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
The White House: South Africa used its G20 presidency to undermine the group's founding principles.
US White House: South Africa refuses to facilitate a smooth transition of the G20 presidency
Al Mayadeen's correspondent in Caracas: Several airlines are canceling flights to and from Venezuela until further notice
Al-Rashq: 'Israel' is fabricating pretexts to evade the agreement and return to a war of extermination, and it is the one that violates the agreement daily and systematically
Al-Rashq: We demand that mediators and the US administration pressure 'Israel' to reveal the identity of the gunman it claims Hamas sent
Hamas political bureau member Izzat al-Rashq: Reports published by Israeli sources regarding Hamas informing Witkoff that the agreement has ended are false
Al-Dali: Various Palestinian factions will participate in these important national meetings
Al-Dali: The visit carries important implications, and this is proof that Hamas attaches particular importance to this visit, given the sensitivity of the current stage
Al-Dali: The goal is to coordinate positions with Arab countries and strengthen the Palestinian national position in the face of Israeli plans
Al-Dali: A Hamas leadership delegation, headed by Khalil al-Hayya, will arrive in Cairo within the next few hours

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

Inside the Epstein-Rothschild web behind 'Israel’s' spy tech empire

Inside the Epstein-Rothschild web behind 'Israel’s' spy tech empire

  • Politics
  • 19 Nov 2025
Hamas fighters stand in formation as they prepare for the ceremony of Israeli captive hand over to the Red Cross in Nuseirat, central Gaza Strip, Feb. 22, 2025. (AP)

US plot for Gaza in shambles amid continued popular support for Hamas

  • Politics
  • 17 Nov 2025
Ukrainian political analyst Mikhail Chaplyha has written that Jolie was ‘called’ to Kherson in order to divert attention from Pokrovsk. (Al Mayadeen English; Illustrated by Zeinab el-Hajj)

Strategic cities fall to Russian forces in Donbass; Ukraine denies what is happening

  • Opinion
  • 16 Nov 2025
Democracy at the civilizational crossroads: Critical analysis of bourgeois Democracy, its alternatives

Democracy at the civilizational crossroads: Critical analysis of bourgeois Democracy, its alternatives

  • Analysis
  • 19 Nov 2025

Coverage

All
In Five

Read Next

All
Nahdlatul Ulama chief faces removal over hosting pro-'Israel' speaker
Asia

Nahdlatul Ulama chief faces removal over hosting pro-'Israel' speaker

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)
Politics

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

Guy Pearce
Asia Pacific

Aussie actor 'disgusted' by Israeli 'disdain' for Palestinian life

Brigadier General Ali Jahanshahi, the newly appointed commander of the Iranian ground forces, in an undated photo (MNA)
Politics

Ali Jahanshahi appointed as new commander of Iran Army Ground Force

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