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March 29, 2017

Marlborough firm explores vision cures and more

Courtesy of Astellas Institute for Regenerative Medicine Astellas Institute for Regenerative Medicine in Marlborough is developing new cures for vision diseases and more after an acquisition by a Japanese company.

Chief Science Officer Dr. Robert Lanza has been with Astellas Institute for Regenerative Medicine (AIRM) since 1999, when the Marlborough biotechnology company—then known as Advanced Cell Technologies Inc. (ACT)—was just five years old. Since then, he’s seen the stock price rise and fall, and CEOs come and go.

But since its acquisition by the Japanese company Astellas Pharma Inc. just over a year ago, Lanza said he’s been feeling more optimistic than he has in a long time about the prospects for developing new therapies, including methods for restoring vision for some people who have lost their sight. He’s even thinking bigger than he used to: about how to help build projects that could take down some of the world’s biggest threats to human health.

Today, Lanza also serves as head of Global Regenerative Medicine for Astellas, a pharmaceutical giant with 17,000 employees in locations around the world. The parent company offers AIRM support it’s never had before, Lanza said.

“We can take advantage of all their wealth of knowledge for development and clinical trials,” he said. “They’ve given us all the resources that we’ve needed—equipment that only a few people in the world have.”

A growing workforce

And so, for the past year, AIRM has been on a hiring binge, doubling its employee count to more than 70 and bringing in leading researchers from all over the world who specialize in the company’s various focus areas.

For years, the company now known as AIRM has been at the forefront of developing therapies based on the sometimes controversial use of embryonic stem cells. In 2006, ACT developed a method for using those cells without destroying embryos, helping to reduce political pushback over the issue.

But, as is often the case with biotech companies taking on ambitious projects, progress was slow and uneven at times. Some investors complained that ACT overpromised in terms of both its research and its business relationships. For some time, it was selling as a penny stock. In early 2014, it got in trouble with the Securities and Exchange Commission for reportedly selling unregistered stock, and one of its CEOs had to leave the company after failing to report stock sales. Later that year, it renamed itself Ocata.

Kevin O’Sullivan, president and CEO of Massachusetts Biomedical Initiatives in Worcester, said Astellas’ decision to acquire Ocata for $379 million speaks to the underlying value of the company’s research.

“I was a bit surprised,” he said. “I said to myself, obviously this science is something that his Japanese company sees merit in.”

In particular, O’Sullivan said Astellas seemed to be investing in Lanza’s work and ideas.

“I still have faith in Rob as a scientist,” he said. “He’s been able to kind of hang in there for a long period of time.”

A fresh start

Since AIRM became part of Astellas, Lanza said, he’s felt more able to get things done.

“The culture, the people, are so pleasant,” he said. “Just before the takeover, with the last couple administrations, it wasn’t quite that way.”

Having the backing of a deep-pocketed pharmaceutical company means there’s little pressure to deliver a quick return on investment.

“This is what’s so great about it, is they’ve identified us as one of the leaders in regenerative medicine,” Lanza said. “So they’re leaving it to us, with our experience, the scientific background, to decide what’s feasible.”

Lanza has high expectations for feasibility, starting with finally commercializing the therapy for vision problems that the company’s been working on for years. The treatment targets two forms of vision loss that currently have no cure, Stargardt’s macular degeneration (SMD) and dry age-related macular degeneration (AMD). So far, Lanza said, 38 people have received treatment for the conditions through clinical trials, and all but two have had their conditions stabilize or improve.

One patient whose story stuck with him was a cowboy from Kansas who had been blind in one eye has had his vision improve to 20/40.

“He can now ride his horses again,” Lanza said. “It makes me feel good to know that (we’re) helping people. People can now go to the airport on their own, read their watch, use a computer.”

Beyond treating those conditions, though, AIRM has bigger plans. Lanza said cell lines the company has been working with have been shown to restore vision in totally blind animals suffering from a disease called retinitis pigmentosa, and to restore function in the eyes of mice with glaucoma.

Other applications

Other cell lines show promise for treating disorders of the immune system including lupus and Crohn’s disease. In 2015, AIRM tested one such therapy on six pet dogs with the canine equivalent of Crohn’s who were patients at the Cummings Veterinary Medical Center at Tufts University. A paper published at the time reported that all the dogs showed improvement, and only two relapsed.

Lanza said Astellas is also thinking bigger. He said he and other research leaders are now collaborating to put resources into therapies for the most deadly ailments—such as kidney, heart, and liver diseases.

“We put together a dream team,” he said.

While he works with researchers all over the world, Lanza said he’s happy to be doing it from Marlborough. For scientists in the Boston area, MetroWest can be either a great place to live or an easy commute from the city, going against the flow of traffic. And the vibrant Boston-area biotech research world is appealing to new hires from other countries and other parts of the U.S., he said.

“You have a lot of the top universities here,” Lanza said.

The proximity to big research institutions is helpful not just in recruiting but also because it allows for more interactions with leaders in the field. It even helps with basic logistics, Lanza said: if AIRM wants to get cell samples from Boston, someone can just drive them over rather than worrying about shipping.

Today, AIRM has reached the maximum capacity of its current building in Marlborough, and it will be expanding as it continues to add to its headcount, Lanza said.

“We’ve already hired some of the top scientists in the field, and we plan to continue expanding here in MetroWest,” he said.

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