A new frontier into advancing treatments for children and adults with bone marrow failure, leukaemia 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.

Image: Professor Andrew Elefanty, Associate Professor Elizabeth Ng, Chief Strategy Officer Ben Fielding, Minister Danny Pearson, Retro Biosciences CEO Joe Betts-LaCroix , Professor Kathryn North AC and Professor Ed Stanley at the agreement signing.

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.

Image: Associate Professor Elizabeth Ng

“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, among others. 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.

Retro Biosciences Co-founder and CEO Joe Betts-LaCroix said; “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.”

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.

Image: Professor Enzo Porrello

Victorian Economic Growth and Jobs Minister Danny Pearson said; “Melbourne is a global leader in medical research as shown by the amazing work done by the Murdoch Children’s Research Institute, this partnership will help them advance their research to provide more treatment options for people with blood disorders around the world.”

Available for interview

• Associate Professor Elizabeth Ng, MCRI Group Leader, Blood Development
• Professor Andrew Elefanty, MCRI Group Leader, Blood Development
• Professor Ed Stanley, MCRI Deputy Stem Cell Medicine Theme Director and Group Leader, Immune Development
• Professor Enzo Porrello, MCRI Theme Director, Stem Cell Medicine
• Joe Betts-LaCroix, Retro Biosciences Co-founder and CEO

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About Murdoch Children’s Research Institute

Murdoch Children's Research Institute is the largest child health research institute in Australia committed to making discoveries and developing treatments to improve child and adolescent health in Australia and around the world. They are pioneering new treatments, trialling better vaccines and improving ways of diagnosing and helping sick babies, children and adolescents. It is one of the only research institutes in Australia to offer genetic testing to find answers for families of children with previously undiagnosed conditions.

About Retro Biosciences

Retro Biosciences is pioneering breakthrough therapies designed to extend healthy human lifespan by a decade. Leveraging advanced biotechnology such as cellular reprogramming, autophagy enhancement, and AI-driven protein engineering, Retro targets the fundamental mechanisms of aging and age-related diseases. Founded in 2021 and backed by leading innovators including OpenAI CEO Sam Altman, Retro sits at the forefront of the rapidly emerging trillion-dollar longevity field. To learn more about how the company's multidisciplinary team is translating cutting-edge science into practical, scalable solutions aimed at redefining human healthspan, visit Retro Biosciences.