SEATTLE, Aug. 6, 2024 /PRNewswire/ — YouthBio Therapeutics (YouthBio), an innovative longevity biotechnology company developing gene therapies based on partial reprogramming, today announced significant results from its collaboration with Dr. Alejandro Ocampo, a professor at the University of Lausanne and a leading figure in aging research whose pioneering work gave rise to the field of partial reprogramming.
The study, titled “Specific Partial Reprogramming of Neurons in the Dentate Gyrus Impacts Behavior in Mice and Ameliorates Age-Related Decline in Memory and Learning,” demonstrates that brain-specific induction of partial reprogramming can attenuate cognitive decline associated with aging. These findings add to the growing body of evidence pointing to the therapeutic potential of in vivo partial reprogramming for the prevention and treatment of neurodegenerative disorders.
Main Conclusions
This study utilized two distinct approaches: a neuron-specific reprogrammable transgenic mouse model and neuron-specific targeted lentiviral delivery of OSKM reprogramming factors. Both methods demonstrated that partial reprogramming of adult neurons in the dentate gyrus of the hippocampus impacts animal behavior and cognitive function, and ameliorates age-related decline in memory and learning.
“These exciting results from our collaboration with Dr. Ocampo validate our approach and highlight the transformative potential of partial reprogramming in combating age-related cognitive decline,” said Yuri Deigin, CEO and co-founder of YouthBio. “We are thrilled to see such promising results and are committed to taking this research forward.”
Dr. Ocampo added, “I am thrilled to be collaborating with YouthBio to accelerate the clinical translation of partial cellular reprogramming to treat age-related diseases, particularly neurodegenerative conditions. It is also very exciting to see other research groups demonstrating lifespan extension in wild-type mice through partial reprogramming. I believe that once we fully understand how to use this approach in a tissue-specific manner—something YouthBio has recognized since the company’s inception—we will see even greater increases in lifespan and more beneficial therapeutic effects across multiple disease models.”
“The potential of partial reprogramming for brain rejuvenation and the treatment of neurodegenerative disorders is very promising,” said João Pedro de Magalhães, Ph.D., Chief Scientific Officer of YouthBio. “Our recent findings suggest that partial reprogramming may play a significant role in the treatment of age-related cognitive decline. While there is still much to learn and refine, these initial results are encouraging and provide a solid foundation for future research. We are cautiously optimistic that with continued research, we may be able to develop effective therapies that may one day benefit patients suffering from conditions such as Alzheimer’s disease.”
Future Plans and Alzheimer’s Research
YouthBio is committed to investigating the use of partial reprogramming in the CNS, with a particular focus on Alzheimer’s disease. In another study using ARTE10 transgenic mice treated with brain-specific inducible OSKM vectors, YouthBio observed reduced levels of beta-amyloid and reduced brain plaque burden in critical areas such as the hippocampus, as well as improved cognitive performance on behavioral tests compared to untreated controls. Based on these promising results, YouthBio has filed a provisional patent application with the USPTO.
YouthBio’s results are independently validated by a team of researchers from Altos Labs, the University of Barcelona and the Max Planck Institute, who demonstrated favorable results in preventing Alzheimer’s symptoms through targeted partial brain reprogramming in a 5xFAD mouse model. Their study, “Neocortex Expansion and Protection against Neurodegeneration by Transient In Vivo Reprogramming,” corroborates YouthBio’s positive findings.
“Our positive Alzheimer’s data, coupled with the positive independent results from Altos Labs and its collaborators, reinforce our confidence in the ability of partial reprogramming to provide a new treatment paradigm for neurodegenerative conditions,” said Deigin. “Our next important milestone is to advance our Alzheimer’s therapy into clinical trials, and we are fully committed to achieving this goal.”
Clinical Trials and Beyond
YouthBio intends to advance its Alzheimer’s disease therapy to clinical trials within three years. During this time, the company will conduct the necessary preclinical studies, GMP manufacturing, and regulatory preparations essential for filing an Investigational New Drug (IND) application with the FDA.
At the same time, YouthBio will continue its discovery program, led by CSO Dr. João Pedro de Magalhães, to identify superior rejuvenation factors beyond Yamanaka factors for use in subsequent generations of its tissue-specific gene therapies. The company will also conduct multiple proof-of-concept animal studies in other CNS indications and will continue its progeria program.
In its progeria program, YouthBio has received a Rare Pediatric Disease Designation from the FDA for YB-001 for the treatment of Hutchinson-Gilford progeria syndrome (HGPS). This designation makes YouthBio eligible to receive a Priority Review Voucher (PRV) if YB-001 is approved to treat progeria. The PRV can be redeemed to receive an accelerated priority review for any subsequent marketing application or sale. Historical transaction values for PRV have exceeded $100 million, reaching $350 million.
About YouthBio Therapeutics, Inc.
YouthBio Therapeutics ( https://youthbiotx.com ) is a longevity company developing gene therapies that leverage partial reprogramming to restore a more youthful gene expression profile in target cells, leading to positive therapeutic effects in corresponding organs. It is currently focused on brain-specific gene therapies targeting CNS diseases, with Alzheimer’s disease being the lead program.
