* . *
  • About
  • Advertise
  • Privacy & Policy
  • Contact
Friday, July 4, 2025
Earth-News
  • Home
  • Business
  • Entertainment
    MAY HER SOUL REST IN PEACE 🙏 Veteran entertainment columnist and talent manager Lolit Solis has passed away. She was 78 years old. https://tinyurl.com/6kumarkx | LatestChika.com – Facebook

    Beloved Entertainment Icon Lolit Solis Passes Away at 78 – A Life Remembered with Love and Respect 🙏

    Neil Young Plays Rare Full-Band ‘Ambulance Blues’ With The Chrome Hearts – Yahoo

    Neil Young Stuns Fans with Rare Full-Band Performance of ‘Ambulance Blues’ Alongside The Chrome Hearts

    BTS Announce Their Big Return and Yes, They Already Have Some Major Plans in the Works – Yahoo

    BTS Announce Their Big Return and Yes, They Already Have Some Major Plans in the Works – Yahoo

    Nantucket Dance Festival opens July 8 – The Inquirer and Mirror

    Nantucket Dance Festival Launches with Thrilling Performances Beginning July 8

    A Secret Society, Ritualistic Killings, and a Century-Old Curse Netflix and YRF Entertainment’s ‘Mandala Murders’ Premieres July 25 – About Netflix

    A Secret Society, Ritualistic Killings, and a Century-Old Curse: Dive into the Chilling World of ‘Mandala Murders’ Premiering July 25

    Susquehanna Raises Penn Entertainment Inc. (PENN) Price Target. – Yahoo Finance

    Susquehanna Raises Price Target for Penn Entertainment Inc. (PENN)

  • General
  • Health
  • News

    Cracking the Code: Why China’s Economic Challenges Aren’t Shaking Markets, Unlike America’s” – Bloomberg

    Trump’s Narrow Window to Spread the Truth About Harris

    Trump’s Narrow Window to Spread the Truth About Harris

    Israel-Gaza war live updates: Hamas leader Ismail Haniyeh assassinated in Iran, group says

    Israel-Gaza war live updates: Hamas leader Ismail Haniyeh assassinated in Iran, group says

    PAP Boss to Niger Delta Youths, Stay Away from the Protest

    PAP Boss to Niger Delta Youths, Stay Away from the Protest

    Court Restricts Protests In Lagos To Freedom, Peace Park

    Court Restricts Protests In Lagos To Freedom, Peace Park

    Fans React to Jazz Jennings’ Inspiring Weight Loss Journey

    Fans React to Jazz Jennings’ Inspiring Weight Loss Journey

    Trending Tags

    • Trump Inauguration
    • United Stated
    • White House
    • Market Stories
    • Election Results
  • Science
  • Sports
  • Technology
    LG Innotek CEO Moon Hyuksoo: “Our Next-gen Substrate Technology Will Change the Industry Paradigm” – TechPowerUp

    LG Innotek CEO Moon Hyuksoo: “Our Next-Gen Substrate Technology Will Revolutionize the Industry” Revolutionizing the Future: LG Innotek’s CEO Unveils Game-Changing Next-Gen Substrate Technology

    Inspira Technologies Secures Landmark $22.5M Deal: Major Revenue Breakthrough After FDA Clearance – Stock Titan

    Inspira Technologies Secures Landmark $22.5M Deal: Major Revenue Breakthrough After FDA Clearance – Stock Titan

    Meiwu Technology Company Limited and Shenzhen Zhinuo – GlobeNewswire

    Meiwu Technology Company Limited and Shenzhen Zhinuo – GlobeNewswire

    Owls inspire new revolutionary noise reduction technology – KTEN

    Owls inspire new revolutionary noise reduction technology – KTEN

    New center coming to Mizzou will focus on energy research and technology – Columbia Missourian

    Mizzou Launches Innovative New Center Dedicated to Energy Research and Technology

    Mirrors in space and underwater curtains: can technology buy us enough time to save the Arctic ice caps? – The Guardian

    Can Technology Like Space Mirrors and Underwater Curtains Buy Us Time to Save the Arctic Ice Caps?

