sickle cell disease

‘Build back better’ cannot mean longer waiting times for change

Global Blood Therapeutics (GBT) UK general manager Nigel Nicholls discusses the impact of COVID-19 for sickle cell patients in the UK. If the COVID-19 pandemic has shown us anything, it has highlighted the extent to which health inequity plagues our society. Although the Government, public and health care system have started to wake up to …

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Joy as sickle cell patients get access to Novartis’ Adakveo after NICE ruling

People living with sickle cell disease (SCD) in England and Wales will be able to get access to treatment with Novartis’ Adakveo, thanks to a special arrangement between the drugmaker and NICE. The decision makes Adakveo (crizanlizumab) the first new treatment for SCD available via the NHS for 20 years, according to the cost-effectiveness agency, …

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Vertex builds in gene editing yet again, with $1.2bn Arbor deal

Vertex Pharma has ramped up its involvement in gene-editing medicines for the third time in a matter of months, agreeing a partnership with CRISPR specialist Arbor Biotechnologies that could be worth up to $1.2 billion. The latest deal allows Vertex to tap into Arbor’s technology platform to develop cell therapies for diseases like type 1 …

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Vertex to pay CRISPR Therapeutics $900M for bigger share of gene-editing alliance

Vertex Pharmaceuticals is amending its collaboration with CRISPR Therapeutics, paying $900 million now for chance to grab a bigger share of a gene-editing therapy’s profits later—if the treatment is approved. Clinical trials evaluating the CRISPR-based therapy are underway in sickle cell disease and beta thalassemia.

For which diseases do broader value elements matter most?

This is the question I–along with co-authors Syvart Dennen, Priti Pednekar , Kelly Birch, Menaka Bhor, Julie Kanter , and Peter Neumann–aim to answer in our new paper in JMCP. The full title is: “For which diseases do broader value elements matter most? An evaluation across 20 ICER evidence reports“. The abstract is below. BACKGROUND: U.S. …

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How do pain crises affect quality of life and productivity among patients with sickle cell disease?

I provide the answer to this question in a recent paper in Current Medical Research & Opinion with co-authors Howard Thom, Edna Keeney, Daisy Gaunt, Lauren Zhao, Menaka Bhor, Avery Rizio, Lanetta Bronté-Hall and Nirmish Shah. The abstract is below. AimSickle cell disease (SCD) is a lifelong blood disorder affecting approximately 100,000 individuals in the …

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Global Blood Therapeutics inks deal for two sickle cell disease drugs from Sanofi

In acquiring the two early-stage Sanofi drugs for sickle cell disease, Global Blood Therapeutics aims to complement Oxbryta, its FDA-approved treatment for the rare blood disorder. Meanwhile, Sanofi can turn its full sickle cell efforts on a gene-editing therapy partnered with Sangamo Therapeutics.

Novartis Collaborates with Bill & Melinda Gates Foundation to Discover and Develop Gene Therapy for Sickle Cell Disease

Shots: The Gates Foundation will provide funding support to discover and develop in vivo gene therapy for SCD. The alliance brings together Novartis drug discovery and gene therapy expertise with Gates’ funding’s The focus of the agreement to address the disparity in access to treatments and to prioritize populations and regions bearing the greatest burden …

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ASH: CRISPR, Vertex’ CTX001 hits the mark in red cell disorders

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting. The data with the CRISPR/Cas9 drug CTX001 is still very early-stage, coming from just seven thalassaemia patients and …

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2nd Gene Therapy for Blood Disorders

2020 has been an exciting year for gene therapies targeting blood disorders. Despite FDA’s recent rejection of BioMarin’s hemophilia gene therapy program, the clinical landscape is progressing quickly with Pfizer and Sangamo kicking off their phase 3 and bluebird bio receiving European approval for their beta-thalassemia candidate. With pipelines advancing and emerging companies joining the …

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