Remove FDA Remove Pharmacokinetics Remove Protein Expression
article thumbnail

Informed Design of Bioanalytical PCR Assay Testing Parameters

PPD

These treatments use a vector to introduce the desired nucleic acid code to replace or modify protein expression or use cells to alter/restore a specific cell type. Food and Drug Administration (FDA). Working with experienced providers can help clients build data sets that satisfy current and future FDA requirements.

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

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

Alta Sciences

Food and Drug Administration (FDA) as a nasal spray called esketamine, for treatment-resistant depression. They work by binding to specific sequences of nucleotides present within the mRNA structure and can induce mechanisms that either decrease, restore, or modify protein expression. Asia, and Europe.

article thumbnail

Stoke Therapeutics Announces Presentations Related to the Company’s Work to Advance STK-001, the First Potential New Medicine to Target the Underlying Cause of Dravet Syndrome at the American Epilepsy Society (AES) 2020 Annual Meeting

The Pharma Data

Safety and Pharmacokinetics of Antisense Oligonucleotide STK-001 in Children and Adolescents with Dravet Syndrome: Single and Multiple Ascending Dose Design for the Open-Label Phase 1/2a MONARCH Study. The primary endpoints are safety, tolerability and pharmacokinetic (PK) profile of STK-001 in Dravet syndrome patients.

article thumbnail

2021 AAN highlight impact and breadth of expanding neuroscience portfolio

The Pharma Data

FDA and global approvals for our groundbreaking therapies in SMA and NMOSD, Roche’s data at AAN reflect our continued commitment to meaningful therapeutic progress for people living with neurological disorders,” said Levi Garraway, M.D., Following U.S. Roche’s Chief Medical Officer and Head of Global Product Development.

article thumbnail

FDA’s Project Optimus: A new era in oncology drug dosing

Drug Target Review

Molecular factors Molecular markers, such as protein expression and signalling pathway activation, also guide therapy choices. For example, PD-L1 expression predicts the response to immune checkpoint inhibitors. Project Optimus: FDA’s Initiative to Reform Oncology Dose Optimization. Food and Drug Administration.