Pre-Conference Workshops - Monday March 7, 2022

Morning Workshops

Workshop A:  9:00am - 12:00pm

Optimizing Key Lentiviral CMC & Analytical Approaches


Several gene therapies are being developed using lentiviral vectors, and it’s clear from regulatory feedback that quality and characterization of lentiviral gene therapy products are an absolute must when gearing up for any approval, and there are learnings to be made across the gene therapy field regardless which vector type being worked in. Whether you work in lentiviral vectors or not, this workshop will give a solid grounding in analytical and CMC techniques to meet regulatory expectations.

Attend this workshop to learn about:
  • Defining key quality attributes for lentiviral vectors
  • How translatable are AAV and lentiviral product characterization methods?
  • Streamlining CMC and regulatory interactions to meet current regulatory guidelines

Workshop B: 9:00am - 12:00pm 

Inputting High Quality Raw Materials Into Gene Therapy Development

  • Dawn Henke Senior Scientific Program Manager , Standards Coordinating Body (SCB)


It’s clear that quality of gene therapies is being scrutinised at every stage to ensure patient safety. Given this context, this workshop will delve into the importance of high quality, standardized raw materials and incorporating these into development, ensuring effective standards and parameters are put in place to maintain quality.

Attend this workshop to learn about:

  • Understanding guidelines on raw material standards that need to be met
  • Transitioning from research to clinical grade materials effectively
  • Effectively managing the supply chain end-to-end

Workshop C: 9:00am - 12:00pm

Accelerating Newborn Screening Programs for Earlier Diagnosis & Intervention

  • Amy Nicole Nayar VP, US Patient Advocacy & Government Affairs , Novartis Gene Therapies
  • Dylan Simon Newborn Screening and Diagnostics Policy Manager, EveryLife Foundation for Rare Diseases


Earlier diagnosis and intervention for severe rare diseases often has significant impacts on improving patient outcomes. This workshop will explore the development and implementation of newborn screening programs for rare diseases, how sponsors can get involved in this process, and ultimately make a significant difference in halting disease progression.

Attend this workshop to learn about:

  • What does it take to get newborn screening on the roster in different geographies?
  • Beginning newborn screening initiatives early on in development
  • Increasing likelihood of better outcomes through early intervention

Workshop D: 9:00am - 12:00pm 

Developing Gene Therapies For Ultra-Rare Diseases


Several of the challenges in rare disease, including patient identification, trial design, and patient access to therapies are all exacerbated when looking to develop gene therapies for ultra-rare diseases. This workshop will delve into the challenges faced by ultra-rare gene therapy development to set up a process going forward to bring these transformative therapies more feasibly to patients in need.

Attend this workshop to learn about:

  • Exploring regulatory guidelines in the context of ultra-rare diseases
  • Utilizing natural history studies and designing trials for even smaller patient numbers
  • Understanding commercial viability for ultra-rare gene therapy development

Workshop E: 9:00am - 12:00pm 

Investigating the Realities Of Outcome-Based Payment Models

  • Nick Li Senior Director, Global/US Market Access Lead , uniQure


Developing a safe, efficacious gene therapy will mean nothing if patients are not able to access them. Several different payment models have been proposed to fit the gene therapy paradigm, and this workshop will delve into the realities of outcome-based payment models and whether they could be a useful solution to several of the market access challenges being faced by the industry.

Attend this workshop to learn about:

  • Building models to reflect potential outcome-based models
  • How well does the theory match to agreements in practice?
  • Exploring different payer perspectives on payment preferences to align

Afternoon Workshops

Workshop F: 1:00pm - 4:00pm 

Determining Product Quality: Establishing Effective Analytics


Defining a comprehensive analytical development strategy is vital to successful gene therapy development. This workshop will examine different assays that can be developed to successfully characterize gene therapy products to ensure programs meet stringent regulatory requirements.

Attend this workshop to learn about:

  • Moving towards high yield and high-quality gene therapy products
  • Exploring assays beyond potency to determine overall product quality
  • How does this match up with current regulatory requirements?

Workshop G: 1:00pm - 4:00pm 

Exploring Progress of Next Generation Gene Therapy Vectors

  • Bernard Kok Senior Scientist, Poseida Therapeutics
  • José Lora Chief Scientific Officer, Intergalactic Therapeutics


Several limitations of current vectors, including immunogenicity concerns and payload size, have the potential to be solved by developing next generation vectors. This workshop will delve into the promise of non-viral vectors, latest viral vector innovations, and overall program implications for progressing novel vectors towards a commercially viable platform.

Attend this workshop to learn about:

  • Gaining insights into the latest innovations for non-viral vectors
  • How are novel viral vectors progressing? AAV & beyond
  • Understanding full implications on program development, including manufacturing and regulatory requirements of next generation vectors

Workshop H: 1:00pm - 4:00pm

Identifying and Establishing Biomarkers for Complex Rare Diseases

  • John Jefferies Professor and Chief, Division of Adult Cardiovascular Diseases , University of Tennessee Health Science Center


Establishing endpoints in gene therapy is always top of mind when designing clinical trials, for which biomarkers can be a highly useful indicator. This workshop will delve into how to better identify appropriate biomarkers in different indications which could be used to define clinical endpoints that demonstrate efficacy and safety.

Attend this workshop to learn about:

  • Validating biomarkers to contribute to defining clinical endpoints
  • Balancing standard biomarkers and novel ones – time versus effectiveness
  • Predicting real-time efficacy with the patient journey at the centre

Workshop I: 1:00pm - 4:00pm

Effectively Navigating Gene Therapy Development in Europe


Gene therapy development is rarely thought of in the context of one region or country, so it’s key to understand the development process on a global level. This workshop will delve into the
specifics of developing gene therapy in the European landscape, including challenges associated with regulatory requirements, GMO guidelines, and interacting on a national level within Europe with different countries.

Attend this workshop to learn about:

  • Interacting with individual countries poses unique challenges
  • Approaching regulatory interactions in the US versus Europe
  • How do market access conversations differ with payers & HTAs

Workshop J: 1:00pm - 4:00pm 

Building a Comprehensive Target Product Profile (TPP)

  • Rebecca Derwin Director, Early Commercial Strategy, Rare Programs , REGENXBIO
  • Cathy Garabedian Senior Director, New Product Planning, Keros Therapeutics


A key cross-functional tool that all companies will need to develop is the Target Product Profile (TPP). TPPs are key to aligning different departments on a common goal, ensuring protocols are being adhered to and understanding the patient and product journey. This workshop will look into when and how to build the TPP, and why it’s important to overall program success.

Attend this workshop to learn about:

  • Standing out in an increasingly crowded market is key to product success
  • Understanding the patient and product journey
  • Beginning the TPP early – what are they key components and what should be done when?