Platelet BioGenesis Receives Contract Worth Up to $56 Million from the Biomedical Advanced Research and Development Authority (BARDA) to Develop Human Stem Cell-Derived Platelets as a Medical Countermeasure to Radiological and Nuclear Exposure
Funding will support clinical development of stem cell-derived platelet technology platform
Platelet BioGenesis, a leader in stem cell-derived, on-demand human platelets (PLTs+™) and genetically engineered platelet-based therapeutics, announced today it was awarded a contract worth $5 million, with the potential to reach $56 million total with options, by the Biomedical Advanced Research and Development Authority (BARDA), an agency of the US government's Department of Health and Human Services' Office of the Assistant Secretary for Preparedness and Response. PBG will use the funding to develop and establish donor-independent platelets as a medical countermeasure for treating victims of a nuclear or radiological event.
As part of national biodefense preparedness, BARDA has prioritized the development and procurement of therapies for trauma resulting from chemical, biological, radiological and nuclear defense threats, including exposure to high doses of radiation. BARDA, with its extensive experience working with organizations focused on blood-related therapies, has evaluated PBG’s technology platform and determined that donor-independent platelets could be a critical medical countermeasure in case of a national threat.
“This is a significant milestone for PBG and a highly valuable and timely validation of the groundbreaking work of our scientific founder, Dr. Jonathan Thon, and the research, development and manufacturing teams at PBG, allowing us to use human induced pluripotent stem cells to manufacture platelets on-demand,” said Sam Rasty, Ph.D., President and CEO of Platelet BioGenesis. “With BARDA’s expertise in the development of blood-related therapies, their decision to award us this significant contract will further bolster the company’s resources to advance this technology into the clinic. The funding will not only help bring our unique donor-independent platelets to patients as a medical countermeasure but will also expedite the advancement of our broader donor-independent PLTs+™ platform.”
“In a radiological or nuclear emergency, impacted communities will face a significant blood product shortage,” explained BARDA Director Rick Bright, Ph.D. “We are exploring donor-independent platelet technology to increase surge capacity within the blood industry. Our nation must find innovative ways to make essential blood products available to save lives in any type of mass casualty incident.”
PBG’s research, development and manufacturing activities under the contract will specifically focus on the development of PLTs+™ for the treatment of thrombocytopenia induced by exposure to nuclear radiation. In addition to the funding, BARDA will provide a comprehensive, integrated portfolio approach through mentorship, the facilitation of future partnerships and the enablement of government collaborations with agencies such as the FDA.
Via BusinessWire PR news services www.plateletbiogenesis.com
Platelet BioGenesis Licenses Induced Pluripotent Stem Cell Technology from iPS Academia Japan
Sven Karlsson, co-founder and president of Platelet BioGenesis, said, “This license from iPS Academia Japan is a major milestone for Platelet BioGenesis because it gives us access to an important processing step needed to produce platelets using our novel and exclusive method. With this agreement in place, we have now secured control of all the underlying technology needed to produce donor-independent, life-saving platelets for transfusion.”
Platelet BioGenesis joins a select group of global life science leaders that have been licensed technology from iPS Academia Japan. Its current licensees include Pfizer, Roche, Sanofi, Bayer and Boehringer Ingelheim.
iPS cells are derived from somatic cells such as skin or blood cells, and have been reprogrammed back into an embryonic-like pluripotent state. This enables the iPS cells to develop into almost any type of human cells. At Platelet BioGenesis, the iPS cells are differentiated into megakaryocytes, the cells in the bone marrow that produce platelets, which are crucial for normal blood clotting and the transport of various factors throughout the body.
iPS Academia Japan is the entity established to commercialize the intellectual property of Kyoto University and other prominent universities and research institutions. The iPS cell patent portfolio primarily issues from the work of Professor Shinya Yamanaka at the Center for iPS Cell Research and Application at Kyoto University.
Via BioSpace PR news services www.plateletbiogenesis.com
Platelet BioGenesis ("PBG") is a Harvard University spinout that is making human platelets from stem cells. PBG have developed and patented a microfluidic bioreactor, and shown that functional platelets can be generated from human pluripotent stem cell cultures (iPSCs). By removing the volunteer donor, PBG can make platelets that are cheaper, safer, and available on demand.
To produce platelets in the body, parent cells called megakaryocytes sit adjacent sinusoidal blood vessels in the bone marrow through which they extend and sequentially release platelets into the circulation. PBG hypothesized that by creating megakaryocytes from hiPSCs using scalable serum- and feeder-free cell culture protocols, and exposing them to the architecture and intravascular shear stresses characteristic of their native microenvironment, PBG could trigger production of functional platelets.
In collaboration with Ocata Therapeutics in 2014, PBG showed that it was feasible to generate functional megakaryocytes from research-grade hiPSC cultures. The decision to begin with a hiPSC line offered genetic control of the product, supporting the future development of HLA-matched platelets that could be customized to recipients and targeted to particular diseases. This method also permitted the cryopreservation of megakaryocyte progenitors, which could be thawed and differentiated to mature megakaryocytes within a few days, facilitating future on-demand production. Most importantly however, this approach employed a serum- and feeder cell layer-free protocol which decreased the risk of an immunogenic reaction in humans, improved scalability, increased time efficiency from megakaryocyte progenitor to platelet and decreased the overall cost of platelet unit generation.
To trigger platelet production, PBG created an integrated microfluidic platform that combined novel concepts in bone marrow physiology with biologically-inspired tissue engineering. By modeling bone marrow architecture ex-vivo, and exposing megakaryocytes to shear stresses characteristic of flowing blood, PBG found that they could reproduce physiological platelet production and increase the overall rate and extent of platelet release.
In order to make platelets in the lab PBG needed to solve the supply chain from stem cell, to megakaryocyte (parent cell), to platelet. While the discovery of thrombopoietin in 1994 drove the generation of the first human platelets by Amgen in 1995, it wasn’t until 2006 that the invention of human induced pluripotent stem cells (hiPSCs) by Dr. Yamanaka alloPBGd for the scalable generation of genetically consistent stem cells. The third major advance was made in 2014, when PBG solved how to trigger megakaryocytes to make platelets at yields necessary for clinical/commercial application.
Source: http://www.plateletbiogenesis.com
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