Pre Conference Workshop Day

Manufacturing

Clinical

Regulatory

Market Preparedness

Workshop A

9.00AM - 12.00PM

Workshop B

9.00AM - 12.00PM

Workshop C

9.00AM - 12.00PM

Workshop D

9.00AM - 12.00PM

Strategies to Successfully Scale Up to Commercial Manufacturing

  • Understanding how to weigh up the decision between outsourcing and developing in-house capabilities
  • Designing processes to be flexible in light of an unpredictable supply chain
  • Analysing the benefits and limitations of the most popular AAV production platforms available
  • Successfully shifting from adherent to suspension cell lines when transitioning from the lab to the clinic
  • Exploring the importance of successful batch execution

Brad Mickey, Director, Manufacturing, MSAT & Engineering, Astellas Gene Therapies

Tristan Marshall, Vice President, Process Development, REGENXBIO

Accelerating Progress Through the Clinic with Effective Endpoint Selection

  • Exploring how biomarkers can be used as validated surrogate endpoints to increase chances of gaining FDA accelerated approval
  • Navigating a lack of available data in the rare disease space to define clinical endpoints
  • Lessons learned from successful clinical trials using novel endpoint selection
  • How can we leverage insight from patient groups to develop endpoints more conducive to gaining conditional approval?

Michael Grosso, Global Head of Clinical Development, Specialty Medicine, Daiichi Sankyo

Oscar Segurado, Chief Medical Officer, ASC Therapeutics

Exploring the Best Practices for Cross-Collaboration Between Regulatory and Other Departments

  • How the key structural differences between biotech and pharma gene therapy teams influence collaborative strategy
  • Analysing key pinch points in regulatory interactions throughout the development timeline: how can we work with other departments to prepare effectively?
  • How can we effectively collaborate with scientific SMEs to prepare for regulatory meetings and write regulatory documents?
  • What does good teamwork look like when preparing for product approval?

Elizabeth Hook, Global Head of Clinical Development, Specialty Medicine, Homology Medicines

Lisa Carlton, Vice President, Global Regulatory Affairs, REGENXBIO

Developing Viable Commercial Models for Gene Therapies

  • Addressing the challenges in education and misinformation in target disease states for future gene therapies in order to protect gene therapy benefits with commercial payers and employers
  • Exploring the different geographical commercial reimbursement landscapes for gene therapies
  • Strategies for successful business models to create sustainable, marketable and commercially viable gene therapy products
  • Establishing effective payment and risk-sharing agreements between sponsors and payers
  • The keys to success in building up a biotech commercial team from scratch
  • Optimizing patient identification to set up for commercial success

Lawrence Kau, Global Commercial Development Lead Spark Therapeutics

Carrie Edwards Wolkoff, Former Spark Gene Therapy National Account Director, , Gov, and Federal US

12.00 pm Lunch & Networking

Manufacturing

Clinical

Regulatory

Market Preparedness

Workshop E

1.00PM - 4.00PM

Workshop F

1.00PM - 4.00PM

Workshop G

1.00PM - 4.00PM

Workshop H

1.00PM - 4.00PM

Finetuning Downstream Processes to Produce Purer AAV Vectors

  • Exploring the latest full-empty capsid separation techniques
  • Optimizing later stage clinical manufacturing processes to comply with cGMP regulations
  • Understanding the intricacies of each downstream purification stage, including clarification, purification, filtration and formulation
  • Analyzing different vector chromatography approaches for purification
  • Overcoming inhibitive costs associated with numerous purification steps
  • Contaminant risk mitigation: strategies to keeping adventitious viruses out of your product

Joe Balleydier, Vice President Process Development, Jaguar Gene Therapy

Chris Williams, Principal Engineer, Roche

Investigating the State-of-Play and Challenges Surrounding Redosing Strategies

  • An overview of the immune response to gene therapies and how this impacts upon potential clinical dosing strategies
  • Exploring the use of novel vectors to control the immune response
  • Investigating the latest approaches to circumvent humoral immunity to gene therapies
  • The importance of measuring full-empty capsid ratios, the strategies behind this, and ongoing advocacy towards FDA guidance

Nicole K. Paulk, Assistant Professor, UCSF

Genine Winslow, Founder & Chief Executive Officer, Chameleon Biosciences

Kei Kishimoto, Chief Scientific Officer, Selecta Biosciences

Expediting Your Gene Therapy Product: Leveraging Regulatory Expertise over FDA Processes

  • Outlining the road to success with Fast Track, Breakthrough Therapy, Accelerated Approval, and Priority Review processes
  • Comprehending the specific situations to which these tools apply
  • A 101 in leveraging pre-IND interactions with the FDA
  • Understanding the challenges in attaining and maintaining Orphan drug designations
  • Common pitfalls and misunderstandings in filings for these expedited programs

Weicheng Wu, Vice President, Global Regulatory Affairs, ASC Therapeutics

A 101 in Demonstrating Value

  • How can we leverage innovative approaches to data collection to demonstrate long-term efficacy and safety, and ultimate drive confidence in gene therapies?
  • Key considerations for demonstrating value to payors, patients, and clinicians
  • Analysing price tolerance differences between European central and U.S. private payer systems.
  • How to leverage understanding of these differences to market your product in both continents.
  • Case study of an approved gene therapy product: how to choose data points to successfully demonstrate value and justify high price points?

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

Sarah Pitluck, Former Head/Vice President, Global Pricing & Reimbursement Strategy, Spark Therapeutics

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