Delivering Genetic Editing Therapies To Market

By Dr. Rajiv Vaidya, Head of Manufacturing Science & Technology, and Dr. Samir Acharya, Associate Director of Process Development, Andelyn Biosciences

In the last decade, breakthrough approaches in genetic engineering have revolutionized therapeutic research and placed deliverable gene editing therapies within patients' grasps. Molecular tools such as CRISPR-Cas machinery and zinc finger nucleases (ZFNs) are providing the possibility for patients to overcome the effects of genetic abnormalities, which are thought to impact around 1% of the global population.

However, before these therapeutics are available to patients, there are a number of hurdles for developers to navigate as their gene editing therapies move toward clinical and commercial production. Patient safety remains at the forefront of genetic engineering practices and is pertinent for not only the application of gene editing machinery, but also the manufacturing processes involved in delivering therapies. Considering scalability and cost as therapies move toward manufacturing will also be essential to make gene editing treatments accessible to a broad number of patients.

Developers and manufacturers will need to demonstrate flexibility while staying abreast of innovations, industry advancements, and evolving regulations as the gene editing industry matures. As your company develops its genetic engineering capabilities, learn more about the steps necessary to allow commercially available gene editing therapeutics to become a reality.