Some patients who have not responded to traditional medicines are now experiencing remarkable recoveries thanks to next-generation immunotherapies. These therapies equip a patient's own immune cells to recognize, target, and destroy cancer cells. To do this, the patient's cells are collected, modified, and re-introduced into their body - a complex procedure currently available to only a small number of people. With major innovations underway, this fast-moving area of science is set to expand the pool of patients who will respond to immunotherapies and other emerging medicines. But there is a bottleneck in the discovery pipeline. Manufacturing backlogs are slowing the production of cells that are essential to research, holding up the availability of new treatments headed for the clinic.
To address these challenges, a group of Massachusetts academic, healthcare, biotech, and biopharma industry leaders have come together to establish a new center.
The new center for advanced biological innovation and manufacturing will explore and cultivate innovations in cell and gene therapy, advance biologic innovation and manufacturing, and accelerate developments in immunotherapy, cell therapies, gene editing, and other technologies that carry the promise of lasting impact on human health globally and boosting the local economy. By fostering collaboration and innovation, it holds the promise of speeding innovation and broadening the universe of patients that can be served by these emerging therapies.
Leaders from Harvard University, Massachusetts Institute of Technology (MIT), Fujifilm Diosynth Biotechnologies (FDB), GE Healthcare Life Sciences, Alexandria Real Estate Equities, Inc., will comprise the Board of Directors, while other contributing members include Beth Israel Deaconess Medical Center, Boston Children's Hospital, Brigham and Women's Hospital, the Dana-Farber Cancer Institute, Massachusetts General Hospital, MilliporeSigma, and the Commonwealth of Massachusetts.
The $50 million center will be located in the greater Boston area and will be named, along with incorporation, in the new year. The expectation is that this will be an independent, separate nonprofit corporation.
Home to a dense concentration of world-leading universities, hospitals, large pharmaceutical companies and small biotech firms, Massachusetts is at the forefront of biomedicine. These organizations are redefining traditional ideas about biomedicine and rapidly advancing discoveries from lab to clinic.
The overarching mission of the newly established consortium is to catalyze the development of transformative therapies by shortening the path between research and clinical application. The consortium will harness world-leading expertise to propel forward fast-emerging and promising science, the cost and risks of which are daunting for any single institution to tackle alone. By housing institutions with strengths in each link in the "chain of innovation" within one facility, the partners believe new innovations in both science and manufacturing will speed the introduction of new medicines to patients.
The ability of scientists to modify cells for therapeutic application, and to alter disease-causing genes, has ushered in a new era in biomedicine. Some of these potential therapies are entering clinical trials, others will soon be in the clinic, and still more are in early stages of investigation. There is strong motivation and acute need to translate these emergent approaches to clinical use. More than 60,000 patients globally are currently participating in clinical trials for new cell and gene therapies, including gene editing.
Currently, major obstacles and bottlenecks to getting new treatments into the clinic include production - specifically, the pressure placed on highly skilled contract manufacturers to deliver customized cells and viral vectors of high quality and regulatory compliance to labs throughout the region. Because of the backlog, scientists may need to wait as long as 18 months for essential products they need to carry out research.
The center will offer three critical services to the Massachusetts life science ecosystem.
It will provide preferred access to a new manufacturing facility at favorable pricing, reducing the wait and cost for researchers at universities, hospitals and start-ups. The facility offers pharma-grade "good manufacturing practices" (GMP) manufacturing capacity in approximately eight cleanrooms for the production of cell and viral vector products and other related raw materials that may be used for phase 1 or phase 2 clinical trials.
The facility will have a shared innovation space where scientists from universities, hospitals, and industry can work side-by-side with dedicated, experienced, professional staff. This will be a unique opportunity to refine new methods rapidly, readying them for first-in-patient clinical trials. With access to manufacturing within the same space, the center will cultivate a community of experts across sectors who share a goal of serving patients, and who are dedicated to innovating collectively in both manufacturing processes and drug development.
