Advancing viral vector development through Quality by Design approach

Parkinson’s disease is a debilitating neurological disorder that affects movement and other functions, and approximately 90,000 people in the U.S. are diagnosed with Parkinson’s each year. Jennifer A. Johnston, PhD, has dedicated her entire career to researching Parkinson’s disease. Most recently, her efforts have focused on Parkin-PD, a genetic form of Parkinson’s with an early onset age.

In our recent webinar, Dr. Johnston discussed her partnership with Thermo Fisher Scientific, which provides crucial CDMO (contract development and manufacturing organization) services and support for NysnoBio's innovative gene therapy. This partnership focuses on viral vector development and manufacturing, aiming to create a one-time treatment to restore movement in Parkin-PD patients.

Pioneering research and NysnoBio’s mission

In 2019, Dr. Johnston co-founded NysnoBio in San Francisco to develop a Parkin gene replacement therapy. Despite the challenges of navigating the complex and highly regulated gene therapy landscape, NysnoBio has made significant strides. The company's efforts were given a major boost in July 2023 when it received a research grant from the Michael J. Fox Foundation. This funding is intended to support product manufacturing necessary for Investigational New Drug (IND) safety studies, which are essential for human clinical trials.

Partnering for success: Thermo Fisher Scientific

Recognizing the need for industry-leading infrastructure and manufacturing capabilities, NysnoBio chose to partner with Thermo Fisher Scientific. This collaboration leverages Thermo Fisher’s expertise in viral vector development and process engineering, ensuring that NysnoBio's groundbreaking work is reproducible, scalable, and scientifically sound. Dr. Johnston emphasized the importance of having a partner who can provide comprehensive support from process development through commercialization, allowing NysnoBio to focus on innovation.

Quality by Design: A systematic approach

As part of the webinar, Dr. Johnston and Dr. Pouria Motevalian, Director of Bioprocess Sciences for Viral Vector Services at Thermo Fisher Scientific, discussed Thermo Fisher Scientific’s "Quality by Design" (QbD) approach to viral vector process and analytical development. This methodology emphasizes predefined objectives, product and process understanding, and process control based on science and quality risk management.

Dr. Motevalian provided insights into how Thermo Fisher Scientific applies QbD standards throughout the product lifecycle, from early-stage research and development to commercialization and manufacturing. He illustrated this approach with a recent example from NysnoBio’s gene therapy program for Parkin-PD, highlighting the commitment to product quality from the onset of the development journey.

Watch the video clip to learn how our QbD approach ensures excellence and cost-effectiveness at every step of development.