The Building of an Enterprise: Regenerative Medicine Poised for World Stage

Diane Gates Wallach has a head for business and a heart for science. When she pursues both – blending her knack for strategy with a desire to better the world – her imagination comes alive. No frontier looks insurmountable when the right talent is involved.

Wallach inherited those traits from her father, Charles C. Gates Jr., who took the helm of the family business, Gates Rubber Co., after the death of its founder, Charles Gates Sr., in 1961. In 2005, having struggled with macular degeneration and battling aggressive pancreatic cancer, Charles Gates Jr. set in motion a pioneering stem cell program at the up-and-coming University of Colorado Anschutz Medical Campus. Charlie, as he was known by many, asked Wallach and her brother John Gates, co-trustees of the Gates Frontiers Fund, to assume the lead on turning the dream – seeded with a $6 million gift – into reality.

Along with their father, who died in August 2005, they launched a journey into a relatively new area of medicine – stem cell therapy, which takes diseased cells and genetically reprograms them to exploit the body’s ability to heal itself. The endeavor has relied on the smarts, collaboration and investment of scores of visionaries – from philanthropists and hospital administrators to scientists and campus leaders.

Their efforts created the pre-eminent regenerative medicine enterprise in the Rocky Mountain region – one that, now 15 years later, is positioned to become a world leader in moving discoveries from the laboratory to the clinic.

From program to institute

The pandemic ushered in a time of reflection and refocus, leading the collection of dedicated people who brought the Gates Center for Regenerative Medicine this far to look to the next horizon. It resulted in a reboot: the establishment of the state-of-the-art Gates Institute, funded by an expanded $200 million partnership between the Gates Frontiers Fund and CU Anschutz over the next five years.

Terry Fry, MD, a pediatric oncologist and renowned expert in CAR-(chimeric antigen receptor) T cell therapy, will become director of the new institute this fall when the expanded business plan is finalized. The original Gates Center director Dennis Roop, PhD, will stay on as associate director.

“I expect they’re ready to take off,” said David Norris, MD, professor and chairman of the Department of Dermatology at the University of Colorado School of Medicine, who was on the team that recruited Roop to CU Anschutz in 2007. “Terry Fry has tremendous experience in both NIH (National Institutes of Health) and industry laboratories and collaborations between campuses and industry, and he will be able to run this operation, with the help of many other capable and experienced people, including Dennis Roop.”

A partnership in lockstep

Wallach and Roop, the inaugural director, have been partners at every juncture of the ambitious enterprise – from the Gates Stem Cell Program in 2007 to the Gates Center for Regenerative Medicine in 2010 to the Gates Institute today.

Press conference with Diane Gates Wallach in August 2006, after Dennis Roop, PhD, accepted the offer from the University of Colorado to direct the Gates Stem Cell Program.

Wallach and scientific colleagues describe Roop, a man with a touch of a Southern drawl (he put in two stints in molecular biology labs in Texas, both at Baylor College of Medicine), as visionary, tenacious, risk-taking, enthusiastic and charismatic. The history of the Gates Center is filled with many “we took a chance” stories, and they begin with Roop.

“I think there’s no question that the entire enterprise, funded by the Gates family and other philanthropists over the last 15 years and led by Dennis Roop, has progressively become a real game changer,” said Norris, who met Roop decades ago at an annual investigative dermatology meeting. “They’ve been able to take unique resources and apply them to a unique project.”

Norris tried to recruit Roop to various positions over the years, but nothing was a fit until the launch of the stem cell program.

Dynamic leader takes the reins

Roop’s first steps included the recruitment of a cadre of talented researchers in dermatology and other disciplines and the expansion of his study into Epidermolysis Bullosa (EB), a fatal genetic skin disease with no cure.

“Dennis tapped resources at the NIH, the NCI (National Cancer Institute) and foundations, including the University of Colorado Foundation, and gave a good example of how you can raise funds to support translational research – even in the so-called valley of death in the life of a translational research project where it’s difficult to translate basic research findings into clinical practice,” Norris said. “His first big accomplishment was great recruitments, building his own research program and then getting others to be involved in various programs at the Gates Center – the Gates Grubstake Fund, the Graduate Program, the Gates Summer Internship Program and other programs.”

