Fierce Pharma

NOVEMBER 7, 2023

Vertex and CRISPR Therapeutics are up first with a Dec. | But on Tuesday bluebird said that Vertex’s price tag will not factor into how the Massachusetts company will price its treatment. Two long-awaited treatments for sickle cell disease (SCD) are on the docket for FDA decisions next month.

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Gene Therapy Gene 86

Pharmaceutical Technology

APRIL 13, 2023

Released on April 12, the report focuses on bluebird bio’s lovotibeglogene autotemcel and Vertex Pharmaceuticals and CRISPR Therapeutics’ exagamglogene autotemcel or exa-cel and their potential use in treating sickle cell disease. If approved, exa-cel would be the first FDA-approved gene therapy based on CRISPR editing.

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Gene Therapy Gene FDA Approval Licensing 147

XTalks

DECEMBER 13, 2023

The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel). Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US. Vertex-CRISPR’s Casgevy has a US list price of $2.2

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Gene Therapy FDA Approval Gene In-Vivo 98

Drug Discovery World

JANUARY 9, 2023

Using gene engineering, we are repurposing cancer cells to develop a therapeutic that kills tumour cells and stimulates the immune system to both destroy primary tumors and prevent cancer.” . CRISPR-Cas9 gene editing . Living tumour cells will travel long distances across the brain to return to the site of their fellow tumour cells.

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Scientist Gene Editing Gene Engineer 52

Pharmaceutical Technology

APRIL 5, 2023

But access to these treatments continues to remain limited due to high price tags and variable availability across regions. This includes the first potential approval of a CRISPR-based gene therapy called exa-cel , which is developed by CRISPR Therapeutics and Vertex Pharmaceuticals.

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Gene Therapy Gene Engineer Production 147

XTalks

FEBRUARY 27, 2024

Technologies like CRISPR-Cas9 have revolutionized the potential for gene editing, offering hope for curative treatments for conditions like Duchenne muscular dystrophy (DMD) and certain types of inherited blindness. Both therapies were awarded their approvals on the same day in December 2023.

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Research Drugs Gene Therapy Clinical Trials 59

XTalks

FEBRUARY 11, 2021

After several such Nobel Prize snubs for female scientists, 2020 saw the Nobel Prize in Chemistry go to a female scientist duo for the first time for their revolutionary discovery of the CRISPR-Cas9 gene editing system. Jennifer Doudna and Emmanuelle Charpentier Pioneer with CRISPR. Emmanuelle Charpentier.

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Genetics DNA Genome Genomics 119