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Cystic fibrosis

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Vertex to expand autoimmune and inflammatory disease pipeline with $4.9bn Alpine acquisition

The company will gain rights to Alpine’s lead molecule, povetacicept, to treat IgA nephropathy

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EC approves Vertex/CRISPR’s Casgevy gene therapy for inherited blood disorders

There are now more than 8,000 patients who are potentially eligible for treatment with Casgevy

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Vertex shares positive late-stage results for triple combination cystic fibrosis treatment

The company compared the treatment to its current triple cystic fibrosis therapy

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FDA approves Vertex/CRISPR’s Casgevy gene therapy for beta thalassaemia

The companies estimate that approximately 1,000 patients in the US will be eligible for the one-time treatment

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CHMP recommends gene therapy for beta thalassemia and sickle cell disease

If approved, Casgevy will be the only genetic therapy in the EU to treat these conditions

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FDA approves two cell-based gene therapies for sickle cell disease

Vertex/CRISPR and bluebird bio’s gene therapies have been approved to treat patients aged 12 years and older

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MHRA extends licence of Vertex’s cystic fibrosis drugs for young children

Kaftrio and Kalydeco are now approved for use in certain patients aged two to five years

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MHRA authorises world-first gene therapy for two inherited blood disorders

Casgevy is now the first licensed treatment that uses the gene-editing tool CRISPR

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FDA completes meeting for potential genetic therapy to treat sickle cell disease

If approved, exa-cel will be the first genetic therapy available to treat patients with severe sickle cell disease

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Pioneering research collaboration seeks cure for cystic fibrosis

Backed by the non-profit Cystic Fibrosis Foundation and venture capital group Flagship, Pioneering Medicines will identify and develop genetic therapies for CF

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Moderna inks R&D agreements with Vertex and Chiesi

Biotech agrees to separate deals for the development of gene-editing and mRNA therapies

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Vertex’s triple combination CF therapy to become available on the NHS

The drug will immediately become available to thousands of cystic fibrosis patients

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