The center will provide a platform for workforce development and training in a rapidly growing field, where there is a critical need for people with specialized skills.
The modular design of the new facility will make it easier for users to adapt quickly to changes in technology. Such flexibility will remove barriers to accessing promising innovations that emerge from improved methods involving gene manipulation, gene editing, oligonucleotides, peptides, and new methods and discoveries as they arise.
While there are many commercial contract manufacturing organizations, shared lab spaces, and even small manufacturing spaces at universities and hospitals in the U.S., this is a first-of-its-kind facility in three respects. First, for its intention to produce both cell and viral vector products within a single physical space. Second, for its unique partnerships between industry, academia, and leading area hospitals. Finally, for its partners' aspirations to provide services to researchers and start-ups that will advance this new area of medicine through collaboration.
"This powerful collaboration embodies the deep and broad world-class expertise in multiple disciplines that exists across this region," said Harvard President Larry Bacow. "We are privileged to be part of this collaborative initiative. It will advance scientific discovery, reaffirm the region's global leadership in the life sciences, and bring forward life-saving and life-changing therapies that will make a difference for people around the world."
"The broad question that we were trying to address was, 'How can we best position our region to be preeminent in the life sciences in the decades to come?'" said Alan M. Garber, Harvard's Provost, who helped conceive of the project more than two years ago and has shepherded it since then. "We have a vibrant life sciences community, with some of the world's greatest hospitals, universities, and life sciences companies of all kinds. We also have a strong financial sector that helps to spawn and support new companies. So the elements for rapid progress in the life sciences -- particularly in the application of the life sciences to human health -- are all here. But with such a rapid pace of innovation, it's easy to fall behind. We wanted to make sure that would not happen here."
"MIT researchers are developing innovative approaches to cell and gene therapy, designing new concepts for such biopharmaceutical medicines as well as new processes to manufacture these products and qualify them for clinical use," said MIT Provost Martin A. Schmidt. "A shared facility to de-risk this innovation, including production, will facilitate even stronger collaborations among local universities, hospitals, and companies -- and ultimately, such a facility can help speed impact and access for patients. MIT appreciates Harvard's lead in convening exploration of this opportunity for the Commonwealth."
Richard McCullough, Harvard's vice provost for research and professor of materials science and engineering, who helped lead the project, said, "the power of facility's partners will accelerate therapeutic discoveries and have the ability to advance biologics from the lab to the bedside."
"It's an exciting time for the life sciences industry with cell and gene therapies in position to revolutionize the global healthcare system. While these therapies are promising, challenges in manufacturing, access and cost must be addressed so they can reach their full potential. Initiatives such as the center are important because they bring together key life sciences stakeholders together to share their capabilities, knowledge and expertise to collaborate and accelerate innovation," said Emmanuel Ligner, CEO and President of GE Healthcare Life Sciences.
"We are very proud to be part of this unparalleled consortium to create an innovative and collaborative center involving advanced technologies as well as next-generation manufacturing. The highly respected partner institutions have the scientific talent and the engineering capabilities to deliver truly novel therapies to patients suffering today from serious and life-threatening diseases and also to design the next-generation processes that will accelerate the translation of tomorrow's cost-effective, lifesaving medicines from bench to bedside," said Joel S. Marcus, executive chairman and founder, Alexandria Real Estate Equities, Inc. and Alexandria Venture Investments.
"We are excited to be a founding member of this consortium. Partnering to get medicines to patients is what we are all about. The opportunity to do this in collaboration with everyone that has come together to make this a reality is something that really meets our core purpose to deliver tomorrow's medicines as a partner for life," said Martin Meeson, President & COO, FUJFILM Diosynth Biotechnologies USA.
