BioWorld. Link to homepage.

Clarivate
  • BioWorld
  • BioWorld MedTech
  • BioWorld Asia
  • BioWorld Science
  • Data Snapshots
    • BioWorld
    • BioWorld MedTech
    • Infographics: Dynamic digital data analysis
  • Special reports
    • Infographics: Dynamic digital data analysis
    • Trump administration impacts
    • Biopharma M&A scorecard
    • BioWorld 2024 review
    • BioWorld MedTech 2024 review
    • BioWorld Science 2024 review
    • Women's health
    • China's GLP-1 landscape
    • PFA re-energizes afib market
    • China CAR T
    • Alzheimer's disease
    • Israel
    • Rise of obesity
    • Radiopharmaceuticals
    • Biosimilars
    • Aging
    • IVDs on the rise
    • Coronavirus
    • Artificial intelligence

BioWorld. Link to homepage.

  • Sign In
  • Sign Out
  • My Account
Subscribe
BioWorld - Sunday, May 11, 2025
Home » Keywords » exagamglogene autotemcel

Items Tagged with 'exagamglogene autotemcel'

ARTICLES

Handshake with DNA, molecules

Orum scores potential $945M deal with Vertex for novel DACs

July 16, 2024
By Marian (YoonJee) Chu
Orum Therapeutics Inc. struck a potential $945 million (₩1.3 trillion) deal with Vertex Pharmaceuticals Inc. to discover novel degrader antibody conjugates (DAC) as targeted conditioning agents for use with gene editing, including Vertex’s gene therapy, Casgevy (exagamglogene autotemcel).
Read More

Biogen’s ataxia drug, Crispr’s sickle cell therapy cleared in EU

Feb. 13, 2024
By Caroline Richards
The European Commission approved two therapies for progressive, genetic diseases: Biogen Inc.’s Friedreich’s ataxia drug, Skyclarys (omaveloxolone), and Crispr Therapeutics AG’s CRISPR/Cas9 gene therapy for sickle cell disease and transfusion-dependent beta-thalassemia, Casgevy (exagamglogene autotemcel, exa-cel).
Read More

FDA approves Vertex’s Casgevy for beta-thalassemia two months early

Jan. 16, 2024
Though the PDUFA date for its BLA wasn’t until March 30, 2024, Vertex Pharmaceuticals Inc. celebrated the U.S. FDA approval Jan. 16 for Casgevy (exagamglogene autotemcel), expanding use the CRISPR/Cas9 gene-edited cell therapy in patients, 12 and older, with transfusion-dependent beta-thalassemia.
Read More
EMA logo on mobile screen, vials, syringes

EMA grants 77 approvals in 2023, outpaces FDA in biosimilars

Jan. 16, 2024
By Nuala Moran
The EMA has once again come in behind the U.S. FDA, granting market access to 77 new products in 2023, fewer than half the 157 approvals the FDA processed in the 11 months from January through December 2023.
Read More
Sickle cells

US FDA approves first CRISPR-based gene therapy Casgevy for sickle cell; Lyfgenia gets nod, too

Dec. 8, 2023
By Karen Carey and Mari Serebrov
Both Vertex Pharmaceuticals Inc.’s Casgevy (exagamglogene autotemcel, exa-cel) and Bluebird Bio Inc.’s Lyfgenia (lovotibeglogene autotemcel, lovo-cel) received U.S. FDA approval Dec. 8, providing 16,000 American sickle cell patients who have recurring vaso-occlusive events with access to the first cell-based gene therapies.
Read More
Red blood cells, DNA

Historic MHRA exa-cel win bagged, Crispr/Vertex await FDA nod

Nov. 16, 2023
By Randy Osborne
With a landmark U.K. approval in hand for Casgevy (exagamglogene autotemcel [exa-cel]) to treat sickle cell disease and transfusion-dependent beta thalassemia, Crispr Therapeutics AG and partner Vertex Therapeutics Inc. are turning their attention to the PDUFA dates set by the U.S. FDA for the treatment in both conditions.
Read More
Sickle cell illustration

Adcom: Don’t sacrifice progress for perfection

Nov. 1, 2023
By Mari Serebrov
Had it been asked to, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee would have voted Oct. 31 to recommend approval of Vertex Pharmaceutical Inc. and Crispr Therapeutics AG’s exagamglogene autotemcel, or exa-cel, as a one-time transformative treatment for severe sickle cell disease in individuals 12 and older.
Read More
Sickle cell disease 3D illustration

No red flags flying ahead of adcom on Vertex’s SCD therapy

Oct. 27, 2023
By Lee Landenberger
It’s just a discussion next week of Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG’s sickle cell disease (SCD) therapy, with no vote expected from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee. The FDA isn’t questioning the drug’s efficacy as much as it is concerned about the effects of off-target editing.
Read More
DNA repair
International Summit on Human Genome Editing

Somatic genome editing pricey, germline editing still risky, researchers conclude

March 9, 2023
By Nuala Moran
Somatic human genome editing has made huge strides in the past five years, but the likely extremely high prices will be unsustainable. A global commitment to affordable, equitable access is urgently needed because the costs and infrastructure needs of this form of treatment are not manageable for either patients or health care systems.
Read More
Handshake with DNA, molecules

Scribe, Vertex add to gene therapy's advance with $1B+ deal, near-term BLA

Sep. 27, 2022
By Michael Fitzhugh
CRISPR-based cell therapies continued to gain steam Sept. 27 with the announcements of a potentially valuable big pharma collaboration and an ambitious global regulatory push.
Read More
More Articles Tagged with 'exagamglogene autotemcel'

Popular Stories

  • Today's news in brief

    BioWorld
    BioWorld briefs for May 9, 2025.
  • Today's news in brief

    BioWorld MedTech
    BioWorld MedTech briefs for May 9, 2025.
  • US flag, stock market chart, White House

    100 days of uncertainty

    BioWorld
    The first 100 days of the Trump administration have been nothing short of chaotic, both in the U.S. and throughout the world. Shining a light through the...
  • AI generated, 3D rendering of protein degradation

    AACR 2025: Induced proximity strategy, beyond degraders

    BioWorld
    Targeted protein degradation has yet to notch its first approval. But with more than two dozen agents now in clinical trials, the strategy’s ultimate clinical...
  • NME Digest: Q1 2025

    BioWorld
    A quarterly dynamic table featuring new molecular entities (NMEs) revealed for the first time in current literature, at congresses and in company communications...
  • BioWorld
    • Today's news
    • Analysis and data insight
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Newco news
    • Opinion
    • Regulatory
    • Science
  • BioWorld MedTech
    • Today's news
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Newco news
    • Opinion
    • Regulatory
    • Science
  • BioWorld Asia
    • Today's news
    • Analysis and data insight
    • Australia
    • China
    • Clinical
    • Deals and M&A
    • Financings
    • Newco news
    • Regulatory
    • Science
  • BioWorld Science
    • Today's news
    • Biomarkers
    • Cancer
    • Conferences
    • Endocrine/Metabolic
    • Immune
    • Infection
    • Neurology/Psychiatric
    • Patents
  • More
    • About
    • Advertise with BioWorld
    • Archives
    • Article reprints and permissions
    • Contact us
    • Cookie policy
    • Copyright notice
    • Data methodology
    • Infographics: Dynamic digital data analysis
    • Podcasts
    • Privacy policy
    • Share your news with BioWorld
    • Staff
    • Terms of use
    • Topic alerts
Follow Us

Copyright ©2025. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing