Guess the Therapy Answer May 8, 2026
What was the first lentiviral vector-based gene therapy to be approved by the FDA for treating pediatric patients with severe Leukocyte Adhesion Deficiency Type I (LAD-I)?
Answer: marnetegragene autotemcel (marketed as Kresladi).
See below for further reading on Kresladi:
April 28, 2026 — Rocket Pharmaceuticals has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $180 million. The PRV was awarded following the FDA's accelerated approval of Kresladi, the company's gene therapy for LAD-I. Proceeds from the sale, which the company characterizes as nondilutive capital, are intended to support advancement of Rocket's clinical-stage cardiovascular gene therapy programs in Danon disease, PKP2-associated arrhythmogenic cardiomyopathy, and BAG3-associated dilated cardiomyopathy, with cash runway now projected to extend into the second quarter of 2028.
“The monetization of our PRV, following the FDA approval of Kresladi, provides meaningful nondilutive capital and extends our cash runway into the second quarter of 2028,” Gaurav Shah, MD, the chief executive officer of Rocket Pharmaceuticals, said in a statement. “This strengthens our ability to advance key clinical milestones across our cardiovascular gene therapy pipeline, with all programs on track.”
March 26, 2026 — The FDA has approved Rocket Pharmaceuticals’ Kresladi, a genetically modified autologous hematopoietic stem cell therapy, for pediatric patients with severe LAD-I caused by biallelic variants in ITGB2 who don’t have an available human leukocyte antigen (HLA)-matched sibling donor for allogeneic hematopoietic stem cell transplant.
“Today’s accelerated approval provides a breakthrough treatment for pediatric patients with severe LAD-I—the first FDA-approved gene therapy to treat this disease,” Vinay Prasad, MD, MPH, the chief medical and scientific officer and director of the FDA’s Center for Biologics Evaluation and Research, said in a statement. “The FDA continues to exercise significant regulatory flexibilities as applicable, during both Chemistry, Manufacturing and Control and Clinical review of licensing applications. For rare diseases, the FDA considers small patient populations in clinical trials and all available sources of evidence to advance life-changing treatments while still meeting its rigorous scientific standards.”
