Harvard Biotech Spinout Lands $10M to Make Platelets From Stem Cells

Xconomy Boston — 

If you’ve donated blood, you probably received a follow-up message asking you to consider a platelet donation. Donation centers must constantly replenish their blood supplies, but the need for platelets is particularly acute. Compared to other components of blood, platelets are fragile and short lived, which puts high demand on the small supply of these cell fragments that are key to stopping bleeding.

Plasma separated from blood can be frozen and red blood cells can be refrigerated for up to 42 days. But platelets, which must be stored at room temperature, last just five days. After pathogen screening and transport, platelets have about two days to reach a patient, says Sven Karlsson, president and co-founder of Boston-based Platelet Biogenesis.

Karlsson’s startup aims to bring hospitals an alternative: platelets produced as needed from stem cells. Platelet BioGenesis, a spinout from Harvard University and Brigham and Women’s Hospital, now has $10 million from a Series A round of funding to support preclinical testing of its regenerative medicine technology.

The body produces the components of blood in bone marrow. Platelets are made by a type of bone marrow cell called a megakaryocyte. Platelet Biogenesis produces its platelets in a two-step process. It first develops megakaryocytes from stem cells in culture, Karlsson says. Next, the megakaryocytes are fed into a device that the company developed that puts the cells through a process the company says is similar to what occurs in natural bone marrow physiology and results in the production of platelets.

“We’re making real, functional platelets,” Karlsson says. “Instead of mimicking the platelet, we’re mimicking the process of making a platelet.”

Platelet transfusions are needed by patients whose bodies don’t produce enough platelets, as well as those who have clotting problems. These transfusions are also used to treat patients who have cancer or are undergoing surgery. Since most platelet donation centers are in urban areas, the short shelf life of platelets makes supplies tight in rural areas. Donations don’t entirely solve the problem, Karlsson says. While donating whole blood takes about 15 minutes, platelet donation takes 90 minutes, which makes it harder to get donors.

Researchers have tried to address the platelet shortage problem by developing synthetic platelets. But Karlsson says that the synthetic versions can cause side effects. The idea for replicating the body’s process of producing platelets stems from more than a decade of research. Jonathan Thon, the CEO and co-founder of Platelet BioGenesis, was pursuing ways to extend the shelf life of platelets. But Karlsson says Thon concluded a better approach might be developing a way to replace human donors. As a post-doctoral researcher at Harvard, he invented a microfluidic device that mimics a human bone marrow.

Before Platelet BioGenesis can bring its platelet-making technology to the market, it will need FDA approval. The regulator treats blood products as therapeutics, which means that the company will need to conduct clinical trials. Karlsson says the funding round will support preclinical research. Within three years, he expects the startup will be able to start Phase 1 safety studies. For those trials, Platelet BioGenesis will need to raise additional funds. The current financing round was led by Qiming US Healthcare Fund and included participation from Vivo Capital, VI Ventures, Adena Partners, eCoast Angels, as well as other unidentified investors.

If Platelet BioGenesis wins FDA approval, Karlsson says the company plans to become a manufacturer, selling its platelets in the existing blood supply chain. It’s early to talk about pricing, but he says that Platelet Biogenesis’ products could be more affordable because they are produced from a pathogen-free source. They could also be produced on demand. While the startup’s platelets should have the same shelf-life as platelets from human donors, Karlsson says that the parent cells that produce platelets can be stored frozen. When platelets are needed, thawed cells can be placed in the company’s microfluidic device, which produces platelets within hours.

Karlsson says Platelet BioGenesis might be able offer another advantage: beating the five-day shelf life of donor platelets. In the body, a platelet lasts 10 days. If all of Platelet BioGenesis’ platelets are “born” on the same day, they should theoretically have the same 10-day life, he says. That’s one of the things the company plans to test in upcoming trials. The company will also evaluate its platelets to make sure that they are comparable to donor platelets.

Image by Wikimedia user Erhabor Osaro via a Creative Commons license.