Children's Hospital of Philadelphia

New Capabilities

Coordinated the PROSpect trial

For more than 30 years, pediatric intensive care doctors have argued over whether turning a desperately ill child face-down, or ventilating them with hundreds of tiny rapid breaths per minute, helps more than standard care. A 424-patient international trial published in The Lancet found neither did.

Updated Nov 2

New Capabilities

As of October 2026: First U.S. site to administer gene therapy for genetic hearing loss

A single injection into the inner ear can now restore hearing in children born deaf. In April 2026 the FDA approved Otarmeni, the first gene therapy for inherited hearing loss, after about 80% of 24 trial participants gained measurable hearing and 42% could pick up whispers.

Updated Oct 3

Rule Changes

As of May 2026: Site of the first personalized CRISPR therapy for Baby KJ

For decades, the FDA required all drugs to prove effectiveness through controlled trials with sufficient sample sizes, a standard that made sense for common diseases but created an impossible barrier for ultra-rare conditions. On February 23, 2026, the FDA issued draft guidance for a new standard—the 'plausible mechanism' framework. Developers of gene-editing and ribonucleic acid (RNA) therapies could win full approval by showing that their treatment targets the root genetic cause, engages it, and improves outcomes compared to the disease's natural course.

Updated May 29