Category | Viral vector services
The promise of gene therapy is well established. By introducing, modifying, or replacing genes in a person’s cells to correct or compensate for genetic abnormalities, gene therapies target the underlying cause of the disease at the cellular level, leading to an effective treatment or cure.
Realizing this promise, however, requires the activation of several scientific, technological, and regulatory enablers. Among the most critical of these is the development of safe and effective delivery systems to transport the nucleic acids that encode the desired therapeutic protein into target cells. Unprotected nucleic acids are not stable in a biological environment, nor can they enter the cytoplasm (RNA) or the nucleus (DNA) by themselves. They need a Trojan horse that can get through the barriers and deliver the effectors where they need to go.
Enter viral vectors. These modified versions of naturally occurring viruses, such as adenoviruses, adeno-associated viruses, or lentiviruses, are engineered to be safe and efficient vehicles for transporting therapeutic genetic material into a cell. However, because viral vectors are inherently complex to produce at scale, the journey from bench to commercialization is rarely straightforward.
Navigating it requires maintaining a laser focus on quality to ensure the vectors are well characterized and consistently manufactured to rigorous tolerances of purity, potency, and safety. This is inherently challenging, both because viral vectors differ significantly in productivity and potency, and because regulatory standards for manufacturing and quality control are still evolving.
For biotech companies developing gene therapies, this means that quality—both of the viral vector itself and the CDMO partnership that enables it—must be a priority consideration when outsourcing viral vector development because it directly affects the efficiency, safety, targeted delivery, scalability, and clinical strategy of the gene therapy.
The characteristics of a quality viral vector can vary depending on the specific application and requirements. However, some universal quality traits include:
At a high level, the defining attributes of a quality CDMO partnership are the same features that have been identified by industry partners as priority considerations across all therapeutic modalities. These include supply robustness, a solid regulatory track record, CAPA effectiveness, open communication, and business consistency, among others. With respect to the unique and specific needs of viral vector manufacturing, the following considerations are integral to a quality partnership.
As evidenced by the defining characteristics, quality in viral vector manufacturing is a holistic endeavor that encompasses product, process, and partnership attributes. Developing gene therapy products that are clinically beneficial, commercially viable, and meaningful to patients requires harnessing all of them.
Learn more about partnership quality in viral vector development and manufacturing services.