    Trending Tags

    • Nintendo Switch
    • CES 2017
    • Playstation 4 Pro
    • Mark Zuckerberg
No Result
View All Result
  • Home
  • Business
  • Entertainment
    MAY HER SOUL REST IN PEACE 🙏 Veteran entertainment columnist and talent manager Lolit Solis has passed away. She was 78 years old. https://tinyurl.com/6kumarkx | LatestChika.com – Facebook

    Beloved Entertainment Icon Lolit Solis Passes Away at 78 – A Life Remembered with Love and Respect 🙏

    Neil Young Plays Rare Full-Band ‘Ambulance Blues’ With The Chrome Hearts – Yahoo

    Neil Young Stuns Fans with Rare Full-Band Performance of ‘Ambulance Blues’ Alongside The Chrome Hearts

    BTS Announce Their Big Return and Yes, They Already Have Some Major Plans in the Works – Yahoo

    BTS Announce Their Big Return and Yes, They Already Have Some Major Plans in the Works – Yahoo

    Nantucket Dance Festival opens July 8 – The Inquirer and Mirror

    Nantucket Dance Festival Launches with Thrilling Performances Beginning July 8

    A Secret Society, Ritualistic Killings, and a Century-Old Curse Netflix and YRF Entertainment’s ‘Mandala Murders’ Premieres July 25 – About Netflix

    A Secret Society, Ritualistic Killings, and a Century-Old Curse: Dive into the Chilling World of ‘Mandala Murders’ Premiering July 25

    Susquehanna Raises Penn Entertainment Inc. (PENN) Price Target. – Yahoo Finance

    Susquehanna Raises Price Target for Penn Entertainment Inc. (PENN)

  • General
  • Health
  • News

    Cracking the Code: Why China’s Economic Challenges Aren’t Shaking Markets, Unlike America’s” – Bloomberg

    Trump’s Narrow Window to Spread the Truth About Harris

    Trump’s Narrow Window to Spread the Truth About Harris

    Israel-Gaza war live updates: Hamas leader Ismail Haniyeh assassinated in Iran, group says

    Israel-Gaza war live updates: Hamas leader Ismail Haniyeh assassinated in Iran, group says

    PAP Boss to Niger Delta Youths, Stay Away from the Protest

    PAP Boss to Niger Delta Youths, Stay Away from the Protest

    Court Restricts Protests In Lagos To Freedom, Peace Park

    Court Restricts Protests In Lagos To Freedom, Peace Park

    Fans React to Jazz Jennings’ Inspiring Weight Loss Journey

    Fans React to Jazz Jennings’ Inspiring Weight Loss Journey

    Trending Tags

    • Trump Inauguration
    • United Stated
    • White House
    • Market Stories
    • Election Results
  • Science
  • Sports
  • Technology
    LG Innotek CEO Moon Hyuksoo: “Our Next-gen Substrate Technology Will Change the Industry Paradigm” – TechPowerUp

    LG Innotek CEO Moon Hyuksoo: “Our Next-Gen Substrate Technology Will Revolutionize the Industry” Revolutionizing the Future: LG Innotek’s CEO Unveils Game-Changing Next-Gen Substrate Technology

    Inspira Technologies Secures Landmark $22.5M Deal: Major Revenue Breakthrough After FDA Clearance – Stock Titan

    Inspira Technologies Secures Landmark $22.5M Deal: Major Revenue Breakthrough After FDA Clearance – Stock Titan

    Meiwu Technology Company Limited and Shenzhen Zhinuo – GlobeNewswire

    Meiwu Technology Company Limited and Shenzhen Zhinuo – GlobeNewswire

    Owls inspire new revolutionary noise reduction technology – KTEN

    Owls inspire new revolutionary noise reduction technology – KTEN

    New center coming to Mizzou will focus on energy research and technology – Columbia Missourian

    Mizzou Launches Innovative New Center Dedicated to Energy Research and Technology

    Mirrors in space and underwater curtains: can technology buy us enough time to save the Arctic ice caps? – The Guardian

    Can Technology Like Space Mirrors and Underwater Curtains Buy Us Time to Save the Arctic Ice Caps?

