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Mayo's Regenerative Medicine Expertise Pays Off For Startup

A collaboration between Mayo and HART is attempting to introduce a bioengineered organ implant system into the market.

The Mayo Clinic’s expertise in regenerative medicine is playing a starring role for a biotech start-up aiming to bioengineer replacement tissue for cancer-ridden human organs. And this week its backers are anxiously awaiting word on some key animal test results that could propel it into the market. 

The initial research focus of a year-old formal collaboration between the Mayo Clinic and Holliston, Massachusetts-based Harvard Apparatus Regeneration Technology (Nasdaq: HART) is large-animal tests on esophagus tissue regeneration.

Should the research ultimately prove successful—and result in the introduction of HART’s bioengineered organ implant system (called Cellframe) into the market—it could evolve into a lucrative business relationship for Mayo: The terms of the collaboration provide that any future patented inventions produced through the partnership “may be licensed by HART and HART will pay royalties to Mayo Clinic.”

After encouraging results in preliminary animal tests conducted last year by Dr. Dennis Wigle, chair of thoracic surgery at Mayo Clinic, and HART Chief Medical Officer Dr. Saverio La Francesca, the company said this month it would issue an update on further test surgeries by early May. The two doctors had been scheduled to perform the new tests through mid-February.

Investors will closely watch the results of those tests, as it is believed that the technology has potential as an alternative to many different kinds of organ transplantation, including the heart and lung transplants. They could also boost to Mayo’s push to establish Rochester as a worldwide hub for regenerative medicine clinical expertise as well as a future industry cluster.

More than 121,000 U.S. patients are on organ transplant waiting lists, but there were just over 15,000 organ donations last year, according to the U.S. Department of Health and Human Services. HART estimates the global market for Cellframe to be $1.5 billion.

Its work with Mayo’s Dr. Wigle is geared toward filing an Investigational New Drug (IND) application with the U.S. Food and Drug Administration in late 2016, and seeking to initiate clinical trials soon thereafter.

Dr. Wigle told TCB he was not at liberty to discuss the latest tests before the company’s release of the information, but characterized the earlier tests as “encouraging, certainly encouraging enough to continue with the large-animal work with a view towards coming up with a product that we’d be able to use for human applications.”

The relationship between Mayo and the Massachusetts biotech first took shape in 2013 with the clinic’s program to develop a synthetic human heart valve. From there the association grew into work on additional organs such as the esophagus, trachea and bronchial airway. The arrangement is meant to be a marriage of HART’s expertise in the device design and Mayo’s expertise in regenerative medicine.

HART’s Cellframe technology consists of placing a patient’s own bone marrow stem cells onto a synthetic biopolymer “scaffold” made from ultrafine plastic fibers. Once distributed onto the fibers – which mimic the properties of human esophagus -- the stem cells begin their growth inside a slowly spinning incubator for three days. Then the biocompatible scaffolding is transplanted into the diseased or damaged organ, where the stem cells complete their transformation into new, organ-specific tissue, hopefully going on to heal the damaged organ.

The company, spun off from Harvard Bioscience Inc. (Nasdaq: HBIO) in 2013, initially concentrated on developing regenerated tracheas while working with Dr. Paolo Macchiarini, then a senior researcher at the Karolinska Institute in Stockholm. He had early success, but soon after the arrival of current CMO Saverio La Francesca in 2014, HART severed its relationship with Macchiarini.

The move followed a damning documentary produced by Swedish broadcaster SVT portraying Macchiarini as a media-obsessed, ethically lax “celebrity surgeon” who used terminally ill European patients as guinea pigs to test HART’s trachea system. In February, the Karolinska Institute announced that while it would not pursue charges of scientific misconduct against Macchiarini, it also was not inviting him back onto its faculty after his current contract expires.

Since then, HART says it has produced a “second generation” scaffolding that La Francesca asserts addresses problems with inflammation in the earlier iterations thanks to “changed chemistry” and “refined microstructural properties.” He added in a statement, “I believe that we have the right combination of characteristics to restore the function of the airways and the esophagus.”

Dr. Wigle said the advances HART has made in the properties of its implantable scaffolding are unique, in part because those efforts are aimed specifically for the development of artificial esophaguses and tracheas.

“The vision and the progress comes with their trying to sort out what materials are most compatible with replacing organs in the digestive tract, and in what specifically the right steps are to producing an artificial esophagus and artificial trachea,” the surgeon said. “I mean, there are lots of new materials all the time, but many of them are coming from companies that are, say, straight chemical companies, or straight biopolymer companies, who don’t have the stated mission of producing replacement organs.”

While the new and improved biopolymers are important, Wigle said, the most exciting thing about Mayo’s work with HART “is just putting the whole package together and doing all the R&D work it takes to figure out what’s the right material, what’s the right stem cell type -- in short, how to do really all the necessary pre-clinical work to prepare for trying to use it in human situations.”

HART shares were trading at $1.63 per share Monday. Its biggest shareholders as of April 2015 included New York healthcare investment manager Sio Capital; Dallas-based investment firm Prosight Partners; and the state of New Jersey’s pension investment fund, according to SEC filings.
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