strategies

2:50 pm Ready for Launch? – Operationalizing your Supply Chain to Deliver Cell Therapies Commercially

Synopsis

• Supply Chain design and key considerations for treating 1000’s of patients
commercially
• The complexity and challenges of planning and operationalizing your supply
chain for commercial launch
• Preparing for what happens next after launching your product, and key
considerations for lifecycle management

3:10 pm Q&A Panel Discussion

Synopsis

Additional Panelists to be Confirmed

3:40 pm Chair’s Closing Remarks

Day One