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Genetic disease

This page shows the latest Genetic disease news and features for those working in and with pharma, biotech and healthcare.

EMA recommends revoking marketing authorisation for Novartis’ sickle cell disease drug

EMA recommends revoking marketing authorisation for Novartis’ sickle cell disease drug

Affecting approximately 100, 000 people in the US, SCD is a life-long, incurable genetic disease causing red blood cells to take a distinct crescent shape, which can block blood vessels ... The disease can cause serious health problems, including anaemia,

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  • Gene therapy, gene editing and perspectives on future use in Parkinson’s disease Gene therapy, gene editing and perspectives on future use in Parkinson’s disease

    systems have the potential to edit and correct faulty genes, preventing them from causing disease. ... The application of this technology is clear in monogenic diseases and its potential is now being investigated for idiopathic diseases without a known

  • Rare disease drug development – real-world evidence innovations with historical controls Rare disease drug development – real-world evidence innovations with historical controls

    By Will Maier. Rare disease drug development poses special challenges that can be overcome by using real-world evidence (RWE). ... Disease natural history describes the demographic, genetic, environmental and other variables (eg treatment modalities,

  • EU cross-border healthcare EU cross-border healthcare

    Time can be a precious luxury to someone diagnosed with a rare genetic disease, so getting timely access to new innovative therapies is critical. ... In the last few years, the promise of gene and cell therapies has finally become tangible for certain

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

    In 2021, I expect there to be an increase in digital health solutions on the whole but particularly in rare genetic disease areas. ... In. rare genetic disease research, there may be a way to apply Bayesian models to analyse historic data to accelerate

  • Biopharma and orphan drugs Biopharma and orphan drugs

    Such support, coupled with new approaches to patient recruitment and outreach have made rare disease R&D much more common. ... Price concerns. Another argument in favour of the support given to the orphan drug industry is that rare disease drugs generate

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Latest appointments

  • New hires at Crescendo, Healx and Galecto New hires at Crescendo, Healx and Galecto

    He co-founded the rare disease patient group support charity Findacure with Nick Sireau, the advisor to and the inspiration behind Healx. ... Hall’s experience in drug development includes significant contributions to the repurposing of nitisinone for

  • BMS appoints new commercial leader, Davies joins Eisai and more BMS appoints new commercial leader, Davies joins Eisai and more

    The field of genomic medicine has the potential to revolutionise our approach to rare genetic disease diagnosis and management, as well as in other areas of personalised medicine.”. ... Sobi appoints head of technical operations. Rare disease

  • Abeona Therapeutics names CEO Abeona Therapeutics names CEO

    Carsten Thiel joins the biopharma from Alexion. Rare genetic disease focused biopharma Abeona Therapeutics has named its chief executive officer in the form of Carsten Thiel. ... Steven Rouhandeh, executive chairman of Abeona, said: “As CEO, Carsten

  • Agios appoints Celgene’s Jackie Fouse Agios appoints Celgene’s Jackie Fouse

    She joins the pharma group’s board of directors. Cancer and rare genetic disease focused group Agios Pharmaceuticals has appointed Jackie Fouse to its board of directors as the company prepares

  • Jonathan Fox leaves AstraZeneca for MyoKardia Jonathan Fox leaves AstraZeneca for MyoKardia

    Will lead programme to target genetic heart disease. MyoKardia has appointed former VP, clinical development at AstraZeneca Jonathan Fox to chief medical officer. ... Commenting on his appointment, he highlighted MyoKardia's potential in treating genetic

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