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The Latest Breaking News from Sickle Cell Disease

Sickle cell disease: nearly 50% of patients receive poor care, says global study

Across 10 countries almost half those surveyed say they were treated like drug addicts and more than third believe their race influenced poor care

The Guardian - UK

Recap Of Thursday's Biotech Catalysts - End Of the Day Summary

Recap Of Thursday's Biotech Catalysts - End Of the Day Summary

The U.S. Food and Drug Administration (FDA) has accepted the Menarini Group and Radius Health’s (NASDAQ:RDUS), New Drug Application (NDA) for elacestrant for patients with ER+/HER2- advanced or metastatic breast cancer. The agency has granted Priority Review designation and assigned a target action date of February 17, 2023. Radius Health…

Recap Of Tuesday's Biotech Catalysts - End of The Day Summary

Recap Of Tuesday's Biotech Catalysts - End of The Day Summary

European Commission (EC) has approved AbbVie’s (NYSE:ABBV) lead asset RINVOQ (upadacitinib 45 mg [induction dose] and 15 mg and 30 mg [maintenance doses]) for the treatment of adult patients with moderately to severely active ulcerative colitis. AbbVie shares traded in a range of $150.33 to $153.56 on a day volume…

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There's no cure for sickle cell disease, but spotting it early can improve treatment

Most of the global cases of sickle cell disease are diagnosed in African countries. The Economist Intelligence Unit – the research and analysis division of the Economist media group – estimated that in 2020 the total annual cost of treatment and impact on the economy of sickle cell disease across…

The Conversation

Why Precision BioSciences Shares Are Surging Today

Precision BioSciences Inc (NASDAQ:DTIL) has entered into an exclusive worldwide in vivo gene editing research and development collaboration and license agreement with Novartis AG (NYSE:NVS) Precision will develop a custom ARCUS nuclease that will be designed to insert, in vivo, a therapeutic transgene at a "safe harbor" location in the genome as a potential one-time…

Read Why Forma Therapeutics' Current Price Level Is 'Attractive Entry Point'

During the European Hematology Association (EHA) conference, Forma Therapeutics Holdings Inc (NASDAQ:FMTX) presented updated results from the 12-week open-label extension (OLE) of etavopivat's Phase 1 trial in sickle cell disease. Treatment with etavopivat 400mg QD elicited a >1g/dL hemoglobin (Hb) increase in 73% of the 15 patients and an average maximal Hb increase of…

FDA Sounds Positive On Bluebird's One Gene Therapy But Doubts Another

The FDA signaled in briefing documents released that it may approve Bluebird bio Inc's (NASDAQ:BLUE) beti-cel gene therapy to treat β-thalassemia for those who require regular blood transfusions. The Prescription Drug User Fee Act (PDUFA) goal dates for beti-cel and eli-cel are August 19 and September 16. Bluebird bio's blood disorder treatment demonstrates "clinically meaningful"…

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Why This Analyst Has 'Little Conviction' On CRISPR's Upcoming Trial Data

Why This Analyst Has 'Little Conviction' On CRISPR's Upcoming Trial Data

RBC Capital Markets says it remains on the sidelines given commercial uptake for CRISPR Therapeutics' (NASDAQ: CRSP) sickle cell disease (SCD)/beta-thalassemia (BT) capped by preconditioning and has little conviction in BCMA/CD70 data within weeks. The analysts lowered its price target to $85 from $95 to reflect higher Opex, with a Sector Perform rating. As recently…

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Food and Drug Administration

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Vertex Pharmaceuticals Incorporated

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Sickle Cell Disease

Ulcerative Colitis

Macular Degeneration

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