New Jersey Innovation Institute's BioCentriq Aims to Usher in a New Era in Cell and Gene Therapies

To overcome production hurdles for cell and gene therapies, a promising new class of biologic medications, NJIT’s New Jersey Innovation Institute (NJII) has opened clinical-scale centers that will work directly with drug developers to streamline the processing of new therapies and manufacture them in large quantities.

NJII’s new company, BioCentriq, was formed in collaboration with leaders in the pharmaceutical industry, top regulators at federal agencies that oversee biologic therapies and state economic development officials. It is the first such enterprise in the nation backed by a university.  

“Our goal is to provide a collaborative space where innovative approaches and technologies can be used to help make emerging therapeutics available to the patients that so desperately need them,” said Haro Hartounian, senior vice president and general manager of NJII’s biopharma division and at BioCentriq, at a virtual ribbon-cutting in late October hosted by the economic development organization, Choose New Jersey.

The two classes of therapies work by reprogramming the body’s immune system at the cellular and genetic level, respectively, to fight diseases such as cancers that don’t respond to other medications. In some cases, they are derived from a patient’s own, or autologous, cells that are extracted from the body, reengineered in the laboratory and then reimplanted. They bind with specific molecules called antigens on the disease cells to neutralize them and alert other defender cells.

The challenges in developing and manufacturing these drugs consistently and at the necessary scale has slowed their entry to the market, however. These difficulties have led to prohibitive costs that keep them out of reach for many patients.

“As unique as the people they come from, each collection of cells behaves differently in the manufacturing process. This variability causes consistency concerns, which is why we are invested in supporting the development of new technologies that help us understand and overcome these differences,” Hartounian explained.

As to transportation challenges, he added, “Autologous therapies use cells that originate from and are directly returned to the patient in what is referred to as the ‘needle-to-needle’ process. Since manufacturing typically occurs at a centralized facility, this means that sensitive biological material is transported from the patient to the manufacturing site and back again, which requires strong coordination between the patient, doctor, specialty currier and manufacturing site.”

BioCentriq seeks to speed up the production of medications, while maintaining the highest clinical safety standards. The team will develop technologies to automate more of the development process and to increase continuous, rather than batch, processing capabilities. Limiting human interactions with materials in the lab will also reduce the risk of contamination.

In 2017, the Food and Drug Administration (FDA) approved the first gene therapies, known as CAR T-cells, to treat acute blood cancers. Calling cell and gene therapies “the wave of the future,” Peter Marks, M.D., the director of the FDA’s Center for Biologics Evaluation and Research, remarked at the virtual opening ceremony that they are poised to “change how we do medicine.”

He added, however, “The science has moved ahead magnificently over the years, but the technology has not kept pace – that is how we go about making these things – and needs to be brought along.”

“The space is so new that we see people making the same mistakes over and over again,” noted Janet Lambert, CEO of the Alliance for Regenerative Medicine, who took part in a panel discussion at the opening. Lambert said she viewed BioCentriq as a “home-base for learning” as companies develop their first biologic drugs in this class.

BioCentriq is composed of two facilities. At a process development center in South Brunswick, the company’s scientists genetically modify cells, inducing them to produce specified proteins, or antibodies, that will bind with antigens on the disease cells. For viral gene therapies, they then extract the viral particles, which are then processed, purified and infused into the patient.

“What we do in South Brunswick is essentially a pilot test of our production process for each drug, which we then move to our cleanroom facilities on the NJIT campus for large-scale clinical manufacturing. It is only in these highly sterile conditions that we can make materials for use in the human body,” Hartounian explained. The clinical manufacturing centers on the NJIT campus, one for gene therapy and one for cell therapy, are FDA-compliant Current Good Manufacturing Practice manufacturing facilities.

The company, which is currently working on three projects with large, public pharmaceutical companies, two projects with mid-sized companies, and one with an innovative startup, takes what Hartounian calls a “hotel” approach,” meaning it will take on full duties as a contractor or invite a company’s own scientists to work alongside BioCentriq technologists in the lab.

“The innovation sector has been and will continue to be key to our recovery,” said Gov. Phil Murphy, at the virtual opening. “New Jersey was Silicon Valley before there was a Silicon Valley. We have more scientist and engineers per square mile than anywhere in the world.”

The company’s advisory committee consists of industry veterans such as Joanne Beck, the chief operating officer of Boston Pharmaceuticals; Kelvin Lee, the director of the National Institute for Innovation in Manufacturing Biopharmaceuticals; and Steve Dziennik, the director of Global Technical Operations Biologics at Merck. Marks serves as a liaison to the FDA.

“There is a massive need for what BioCentriq can provide, and our ability to fill that gap will translate into results that literally save and improve the quality of countless lives,” said NJIT President Joel Bloom. “We have the capacity to help companies develop processes, conduct clinical production trials and train employees.”

In conjunction with BioCentriq, NJIT has also launched a 30-credit master’s program in cell and gene therapy, as well as a related four-course, 18-month apprenticeship certificate program.

“In addition, we have ongoing collaborations with big pharma clients to train their incoming workforce. These programs have been tremendously successful and have been going on for more than a year and a half,” said Kevin Belfield, dean of NJIT’s College of Science and Liberal Arts.

“Access to workforce is really limiting. You see the fierce competition for anybody of talent,” noted Lee.

NJII also offers an incubator program called the BioFoundry, which helps startups and other companies scale up production and connects them with capital, vetted workers and technology professionals who can provide mentoring.