Pre-Conference Workshops - Monday March 7, 2022

Morning Workshops

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

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 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?