Held by 2 specialist biotech funds
The FDA has approved Kresladi (marnetegragene autotemcel) as the first gene therapy for severe leukocyte adhesion deficiency-I. The approval covers pediatric patients with severe LAD-I due to biallelic ITGB2 variants lacking an HLA-matched sibling donor.
**Signal Note: RTW Investments initiates $62.1M stake in RCKT** RTW's entry into Rocket Pharmaceuticals signals confidence in the company's late-stage pipeline, likely driven by upcoming clinical readouts or regulatory catalysts in their lentiviral gene therapy programs (notably RP-L102 for frangible bone disease and RP-M01 for mucopolysaccharidosis). The $62.1M position (18M shares) represents a meaningful conviction bet from a data-focused investor, typically indicating visible near-term milestones or competitive moat thesis rather than speculative upside.