Episode 37: Let’s Talk About Gene Therapy

<p><strong>Episode 37: Let’s Talk About Gene Therapy</strong></p><p>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.</p><p>Episode recorded March 18, 2026.</p><p><br></p><p><strong>SHOW NOTES</strong></p><p>Check out our website and sign up for our quarterly newsletter at <a href="http://www.cdkl5incolor.com" target="_blank" rel="ugc noopener noreferrer"><strong>www.CDKL5inColor.com</strong></a></p><p>Follow us @CDKL5inColor on Instagram, Facebook, LinkedIn, Blue Sky, &amp; YouTube!</p><p>Email us to be on the show! <a href="mailto:CDKL5inColor@gmail.com" target="_blank" rel="ugc noopener noreferrer">CDKL5inColor@gmail.com</a></p><p>Thank you to our 2026 Airwave Ally Sponsor - <a href="https://www.ucb-usa.com/Patients"><u>UCB Pharma</u></a>!<strong>LINKS:</strong></p><p>Ultragenyx Gene Therapy</p><p><a href="https://www.ultragenyx.com/our-research/our-gene-therapy-platform/" target="_blank" rel="ugc noopener noreferrer">Ultragenyx Website</a></p><p><a href="https://www.youtube.com/live/xkOxzwX9O-k?si=a93WbxWaTWbfc8Ag" target="_blank" rel="ugc noopener noreferrer">November 2021 Webinar - Gene Therapy: A Conversation With Dr Benke &amp; Ultragenyx</a></p><p><a href="https://www.youtube.com/live/POXGaoy09x4?si=RHg9HMUo9_xftU8p" target="_blank" rel="ugc noopener noreferrer">August 2022 Webinar - CDD Research Updates from Ultragenyx</a></p><p><a href="https://www.cdkl5.com/wp-content/uploads/2023/11/CDD-Community-Update_Q4-2022-MRCC-UX055-00002.pdf" target="_blank" rel="ugc noopener noreferrer">November 2022 - Ultragenyx Community Update Letter</a></p><p><a href="https://youtu.be/K3Y-jvVxDWc?si=fytq8A7EyA8zTlp4" target="_blank" rel="ugc noopener noreferrer">February 2023 Webinar - Ultragenyx Preclinical UXO55 Update</a></p><p><a href="https://www.cdkl5.com/wp-content/uploads/2023/11/CDD-Community-Update-Letter_Q4-2023-MRCM-UX055-00019_APPROVED.pdf" target="_blank" rel="ugc noopener noreferrer">November 2023 - Ultragenyx Community Update Letter</a></p><p><a href="https://www.cdkl5.com/wp-content/uploads/2024/04/UX055-Community-Update-April-2024-MRCM-UX055-00021_FINAL-MRC-APPROVED.pdf" target="_blank" rel="ugc noopener noreferrer">April 2024 - Ultragenyx Community Update Letter</a></p><p><a href="https://www.facebook.com/share/p/1J1aFtf8mX/" target="_blank" rel="ugc noopener noreferrer">September 2025 Facebook Post - PAG Community Letter re: Ultragenyx</a></p><p><br></p><p>Elaaj Bio Gene Therapy</p><p><a href="https://www.elaajbio.com/" target="_blank" rel="ugc noopener noreferrer">Elaaj Bio Website</a></p><p><a href="https://youtu.be/r11s9tUHEcA?si=kD-G459aiv-Wk7HB" target="_blank" rel="ugc noopener noreferrer">February 2026 Webinar: Update on ELJ-101: A gene therapy for CDD</a></p><p><br></p><p><a href="https://www.draccon.com/dracaena-report/2025forum" target="_blank" rel="ugc noopener noreferrer">CDKL5 Forum 2025 Summary (includes links to prior year summaries)</a></p><p><a href="https://www.cdkl5.com/wp-content/uploads/2023/05/CDD-VoP-REPORT.pdf" target="_blank" rel="ugc noopener noreferrer">⁠Patient-Focused Drug Development (PFDD) meeting with the FDA &quot;Voice of the Patient Report&quot;⁠</a></p><p><br></p><p>CDKL5 Gene Therapy (Jainu Jogani)</p><p><a href="http://www.cdkl5genetherapy.com" target="_blank" rel="ugc noopener