Rocket Pharma gets FDA nod for first pediatric gene therapy targeting severe LAD-I
The approval covers pediatric patients without a matched sibling donor for stem cell transplant
The approval covers pediatric patients without a matched sibling donor for stem cell transplant
The therapy works by targeting interleukin-13, a key driver of inflammation behind eczema symptoms such as itching, skin damage, and infection
The milestone moves the company closer to its previously announced $120 million US expansion, with groundbreaking slated for late 2026
The Trust’s shift from single-use compostable cups to a reusable system was driven by the need to reduce environmental impact and control long-term costs
They stressed that while the drug is clinically proven to support weight and blood sugar control, it must be prescribed only after careful patient evaluation and used under strict medical supervision.
The patent protects antiviral compounds developed by the company that inhibit Vacuolar ATPase (V-ATPase) activity in cells, a mechanism involved in viral infection pathways
This linear, covalently closed, double-stranded DNA offers a rapid, cell-free alternative to traditional plasmid DNA, addressing a critical bottleneck in nucleic acid therapeutics development
The study included four independent genetic substudies targeting obesity caused by heterozygous variants in the POMC/PCSK1, LEPR, SRC1 (NCOA1), and SH2B1 genes
The drug also showed a manageable safety profile, with just 6.4% of patients stopping treatment due to side effects. No new safety concerns emerged
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