Massachusetts' new center for advanced biological innovation and manufacturing will focus first on emergent areas such as cell therapies and gene therapies, and other advanced therapy medicinal products. Cell therapies that help a patient's own immune system target cancer cells have been remarkably successful. One example is CAR-T cell therapy, in which a patient's own T cells are modified to identify and attack cancer cells in the blood more easily. But immunotherapy is not restricted to treating cancers. Scientists are finding new ways to harness the immune system to treat a broad spectrum of diseases, including type 1 diabetes and many others. Cell therapies more broadly - harnessing unique properties of adult stem cells, for example - are under wide consideration for regenerative medicine, including joint tissue repair and neurodegeneration.
Gene therapies offer new hope to patients, often children, who suffer from debilitating inherited diseases. They involve introducing, removing, or changing a targeted gene within a patient's cells. The goal is to make the patient's cells produce disease-fighting proteins, or to stop them from producing disease-causing versions of a protein. Gene-editing research is progressing very rapidly, but there is a marked shortage of capability for manufacturing the gene delivery vectors.
Hospitals need to be able to create customized therapeutics for their patients, but most do not have manufacturing facilities on-site. Beyond the constraint of limited facilities to produce potential new treatments, much technological innovation is required to produce these medicines more efficiently - in terms of time, labor, and cost - and in accordance with regulatory guidance. The new center would be equipped to handle some of this work for technology innovation and early stage clinical trial-scale production, which would directly help bring promising solutions to patients sooner.
"Scientific breakthroughs in cellular, immune and gene therapies from just the past few years are now saving lives and represent a truly revolutionary time in medicine," said Laurie H. Glimcher, MD, president and CEO of Dana-Farber Cancer Institute. "By bringing together the talent that exists only in the Massachusetts life sciences ecosystem and fostering collaboration, this new manufacturing center will help to extend the benefit of these technologies to more patients and accelerate discoveries to effectively treat more diseases."
"We need more manufacturing capability in order to translate our work, especially in the stem cell field," said Leonard Zon, MD, director of the Stem Cell Research Program at Boston Children's Hospital. "For academic investigators who want to see their basic science advance into the clinic space, it's important to have a manufacturing facility collaborate on protocols. Researchers can then exchange information directly with the facility, optimizing protocols and working smarter."
"This collaboration represents an exciting opportunity to harness the collective efforts of leading academic, industrial and clinical institutions to further explore exciting new technologies and therapies that are inspiring scientists and offering new hope to our patients," says Peter L. Slavin, MD, MGH president. "New scientific fields like regenerative medicine, gene editing and immunotherapy are unlocking clues to understanding disease which can lead to better treatments and ultimately, richer, more healthy lives for our patients and their families."
"Our mission at Beth Israel Deaconess Medical Center is to provide extraordinary care supported by world-class research and education," said Peter J. Healy, president of Beth Israel Deaconess Medical Center. "We are happy to be a founding member of this innovative consortium, which will allow us to work collaboratively across the diverse health care ecosystem. Together, we will propel the fields of cell therapy, gene therapy and gene editing forward with the shared goal of transforming how we care for patients right here in Boston and around the world."
"Boston is an epicenter of biomedical research and innovation," said Brigham Health president Elizabeth G. Nabel, MD. "In furthering the Brigham's commitment to advancing development and delivery of cell and gene therapies, this unique collaboration is an opportunity to accelerate the pace and broaden the manufacturing capacity for therapies that have the potential to significantly improve patient outcomes."
"Never before have we had so many breakthroughs available in the clinic. However, it can take up to 30 days, 'needle to needle,' to deliver a CAR-T therapy to a patient, and that does not take into account any of the bottlenecks in the supply chain that could occur along the way. It is our collective responsibility to eliminate any barriers to making these life-saving medicines accessible to patients everywhere," said Udit Batra, CEO, MilliporeSigma.
"The Commonwealth's life sciences ecosystem is thriving because of the strength of the academic, research and industry partners that call Massachusetts home, and their commitment to collaboration," said Secretary of Housing and Economic Development Mike Kennealy. "Combining a manufacturing facility, co-working labs, and workforce development and training in this first-in-the-nation center will boost the regional economy, create jobs and accelerate the delivery of next-generation therapies."