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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.

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

Latest news

  • 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

  • FDA lifts hold on Vertex and CRISPR Therapeutics sickle cell therapy FDA lifts hold on Vertex and CRISPR Therapeutics sickle cell therapy

    Trial could begin by year end. The FDA has lifted the clinical hold on the trial of a CRISPR gene-editing therapy CTX001 in patients with of sickle cell disease (SCD), ... The partners are developing it for both β-thalassemia and sickle cell disease (SCD

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

    LentiGlobin has, however, been shown to reduce or even eliminate entirely the need for blood transfusions, freeing patients from the disease. ... cell disease, the latter being another therapy area that Bluebird is looking to treat with its gene therapy.

  • Allergan builds on gene editing deal Allergan builds on gene editing deal

    That existing deal gives Allergan exclusive access, along with the option to license up to five of Editas’ genome editing programmes for ocular disease, including EDIT-101. ... Back in March, the US regulator halted Vertex Pharma’s planned trial

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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

    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.

  • Deal Watch November 2016 Deal Watch November 2016

    antibody, SelG1, after the completion of a phase II study in patients with sickle cell disease (SCD). ... lead product - SelG1, anti-P-selection mAb for reduction of vaso-occlusive pain crises in patients with sickle cell.

  • Deal Watch October 2015 Deal Watch October 2015

    The collaboration between CRISPR Therapeutics and Vertex will evaluate the use of CRISPR/Cas9 in a number of diseases with defined genetic targets, including cystic fibrosis and sickle cell disease. ... 17, 200. CRISPR Therapeutics/ Vertex Pharmaceuticals

  • Pharma deals during August 2014 Pharma deals during August 2014

    It also has potential utility in treating systemic sclerosis-related Interstitial Lung Disease. ... It is currently being investigated as the sole stem cell source in a phase I/II study to treat haematological malignancies such as leukaemia and lymphoma

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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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