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sickle cell disease

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

Sanofi exits R&D pact with Alnylam on rare disease

Sanofi exits R&D pact with Alnylam on rare disease

Exit also frees up $700m equity investment. Sanofi is bowing out of a five-year-old R&D alliance with Alnylam on rare disease therapies, but the partners will continue to ... therapies for beta thalassemia and sickle cell disease.

Latest news

  • Blood disorder specialists Imara raises $63m Blood disorder specialists Imara raises $63m

    The company said the investment is reserved for its orally administered sickle cell disease (SCD) candidate known as IMR-687. ... It’s currently in a phase 2 clinical trial, but early data suggests the drug can improve health outcomes and possibly

  • Novartis aims for 10 blockbuster launches by 2020 Novartis aims for 10 blockbuster launches by 2020

    Entresto - Heart failure with preserved ejection fraction (HFpEF). INC280 – non-small cell lung cancer. ... SEG101 – sickle cell disease. The company is looking to be a pioneer in agreeing new payment methods for cell and gene therapies, as well as

  • Novartis gets breakthrough tag from FDA for sickle cell drug Novartis gets breakthrough tag from FDA for sickle cell drug

    Latest pipeline therapy for painful blood disorder. A monthly antibody drug to treat sickle cell disease, developed by Novartis, has won a coveted breakthrough designation from the FDA as it heads ... Crizanlizumab is just one of a series of new dugs

  • AZ and CRUK launch Cambridge genomics centre AZ and CRUK launch Cambridge genomics centre

    disease - and how this knowledge can result in faster, more accurate drug discovery. ... While CRISPR has attracted huge interest as being used in potential therapies, (such as Vertex and CRISPR Therapeutic's sickle cell disease study) AZ has focused on

  • Novartis: pipeline will generate 60 filings in three years Novartis: pipeline will generate 60 filings in three years

    name. Rebadged as OMB157, Novartis describes the drug as a next generation B-cell depletor with a “potentially favourable safety profile from faster b-cell repletion and preserved immunity, and with ... oral dosing – as well as VEGF inhibitor

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Latest Intelligence

  • Vertex: a vision of treating all CF patients worldwide draws closer Vertex: a vision of treating all CF patients worldwide draws closer

    Luckily, that plan B was waiting in the wings: Kalydeco (ivacaftor), the first ever disease-modifying treatment for cystic fibrosis gained approval in 2012. ... with sickle cell disease or beta thalassemia with FDA Fast Track Designation.

  • The Orkambi blame game The Orkambi blame game

    for other diseases, such as sickle cell disease and alpha-1 antitrypsin, which we are working on. ... for our disease.”.

  • 2019: CRISPR and therapeutic gene editing comes of age 2019: CRISPR and therapeutic gene editing comes of age

    The FDA placed the CTX001 programmed in beta thalassemia and a planned trial in sickle cell disease on a clinical hold shortly afterwards, but relaxed that last October. ... consequences – for example if it led to the expression of a disease gene.

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

    While previous read-outs had prepared the ground for this remarkable result, a similar success in sickle cell disease also unveiled at EHA came as a very welcome surprise for the ... sickle cell disease.

  • Pharma deals continue to slide Pharma deals continue to slide

    Gevokizumab plus IP for use of IL-1 beta targeting antibodies for cardiovascular disease. ... 425 (upfront 80). Calimmune (US). CSL (AU). Company acquisition. Preclinical haematopoietic stem cell therapy for sickle cell disease.

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Latest appointments

  • Cellerant appoints William Reed as VP clinical development Cellerant appoints William Reed as VP clinical development

    Will lead development of stem cell-based treatments for blood conditions and cancer. ... He has previous experience in treatments for blood conditions, spending time as director, clinical research and medical affairs at Cerus, where he worked on a

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