BIOTECNIKA BLOG

  New center for innovating and manufacturing next-generation
medicines planned for metro Boston

Harvard, MIT, teaching hospitals, industry partners pool resources to
create a central facility for developing regenerative therapies

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 an independent non-profit organization
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 consortia. 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."

For more information about the types of research and biotechnology
that may occur at the Center, please see the following story in the
Harvard Gazette.

https://www.harvard.edu/media-relations/new-center-for-innovating-and-manufacturing-next-generation-medicines-planned-for