    Trending Tags

    • Nintendo Switch
    • CES 2017
    • Playstation 4 Pro
    • Mark Zuckerberg
No Result
View All Result
Earth-News
No Result
View All Result
Home Science

FDA approves the first gene-editing treatment in the US

December 9, 2023
in Science
FDA approves the first gene-editing treatment in the US
Share on FacebookShare on Twitter

Today, the United States Food and Drug Administration (FDA) approved a historic treatment for sickle cell disease. Casgevy is the first medicine based on CRISPR gene-editing technology. The procedure was approved for use in the United Kingdom in November. 

[Related: The UK becomes the first country to approve CRISPR treatment.]

“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” Doctor Nicole Verdun, director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research, said in a statement. “Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.”

The life-long genetic condition is caused by errors in the genes for the protein hemoglobin. Red blood cells use hemoglobin to carry oxygen through the body. The abnormal hemoglobin makes the blood cells crescent-shaped and hard. The misshapen cells then clump together and block the flow of oxygen to the organs, which causes extreme pain. The cells can then die off early, which leads to anemia.

Sickle cell disease is particularly common among people with Caribbean or African ancestry and affects more than 100,000 Americans. Previously, the only known cure for sickle cell disease was a bone marrow transplant from a donor. These procedures come with the risk of rejection by the patient’s immune system and finding a matching donor is a difficult process. 

How Casgevy works

Casgevy uses CRISPR-Cas9 gene editing technique, which allows scientists to make precise alterations to human DNA. French microbiologist, geneticist, and biochemist Emmanuelle Charpentier and American biochemist Jennifer A. Doudna shared the 2020 Nobel Prize in Chemistry for their work developing the technique.

The procedure uses stem cells taken from a patient’s bone marrow. The cells are then brought into a lab and the genes that are meant to switch on a functioning version of hemoglobin are edited with CRISPR. Patients must then go through a “conditioning treatment.” This can involve taking a drug that suppresses the immune system, radiotherapy, or chemotherapy to get the body ready for an infusion of CRISPR-modified cells back into the body. The new treatment does not come with the risk of graft versus host disease the way that a traditional bone marrow transplant does.

The approval comes on the heels of a clinical trial with 45 patients. Of these, 29 patients have been in the trial long enough for the researchers to gauge how effective Casgevy is. For at least 12 months after treatment, 28 were free of severe pain caused by the disease.

[Related: These organisms have a natural gene-editing system that could be more useful than CRISPR.]

How Lyfgenia works

The FDA also approved another sickle cell treatment called Lyfgenia. The gene therapy from Bluebird Bio. also modifies a patient’s stem cells. Both Lyfgenia and Casgevy require a conditioning treatment that removes cells and bone marrow so that they can be replaced with the modified cells.

According to the FDA,  patient’s blood stem cells are then genetically modified to produce HbAT87Q. This gene-therapy-derived hemoglobin functions similarly to hemoglobin A, or the normal adult hemoglobin produced by those who do not have sickle cell disease. Red blood cells that have HbAT87Q come with a lower risk of sickling and occluding blood flow. The modified stem cells are then delivered to the patient in a one-time, single-dose infusion. 

Lyfgenia’s safety and analysis is based on data from a 24-month study. Of the 32 patients in the study, 88 percent did not have a recurrent vaso-occlusive crises. These hallmarks of the disease occur when blood microcirculation is obstructed by sickled red blood cells, injuring organs and causing extreme pain.  It does have a risk of blood cancer and a black box warning is included in the label. The FDA also advises that patients should have continued monitoring for blood cnacer

Questions for the future

These groundbreaking therapies are expected to be incredibly expensive. A report from a fair prices nonprofit the Institute for Clinical and Economic Review, a nonprofit group that helps evaluate the value of medical tests and treatments found that the therapy could cost about $2 million per patient. It could be out of reach for many families and does not include additional care costs. 

“We really have to make sure that it is accessible,” Cleveland Clinic pediatric hematologist-oncologist Rabi told NBC News. “This could be an equalizer for people with sickle cell because many patients cannot pursue career options because of the illness.” Hanna previously served on the advisory board for Vertex, who makes Casgevy.

The treatments are also very time consuming. Between the pre-treatment conditioning, treatment itself, and follow up, patients have to spend weeks and possibly months in the hospital.

“I think there will be immediate uptake in some portion of the patients who have more severe disease—frequent pain episodes that cause hospitalization and require opioids for the treatment of pain,” said Boston Children’s Hospital hematologist and oncologist David Williams, told Stat. He added that there could be “bumps in the road” if pharmaceutical companies run into trouble manufacturing these complicated treatments or if the price tag keeps it out of reach for many patients. 

