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PTAC/CFTR
Symposium

S11--Nucleotide-Based Therapeutics Across Diverse Populations

Thursday, September 26, 2024
2:30 PM – 4:30 PM ET
Location:157 A-C

Recorded Session

Critical efforts are needed to address the unmet need for people with CF (pwCF) from diverse populations who are ineligible for, intolerant to, or lack access to CFTR modulator therapies. A pipeline of nucleic acid-based therapies (NABTs) aimed at restoring full function of the cystic fibrosis transmembrane (CFTR) protein are in early-stage clinical development and will be focusing innovative clinical trials within this population. To successfully advance this pipeline to ensure that all pwCF can benefit, development efforts must consider clinical trial designs which allow for testing of multiple therapies with smaller population sizes, and recruitment and engagement strategies which decrease barriers to trial participation and build a culture of trust in research across diverse racial, ethnic, gender, socioeconomic and geographic communities. Unique aspects related to long term safety follow-up for these therapies and the progression of clinical development from adult to pediatric populations will be discussed. Planning for the successful regulatory approval of an NABT will need to consider lessons learned from other diseases in the adoption of these therapies across diverse populations and highlight the ongoing challenges to access that must be overcome to ensure all people with CF will benefit from a CFTR directed therapy.

Learning Objectives:

  • Describe the pipeline of nucleic acid-based therapies and the path to ensuring that CFTR directed therapies will benefit all people with CF including those with high unmet need from diverse racial, ethnic, gender, socioeconomic and geographic communities
  • Review the clinical trial designs and recruitment approaches that must be considered to support the development of an NABT and their regulatory approval across diverse populations
  • Utilizing knowledge developed in other disease settings and based on current regulatory guidelines, understand the requirements for long term safety follow up of NABTs, anticipated progression of these therapies from adult to pediatric populations, and the opportunities and challenges with the adoption of these therapies across diverse populations

Presentations:

  • 2:30 PM – 4:30 PM ET
    S11.1- The Final Frontier for All PwCF: NABTs from the Investigator & Community Perspective

    Speaker: Scott C. Bell, MBBS MD (he/him/his) – University of Queensland

    Community Speaker: Steph Hansen (she/her or they/them) – Patient Advocate

  • 2:30 PM – 4:30 PM ET
    S11.2- Clinical Trial Design & Recruitment Considerations to Advance a Pipeline of NABTs

    Speaker: Nicole Mayer Hamblett, PhD (she/her/hers) – University of Washington

  • 2:30 PM – 4:30 PM ET
    S11.3- Forecasting the Future: Long-Term Follow-up & Including Children in NABT Trials

    Speaker: Jane C. Davies, MD MBChB MRCP FMedSci (she/her/hers) – National Heart and Lung Institute, Imperial College London

    Community Speaker: Rena Barrow, CPhT – CVS Health/ CEO and Founder of Two Salty Okes

  • 2:30 PM – 4:30 PM ET
    S11.4- Towards CFTR Directed Therapies for All: From Development to Access

    Speaker: Damian Downey, MD – Wellcome-Wolfson Institute for Experimental Medicine, Queens University Belfast, Northern Ireland