CHILDREN'S HOSPITAL OF PHILADELPHIA

The FDA's Division of Cardiology and Nephrology rejected elamipretide's new drug application on May 29, 2025, despite reported improvements in patients with rare mitochondrial diseases.

Researchers from the University of Pennsylvania, including the School of Veterinary Medicine, Perelman School of Medicine, and Children's Hospital of Philadelphia, have developed modified mRNA vaccines incorporating cytokine IL-12 that significantly enhance CD8+ T cell responses.

Four members of the CDC's Advisory Committee on Immunization Practices have received termination notices as special government employees, raising concerns about potential political interference in vaccine policy oversight.

Pharmacists are uniquely positioned to lead the implementation of advanced cell and gene therapies, serving as both clinical and operational experts in health systems managing these complex treatments.

Researchers at Children's Hospital of Philadelphia have engineered new adeno-associated viral (AAV) vectors that can target brain cells at significantly lower doses than current therapies, potentially improving safety and reducing costs.

• Acuitas Therapeutics, Children's Hospital of Philadelphia, and University of Pennsylvania successfully delivered the world's first personalized LNP-delivered CRISPR gene-editing therapy to an infant with urea cycle disorder, with no adverse events reported. • The groundbreaking therapy was developed, manufactured, and delivered in just six months, establishing a new model for rapid development of personalized gene therapies through cross-functional partnerships. • Acuitas also presented advances in targeted LNP delivery, including DARPin-conjugated formulations achieving up to 98% binding and 90% expression in human CD8+ T cells, expanding therapeutic applications beyond the liver.

The world's first patient has been successfully treated with personalized CRISPR gene editing therapy at Children's Hospital of Philadelphia, marking a historic milestone in precision medicine.

Aldevron and Integrated DNA Technologies have manufactured the world's first personalized CRISPR gene editing therapy for an infant with urea cycle disorder, completing the process in just six months—three times faster than standard timelines.

Doctors at Children's Hospital of Philadelphia successfully treated a baby with severe CPS1 deficiency using a personalized CRISPR base-editing therapy, marking a first-of-its-kind approach for this rare metabolic disorder.

Researchers at Children's Hospital of Philadelphia have successfully treated an infant with a severe metabolic disorder using a customized gene editing therapy, as reported at ASGCT 2025.