The long term effects are also still not known. The FDA says that patients who received Casgevy or Lyfgenia will be followed in a long-term study to evaluate safety and effectiveness.

Laura Baisas

>>> Read full article>>>
Copyright for syndicated content belongs to the linked Source : Popular Science – https://www.popsci.com/health/fda-crispr-sickle-cell-disease/

Tags: ApprovesFirstscience
Previous Post

Leaning into the thrills: Audio-Technica microphones and the dynamic soundscapes of MotoGP

Next Post

Bacterial ‘blood’ could heal cracks in concrete

How UMich computer science students are navigating a shifting job market – The Michigan Daily

How UMich computer science students are navigating a shifting job market – The Michigan Daily

July 4, 2025
Genoa Central Junior High Student Places in the 2025 Soybean Science Challenge – TXK Today

Genoa Central Junior High Student Excels in 2025 Soybean Science Challenge

July 4, 2025
Maison & Objet, the Paris-based home and lifestyle trade show, announces leadership change – FashionNetwork India

Maison & Objet Reveals Dynamic New Leadership to Transform the Future of Home and Lifestyle

July 4, 2025
World’s biggest climate fund ramps up investment plans – Reuters

World’s Largest Climate Fund Accelerates Ambitious Investment Plans

July 4, 2025
US economy ‘on wobbly footing’: Why Wall Street strategists are cautious about stock market’s recent records – Yahoo Finance

US Economy on Shaky Ground: Why Wall Street Strategists Are Cautious Despite Stock Market Records

July 4, 2025
MAY HER SOUL REST IN PEACE 🙏 Veteran entertainment columnist and talent manager Lolit Solis has passed away. She was 78 years old. https://tinyurl.com/6kumarkx | LatestChika.com – Facebook

Beloved Entertainment Icon Lolit Solis Passes Away at 78 – A Life Remembered with Love and Respect 🙏

July 4, 2025
Supreme Court declines to hear case challenging parental consent for abortion – CNN

Supreme Court declines to hear case challenging parental consent for abortion – CNN

July 4, 2025
LG Innotek CEO Moon Hyuksoo: “Our Next-gen Substrate Technology Will Change the Industry Paradigm” – TechPowerUp

LG Innotek CEO Moon Hyuksoo: “Our Next-Gen Substrate Technology Will Revolutionize the Industry” Revolutionizing the Future: LG Innotek’s CEO Unveils Game-Changing Next-Gen Substrate Technology

July 3, 2025
Diego Luna strikes shaky USMNT to win over Guatemala, and into Gold Cup final – Yahoo Sports

Diego Luna Propels USMNT to Thrilling Win Over Guatemala, Punching Ticket to Gold Cup Final

July 3, 2025
Multiple hunting displays in wild broadclub cuttlefish – How – 2025 – Ecology – ESA Journals

Mind-Blowing Hunting Tactics Uncovered in Wild Broadclub Cuttlefish

July 3, 2025

Categories

Archives

July 2025
MTWTFSS
 123456
78910111213
14151617181920
21222324252627
28293031 
« Jun    
Earth-News.info

The Earth News is an independent English-language daily published Website from all around the World News

Browse by Category

  • Business (20,132)
  • Ecology (703)
  • Economy (730)
  • Entertainment (21,618)
  • General (15,699)
  • Health (9,768)
  • Lifestyle (734)
  • News (22,149)
  • People (730)
  • Politics (737)
  • Science (15,947)
  • Sports (21,227)
  • Technology (15,713)
  • World (710)

Recent News

How UMich computer science students are navigating a shifting job market – The Michigan Daily

How UMich computer science students are navigating a shifting job market – The Michigan Daily

July 4, 2025
Genoa Central Junior High Student Places in the 2025 Soybean Science Challenge – TXK Today

Genoa Central Junior High Student Excels in 2025 Soybean Science Challenge

July 4, 2025
  • About
  • Advertise
  • Privacy & Policy
  • Contact

© 2023 earth-news.info

No Result
View All Result

© 2023 earth-news.info

No Result
View All Result

© 2023 earth-news.info

Go to mobile version