Gene Therapy Testing Services

BA Sciences is proud to be the source trusted by many in the pharmaceutical industry for testing services aimed at Advanced Therapeutic Medicinal Products (ATMP) including gene therapy treatments. Backed by deep scientific expertise and purpose-built cell culture facilities, we provide comprehensive analytical support for gene therapy drug substances and final drug products.

Our team provides comprehensive support throughout every phase of the clinical evaluation process, enabling clients to generate the data needed to demonstrate the safety and efficacy of their gene therapy products. The level of experience delivered is especially critical because gene therapy products often produce long-lasting and even permanent biological effects in patients. BA Sciences offers the extra level of monitoring and analysis needed to ensure these therapies deliver the desired effect.

Gene therapy is a form of treatment that alters, regulates, or replaces genes in the patient’s cells to treat the underlying cause of certain diseases. For instance, gene therapy may be used to silence or suppress the expression of a gene that contributes to disease or other adverse effects, introduce a new gene to produce a therapeutic protein, or replace a nonfunctional or mutated gene to restore normal cellular function.

The process uses vectors to transport new genetic code into the target cells. These can be engineered viruses or non-viral vectors such as lipid nanoparticles or polymer-based systems. Today, advanced technologies such as CRISPR-Cas9 have made targeted modification of DNA in specific locations much more accurate and versatile.

BA Sciences has the gene therapy analytical services needed to support our clients through every phase of clinical evaluation for gene therapies. These stages are designed to assess the safety of these products as well as their proper dosage and long-term performance. Here is a brief rundown of the three main phases of clinical trials for gene therapies:

Phase I — These are the earliest tests, aimed at evaluating the initial safety of the therapy and determining the dose ranges. The key objectives during Phase I include identifying dose-limiting toxicities, assessing the vector behavior in the body, and documenting any short-term side effects.

Phase II — During this phase, the participant population has expanded. Examples of programs that typically take place during Phase II include dose-expansion cohorts, assessments of biological activity, and evaluations of durability over time. This process also may involve additional measurements such as vector shedding, immunogenicity, or biomarker responses.

Phase III — These confirmatory studies are used to compare the efficacy of the gene therapy with standard care methods. The trials during this phase are intended to evaluate clinical endpoints across broader and more-diverse populations, monitor adverse events over longer timeframes, and generate the data needed for regulatory submissions.

We work closely with gene therapy developers by offering analytical testing and characterization of ATMPs. Our gene therapy services include method development, validation, and release-support testing that is tailored specifically to advanced modalities. With our help, clients in development and manufacturing have the support they need to ensure the quality, consistency, and regulatory readiness of what they produce.

To learn more about how our gene therapy solutions can help with your analytical needs or want to discuss an upcoming program with our experts, reach out and connect with a member of our experienced team today.