MegaKaryon Corporation

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MegaKaryon Corporation

The three major methods in MegaKaryon's production of platelets based on iPS cells include introducing three different genes into an iPS cell, establishing megakaryocyte precursor cells, freezing these precursor cells, and irradiating the cells to ensure they a sterile/clean. Additionally, Megakaryon is internationally the first and only company that owns proprietary technology to manufacture large quantities of iPS cell-originated platelets.


HOW MEGAKARYON USES IPS CELLS TO PRODUCE PLATELETS

STEP 1
  • Three genes are introduced into an iPS cell, establishing megakaryocyte precursor cells.
  • These three genes have the functions of multiplying cells, suppressing cell aging and preventing cell death.
  • The result is a precursor cell that can be safely multiplied.

STEP 2
  • These precursor cells are frozen and stored for prolonged periods of time.
  • This allows MegaKaryon to produce and provide varying quantities of platelets on an as-needed basis.
  • This is significant because, previously, platelets could only be stored for four days and needed to be constantly resupplied through human donation.

STEP 3

MAJOR ACCOMPLISHMENTS

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MegaKaryon Competitors

After an exhaustive search through academic journals, professional associations in medical research, magazines publishing research on biomedical engineering, platelets, iPS cells, hematology and transfusion medicine and blood, as well as companies working in these fields, information on direct competitors of Megakyron was unavailable. This is because no related company was found to use the same process of mass production of platelets based on iPS cells which Megakyron uses. However, below is a description of two companies that use parts of the process and could become competitors if they opt to expand or shift focus.

#1: CYNATA

  • Cynata, a company based in Australia, uses its patented Cymerus technology to generate cell-based products from intermediate cells, known as mesenchymoangioblasts (MCAs), derived from induced pluripotent stem cells (iPSCs).
  • According to the company, its technology is "ideal for the commercial development of cell-based therapeutics."
  • Its manufacturing process guarantees the production of unlimited quantities of cells for therapeutic use from a single donor, implying that the company will not have to seek out fresh donors to meet its manufacturing demands consistently. This gives Cynata a unique differentiator and a competitive advantage, with the potential to create a new standard in the new field of regenerative medicine and stem cell therapeutics.
  • The Cymerus technology has advantages, including that it is versatile, scalable in manufacturing, cost-effective, safe, and provides clinical predictability.
  • The company, therefore, has the potential to become a competitor of Megakaryon in the future, but at the moment they are focused graft-vs-host disease, asthma, heart attacks, diabetic wounds, coronary heart disease, and brain cancer, among others, none of which involve platelets for treatment.

#2: RUBIUS

  • Rubius does not generate platelets, but red blood cells, and genetically engineers them into a new class of cellular medicines, called Red Cell Therapeutics (RCTs).
  • Like Megakaryon, Rubius focuses on transfusion medicine.
  • It currently focuses on treating enzyme deficiencies, activating the immune system to stop cancer — both solid tumors and hematological cancers — and to defeat autoimmune diseases such as Type I Diabetes.
  • It currently has ten product candidates, with the US Food and Drug Administration clearing an Investigational New Drug application (IND) for the company's first clinical candidate, RTX-134, for the treatment of phenylketonuria.
  • Rubius also plans to file an IND for RTX-240 for the treatment of solid tumors by early 2020.
  • In total, it plans to file 4-5 IND applications in 2019 and 2020.

RESEARCH STRATEGY

The research team began by searching for literature on the mass production of platelets based on iPS cells. During our search, we found some sites with information about Megakaryon and the genetic research that led to their success. However, we couldn't find info about the company's competitors.

We then researched the process Megakaryon uses to mass-produce these platelets, hoping that by finding and understanding the process, we would be able to narrow down to a competitor. To do this, we searched through available academic peer-reviewed journals and found a research paper that addressed some of what MegaKaryon does. However, we did not find competitors.

We then looked for clinical trials in the area of mass production of platelets based on iPS cells, in hopes that we might be able to trace the trial to a company that uses the results commercially. We found a map that showed by countries where related trials were taking place. We then examined each trial on the list to determine if they were affiliated with a company, but they were all funded by their respective governments, including the US, France, Italy, Israel, and China.

We found an academic paper that provided an end-to-end description of the process to create platelets. The process was confirmed through an interview with Megakaryon's CEO, which he stated that "Megakaryon is internationally the first and only company that owns proprietary technology to manufacture large quantities of iPS cell-originated platelets." We then opted to look for companies that could be using parts of the same process MegaKyron uses. The aim was to identify companies that could become future competitors if they decided to expand their platforms, products, or diseases they were targeting. We were able to provide descriptions of two of those companies as presented above.
Sources
Sources