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The Latest Breaking News from Sickle Cell Disease

The World's First CRISPR Gene Therapy Just Got Approved for Children as Young as 2 with Sickle Cell Disease

The FDA on July 1, 2026, expanded Casgevy CRISPR gene therapy to children as young as 2 with sickle cell disease. Here is what families need to know about eligibility and access.

Medical Daily

FDA Expands CRISPR Gene Therapy Casgevy to Children as Young as 2 with Sickle Cell Disease

FDA approved Casgevy CRISPR gene therapy for children as young as 2 with sickle cell disease on July 1, 2026. Here's what families need to know about this milestone.

Medical Daily

FDA drug approval affects healthcare around the world, but political shortcuts could hurt the agency’s international reputation

At the beginning of July 2026, the U.S. Food and Drug Administration approved the first gene therapy for young children with sickle cell disease.

The Conversation

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Medicaid Will Cover Sickle Cell Gene Therapy, but Only When It Actually Works for the Patient

CMS's CGT Access Model now covers Casgevy and Lyfgenia for sickle cell through 33 state Medicaid programs under outcomes-based contracts. Here's what patients need to know.

Medical Daily

A 23-Year-Old Louisiana Man Became the First Person in the Gulf South to Be Functionally Cured of Sickle Cell Disease

Daniel Cressy, 23, is the first person in the Gulf South functionally cured of sickle cell disease using Casgevy CRISPR gene therapy at Manning Family Children's Hospital in New Orleans.

Medical Daily

Louisiana man becomes first in region functionally cured of sickle cell disease

Daniel Cressy, 23, says path leading to completion of curative gene therapy is his ‘greatest blessing’

The Guardian - US

Aflac general counsel: Georgia lawmakers took a crucial step forward on sickle cell disease – but there's more work to be done

A new Georgia law requires Medicaid to keep pace with sickle cell treatments. With World Sickle Cell Day on June 19, here's why every state needs to act now.

Fortune

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India can eradicate sickle cell disease before 2047 through collective efforts: President Murmu

President Droupadi Murmu believes India will eliminate sickle cell disease before the 2047 target. She stressed the need for increased awareness, particularly within tribal communities. The President stated that collective efforts from all states will achieve this national goal. She highlighted that treatment is possible and the disease can be…

The Economic Times

CRISPR Gene Editing Achieves a Functional Cure for Sickle Cell Disease in 96 Percent of Patients — RUBY Trial Results in the New England Journal of Medicine Are Historic

The RUBY Trial (NEJM, April 2026): CRISPR-Cas12a gene editing therapy reni-cel (renizgamglogene autogedtemcel) achieved a functional cure in 27 of 28 (96%) severe sickle cell disease patients, with no painful crises for up to two years after treatment. Led by Dr. Rabi Hanna at Cleveland Clinic.

Medical Daily

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

$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

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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

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.

Evening Standard

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

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The Guardian - UK

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

LiveScience

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.

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

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