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gene therapy

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

FDA lifts hold on Vertex and CRISPR Therapeutics sickle cell therapy

FDA lifts hold on Vertex and CRISPR Therapeutics sickle cell therapy

CTX001 is an investigational, autologous, gene-edited haematopoietic stem cell therapy for patients suffering from severe haemoglobinopathies. ... It already has a rival gene therapy ahead of it: Bluebird’s LentiGlobin has just been submitted to the

Latest news

  • EMA to review Bluebird’s blood disorder gene therapy EMA to review Bluebird’s blood disorder gene therapy

    EMA to review Bluebird’ s blood disorder gene therapy. LentiGlobin could free patients of regular blood transfusions. ... although this won't produce the potentially curative results seen with Bluebird's gene therapy.

  • Orchard plans IPO as gene therapy pipeline advances Orchard plans IPO as gene therapy pipeline advances

    Orchard plans IPO as gene therapy pipeline advances. UK biotech seeks funds for expanded rare disease portfolio. ... an alternative gene therapy, such as our OTL-101 product candidate, is commercially available.”.

  • Leadiant gets FDA OK for bubble boy disease drug Leadiant gets FDA OK for bubble boy disease drug

    patients. Since 2016, ADA-SCID patients in Europe have been able to get access to a one-shot gene therapy that can replace the defective gene coding for ADA – GlaxoSmithKline’s ... Meanwhile, it is also developing another gene therapy – OTL-101 –

  • Roche drug hits the mark in spinal muscular atrophy studies Roche drug hits the mark in spinal muscular atrophy studies

    patients. The commercial potential of both Spinraza and risdiplam could however be short-lived if a gene therapy for SMA – which aims to treat the disease by the one-off administration ... Earlier this year, US biotech AveXis revealed data for its gene

  • New chair for Europe’s key medicines committee New chair for Europe’s key medicines committee

    Like other major regulators, such as the US FDA, the EMA is updating its structures and review processes as new technologies such as gene and cell therapy emerge from industry pipelines. ... The CHMP has just recommended Luxturna, Spark's gene therapy,

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Latest Intelligence

  • Bluebird Bio: on the cusp of a gene therapy revolution Bluebird Bio: on the cusp of a gene therapy revolution

    drugs, Novartis’Kymriah and Gilead/Kite’s Yescarta and Spark Therapeutics’gene therapy Luxturna all reaching the market last year. ... Prasad says Bluebird’s continued focus on improving its product is building confidence in it and the broader

  • The NHS and ABPI at 70: inching closer to the triple win The NHS and ABPI at 70: inching closer to the triple win

    Meanwhile, the pharma industry is hitting new heights in drug innovation, including cures for diseases such as hepatitis C, haemophilia and huge progress in cancer and cell and gene therapy - but ... This, and near-perpetual reform, continues to confound

  • Leo has its eyes on the prize Leo has its eyes on the prize

    Niels Ryber Finsen received the accolade for his pioneering ultraviolet light therapy in 1903 and experts in the field believe it is time the spotlight was once again turned on the ... The Leo surge will include gene therapy and the flexibility to push

  • Health on instalment Health on instalment

    Standing ready. If all goes to plan, gene therapy, a loose umbrella term for modalities that alter genes to treat or prevent disease, stands at the threshold of enormous clinical success. ... But wait, there’s more: well established and respected

  • The rise of real-world evidence The rise of real-world evidence

    Novartis secured such an outcome-based model for its CAR-T therapy Kymriah. ... Spark Therapeutics has a similar arrangement for its gene therapy. These agreements make real-world efficacy a requirement for full payment for a drug.

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Latest appointments

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Latest from PMHub

  • The gene therapy revolution

    The gene therapy revolution. Gene therapies are bringing new hope for many people with diseases caused by genetic disorders. ... https://www.technologyreview.com/s/602113/gene-therapy-cure-has-money-back-guarantee/ (accessed September 2018). Marian Byrt.

  • Training the next generation of biotech leaders

    Training the next generation of biotech leaders. Cell and gene therapy is no longer science fiction. ... As a result, he is at the forefront of inspiring the next generation of scientists to make breakthroughs in cell and gene therapy.

  • AMICULUM boosts global healthcare communications offering with a new specialist agency, Seques®

    AMICULUM boosts global healthcare communications offering with a new specialist agency, Seques. AMICULUM is entering its 18th year of growth as an independent healthcare communications and consulting business. Today we are delighted to announce the

  • Will gene therapies finally be commercially viable to pharma?

    Will gene therapies finally be commercially viable to pharma? Our latest Free Thinking white paper explores the science behind the idea of gene therapy and takes a look at some of ... We review gene therapy’ s chequered past, look at recent

  • Healthcare in the Digital Age

    Exciting developments in immunotherapy and oncology, and huge strides in gene and stem-cell therapy are matched by powerhouse collaborations between academia and industry that point to a golden age for

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