International investment to advance cell therapies for blood disease

Progress towards personalised blood stem cell treatments for children and adults with bone marrow failure, leukaemia and other blood disorders will be advanced through a new partnership between the Melbourne node of the Novo Nordisk Foundation Center for Stem Cell Medicine, reNEW, based at Murdoch Children’s Research Institute in Australia, and US biotechnology company Retro Biosciences.

Today, the significant research and commercial agreement with Retro Biosciences was announced, injecting more than 10 million Australian dollars ($5.7 million euros) into reNEW Melbourne’s research to advance their world-first discovery to grow human blood stem cells in the lab towards a novel autologous cell therapy. The overall partnership could be worth more than US$35 million.

Globally, 90,000 blood stem cell transplants are performed every year to treat blood diseases. Of these, over 40% involve transplants from donors but not all patients receive a perfectly matched transplant. This can result in severe illness or death, as mismatched donor cells may attack the patient’s own tissues.

In 2024, researchers at reNEW Melbourne achieved a world-first breakthrough by creating blood stem cells in the lab that closely mimic those in the human embryo.

Lead researcher Associate Professor Elizabeth Ng said, “In work published in 2024, we showed that we could  take any cell from a patient, reprogram it into a stem cell and then turn these into specifically matched blood cells for transplantation.

“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” she said.

“A goal of the partnership is to progress this research into the first-in-human 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.

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.”

“reNEW supports the very best stem cell research upon which we hope to deliver stem cell medicine to the patients that need it” said reNEW CEO Melissa Little.

“This agreement with Retro is just such an example where a world-first, novel cell therapy has attracted an international commercial partnership to move forward as a product,” she said.

“This is a resounding success for reNEW and is the blueprint for many more partnerships to come”.

About reNEW
The Novo Nordisk Foundation Center for Stem Cell Medicine, reNEW, is a unique global collaboration focusing on stem cell research involving three leading research institutions: the University of Copenhagen in Denmark, the Leiden University Medical Center in the Netherlands, and the Murdoch Children’s Research Institute in Australia. reNEW strives to advance a new generation of effective and safe stem cell-based therapies, built on this global collaborative network of excellence in targeted biomedical research. Its funding is provided by the Novo Nordisk Foundation, an international philanthropic foundation based in Denmark. The 300 million euro grant runs over a 10-year period from 2022.

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 www.retro.bio