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Ionis Pharmaceuticals

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MHRA approves TMC Pharma’s Nulibry to treat rare genetic disease MoCD type A

Molybdenum cofactor deficiency is estimated to affect one in 100,000 to 200,000 newborns worldwide

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Ionis shares positive late-stage results for olezarsen in familial chylomicronaemia syndrome

The rare genetic disease is estimated to affect up to 13 people per million in the US

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FDA approves Orchard’s Lenmeldy gene therapy for metachromatic leukodystrophy

The rare genetic disease is estimated to affect one in every 40,000 people in the US

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Sanofi presents positive results for Pompe disease enzyme replacement therapy

The rare genetic disorder is estimated to affect around one in every 40,000 people in the UK

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Regenxbio’s investigational gene therapy shows promise in Hunter syndrome

The rare genetic disease is estimated to affect one in every 100,000 to 170,000 births

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Ionis shares positive phase 3 results for hereditary angioedema drug candidate

More than 20,000 people in the US and Europe are affected by the rare genetic disease

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Genomics England receives government funding for cutting-edge genomics research

£105m will be used to accelerate the diagnosis of rare genetic diseases in newborns

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Ionis reports positive phase 2b results for fesomersen in thrombosis prevention

The company also announced it will regain rights to the antisense medicine from Bayer

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AstraZeneca and Ionis sign $3.6bn deal for eplontersen

The liver-targeted antisense therapy is in phase 3 trials for the treatment of transthyretin amyloidosis, a systemic, progressive and fatal condition

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Rare genetic diseases – what’s in store for 2021?

Why personalised medicine based on our own genomes is the future of healthcare

- PMLiVE

GSK’s Ionis-partnered hepatitis B drug hits the mark in phase 2

Treatment could be transformative for around 260 million people living with disease

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