Cystic fibrosis
Vertex is kicking off a Phase I trial assessing VX-522, an mRNA therapy designed to treat the underlying cause of cystic fibrosis lung disease, while SpliSense is targeting a specific mutation.
Sionna hit the ground with a pipeline of first-in-class small molecules designed to fully restore the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein in CF patients.
Vertex has made a point over the past decade to make research and development a priority, investing over 70% of its expenses back into it.
Suliman anticipates filing an Investigational New Drug application with the FDA for the PCD asset this year, and next year filing one for the cystic fibrosis asset.
Avilar Therapeutics officially joined the game with $60 million in seed financing from RA Capital Management.
This first-of-its-kind collaboration will leverage the talent and innovation at companies such as Tessera Therapeutics to advance multiple candidates to human proof of concept.
Mammoth Biosciences just announced a collaboration with Vertex to develop in vivo gene-editing therapeutics for patients with either of two serious, but not-yet-disclosed diseases.
ReCode is aiming to advance its lead candidates for cystic fibrosis and primary ciliary dyskinesia (PCD).
Choosing to exercise its options with a group of research and development partners, the pharma giant will accelerate a promising new C.F. gene therapy.
The data showed that both experimental therapies restored function in the cystic fibrosis transmembrane conductance regulator gene.
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