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Breakthrough Gene Therapy for Rare CDKL5 Disorder Unveiled

9/25/2025

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Summary

Story summary

CDKL5 deficiency disorder is a rare disease caused by CDKL5 mutations.
Jainu Jogani of Child’s Cure Genetic Research discussed Reyna at the American Society of Gene & Cell Therapy annual meeting.
The organization is developing an AAV9-based gene therapy and exploring a CDKL2-targeting small molecule.
Families rely on therapies as options remain limited, underscoring the need for faster development.

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Story summary

CDKL5 deficiency disorder is a rare disease caused by CDKL5 mutations.
Jainu Jogani of Child’s Cure Genetic Research discussed Reyna at the American Society of Gene & Cell Therapy annual meeting.
The organization is developing an AAV9-based gene therapy and exploring a CDKL2-targeting small molecule.
Families rely on therapies as options remain limited, underscoring the need for faster development.