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Drug Development In-Depth Focus 2022
Features in this in-depth focus highlight the opportunities presented by targeted protein degradation therapeutics, as well as the breakthroughs in developing treatments for sickle cell disease.
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sickle cell disease
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Experimental gene therapy reverses sickle cell disease for three years
The gene therapy corrected the shape of some sickle cell patients' red blood cells and eliminated episodes of severe pain.
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NICE recommends first sickle cell disease treatment in 20 years
Crizanlizumab recommended as a treatment option for preventing recurrent sickle cell crises for people aged 16 and over in the UK.
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WHO publishes recommendations on human genome editing
To promote the safe and ethical use of genome editing for the treatment of disease and genetic disorders WHO has published the first global recommendations.
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Oxbryta® significantly improves haemoglobin levels in paediatric sickle cell disease patients
Data shows treating sickle cell disease patients aged four to 11 years with Oxbryta® (voxelotor) results in increases in haemoglobin associated with improved clinical status.
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CRISPR therapies could be one of the most transformative in human history, says GlobalData
Adam Pearson, Senior Oncology Analyst at GlobalData, comments on the development of CRISPR-based therapies and outlines potential opportunities and challenges in the space.
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Gates Foundation to fund Novartis’ development of a sickle cell disease gene therapy
The Gates Foundation will fund Novartis’ discovery and development of a gene therapy for sickle cell disease that is accessible for low- and middle-income countries.
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DECODR app identifies unintentional DNA mutations from CRISPR gene therapies
The DECODR app was developed to accelerate the development of CRISPR gene therapies, producing similar data as deep sequencing processes in a shorter timeframe and at less cost.
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EMA accepts MAA for Oxbryta to treat haemolytic anaemia in sickle cell disease
The EMA has accepted Global Blood Therapeutics' marketing authorisation application for Oxbryta (voxelotor) to treat haemolytic anaemia in sickle cell disease.
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EMA recommends 11 new medicines for approval following July meeting
Dapivirine Vaginal Ring (dapivirine) and Blenrep (belantamab mafodotin) have been recommended for approval by the EMA's CHMP, along with nine other medicines.
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PhRMA report reveals nearly 600 drugs in US paediatric pipeline
New research from PhRMA found that in the US, there are almost 600 paediatric medicines in development in over 2,000 clinical trials.
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Therapy approved for patients with sickle cell disease complication
Adakveo has been approved by the FDA to reduce the frequency of vaso-occlusive crisis, a painful complication of sickle cell disease.
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‘Revolutionary’ liquid sickle cell treatment has been unveiled
A new liquid formulation of hydroxycarbamide to treat sickle cell disease will “enable doctors to personalise doses in children".
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Sickle cell drug wins Priority Review by the FDA
The US Food and Drug Administration (FDA) has given an investigational sickle cell medicine, crizanlizumab (SEG101), Priority Review.
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Novartis’ Phase II antibody trial reduces frequency of sickle cell pain crises
6 December 2016 | By Niamh Louise Marriott, Digital Editor
Results from the Phase II study show that SEG101 an anti-P-selectin antibody, reduced the median annual rate of sickle cell-related pain crises by 45.3%...
