PDA-002

Robert Hariri/Celularity photo Iovance Biotherapeutics priced its recently approved Amtagvi, the first-ever cell therapy of its type, at $515,000. Bristol Myers Squibb’s CAR-T treatment Breyanzi, which received FDA approval for two additional indications in March, carries a list price of about $487,000. Those prices do not sit well with regenerative medicine pioneer Robert Hariri, who told BioSpace that “cell therapy will go the way of the dinosaur if we expect each treatment to cost a half a million dollars.” Hariri, currently CEO, chairman and founder of placenta-derived stem cell developer Celularity, specifically said that CAR-T therapy involving patients’ own cells—a type of therapy that has been on the market since 2017 and now features six commercial products—is “not a scalable revolution in healthcare until it can fit the economic constraints.” Instead, he said he believes in the potential of cell therapies derived from donor cells, especially from placentas. “The placenta is nature’s professional and universal donor,” Hariri said. A former surgeon, Hariri discovered placental pluripotent stem cells in 2000 while building Anthrogenesis, a company bought by Celgene two years later. After the buyout, Hariri served as CEO of Celgene Cellular Therapeutics. During his time there, he became interested in applying stem cells to slow the aging process, and joined with Craig Venter and Peter Diamandis to found Human Longevity in 2013. Celgene spun off Florham Park, N.J.–based Celularity in 2016, and Hairiri joined the company. He's now using placental stem cells to target cancer, autoimmune disease and more, ushering therapies for Crohn’s disease and facioscapulohumeral muscular dystrophy through early-stage trials. Hariri said he believes that cell therapies will supercede biologics for autoimmune diseases in the next few years. “Had there not been such a rush to chase oncology because of the CAR-T revolution, we would be a lot further along in treating autoimmune diseases,” he said. On the side, Hariri is an aerospace entrepreneur and licensed pilot who has flown with legendary former astronaut Buzz Aldrin. On a Mission Hariri, once a practicing physician, left the hospital world after his fellowship and has been following an entrepreneurial path ever since. His mission is to create what he calls “living-cell medicine”—and to make it cost-effective. “For 30 years, I have focused on the fact that for cell therapy to have a meaningful role in medicine, it has to do more than just transform the way the patients are treated and their outcomes,” Hariri said. “It has to fit in an already stressed healthcare system economically.” When human embryonic stem cells were discovered in the late 1990s, Hariri was a neurosurgery fellow at what is now Weill Cornell Medical Center in New York, specializing in head and spinal cord injuries. He saw embryonic stem cells as a potential way to restore motor function in severely injured patients. But those cells came from discarded embryos from in vitro fertilization, which drew the ire of anti-abortion activists. “It’s very tough to commercialize a product when half the population is opposed to it,” Hariri noted. It also was difficult to control the quality of the supply of embryos, which is problematic in a heavily regulated industry like biopharma manufacturing, Hariri said. “Looking at all the science around embryonic-derived cells as well as fetal-derived material [from abortions], I was very concerned about the qualitative drift from donor to donor to donor.” Hariri found that there was “tremendous consistency” in the quality of cells derived from postpartum placentas, however. Given that hospitals typically have placental residue incinerated, repurposing it to harvest stem cells is a “green” technology in Hariri’s eyes. “We are saving hospitals from having to pay to incinerate this waste material and we’re turning it into a range of useful therapeutic products,” he said. The idea of harvesting stem cells from placentas rather than embryos to create new therapies eventually caught the attention of the staunchly anti-abortion Roman Catholic Church, and in 2018, Pope Francis presented Hariri with the Pontifical Medal for Innovation. “The Vatican recognized this because they saw it as a superior alternative to the destruction of embryos,” Hariri said. Celularity has also received halal certification for its placental stem cell technology, allowing the company to operate in Muslim countries. “Medical approaches to treating disease have to fit into the social norms in order to be deployable without resistance, without opposition,” Hariri said. “You can’t operate if you’re going to offend or potentially violate some of those social standards.” Celularity’s Studies and Struggles Hariri said that Crohn’s was an easy choice for developing placenta-derived treatments because many women see autoimmune diseases go into remission during pregnancy and then flare up after giving birth. In a Phase I trial, Celularity’s stromal cells generated from placenta-derived stem cells helped reduce inflammation and limit fistulas in a small population of patients with Crohn’s. Celularity also has a clinical-stage placenta-based stem cell therapy for facioscapulohumeral muscular dystrophy, a genetic disorder that affects the muscles of the face, shoulders and upper arms. The asset, called PDA-002, is set to begin a Phase I/II study later this year, and the company recently requested FDA orphan drug designation to expedite its development. Celularity has had some financial struggles, though. The company laid off 188 workers in March 2023 as part of a strategic review after its stock price plummeted. This year, with the price languishing below Nasdaq’s $1 minimum, the company was forced to execute a 1-for-10 reverse stock split in February to avoid delisting. While one forecast estimated that the worldwide placental stem cell market would grow by 22% annually between 2022 and 2030, no such stem cell therapies have received FDA approval. Everything in commercial stage to date has been derived from other sources. Reversing Aging Hariri also sees placental cells as a fountain of youth of sorts, helping to stave off the ravages of aging, which was the reason why he, Venter and Diamandis founded Human Longevity. He has long been interested in the potential of natural killer (NK) cells, and Celularity will present preclinical data next month about how its placenta-derived NK cells might be able to remove senescent and other harmful cells from the body. “If we can use cell therapy in a number of different ways to control the degenerative processes in aging . . . then we may in fact be able to shift much of the disease burden that occurs in our 50s, 60s and 70s,” he said. “If we can tune up your immune system as you age, that almost by definition is going to have beneficial consequences for your health.” Neil Versel is the business editor at BioSpace. You can reach him at neil.versel@biospace.com. Follow him on LinkedIn or X.