PPD

OCTOBER 12, 2023

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. Cell and gene therapies (CGTs) are one of the fastest growing areas in human therapeutics.

Laboratories 52

Laboratories Clinical Trials Protein Expression Pharmacokinetics 52

The Pharma Data

NOVEMBER 26, 2020

(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

Disease Treatment Clinical Trials FDA 52

The Pharma Data

DECEMBER 29, 2020

(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.,

Clinical Trials 52

Clinical Trials Disease Treatment Trials 52

Alta Sciences

APRIL 17, 2024

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. And since proteins are often linked to disease, there's huge potential to treat a broad range of diseases with this technology.

Drug Development 52

Drug Development Treatment FDA Approval Drugs 52

The Pharma Data

DECEMBER 3, 2020

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.

Clinical Trials 52

Clinical Trials Protein Expression Pharmacokinetics Trials 52

The Pharma Data

APRIL 8, 2021

Based on preclinical studies, OCREVUS binds to CD20 cell surface proteins expressed on certain B cells, but not on stem cells or plasma cells, suggesting that important functions of the immune system may be preserved. This nerve cell damage can lead to disability in people with MS.

Clinical Trials 52

Clinical Trials Trials Disease Therapies 52

Drug Target Review

JANUARY 20, 2025

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. This approach underlies the identification of the OBD, backed by both clinical efficacy and drug tolerability data.

Clinical Trials 52

Clinical Trials Trials Drugs Drug Development 52