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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STAT

DECEMBER 12, 2023

The news comes as researchers are still processing the Food and Drug Administration’s landmark approval of two cutting-edge sickle cell therapies, one made by Vertex Pharmaceuticals and CRISPR Therapeutics and the other by Bluebird Bio. These drugs have price tags of $2.2 million and $3.1 million, respectively.

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PharmaShots

MAY 19, 2023

Alimera Acquires Rights from EyePoint Pharmaceuticals to Commercialise Yutiq in the US Date: May 19, 2023 | Tags: Alimera, EyePoint Pharmaceuticals, Yutiq, Iluvien, chronic non-infectious uveitis, US, Pharma Sobi Reports EMA’s Validation of MAA for Efanesoctocog Alfa to Treat Haemophilia A Date: May 19, 2023 | Tags: Sobi, Efanesoctocog Alfa, (..)

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PharmaShots

MARCH 31, 2023

PDS Biotech to Initiate P-III Trial (VERSATILE-003) of PDS0101 + Keytruda (pembrolizumab) for Head and Neck Cancer Date: Mar 31, 2023 | Tags: PDS Biotech, PDS0101, Keytruda, pembrolizumab, Head, Neck Cancer, Clinical Trial, P-III, VERSATILE-003 Trial Omega Therapeutics Entered into a Clinical Supply Agreement with Roche to Evaluate OTX-2002 for Hepatocellular (..)

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PharmaShots

MAY 5, 2023

Chiesi Global Rare Diseases and Protalix BioTherapeutics Receive EC’s Marketing Authorization of PRX-102 (pegunigalsidase alfa) for the Treatment of Fabry Disease Date: May 05, 2023 | Tags: Chiesi Global Rare Diseases, Protalix BioTherapeutics, PRX-102, pegunigalsidase alfa, Fabry Disease, Regulatory, EC, Marketing Authorization Kinoxis Therapeutics (..)

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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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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. There has been extremely rapid progress.

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