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Enzyvant Stories | February 2021

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Leading the Way in Regenerative Medicine Manufacturing

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Larry Weiner
Vice President Pharmaceutical Development and Manufacturing at Enzyvant

Larry Weiner leads Enzyvant teams at the forefront of regenerative medicine manufacturing, preparing for the potential shift from manufacturing an investigational tissue-based regenerative therapy used in clinical trials to one that is commercially available. Speed matters as these teams urgently develop the processes, tests and measures, people and facilities to comprise a robust manufacturing and quality system that meets the rigorous GMP (Good Manufacturing Practices) standards of the U.S. Food and Drug Administration (FDA).

“It is important to me that my work touches lives and makes a meaningful impact on the world.”

Larry Weiner has had a lifelong passion for science and the ways it can be applied to help improve lives. After graduating from Tufts University, he went on to work in one of the earliest commercial biotech companies in the United States. “I really wanted to do something that was going to help society and I began to see how engineering and biology could come together to deliver really important therapies for patients,” says Larry.  “It was impactful work because the industry as a whole was figuring out how to apply engineering to biotech. I was fortunate enough to be in the first wave of technology scale-up at a company that was already getting innovative biotech products approved and accessible to patients.”

In a career that has spanned three decades, Larry has developed game-changing manufacturing for novel pharmaceuticals and biotechnologies. Now his significant experience is being applied to the innovation of manufacturing and quality solutions in the rapidly emerging biotech sector of regenerative medicine.  At Enzyvant, the investigational regenerative therapy is tissue-based and the biomaterials are highly complex. This potential allogeneic therapy is made for one patient at a time and handling of the tissue is a meticulous, complicated process that adapts cell culture and analytical methods to multicellular tissue.  Because this type of regenerative therapy represents new science, a tremendous amount of innovation is also happening around the manufacturing processes, as well as tests for process validation and quality control.  Often with science that is this new, it is not only the tests themselves that must be developed but also the markers that measure consistency, potency and quality that must be identified.

“Being part of a winning team with significant goals makes work exciting and meaningful. The work we do at Enzyvant has so much promise because when we achieve our goals, we can help change children’s lives. As a parent, I ask myself ‘What could possibly matter more?’”

All the tasks, projects and detail that go into bringing a complex manufacturing system online add up to a dramatic scope of work. With any meaningful goal worth pursuing and one this intricate, there are huge challenges that are greater than any one person alone can overcome.  However, Larry points out that big challenges don’t always mean you need a large team with enormous resources. “Big challenges need diverse teams with many different perspectives, backgrounds, skills, and ideas, with a passion to be far more than just the sum of their parts” he says.  “Talented teams that are motivated to achieve something truly extraordinary for patients will solve the one big thing today and another tomorrow and so on until we reach the audacious goal. When that happens, it’s incredibly satisfying.”

“Developing regenerative medicines quickly and adhering to very high standards in manufacturing and quality are not tradeoffs. We must do both and we should because it’s the right thing to do for patients and because doing things right the first time is much more efficient.”

Regenerative medicines are receiving remarkable attention right now because of their potential to be curative or near-curative.  Designations, such as RMAT (Regenerative Medicine Advanced Therapy) were created by the FDA to provide expedited review and approval pathways for regenerative medicines because of the promise they represent to patients with rare diseases who have few to no treatment options today.  Speed matters to patients in desperate need.  As Larry and his team work to prepare Enzyvant manufacturing to meet the intense GMP standards of the FDA, they must do so as fast as possible. “Regulators are doing a great job holding us to very high standards and we have to meet those standards,” says Larry.  “That’s a big part of my focus and the work our teams are doing every day.  Both the company developers of regenerative medicines and the regulators have shared goals here. We all want to get manufacturing and quality systems to the best possible place in terms of consistency, reliability, repeatability, safety and efficacy.”

For Larry and the teams working to make regenerative medicines available to patients, the urgent need they are working feverishly to address is palpable. “Our investigative therapy, if approved, will be for infants and very young children with a devastating rare disease,” says Larry. “I’m a parent and it’s on my mind every day that children’s lives are on the line. Families are counting on us and we must deliver on this work quickly and with excellence.”

In the biotechnology industry, being the first to bring a breakthrough new treatment over the finish line is a meaningful accomplishment not only for patients but also to many other stakeholders, including investors. “You can only win that race if companies put quality before schedule or cost,” says Larry.  “It sounds counterintuitive but if you focus on schedule or cost first it tends to eclipse quality, you have more problems, and it takes longer and costs more in the end.  If you invest in manufacturing and quality systems early on in the development process you’re putting quality first and that actually gets you across the finish line stronger and faster.” Regenerative medicines are made from cells, genes, or tissues. Enzyvant’s investigational regenerative therapy is made from human tissue for each individual patient, so timing and precision are crucial. “If a manufacturing batch fails, a child cannot be treated at that time and that is unacceptable,” says Larry. “Our manufacturing processes must be reliably precise each and every time because precious lives are at stake.”

“The projects and challenges we face in regenerative medicine are not easy things. The people who can challenge the status quo, who have different ways of thinking, the skill sets to get to solutions, and the ability to execute efficiently are the ones who really thrive in this industry.”

Collaboration is fundamental to innovation in biotech. Diverse and nimble teams can break down complex projects into discreet activities and component parts.  This requires trust and support, breaking the whole into achievable milestones, efficient decision-making, great communication, working from the right data, and weeding out activities that don’t add value to the outcome.  But it can be challenging to keep teams invested in goals over long time horizons often spanning years so they stay inspired to win the ultimate prize.  According to Larry, painting a clear and tangible picture of what great looks like is the best way to keep teams inspired. “When we see what’s ahead with absolute clarity, that the ultimate stage of the process is about bringing safe and effective treatments to patients in need, everything comes into frame,” he says. “Then it’s not a line in the sand that can be moved.  You can make the hard decisions required to accomplish the mission. When the lens is in full focus for teams, you see clearly the faces of all the people who need our help. You see what really matters and then we’re unstoppable.”

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