New partnership aims to advance treatments for blood disorders and leukemia

A new frontier into advancing treatments for children and adults with bone marrow failure, leukemia and other blood disorders will be achieved under a new partnership between Murdoch Children's Research Institute (MCRI) and US biotechnology company Retro Biosciences.

MCRI, a flagship member of the Melbourne Biomedical Precinct and the Melbourne arm of the international research consortium, the Novo Nordisk Foundation Center for Stem Cell Medicine, reNEW, has today announced a significant research and commercial licensing agreement with Retro Biosciences. The agreement will advance a blood stem cell breakthrough, discovered by MCRI scientists last year, that could pave the way for new therapies for patients around the world.

The partnership, worth over US$35 million, will further develop MCRI's field-leading research and technology by licensing the blood stem cell discovery IP to Retro Biosciences for the development of new, personalised therapies.

More than 90,000 blood stem cell transplants are performed globally every year to treat blood diseases. Of these, over 36,000 involve transplants from donors to treat leukaemia or bone marrow failure syndromes. But not all patients receive a perfectly matched transplant, which can result in severe illness or death, as mismatched donor cells may attack the patient's own tissues.

In a world-first discovery in 2024, MCRI researchers created blood stem cells from human cells in the lab. These cells closely resembled those in the human body. The development lays the groundwork for a patient's own perfectly matched cells to be used in blood stem cell and bone marrow transplants.

MCRI Associate Professor Elizabeth Ng, whose team was behind the research, said; "We have shown that we can take any cell from a patient, reprogram it into a stem cell and then turn these into specifically matched blood cells for transplant, preventing complications from mismatched donors.

"By joining forces with Retro Biosciences, we are now on our way to providing personalised, patient-specific blood stem cells to treat children and adults with blood diseases. A goal of the partnership is to progress this research into the first-in-human clinical trials within the next five years."

Retro Biosciences' mission is to add 10 years to the healthy human lifespan with programs that strive to replace malfunctioning cells through the use of stem cell technologies. Their licensing of MCRI's breakthrough technology and investment in the organisation's world-class stem cell research not only addresses urgent unmet needs in hematology but also represents a critical step toward enabling regenerative solutions that support a longer, healthier lifespan.

For decades, it's been a dream of the stem cell field to convert pluripotent stem cells into blood stem cells that engraft permanently and produce all the needed blood lineages to maintain a person's health.

Last year, the principal investigators at MCRI finally cracked this code, and we immediately saw the potential for sustaining a healthy blood system into late life. Our team at Retro is incredibly excited to be the exclusive licensee for this new generation of autologous therapies."

Joe Betts-LaCroix, Retro Biosciences Co-founder and CEO 

MCRI Professor Enzo Porrello, Director of Stem Cell Medicine, said the partnership demonstrated the value of early investment in transformative technologies and the importance of industry partnerships to drive innovative therapies towards clinical application.