Al Mayadeen English

  • Ar
  • Es
  • x
Al Mayadeen English

Slogan

  • News
    • Politics
    • Economy
    • Sports
    • Arts&Culture
    • Health
    • Miscellaneous
    • Technology
    • Environment
  • Articles
    • Opinion
    • Analysis
    • Blog
    • Features
  • Videos
    • NewsFeed
    • Video Features
    • Explainers
    • TV
    • Digital Series
  • Infographs
  • In Pictures
  • • LIVE
News
  • Politics
  • Economy
  • Sports
  • Arts&Culture
  • Health
  • Miscellaneous
  • Technology
  • Environment
Articles
  • Opinion
  • Analysis
  • Blog
  • Features
Videos
  • NewsFeed
  • Video Features
  • Explainers
  • TV
  • Digital Series
Infographs
In Pictures
  • Africa
  • Asia
  • Asia-Pacific
  • Europe
  • Latin America
  • MENA
  • Palestine
  • US & Canada
BREAKING
Trump claims US significantly ahead of Russia, China, in space
US National Guard female member injured in Washington attack has died: Trump
Israeli media outlet reports one injury in a shooting targeting a bus at the Tunnels checkpoint in al-Quds
Sayyed Khamenei: In the 12-day war, the Iranian people defeated the Americans and the Zionists without a doubt
Sayyed Khamenei: We must enhance mobilization and pass on its concept to future generations, and I have always advised all government agencies to operate in a mobilization-oriented manner
Sayyed Khamenei: The scientists who were martyred in the 12-day aggression were not involved in the mobilization, but they embodied the true image of being part of the mobilization move
Sayyed Khamenei: The IRGC may be the official side concerned with mobilization, but any zealous group that wants to work for the sake of this country is part of this mobilization
Sayyed Khamenei: The phenomenon of resistance will continue and grow, and the oppressed people of the world will feel that someone supports them and that there is a power that amplifies their voice
Addressing the Iranian nation, Iranian leader Sayyed Ali Khamenei: A country like Iran needs mobilization more than any other country in light of foreign ambitions and interventions [affecting it]
Larijani to Pakistan's HUM TV: We are not seeking to acquire nuclear weapons, but we will continue to seriously develop our peaceful nuclear program

Bluebird bio's gene therapy for blood disorder wins FDA panel backing

  • By Al Mayadeen English
  • Source: Agencies
  • 12 Jun 2022 22:25
  • 1 Shares
2 Min Read

FDA panel backs Bluebird gene therapy for blood disorder hours after endorsing another Bluebird therapy for a rare neurological disease despite safety risks.

  • x
  • Bluebird bio
    Bluebird bio (Ruby Wallau)

Advisors to the US Food and Drug Administration backed on Friday the Bluebird bio's (BLUE.O) treatment for a rare blood disorder, in a vote of confidence in gene therapies and bolstering the company beset by regulatory setbacks.

The backing comes less than 24 hours after the same committee endorsed approval of bluebird's gene therapy, eli-cel, for a rare neurological disease even though there have been some concerns that the therapy may lead to cancer.

The panel of 13 outside experts voted unanimously on Friday for beti-cel, as a one-time gene therapy that treats Beta-thalassemia patients who rely on blood transfusions.

The FDA is to decide on beti-cel's fate by August 19 and eli-cel's by September 16. The agency is not required to abide by its advisors' recommendations, although it normally does.

Both products are expected to be launched this year if the FDA approves the therapies.

"Our expectation is that the product should be treating first patients by early fourth quarter," Chief Executive Officer Andrew Obenshain told Reuters.

The therapy's efficacy data was "outstanding", as per Panel member Jeannette Yen Lee, who added that "the opportunity to be transplant-independent is really life-changing for the patient."

Sarah Baqueri-Connolly, a parent of a Beta-thalassemia patient who died in 2015, said, "We've personally watched friends and family participate in gene therapy trials and be cured of this painful disease. They no longer need blood transfusions."

“Hopefully, with approval will come some priority review vouchers for these products that will provide some non-dilutive funding. And we'll also look at other ways of raising funding," CEO Obenshain said.

  • Blood disorder
  • FDA
  • Food and Drug Administration (FDA)

Most Read

Hezbollah announces the martyrdom of Haitham al-Tabatabai

Hezbollah announces the martyrdom of commander Haitham Tabatabai

  • West Asia
  • 23 Nov 2025
Hezbollah publishes biography of martyred leader Haitham al-Tabatabai

Hezbollah publishes biography of martyred leader Haitham Tabatabai

  • Politics
  • 23 Nov 2025
Hezbollah releases the names of the four martyred with Tabatabai

Hezbollah releases the names of the 4 martyred alongside Tabatabai

  • West Asia
  • 24 Nov 2025
A boy tries to stand near missiles displayed in the National Aerospace Park of the Revolutionary Guard, just outside Tehran, Iran, Thursday, Nov. 13, 2025 (AP)

Key Israeli sites destroyed, nuclear docs moved to Tehran: Minister

  • Politics
  • 22 Nov 2025

Coverage

All
In Five

Read Next

All
AI weaponized: 'Israel' scrubs soldiers’ posts to hide atrocities
Technology

AI weaponized: 'Israel' scrubs soldiers’ posts to hide atrocities

z
Politics

Petro: Oil is key to US pressure on Venezuela, not drug trade

The Lafarge cement plant facility is seen Tuesday, October 18, 2022, in Ravena, N.Y. (AP Photo/Hans Pennink)
Politics

Lafarge trial focuses on talks with Syrian armed groups, ISIS links

Denmark sets up Trump ‘night watch’ after Greenland tensions flare
Politics

Denmark sets up Trump ‘night watch’ after Greenland tensions flare

Al Mayadeen English

Al Mayadeen is an Arab Independent Media Satellite Channel.

All Rights Reserved

  • x
  • Privacy Policy
  • About Us
  • Contact Us
  • Authors
Android
iOS