Platelet BioGenesis, Inc. (PBG), a leader in stem cell-derived, on-demand human platelets (PLTs+™) and platelet-based therapeutics raised more than $26 million in Series A-1 financing. This round—in addition to the Series A financing and numerous grants—brings PBG’s total funding to approximately $45 million.
TrialSite News breaks down the update and the new venture.
What year was PBG Founded?
PBG was founded in 2014 and is based in the Boston, MA area.
Who are the latest investors in PBG?
Ziff Capital Partners (ZCP) and Qiming Venture Partners USA co-led the Series A-1 and existing investors Nest Bio Ventures and eCoast Angeles also participated among others.
Dr. Steven Altschuler, MD with ZCP, chairman of the board of Spark Therapeutics, a company he co-founded while serving as CEO of Children’s Hospital, Philadelphia, will join PBG’s board. Dr. Altschuler also sites on the boards of several other prominent companies including AsclepiX Therapeutics and ImVaX, Inc.
What is the PBG Mission?
PBG seeks to extend the healing and therapeutic power of platelets by advancing and commercializing the science of making functional human platelets on-demand from induced pluripotent stem cells.
They seek to be the world leader in making platelets. Why? Because they see a future where minor disruptions, natural disasters, and major emergencies do not generate urgent calls for blood donations, and where doctors have a new tool to treat diseases that do not respond to current therapies.
What is the Use of Funds?
They will accelerate the development of its donor-independent platelet manufacturing capabilities and advance its proof of concept work around platelet-based therapeutics. The company plans to double its staff from 25 to 50 in 2020.
What are Platelets?
They are a component of blood whose function (along with the coagulation factors) is to react to bleeding from blood vessel injury by clumping, thereby initiating a blood clot. Platelets have no cell nucleus—they are fragments of cytoplasm that are derived from the megakaryocytes of the bone marrow and enter the circulation. Circulating inactivated platelets are biconvex discoid (lens-shaped) structures. Activated platelets have cell membrane projections covering their surface. They are only found in mammals, whereas in other animals (e.g. birds, amphibians) thrombocytes circulate as intact mononuclear cells.
Core PBG Solution
PBG reports that platelets are the band-aids of the bloodstream. The problem with platelets is, first of all, there are insufficient numbers of platelets to meet current demand. Any professional in the blood space understand that platelets are a major pain point—for hospitals, physicians or patients they affect. People don’t give as much blood as we would like and providers generally experience shortages. The solution: we need an unlimited supply of platelets—essentially, platelets “on-demand.” Platelets come uniquely form human volunteer donors. The way to solve the problem is by “disconnecting the product from the donor” and producing platelets from stem cells.
PBG Platelet Production
PBG makes platelets in a patented process that mimics the conditions inside bone marrow, where platelets are normally produced. They are being with induced pluripotent stem cells, which can develop into almost any kind of tissue in the body. They create megakaryocytes, the progenitor cells within the bone marrow that produce platelets. Inside their patented platelet bioreactor, megakaryocytes are exposed to environmental conditions found inside bone marrow. These conditions include a permeable membrane through which the cells extend arms called proplatelets—as they would extend arms through the walls of blood vessels—and flowing media that generates the shear stresses similar to those found inside a blood vessel. These induce megakaryocytes to be producing platelets that are collected for use.
A Watershed Moment Paving the Way toward first Clinical Trial based on stem cell-derived platelets
The Series A-1 round is a watershed moment for Platelet BioGenesis and constitutes a significant step toward supporting our goal of designing and launching our first clinical trial using stem cell-derived platelets.” He continued By developing a targeted mechanism that harnesses donor-independent platelets as a new treatment modality, we plan to advance platelet-based therapy to effectively treat a broad range of human diseases. Our technology is unlike anything on the market, and we believe that our therapeutic capabilities have the potential to build on previous advancements in the gene and cell therapy fields and lead to transformative medicines.”
Clinical Hopes and Commercialization Vision
The company seeks to move its technology into clinical trials by 2021 if all goes according to plan. The hope is that in addition to changing the game for treating patients who need a platelet infusion they will also transform drug delivery. An on-demand platelet program has significant interest from the U.S. Department of Defense as they entered into a partnership with PBG with a $3.5 million grant to produce functional human platelets on-demand. The Goal: demonstrate the venture can produce platelets for critical life-saving transfusions to wounded soldiers and civilians.