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# Complete Response on Rocket Gene Therapy
- URL: https://www.fdaweb.com/complete-response-on-rocket-gene-therapy/
- Published: 2024-06-28T12:00:00.000Z
- Updated: 2026-09-14T14:34:31.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5157275

FDA has issued Rocket Pharmaceuticals a complete response letter on its BLA for Kresladi (marnetegragene autotemcel; marne-cel), a lentiviral vector-based gene therapy for treating severe leukocyte adhesion deficiency-I (LAD-I). According to the company, the agency letter requested “limited additional” chemistry manufacturing and controls (CMC) information to complete its review.

Rocket says it recently met with CBER senior leaders to align on the limited scope of additional CMC information needed to support the gene therapy’s approval as quickly as possible. “It is reassuring to have the FDA as a close collaborator who understands the high unmet medical need, clear clinical benefit and importance of timely patient access,” it says.

The submission is based on data from a global Phase 1/2 study that demonstrated 100% overall survival at 12 months post-infusion (and for the entire duration of follow-up) for all nine LAD-I patients with 18 to 42 months of available follow-up, Rocket Pharma says. “Data also showed large decreases compared with pre-treatment history in the incidences of significant infections, combined with evidence of resolution of LAD-I-related skin lesions and restoration of wound repair capabilities,” it adds.

LAD-I is described as a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18\. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections.