United Pharmaceuticals Inc. supports the development of cell and gene therapies with integrated preclinical and bioanalytical solutions designed to address scientific, technical, and regulatory complexity. From early safety evaluations to advanced analytical support, our teams provide the expertise required to move innovative genetic medicines toward clinical success. Together with our partners, we help translate breakthrough therapies into meaningful improvements in patient outcomes and quality of life.
Begin your cell or gene therapy development journey with a trusted preclinical partner. Contact our experts to learn how our tailored solutions can support your program from early research through clinical readiness.
Cell and gene therapy programs present unique development considerations, from biodistribution and long-term safety to immune responses and regulatory expectations. Our dedicated pharmacology, toxicology, and bioanalytical specialists design and execute studies that support efficient progression while maintaining scientific rigor.
Sponsors often seek support in areas such as regulatory-aligned safety and efficacy assessment, reliable access to nonhuman primates, and carefully designed studies evaluating biodistribution, persistence, and potential risks such as germline integration. Additional expertise may be required for specialized routes of administration, including intravenous, intrathecal, intravitreal, or suprachoroidal delivery, as well as for therapeutic-area-specific safety evaluations. United Pharmaceuticals Inc. provides comprehensive solutions to address each of these needs under one integrated platform.
Over the past several years, our teams have completed a significant volume of in-life studies supporting cell and gene therapy programs, with the majority conducted in nonhuman primates. Our experience spans investigational new drug–enabling studies for rare diseases and advanced genome-editing technologies, including work with clustered regularly interspaced short palindromic repeats platforms. This depth of experience enables us to guide sponsors through complex regulatory landscapes and support strong, well-justified submissions.
Our good laboratory practice–compliant bioanalytical laboratories deliver precise biodistribution and molecular analysis to support cell and gene therapy development. On-site polymerase chain reaction capabilities enable accurate quantification of vector DNA and transgene ribonucleic acid. Additional bioanalytical services include protein expression analysis, immunogenicity evaluation, cellular immune response characterization, and full method development and validation tailored to each therapeutic platform.
We support complex routes of administration and long-term study designs, including work in juvenile nonhuman primates and specialized surgical biopsy procedures. Purpose-built facilities support discovery-stage research, while dedicated early-development sites focus on translational studies across adeno-associated virus, lentiviral, and genome-editing platforms. These capabilities enable precise dosing, targeted delivery, and clinically relevant data generation.
The tables below outline the range of pharmacology and toxicology studies conducted across our preclinical sites, highlighting evaluated organ systems and applied methodologies.
Potential germline integration remains a critical concern in gene therapy research. United Pharmaceuticals Inc. incorporates scientifically sound and regulator-aligned approaches to assess and mitigate this risk. Our study designs address biodistribution, persistence, and reproductive tissue exposure, ensuring data packages meet evolving regulatory expectations while supporting responsible development.
Pre-existing immunity to adeno-associated viruses can significantly affect study outcomes, particularly in nonhuman primates previously exposed to viral vectors. Neutralizing antibodies may reduce transduction efficiency and confound the interpretation of toxicology or biodistribution data.
Given the high seropositivity rates observed for certain adeno-associated virus serotypes, pre-screening animals for anti-vector antibodies is an essential step before study initiation. Identifying animals with elevated antibody levels helps ensure appropriate model selection and improves data reliability.
Our validated assays support both good laboratory practice and non-good laboratory practice studies and can be adapted across serotypes and species. These methods also support in-study immunogenicity assessments, providing flexibility across development phases.
With multiple advanced laboratories across North America, United Pharmaceuticals Inc. delivers comprehensive bioanalytical support for cell and gene therapy programs. Our scientists bring extensive expertise in molecular biology, flow cytometry, neutralizing antibody assays, viral vector analytics, and immune profiling.
We provide tailored methodologies to support critical development milestones, including nucleic acid purification from complex matrices, reproducible protein extraction, and pharmacokinetic assay development using advanced analytical platforms. Each service is aligned with regulatory expectations and study objectives.
Our bioanalytical capabilities support biodistribution analysis, transgene and protein expression profiling, immunogenicity assessment, and cellular immune response evaluation. We also offer full method development, qualification, and validation to ensure robust data generation across discovery and development stages.
From study design and scope planning to advanced molecular and bioanalytical services, United Pharmaceuticals Inc. ensures every step aligns with your therapeutic goals and regulatory requirements. Our integrated approach delivers high-quality, reliable data to support informed decision-making throughout cell and gene therapy development.