Advances In Manufacturing Key To Making Cell And Gene Therapies Accessible

According to a June 2019 report published by a team from the Center for Biomedical Innovation at Massachusetts Institute of Technology, about 350,000 patients will have been treated by 30 to 60 cell and gene therapies by 2030. At the time of the report, The Citeline Pharmaprojects database provided a pipeline of 628 active, individual, U.S.-based clinical trial programs for durable gene and cellular therapies comprising 211 indications, 335 preclinical studies, 91 phase I studies, 174 phase II studies, and 19 phase III studies.

For these innovative and novel therapies to become accessible to the patients who desperately need them, they must successfully move through all phases of clinical trial and ultimately, prove to be commercially viable.

The challenges in the development and manufacturing of cell and gene therapies are not insignificant, however.

Understanding Industry Challenges

I was approached in 2015 by the New Jersey Institution of Technology, one of only 32 polytechnic universities in the United States, to work with the New Jersey Innovation Institute (NJII) a NJIT-owned 501(c)(3) company. I was given the task of exploring how we could leverage the vast resources of the university to help advance the biopharma industry and bring more regenerative medicine to market.

The first thing I did was talk to my colleagues. After 25 years in the industry, I am fortunate to call many of the leaders in this growing industry my friends. I knew I could rely on them to provide me with the insight I needed to figure out where we should focus our energy and time.

To a person, they all told me the primary challenges they faced were in the manufacturing of cell and gene therapies. These showed great promise, but to this day, are labor-intensive, manual, highly dependent on skilled resources, and very expensive.

We decided to focus on providing a viable alternative to traditional contract manufacturing options for both process development and clinical manufacturing. In addition to that, we added to our mission, working to support and build the workforce and companies of the future.

As we all know, once a therapy is developed in the lab, there is a big leap in cost, staffing, and equipment needed to scale-up production and move to clinical manufacturing. Handling human trials safely and effectively requires experts to help with process development, then more experts and well-equipped, validated labs to help with clinical manufacturing.

We also recognized that cutting-edge cell and gene therapies are slated for years of growth, so capacity is going to be an ongoing issue. And a tremendous challenge for this growing segment is cost. The list price per patient right now ranges from $500,000 to $4 million — a price point that is clearly challenging for payers to absorb.

Developing Specific Solutions

We examined these challenges extensively with industry veterans, academics, and government regulators, as well as our own advisory committee, led by Chairperson Dr. Joanne Beck, the chief operating officer of Boston Pharmaceuticals.

The committee includes luminaries such as Dr. Kelvin Lee, the director of the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL); Mayo Pujols, the global head of NTO Cell & Gene Therapy, Oncology, at Novartis; and Dr. Steve Dziennik, the director of Global Technical Operations Biologics at Merck. Peter Marks, the director of the FDA’s Center for Biologics Evaluation and Research (CBER), serves as a liaison to the agency and shares his thoughts.

With their support and the support of our parent University, NJIT, the decision was made to build  BioCentriq, an industry-centric cell and gene therapy development and manufacturing center with a mission far beyond achieving target profit margins.

BioCentriq operates differently than any other CDMO or university research center in three key ways.

First, we are a non-profit. Our mission to advance the industry is reflected in our projects, partners and pricing models. Our primary objective is to support the future of the industry and its members. We are driven by far more than profit margins. We welcome discussions with all members of this ecosystem.

Second, our goal is to find opportunities for improvement in efficiency and effectiveness through the application of innovative technologies, processes and approaches. For that reason, we are vendor-agnostic and fortunate to host equipment and technology from all the major suppliers, many of who made generous in kind donations to our center.

Third, it is clear to us that one of the specific challenges for the industry is attracting, training and retain talent. For that reason, our clients can engage us in a traditional fee-for-service manner or a “hybrid” team model where our experts work collaboratively with theirs to ensure knowledge transfer. Embracing transparency and involving stakeholders in manufacturing, and the process development work that precedes it not only builds trust, but also aides in the development and sharing of knowledge.

We’ve also worked directly with our parent NJIT to develop both a Professional Science Masters (PSM) degree program and a professional graduate certificate in cell and gene therapy. Lastly, we offer customized training programs for many of our partners.

Fueling Advances in Manufacturing

BioCentriq is the only process development and clinical manufacturing organization (CDMO) in America backed by a university, and the first facility of its kind in Newark, NJ.

Cell and gene therapies are revolutionizing the field of medicine by treating chronic and deadly diseases by targeting root causes. But the industry is hindered by high costs and underdeveloped manufacturing models. Efficacious, cost effective product development starts by building a lean manufacturing process.

We partner with biotech startups as well as multinational pharmaceutical conglomerates and help them both address the specific process development and manufacturing challenges for their projects and their indications.

Our labs are brand new and very well equipped, and since any new work we take on is an opportunity for our professional and student staff to expand their knowledge, we’re also willing to procure specialty equipment should a client need it for a project.

This is how, as an industry, we will advance manufacturing process and technologies to efficiently and effectively produce both cell and gene therapies.

Creating an Economic Impact

In addition to the collaborative efforts underway inside our organization to solve today’s challenge, NJII is home to the BioFoundry, created as a result of a grant we received from the US Economic Development Administration. Through the BioFoundry, we bring together a network of partners to expand a nascent cluster of biopharmaceutical startups in New Jersey, including those working on cell and gene therapies and biologics.

The network will: 1) scout and match emerging research discoveries at top universities in the region and throughout the US; 2) nurture and curate new ventures with both technical and business support; and 3) connect and expand the innovation ecosystem – including funding, facilities, and mentoring – to support increasing economic activity within New Jersey in this fast-growing sector.

The project’s efforts will concentrate on the “commercialization” stage of technology innovation while maintaining tight connections with the many “proof of concept” resources in the state to ensure a continuum of new and growing businesses in the cluster. By the end of the three-year grant period, NJII anticipates the project will have created 15 new companies that support 75 new jobs, with another 45 new companies in the pipeline.

Improving Accessibility and Patient Outcomes

It is glaringly clear that in order for these innovative therapies to be accessible to patients the manufacturing process has to become more efficient, the workforce must be capable of meeting demand and the cost of treatment must be plausible. We believe that the approach we are taking to create an innovative development and clinical manufacturing center with heavy input on design and technology from a broad range of industry leaders will help.

In addition, we are committed to doing what we can to address the skills shortage and talent gap. Lastly, we are funded and dedicated to supporting the newcomers and start-ups that enter this dynamic and growing field. Ultimately, we hope to help the industry deliver these promising treatments and improve the outcomes for hundreds of thousands of patients suffering from currently untreatable disease.

About the Author

Dr. Haro Hartounian leads the NJII’s Biopharma Division, working with colleagues in industry and academia to help biopharmaceutical companies transform discovery into commercial production. He has 30 years experience in biotechnology working for companies such as DuPont, Monsanto, SkyePharma and Vyteris. He contributed to three FDA-approved product launches ⁠— DepoCyt and DepoMorphine. He also founded the companies MicroIslet, Protagenic Therapeutics, and DiaVacs and has worked in a variety of product lines ⁠— including cell therapy, oncology, women’s health, pain management and drug delivery.  Visit www.biocentriq.com or email biopharma@njii.com to reach him or learn more about the work underway at NJII.