Roop’s indefatigable enthusiasm fueled the innovative programs and ultimately the linchpin milestone – development of the Charles C. Gates Biomanufacturing Facility.

Stuart Yuspa, MD, a mentor of Roop’s at the NIH, where Roop ascended from post-doc to tenured faculty over eight years, said Roop was a personable and upbeat colleague as well as a pioneer in molecular skin biology. “It wasn’t just the science he brought to the lab; he brought a charisma that changed everybody’s behavior,” Yuspa said. “He generated a lot of excitement every day, a lot of laughter. He sometimes broke the rules” for the sake of team building, including his famed Friday afternoon on-campus happy hours.

‘It went from nothing to a $200 million state-of-the-art pioneering institute that

will shape medical therapy and research for decades.’ – Stuart Yuspa, referring to

the Gates Stem Cell Program and Gates Center under Dennis Roop’s leadership.

While sad to lose his dynamic colleague, Yuspa said he knew Roop’s personality wasn’t suited for the NIH’s restrictive atmosphere. “We had a lot of advantages, a lot of benefits,” Yuspa said, “but we also had a lot of restrictions, so Dennis went to Baylor and started his own lab there.”

Yuspa said “it’s simply remarkable” what has happened at the Gates Stem Cell Therapy program and Gates Center since Roop became director. “It went from nothing to a $200 million state-of-the-art pioneering institute that will shape medical therapy and research for decades,” he said.

Roop arrived at CU Anschutz from the Baylor College of Medicine in Houston, which had a GMP (Good Manufacturing Practices) facility that manufactured cellular and gene therapies. He knew CU Anschutz would need an in-house facility as well, especially as pioneering discoveries, such as Fry’s CAR-T cell therapy, were emerging and required the GMP clean-room technology.

Recruiting talent, pioneering discoveries

Roop and Wallach developed a strong partnership in which the scientist would candidly tell the philanthropist when an infusion of money was needed to lubricate the center’s gears to keep things moving.

“Dennis chipped away at this stuff. He was just dogged; he didn’t let go,” Wallach said. “He came to me multiple times along the way and said, ‘Look, we just have to have this person, or we have to do this now, or we’ve got to take a chance doing it two years earlier than we thought we were going to do it, because there’s this little window of opportunity.’ And we would just lock our arms and take those next steps.”

His recruits included Valeria Canto-Soler, PhD, whose research aims to develop cell-based treatments for patients suffering from vision loss, including macular degeneration, and the dermatological research team of Ganna Bilousova, PhD, and Igor Kogut, PhD, who developed a more efficient approach to reprogramming a patient’s diseased skin cells into stem cells.

The creation of the Charles C. Gates Biomanufacturing Facility (GBF) was necessary to attract a scientist of Fry’s caliber – and it’s a story unto itself.

Getting on the map

In 2013, when a company located in a new wing of the Bioscience 1 Building in the Fitzsimons Innovation Community suddenly was unable to obtain next-round funding and had to vacate the newly renovated space, Roop saw a window. Wallach was headed out of town on a trip, but Roop called and said, “We now have the space (for a GMP).”

Wallach knew the GMP was essential. “We can’t be on the map without one, but we never thought we had the resources to do it sooner instead of later. And then this whole opportunity opened up, and Dennis said, ‘We have to do this. I know it’s risky, we’re going to stumble, it’s going to be messy, but we have to do this.’ And so, we did it.”

It began a six-year process, including the securing of key on-campus investment partners: UCHealth University of Colorado Hospital, Children’s Hospital Colorado and the University of Colorado School of Medicine. The Gates Center secured a 15-year lease and $8.8 million in commitments from donors, foundations and campus partners to renovate, equip, retain and operate the GBF through 2018. Today, the GBF is cost-neutral, covering operations through contracts with nonprofits and for-profit industry (made possible through its Fitzsimons Innovation Community location) and producing both cell-based therapies and protein-based therapies (biologics).

