The recent expansion of pharmaceutical company Roivant Sciences’ Durham office marks the growing relationship between the company and the Duke community.

Led by CEO and founder Vivek Ramaswamy, Roivant has drawn significant attention in recent years for its daring approach to biotechnology and pharmaceuticals. The company's Durham employees celebrated an expansion of their branch March 3, opening a new center on Blackwell Street.

Several members of the Duke community who have collaborated with Roivant praised its innovative strategy and mission, expressing their enthusiasm for the company’s goals.

"Roivant has a very different model—it's trying to maximize and utilize products that have gone very far in the drug development process and then, for whatever reason, [they're] dropped," said Dr. Priya Kishnani, Chen Family professor of pediatrics and chair of the division of medical genetics. "So I think they're finding ways to re-purpose them in some way and find a meaningful use and shorten all of these timelines and bring more therapies and interventions to the patient. So far the progress has remained excellent, so I do remain hopeful that the model works."

Kishnani—who has also spearheaded recent breakthroughs in Pompe disease—described her collaboration with Roivant and its subsidiaries in investigating treatment options for Down syndrome, a genetic condition that Kishnani said is challenging to study.

"Down syndrome is basically a whole chromosome disorder, with 220 genes on it," Kishnani said. "It's not a single gene disorder where you can still have some kind of handle when you're designing a [clinical trial]. But here you've got so many variabilities, with some individuals as very [high functioning] and then we have another extreme where the individual is almost non-verbal and just not doing as well."

In 2002, Kishnani and her collaborators found preliminary evidence that adults diagnosed with Down syndrome showed some benefit in their communication abilities from treatment with the Alzheimer’s medication Aricept, used primarily to slow down dementia.

Carrying these findings into her current work, Kishnani said she hopes to explore newer Alzheimer's products to better evaluate whether such therapeutics could benefit children with Down syndrome.

In a field where 99.6 percent of new drug therapies have failed, the drug RVT-101—held by the Roivant subsidiary Axovant—appears promising, Kishnani explained. Success with RVT-101 could also herald a new era in drug development and research, she added.

"I think [Axovant is the] furthest along in that field, and so it would be a huge breakthrough," she said. "[Alzheimer's disease] is already a huge market, but also selfishly for someone like me, [success] could then open it up for some of the rare diseases or some of the genetic diseases where they'd start to pay more attention to that."

According to a 2015 report by Forbes Magazine, the specifics of Kishnani's work with Roivant remain under wraps.

Kishnani offered, however, that some of their collaborative work builds on her primary interests in glycogen storage diseases, for which she and her department have already been successful in developing preliminary treatment options .

"We're working hard on different therapeutic strategies and approaches for glycogen storage disorders, and that's a topic very near and dear to my heart," Kishnani said. "Duke is nationally and internationally recognized for its work on glycogen storage diseases, of which there are many. And so my hope is that within the next decade or so, we can find a way for a treatment or advances for them, and so the partnership with Roivant started there."

Enzyvant, another Roivant subsidiary, has applied its particular interest in rare diseases by working to release a therapy for DiGeorge syndrome first developed almost 20 years ago by Dr. Mary Louise Markert, a Duke professor of pediatrics and immunology.

Markert explained that children with DiGeorge typically carry significant immune deficiencies that can end up life-threatening. To address this, Markert said she and her colleagues began implanting afflicted children with modified thymuses—or organs of the immune system—which they found to markedly improve life expectancies to more than 75 percent survival.

Now in the final stages of the U.S. Food and Drug Administration’s review process, Markert’s therapy has been developed into a biologic product, and is expected to be soon accessible to a wider set of DiGeorge syndrome patients across the country.

"I'm looking at a wall of baby pictures, and they're not all babies anymore—it's amazing," Markert said, referring to some of her first transplant patients. "The average age of the survivors is now over 10, and now I'm so happy because the future children are going to have a therapy that will be [readily] available."

Despite current controversy surrounding the pharmaceutical industry, both Markert and Kishnani agreed that partnering with pharmaceutical companies can provide a strong catalyst for medical advancement.

"It doesn't mean that if you partner with them, you [lose] your integrity as a scientist," Kishnani said. "It's a fine balance, but it's definitely possible to do. You partner with them, you educate them, you learn with them and from them, and they have the financial backing to get experts together and move things forward."

Melissa Rhodes, Roivant's vice president of nonclinical research and a Duke alum, explained that not only will the new Durham office help widen Roivant’s research collaborations with Duke investigators, but that the company is also planning to grow its educational partnerships with the University.

Among these initiatives is an internship program training students in the Graduate School, she noted. Although the program has only trained pharmacology students thus far, Rhodes noted that their Durham office plans to bring in a wider variety of students across other departments down the line.

"I would love to see us have young talent coming through with fresh ideas, and vice versa for us to be able to teach," Rhodes said. "We want to provide students with an experience to learn about drug development so that they can determine if they would like to take their careers in that direction."