Therapies - Drug Discovery Digest

Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment

Science Daily: Pharmacology News

MAY 15, 2025

A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the rare condition carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after birth, has responded positively to the treatment.

Treatment

Treatment Therapies Disease Research

FDA meeting gives window into gene therapy field’s angst

BioPharma Drive: Drug Pricing

JUNE 6, 2025

Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a regulatory forum Thursday.

Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment

FDA meeting gives window into gene therapy field’s angst

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