Vertex Pharmaceuticals
NEWS
The regulator’s advisory committee on Tuesday found Vertex Pharmaceuticals and CRISPR Therapeutics’ off-target analysis for its sickle cell disease candidate exa-cel to be sufficient.
In a briefing document for Tuesday’s advisory committee meeting, the FDA raised concerns about the potential off-target effects of Vertex Pharmaceuticals and CRISPR Therapeutics’ investigational gene edited therapy.
New Phase I/II trial results show that one more type 1 diabetes patient achieved insulin independence after treatment with Vertex Pharmaceuticals’ investigational stem cell therapy VX-880.
While some biopharma companies seek an effective and non-addictive alternative to opioids, others are looking to make opioids themselves safer.
The biopharma company reported sales of $2.24 billion in the second quarter for the cystic fibrosis treatment, beating analysts’ estimate, after it won FDA approval in children ages two to five in April.
The FDA recently approved the first cellular therapy for Type 1 diabetes and others may not be far behind. But experts say challenges still exist to the widespread application of these treatments.
Eli Lilly announced Thursday it will acquire former collaborative partner Sigilon Therapeutics to deepen its diabetic foothold with a potentially functional cure for Type 1.
After a series of milestone approvals in the first half of 2023, the FDA is slated to decide on four more firsts before the year’s end.
A year after treatment, Vertex’s VX-880 has eliminated the need for insulin injections in two Type 1 diabetes patients in a Phase I/II clinical trial of its investigational stem cell-based therapy.
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