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The Latest Breaking News from Sickle Cell Disease

Beam Therapeutics Eyes Sickle Cell BLA as Alpha-1 Gene-Editing Program Advances

Beam Therapeutics (NASDAQ:BEAM) Chief Executive Officer John Evans said the company is advancing its base-editing platform across hematology and liver disease programs, with a potential biologics license application for its sickle cell disease candidate possible as early as the end of this year. Sp

MarketBeat

Agios Pharmaceuticals Touts Strong AQVESME Launch as Sickle Cell Filing Advances

Agios Pharmaceuticals (NASDAQ:AGIO) executives said the company is focused on advancing mitapivat across rare hematologic diseases, highlighting early launch traction in thalassemia and a recently submitted supplemental new drug application for sickle cell disease during a presentation at the 2026 R

MarketBeat

Rise in sickle cell disease prompts NHS call for more Black blood donors

Exclusive: Demand for blood to treat the rare disorder has soared by 132% in 10 years

The Guardian - UK

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$3 million prize goes to duo whose research led to first sickle cell CRISPR therapy

Dr. Swee Lay Thein and Dr. Stuart Orkin won the $3 million Breakthrough Prize in Life Sciences for their work toward a functional cure for the deadly blood disorders sickle cell disease and beta thalassemia.

LiveScience

Fulcrum Therapeutics Stock Rally Ignited By Promising Sickle Cell Results

Fulcrum Therapeutics Inc. (NASDAQ:FULC) stock is surging on Monday, after the company released initial results from the ongoing 20 mg dose cohort of the Phase 1b PIONEER trial of pociredir in sickle cell disease.

Benzinga

Vertex's Gene Therapy Works In Children With Blood Disorders

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) on Saturday presented data from multiple studies demonstrating the clinical benefits of Casgevy (exagamglogene autotemcel) in patients aged 5 years and older with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT).

Benzinga

Why Is Agios Pharmaceuticals Stock Sinking Today?

Agios Pharmaceuticals Inc. (NASDAQ:AGIO) stock plunged on Wednesday after the company released topline results from RISE UP Phase 3 data for mitapivat in sickle cell disease.

Benzinga

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Pfizer's Investigational Drug Flunks Late-Stage For Sickle Cell Disease

Pfizer Inc. (NYSE:PFE) on Friday released results from the Phase 3 THRIVE-131 study evaluating inclacumab, an investigational P-selectin inhibitor, in patients 16 years of age and older with sickle cell disease.

Benzinga

Student wins Hugh Cudlipp Student journalism award for work on sickle cell disease

The prize named after the late Mirror editor is dedicated to recognising outstanding journalistic work by editorial students

Evening Standard

UK sickle cell patients ‘get worse care than sufferers of similar disorders’

Study commissioned by NHS finds that sickle cell disease care lags behind that for conditions such as cystic fibrosis

The Guardian - UK

Sickle cell patients to have quicker and more accessible treatment in England

Government announces £9m funding to make specialist blood machines more widely available across NHS

The Guardian - UK

Nottinghamshire families left unaware of babies’ blood test results in second NHS error

About 300 families may not have been told whether children carry trait for genetic disorders such as sickle cell disease

The Guardian - UK

Graham Serjeant obituary

Physician and researcher who dedicated his life to the study of sickle cell disease and its variants

The Guardian - UK

Dangerous 'crises' in sickle cell disease may be amplified by menstrual cycle

In a new study, researchers found that the inflammation behind painful episodes in sickle cell disease are correlated with the menstrual cycle.

LiveScience

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The Conversation

Sickle cell disease is a genetic disorder that causes lifelong suffering – here’s what you need to know

Right now, approximately 20 billion red blood cells are busy travelling through your blood vessels. They are delivering oxygen to all the different tissues in your body and removing carbon dioxide to be breathed out of your lungs.

The Guardian - UK

‘Groundbreaking’ sickle cell disease treatment approved for NHS use in England

Clinical trials find one-time gene therapy exa-cel offers ‘functional cure’ in 96.6% of patients

The Independent UK

£1.65m gene-editing therapy offers hope of cure for some blood disorder patients

The treatment uses gene-editing tool Crispr, which earned its inventors the Nobel Prize in chemistry in 2020.

The Guardian - UK

NHS error meant hundreds of parents did not know children’s sickle cell status

More than 800 African-Caribbean heritage families in Derbyshire were not sent results of tests on their babies

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Benzinga

Editas Medicine Presents Data From RUBY Trial At ASH 2024, Showing Reni-Cel Achieves 96% VOE-Free Rate, Sustained Increases In Hemoglobin And Fetal Hemoglobin, And Clinically Meaningful Pain And Function Improvements In Severe Sickle Cell Patients; Safety Profile Consistent With Myeloablative Conditioning

Editas Medicine Reports Updated Clinical Data from the RUBY Trial of Reni-cel in Patients with Severe Sickle Cell Disease at the American Society of Hematology (ASH) Annual MeetingGlobe NewswirePoster presentation at ASH

Benzinga

Beam Therapeutics Unveils New Safety, Efficacy Data From Sickle Cell Disease Candidate, Stock Gains

Beam Therapeutics unveiled BEAM-101 trial data at ASH, showing improved outcomes in sickle cell patients and promising preclinical BEAM-103 results.

The Independent UK

Black man with sickle cell disease died ‘naked and alone’ in cell after complaining of breathing difficulties

The 43-year-old was found unresponsive in a cell on the healthcare unit at HMP Pentonville on 17 May 2022

The Independent UK

Health chiefs launch review to tackle ‘poor care’ faced by many young sickle cell disease sufferers

It is estimated that around 300 babies are born in the UK with sickle cell disease each year

The Times of India

Sickle Cell Disease: Symptoms, risks and understanding those living with sickle cell disease

Sickle Cell Disease (SCD) is an inherited blood disorder affecting millions worldwide and significantly burdening India's healthcare system. With over 40,000 newborns estimated to have been born with sickle cell anemia in India in 2010, the country ranks second only to Africa in prevalence. Despite advancements in treatment, many patients,…

The Times of India

Sickle Cell Disease: 5 diet mantras that one must follow

Imagine waking up with the feeling that your body could betray you any time of the day. And, as the day passes, you feel excruciating pain, fatigue, and other debilitating symptoms. While the imagination is harsh, this is a reality to millions of people who are forced to make do…

The Times of India

How sickle cell disease is a huge burden upon India’s public health

Every year, approximately 150,000 to 200,000 children are born with sickle cell disease in India. India ranks second when it comes to this disease burden and about 50-80 percent of the patients struggle to reach the age of five. According to the Indian Journal of Medical Research, India is home…

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