Exhaustive rounds of number crunching, strategic planning and negotiation undergirded the facility, the only one of its kind within a 500-mile radius. Patrick Gaines, former executive director of the Gates Center, recalled the process as being a pivotal juncture in the center’s history. He huddled repeatedly with Tim Gardner, then-Gates Center chief financial officer, and Jill Cowperthwaite, Gates Center executive director, to create a GBF business plan.

“Dr. Roop relied on us to do market studies and research, and it’s proven to be accurate,” Gaines said. “In the early stages, around 2015, there were some competing voices out there saying, ‘We can do this (for you). We can do it cheaper.’ And after running due diligence, we turned out to be right: (outside entities) didn’t have quite the capabilities that we needed, and now Dr. Fry bringing his lab here was a big part of validating that.”

Roop provided his usual balance of scientist, business director and medical liaison during the negotiations, Gaines said. “He has a unique blend of skills and an ability to talk face to face with donors and understand their needs.”

‘Making a difference’

The key donor, as usual, was the Gates Frontiers Fund, but there were other community leaders who also stepped up, expanding the sources of support.

“It was very difficult to get the GBF launched and certified, but now you look at it, and it’s just essential, and they’re doing a great job,” Wallach said. “They’re making a difference – contributing to clinical trials and Terry’s work. It’s worked out well.”

The Gates Center currently has 126 member investigators who are affiliated with CU Anschutz, CU Denver, CU Boulder, Colorado State University, National Jewish Health, Colorado School of Mines and private industry.

The Gates Institute will offer a robust regulatory team that “removes friction from the system” and allows the safe and efficient practice of translational science, Roop said. “There is no reason beyond appropriate safety and scientific protocols that investigators at the Gates Institute should have to wait to get their potential therapy into clinic. It should be a well-oiled machine that you just slot into. You’ll have the people with the expertise to help draft the FDA (Food and Drug Administration) documents you need to get approval for a clinical trial.”

Dennis Roop, PhD, founding director of the Gates Stem Cell Program, with Terry Fry, MD, who will become director of the new Gates Institute this fall. Roop will continue with his stem cell research projects, including his groundbreaking work toward therapies for inherited skin fragility syndromes, in the position of institute associate director.

Fry is excited about the substantial groundwork that’s been laid by Roop and his team. “We’re not starting from scratch,” he said. “What we’re really doing is connecting all of these strengths to launch an institute that is going to be amongst the leading programs in the world.”

Norris said Roop’s leadership and contagious enthusiasm has guided the Gates Center through regulatory hoops, lab-space problems, budgetary issues and other challenges. He has also been helped by the steadfast support of CU Anschutz Chancellor Don Elliman and John J. Reilly Jr., dean of the CU School of Medicine and vice chancellor for health affairs.

A promising future, reaching the next level

“We’re now looking at a future of energized leadership, the continuity with the leadership of Dennis Roop, and a facility that will service multiple investigators and programs on campus,” Norris said.

Wallach is likewise thrilled to see cutting-edge clinical trials rolling out in her home state and the promise of beating the most serious and stubborn diseases – macular degeneration and various cancers among them – with these discoveries. The Gates Institute is envisioned to further break down silos and operate in an integrated and entrepreneurial environment that’s needed to take stem cell therapy to the next level.

“This was a baby program when we started – I mean $6 million sounds like a lot of money, but it takes big investments to really move the needle in medicine,” she said. “It was a start, and everything we saw in Dennis suggested he would be great – and he has been great. Anytime you start something, you can’t anticipate all the roadblocks that are going to come your way. This is just a new space.”

In the still-fresh space, Wallach is certain her father would be ecstatic to see the fruits of his vision. “I just know he’d be tickled seeing that this endeavor is still going, and we’re really dipping into the things that he wanted to see happen in big ways.”

Photo at top: From left, CU Anschutz Chancellor Don Elliman; Terry Fry, MD; Dennis Roop, PhD; and Diane Gates Wallach at the new Anschutz Health Sciences Building.