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Episode 37: Let’s Talk About Gene Therapy
Episode 37: Let’s Talk About Gene Therapy
Gene therapy is on people’s minds, so let’s talk about it! What is known, what is unknown, and how are we meant to make sense of it all? We know from the quiz that we put out in September that, while most respondents had a decent understanding of the basics of gene therapy as a treatment approach, the majority did not understand the CDKL5 gene therapy landscape nor the average timeline and costs associated with bringing a treatment like this to humans. In this episode, we talk about what we see happening and share our thoughts about these treatments that aim to get at the root cause of CDKL5 deficiency disorder. We also talk about what we can be doing as caregivers to support our loved ones in the here and now while we await the treatments of the future.
Episode recorded March 18, 2026.
SHOW NOTES
Check out our website and sign up for our quarterly newsletter at www.CDKL5inColor.com
Follow us @CDKL5inColor on Instagram, Facebook, LinkedIn, Blue Sky, & YouTube!
Email us to be on the show! [email protected]
Thank you to our 2026 Airwave Ally Sponsor - UCB Pharma!LINKS:
Ultragenyx Gene Therapy
Ultragenyx Website
November 2021 Webinar - Gene Therapy: A Conversation With Dr Benke & Ultragenyx
August 2022 Webinar - CDD Research Updates from Ultragenyx
November 2022 - Ultragenyx Community Update Letter
February 2023 Webinar - Ultragenyx Preclinical UXO55 Update
November 2023 - Ultragenyx Community Update Letter
April 2024 - Ultragenyx Community Update Letter
September 2025 Facebook Post - PAG Community Letter re: Ultragenyx
Elaaj Bio Gene Therapy
Elaaj Bio Website
February 2026 Webinar: Update on ELJ-101: A gene therapy for CDD
CDKL5 Forum 2025 Summary (includes links to prior year summaries)
Patient-Focused Drug Development (PFDD) meeting with the FDA "Voice of the Patient Report"
CDKL5 Gene Therapy (Jainu Jogani)
www.CDKL5GeneTherapy.com
October 2025 Webinar: Gene Therapy for CDKL5: The History, Progress & Next Steps
Resources for Caregivers
American Society for Gene & Cell Therapy Patient Education
Enroll in the International CDKL5 Disorder Database!
Episode 16: Acceptance
DEE-P Connections Webinars
Book: Caring for Children Who Have Severe Neurological Impairment: A Life with Grace by Julie M. Hauer, M.D.
View all episodes
By Marissa BishopEpisode 37: Let’s Talk About Gene Therapy
Gene therapy is on people’s minds, so let’s talk about it! What is known, what is unknown, and how are we meant to make sense of it all? We know from the quiz that we put out in September that, while most respondents had a decent understanding of the basics of gene therapy as a treatment approach, the majority did not understand the CDKL5 gene therapy landscape nor the average timeline and costs associated with bringing a treatment like this to humans. In this episode, we talk about what we see happening and share our thoughts about these treatments that aim to get at the root cause of CDKL5 deficiency disorder. We also talk about what we can be doing as caregivers to support our loved ones in the here and now while we await the treatments of the future.
Episode recorded March 18, 2026.
SHOW NOTES
Check out our website and sign up for our quarterly newsletter at www.CDKL5inColor.com
Follow us @CDKL5inColor on Instagram, Facebook, LinkedIn, Blue Sky, & YouTube!
Email us to be on the show! [email protected]
Thank you to our 2026 Airwave Ally Sponsor - UCB Pharma!LINKS:
Ultragenyx Gene Therapy
Ultragenyx Website
November 2021 Webinar - Gene Therapy: A Conversation With Dr Benke & Ultragenyx
August 2022 Webinar - CDD Research Updates from Ultragenyx
November 2022 - Ultragenyx Community Update Letter
February 2023 Webinar - Ultragenyx Preclinical UXO55 Update
November 2023 - Ultragenyx Community Update Letter
April 2024 - Ultragenyx Community Update Letter
September 2025 Facebook Post - PAG Community Letter re: Ultragenyx
Elaaj Bio Gene Therapy
Elaaj Bio Website
February 2026 Webinar: Update on ELJ-101: A gene therapy for CDD
CDKL5 Forum 2025 Summary (includes links to prior year summaries)
Patient-Focused Drug Development (PFDD) meeting with the FDA "Voice of the Patient Report"
CDKL5 Gene Therapy (Jainu Jogani)
www.CDKL5GeneTherapy.com
October 2025 Webinar: Gene Therapy for CDKL5: The History, Progress & Next Steps
Resources for Caregivers
American Society for Gene & Cell Therapy Patient Education
Enroll in the International CDKL5 Disorder Database!
Episode 16: Acceptance
DEE-P Connections Webinars
Book: Caring for Children Who Have Severe Neurological Impairment: A Life with Grace by Julie M. Hauer, M.D.