CMC Pharmaceutical Development Flashcards
What is CMC Pharmaceutical Development?
Pre-formulation characterization and formulation development services for complex pharmaceuticals and biologic therapies. GMP clinical trial materials manufacturing w/ specialty services.
What is CMC?
Chemistry, Manufacturing, and Controls (FDA standard)
What are gene therapies?
experimental techniques that use genes to treat or prevent diseases. Is is used to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein.
What gene therapies does Pace support?
- Native and modified DNA(single and double stranded), mRNA, circRNA, tRNA
- Small interfering RNA (siRNA)
- O-methyl/locked nucleic acids (LNA)
- Peptidic nucleic acids (PNA)
- Phosphorodiamidate morpholino oligos (PMOs)
What assessments can Pace deliver to the therapeutic molecule?
- molecular weight (DNA/RNA)
- conformation of nucleic acids
- thermal and chemical stability
- determination of molar extinction coefficient
- capsid integrity and protein composition
- lipid nanoparticle (LNP) composition and size distribution
- encapsulation efficiency (LNP/VLP)
What does Pace mean by “dedicated analytical strategies”?
What gene therapy development services does Pace provide?
- secondary structure assessment and sizing
- oligomeric state of nucleic acid particles in solution
- purity analysis and quantitative detection of primary components
- degradation products in raw materials
- process samples and final drug substances
What gene therapy analytical services does Pace provide?
- analytical methods
- physiochemical characterization
- biophysical characterization
- biopharmaceutical characterization
What are small molecules (and examples)?
What are biologics (and examples)?
What are protein drug conjugates (and examples)?
What are oligonucleotides (and examples)?
Oligonucleotides is a broad term for molecules with different chemical compositions and covering a variety of molecular weights, ranging between 15-20 nucleotides (siRNAs) to as many as thousands of nucleotides (mRNA, DNA).
