BioTether Sciences specialize in development support for biopharmaceuticals as well as cell and gene therapies. Our team is experienced with the latest techniques to study cell and gene therapy quality attributes, clinical biomarkers, pharmacokinetics, and anti-drug antibodies.
Our services include generation of stable cell lines, reporter assays, protein and nucleic acid characterization, and bioanalysis. We can develop and validate novel test methods for potency, identity, and purity testing. We support non-clinical and clinical studies as well as process validation efforts.
Cell and Gene Therapies (CGT) offer unique requirements and challenges for quality control testing and process validation. Cell and tissue derived products may be used for cancer therapy, regenerative medicine and rare diseases. Gene therapies include complex viral vectors, and engineered nanoparticles used to treat hereditary and metabolic diseases. During process validation the quality attributes of these biopharmaceuticals must be defined and carefully tested because they may impact the safety and efficacy of the product. The raw materials and reference materials used to prepare the CGT should be well characterized. For example, growth factors, cytokines, and production conditions need to be well controlled. In order to monitor the manufacturing process biochemical testing and bioassays are needed. Engineered reporter cell lines and specialty assays are often required to test identity, purity, and potency of critical reagents. The suite of tests suggested by CGT regulators may challenge previous expectations for quality control testing in a classical analytical laboratory. Due to the biologic nature of CGT, a greater emphasis is on developing tools to assess nucleic acids, membranes, bioactivity, immunogenicity, and protein stability. Biosafety assessments such as sterility, endotoxin testing and opportunistic virus testing are important. The Quality Control laboratory testing platforms must now include Flow Cytometry, Next Generation Sequencing, ELISA, and customized bioassays. These instruments and know-how is now added to the list of classical analytical approaches such as HPLC, Mass Spectrometry and physico-chemical testing familiar to developers of small molecule and protein therapeutics. Pre-clinical studies of CGT are more challenging and should include cell and tissue biodistribution, off-target effects (genome editing), immune system response, and other complex assessments of safety and efficacy. The FDA and EMA have recently provided guidance on these topics to CGT developers that are pushing at the frontier of medicine.
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/human-gene-therapy-products-incorporating-human-genome-editing
https://www.ema.europa.eu/en/human-regulatory/research-development/scientific-guidelines/multidisciplinary/multidisciplinary-cell-therapy-tissue-engineering#share
