Disease and 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

More Evidence Gene Therapy Might Cure Sickle Cell Disease

Drugs.com

JUNE 12, 2023

MONDAY, June 12, 2023 -- A clinical trial that’s attempting to discover a cure for sickle cell disease has found a new gene therapy to be safe and successful in four patients. Two of the patients were treated at Cleveland Clinic Children’s in.

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

More Evidence Gene Therapy Might Cure Sickle Cell Disease

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