CLSA And BIO Hosted A Briefing “The Transformative Potential Of Gene Therapies”


On Tuesday, January 26th CLSA and BIO hosted a briefing titled, “The Transformative Potential of Gene Therapies” in partnership with the California Select Committee on Biotechnology and the California Rare Disease Caucus. This briefing was moderated by Assemblymember Kevin Mullin and was attended by legislators, legislative staff and members of the rare disease community who are interested in learning more about gene therapies. The briefing focused on the science behind gene therapies, the incredible potential they hold for positively impacting patients’ lives, and how to best promote access to these transformative therapies. Speakers included:

  • Mike Hu, Caregiver/Patient Advocate – shared his experience as a father of two sons with rare diseases – he also discussed the life-saving impacts that therapies have provided for his family.
  • Scott McGoohan, Senior Director of Policy and Alliance Development at Vertex – provided an overview of the gene therapy process, while also touching on the importance of patient access.
  • Mark Trusheim, Strategic Director at MIT – highlighted potential state financing structures to ensure that patients have access to these live saving cures.

This briefing had a total of 18 legislators in attendance. A total of 84 attendees out of 101 that registered were present on the webinar, an amazing turnout!

CLSA will continue to host events to educate the legislature and rare disease community on the science/effects that these therapies have on patient’s lives.