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Breakthrough CRISPR Therapy Offers Hope for 'Baby KJ'

10/25/2025

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Summary

Story summary

Mark Wetzel, vice president at Aldevron, describes a rapid, personalized CRISPR therapy for K.J. Muldoon, known as 'Baby KJ'.
K.J. Muldoon has a urea cycle disorder that prevents ammonia elimination and causes severe health risks.
The therapy targets a CPS1 gene mutation and was developed in six months through collaboration among industry partners.
Its success offers hope and could set a precedent for future personalized gene therapies.

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Story summary

Mark Wetzel, vice president at Aldevron, describes a rapid, personalized CRISPR therapy for K.J. Muldoon, known as 'Baby KJ'.
K.J. Muldoon has a urea cycle disorder that prevents ammonia elimination and causes severe health risks.
The therapy targets a CPS1 gene mutation and was developed in six months through collaboration among industry partners.
Its success offers hope and could set a precedent for future personalized gene therapies.