Gene Expression Gene Therapy Genetic Disease Immune Response 
Camargo
JULY 27, 2021
How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.
The Pharma Data
DECEMBER 27, 2020
STP705 is a small interfering RNA (siRNA) therapy that leverages a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to knock down both TGF-beta1 and COX-2 gene expression. RESTORE-1 is a Phase II trial of an intracerebral AAV-based gene therapy for Parkinson’s disease.
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