"So the future is one IV infusion, likely no chemotherapy, and that'll cure our IEIs." — Dr. Nicola Wright

For children born with inborn errors of immunity, bone marrow transplant has long been the closest thing medicine had to a cure. It works — but it comes with chemotherapy, graft-versus-host disease, and a donor search that doesn't always end well. Gene therapy is changing that calculus. Dr. Mariam Hanna speaks with Dr. Nicola Wright, a pediatric hematologist and clinical immunologist at the Alberta Children's Hospital and holder of the Barb Ibbotson Chair of Pediatric Hematology, whose research focuses on developing gene editing platforms for blood and immune disorders.

On this episode, they discuss:

• Why bone marrow transplant is, in Dr. Wright's words, "almost a poor man's gene therapy" and what gene therapy offers instead
• The spectrum of technologies: lentiviral insertion, CRISPR, base editing, and prime editing. What each does, and where each falls short
• Immune reconstitution outcomes across diseases, including over 90% good immune reconstitution in ADA-deficient SCID treated with lentiviral therapy
• How to counsel a family when gene therapy might be an option and why most patients still can't access it
• The "valley of death": why therapies that work in trials are failing to reach patients, and what it will take to cross it
• CAR T-cell therapy in IEI, including a Canadian trial underway for refractory autoimmune disease
• What long-term follow-up looks like and why 15-year post-trial monitoring is now an FDA requirement

The science is outpacing the infrastructure. Dr. Wright's vision of shipping cells instead of patients, in vivo delivery via lipid nanoparticle, no chemotherapy required isn't speculative. The runway is being built. The plane is already flying.

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The Allergist is produced for CSACI by PodCraft Productions