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Gene Therapy Drug Development Services
Establishing a robust process for gene therapy drug production enables pre-clinical development and ultimately, formulation development for clinical trial materials. To support this process, specific strategies need to be developed for: 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.
Optimal drug product composition is then formulated within a range of boundary conditions informed through the process development activities. Vehicles, including liquid solution or suspension, lipid nanoparticles, and virus-like particles allow options for administration, such as intravenous (IV), subcutaneous, or inhalation delivery. When long-term stability in an aqueous buffer is not possible, lyophilization of oligos enables storage at ambient conditions and simplifies preparation of the administrable dosage form.
A final significant consideration is the bioanalytical evaluation of preclinical and clinical samples. While FDA regulatory guidelines are not specific for oligonucleotide drugs, these substances are often treated as small molecules due to the synthetic manufacturing processes used in their production, despite their large size and biochemical properties. Methods to determine the fate of the molecular entity, metabolites, and the unique delivery vehicles is a frequent consideration to satisfy regulators.