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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Laboratories Protein Expression Clinical Trials Pharmacokinetics 52

Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

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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. .–( BUSINESS WIRE )– Stoke Therapeutics , Inc. MONARCH Phase 1/2a Current Study Design. Lori Isom, Ph.D.,

Protein Expression 52

Protein Expression Clinical Trials Pharmacokinetics Trials 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

APRIL 8, 2021

today announced that new data for its approved and investigational medicines for the treatment of neurological disorders will be presented at the 73rd American Academy of Neurology (AAN) Annual Meeting being held virtually April 17-22, 2021. Multiple Sclerosis (MS). Neuromyelitis Optica Spectrum Disorder (NMOSD).

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Clinical Trials Trials Disease Nurses 52

The Pharma Data

DECEMBER 29, 2020

Phase III BRIDGE open-label, switch-over clinical trial met key objectives for safety and efficacy. Pegunigalsidase alfa is a plant cell-expressed recombinant, PEGylated, cross-linked ?-galactosidase-A galactosidase-A product candidate under development for the treatment of Fabry disease.

Clinical Trials 52

Clinical Trials Disease Treatment Trials 52