Biotech Connector reimagines the future of gene and cell-based therapies

The recent Biotech Connector showcased exciting advancements in gene and cell-based therapies in the Frederick area. More than 80 attendees gathered in-person and online to hear experts in the field discuss their work manufacturing complex therapies for clinical trials, reimagining the supply chain to reduce development timelines, and leveraging automation to reduce risk and save money.  

Cancer therapies 

Vinay Vyas, Ph.D., associate director of the Biopharmaceutical Development Program at the Frederick National Laboratory for Cancer Research, presented ongoing efforts to develop chimeric antigen receptor T cell (CAR-T) and T cell receptor T cell (TCR-T) cancer therapies. 

CAR-T cell therapies genetically modify a patient’s own immune cells (T cells) to target and destroy cancer cells. TCR-T cell therapies harness the power of T cell receptors to recognize and eliminate cancer cells. Manufacturing these therapies requires extensive expertise and resources. 

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“We work like a boutique biopharmaceutical company,” Vyas said. “We have our own quality control. We have our own quality assurance. We have a manufacturing unit and a process development unit.” 

As a result, the Biopharmaceutical Development Program can turn the collected patient’s blood cells into a usable CAR-T therapy in about three weeks.   

Vyas discussed 13 of the program's CAR-T/TCR-T projects, several of which are supporting ongoing clinical studies at the National Institutes of Health Clinical Center. For the first effort, which launched in 2018, the Biopharmaceutical Development Program used an externally developed viral vector, but since then, the program has developed its own viral vectors, both lentiviral vector and retroviral vectors, in house. Viral vectors are modified viruses that deliver the genetic alteration to the T cells. 

“The engine of CAR-T is typically a viral vector,” Vyas said.   

Scaling manufacturing and supply chain 

Jon A. Rowley, Ph.D., founder and chief product officer of RoosterBio Inc., focused on how to make cell and gene therapy technologies scalable, efficient, and reproducible.  

“We started Rooster Bio really to reimagine a highly fragmented supply chain in cell gene therapy,” he said.  

His talk focused on mesenchymal stromal cells (MSCs), which are stem cells that can differentiate into a variety of cell types, including bone, cartilage, muscle, and fat cells.  

“MSCs are really a workhorse of regenerative medicine,” Rowley said.  

He explained how good manufacturing practice (GMP) plug-and-play bioprocess platforms, such as those created by Rooster Bio, are enabling rapid translation of MSC product concepts from idea to the clinic, shortening the overline development timeline and reducing production costs. 

The typical timeline to get advanced therapy products to first-in-human testing is about seven to 11 years. However, he said using a scalable GMP MSC platform could shorten the timeline to two to four years.  

Automation of manufacturing processes  

Manufacturing cell and gene therapies requires multiple steps, and manually handling and transferring between steps and devices can increase the risk of errors and drive up costs.  

Brian Paszkiet,  principal clinical specialist at Miltenyi Biotec, presented on how automation can reduce these risks and improve efficiency, consistency, and scalability of cell and gene therapy processes. He focused on the CliniMACS Prodigy®, which offers end-to-end enclosed automation. 

“It automates everything from the beginning to the end of the process, starting with a leukapheresis product or a blood product; washing it; labeling it with the magnetic beads; isolating the different fractions of cells; also enabling activation, transduction, and cell culture; and then final harvest and formulation,” Paszkiet said.  

He said the automated plug-in-play platform can be used for a variety of cell types, including CART-T cells, natural killer cells, and engineered hematopoietic stem cells, and it comes with pre-installed standard manufacturing processes.  

Paszkiet said, “I could train your kid how to do it… it’s really engineered in a way that makes it very easy to install, and it’s all guided on the screen.” 

Join us in February  

The Frederick National Laboratory for Cancer Research and the Frederick County Chamber of Commerce organize the quarterly Biotech Connector Speaker Series. This event provides an inside look at local advances in Frederick County and the surrounding areas’ biotech and bioscience community. Save the date for our next event on Thursday, February 19, which will cover AI-powered and computer-driven drug development.