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. There are over 2,000 CGTs currently being evaluated in clinical trials, with more than 200 in Phase III and 10-20 per year estimated to come to market by 2025.

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The Pharma Data

DECEMBER 29, 2020

Phase III BRIDGE open-label, switch-over clinical trial met key objectives for safety and efficacy. Pegunigalsidase alfa (PRX-102) is an investigational, plant cell culture-expressed, and chemically modified stabilized version of the recombinant ?-Galactosidase-A CARMIEL, Israel and BOSTON , Dec.

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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

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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

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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. Clinical Trials. protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene to restore physiological Na V 1.1

Clinical Trials 52

Clinical Trials Protein Expression Pharmacokinetics Trials 52

The Pharma Data

APRIL 8, 2021

New 2-year findings from Part 2 of the Phase II/III FIREFISH trial show longer-term efficacy and safety of EVRYSDI in infants with symptomatic Type 1 SMA treated with EVRYSDI. P6: Neuromuscular Disorders and Clinical Trials. P6: Neuromuscular Disorders and Clinical Trials. Multiple Sclerosis (MS).

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Drug Target Review

JANUARY 20, 2025

These advances are reshaping how pharmaceutical and biotechnology companies approach clinical trial design, with a focus on patient-centric dosing strategies. This article explores how innovations in precision medicine are reshaping clinical trials, followed by a discussion on Project Optimus and its impact on dose optimisation.

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