Remove FDA Approval Remove Pharmacokinetics Remove Protein Expression
article thumbnail

Five Promising Treatment Areas in Early-Phase Drug Development in 2024

Alta Sciences

The FDA approved the first gene therapy in 2017 for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL), and as of December 2023, there are over 30 approved cell and gene therapies on the market in the U.S.A. Asia, and Europe.

article thumbnail

Protalix BioTherapeutics and Chiesi Global Rare Diseases Announce Extension of PDUFA Date for Pegunigalsidase Alfa for the Proposed Treatment of Fabry Disease

The Pharma Data

(NYSE American: PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based protein expression system, and Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A.,

Disease 52
Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Protalix BioTherapeutics and Chiesi Global Rare Diseases Announce Final Results of BRIDGE Phase III Open-Label, Switch-Over Clinical Trial Evaluating Pegunigalsidase Alfa for the Treatment of Fabry Disease

The Pharma Data

(NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based protein expression system, and Chiesi Global Rare Diseases , a business unit of Chiesi Farmaceutici S.p.A.,

article thumbnail

2021 AAN highlight impact and breadth of expanding neuroscience portfolio

The Pharma Data

SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement. Food and Drug Administration (FDA) approved EVRYSDI for the treatment of SMA in adults and children 2 months of age and older. EVRYSDI is administered daily at home in liquid form by mouth or by feeding tube.