Gene and cell therapies (GCTs) are capturing the attention of players from across the pharmaceutical industry. And with rapid growth comes the need for support from specialized CDMOs. Here, Brian Min, CEO of GenScript ProBio, explains how the company is gearing up to help the global GCT community deliver on the promise of these exciting therapies.
We live in a world where 4 million people are diagnosed with cancer each year; ultimately, our goal is to see as many successful GCT products reach patients as possible. It’s an area with an upward trajectory. Though GCTs were first developed in the USA, there is a massive opportunity for companies around the world to get involved with the progression of the field. China, for example, was quick to adopt the concept and now runs nearly as many CAR T trials as the USA. The problem the market now faces is that, despite the huge network of talented companies, the number of CDMOs that are able to help in GCT product development are few and far between. We can help companies achieve their goals through our expedited services. GenScript has supplied R&D grade plasmids to GCT companies since 2004, and continues to serve these companies with process development and GMP manufacturing.
We are a one-stop-shop provider for plasmid and viral vectors, with products ranging from R&D to GMP grade. Our total GCT solutions cover chemistry, manufacturing and control of plasmid and virus for IND filings, as well as clinical and commercial manufacturing. We also ensure phase appropriate compliance, data integrity, and traceability with robust quality management systems.
We currently offer R&D to GMP grade plasmid and viral vectors to support pre-clinical and clinical studies, and in 2022– 2023 our GMP commercial center will begin supporting commercial-scale productions for our increasing pool of customers and projects. These facilities will help us meet customer needs, and signpost our commitment to shortening development timelines – significantly lowering R&D costs and building a healthier future.
Viral vectors are crucial for introducing genetic material into cells – there are over 2,600 ongoing gene therapy clinical trials that rely on them. Developers need a steady supply of lentiviruses and adeno-associated viruses (AAVs), and other viral vectors such as vaccinia, retro, adeno and herpes can be produced with protocol transfer from customer. Stable supply might not be a problem in early clinical trials, but it can become an issue when mass production is needed to address larger trial cohorts. And I believe this could be the biggest bottleneck for the field.
Another challenge companies face is that, though lentiviruses and AAVs are showing promise in the clinic, there are concerns around the potential for side effects. Companies simply must select vectors with ensured safety and purity for their GCT products.
The manufacture of viral vectors is likely to be one of the biggest constraints that developers will face as they shift from clinical trials to commercialization. Avoiding this particular bottleneck is directly linked to accelerated development. Many CDMOs have longer wait and lead times to conduct the GCT product development and manufacturing, and we are able to substantially shorten these time frames. Though some companies may have in-house facilities, choosing an experienced CDMO partner is also important for ensuring regulatory and clinical success while minimizing the burdens of capital investment and running costs.
In the first half of 2020, the number of companies working on regenerative medicine therapies globally passed the 1,000 mark for the first time, with 415 companies entering clinical development. Of these, 515 are developing gene therapies and 632 are developing cell therapies. But the pandemic has affected the progress of many clinical trials, causing delays to regulatory applications and approvals. Even with these challenges, the market is expected to pick up in the second half of the year. At GenScript ProBio, our customers benefit from reliable and uninterrupted GCT manufacturing thanks to our manufacturing capacity located across the globe including the US and China, and we will be there to support our customers through it all.