The PennZone

SEATTLE - PennZone -- YouthBio Therapeutics (YouthBio), a pioneering longevity biotechnology company developing partial reprogramming-based gene therapies, today announced significant research results from its collaboration with Dr. Alejandro Ocampo, Professor at the University of Lausanne and a leading figure in aging research whose pioneering work has given rise to the partial reprogramming field.

The study, titled "Neuron-specific partial reprogramming in the dentate gyrus impacts mouse behavior and ameliorates age-related decline in memory and learning," demonstrates that brain-specific induction of partial reprogramming can ameliorate cognitive decline associated with aging. These findings add to the growing body of evidence for the therapeutic potential of in vivo partial reprogramming for the prevention and treatment of neurodegenerative disorders.

Key Findings
This study used two different approaches: a neuron-specific transgenic reprogrammable mouse model and a neuron-specific targeted lentiviral delivery of OSKM reprogramming factors. Both methods showed that partial reprogramming of adult neurons in the dentate gyrus of the hippocampus impacts animal behavior and cognitive function, and mitigates age-related decline in memory and learning.

"These exciting findings 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 advancing this research further."

Dr. Ocampo added, "I am delighted to collaborate with YouthBio to accelerate clinical translation of partial cellular reprogramming for treating age-related diseases, particularly neurodegenerative conditions. It is also very exciting to see other research groups demonstrating lifespan extension in wildtype mice through partial reprogramming. I believe that once we fully understand how to use this approach in a tissue-specific manner – something that YouthBio has recognized since the company's inception – we will see even greater increases in lifespan and more beneficial therapeutic effects in various disease models."

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"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 could play a significant role in addressing age-related cognitive decline. While there is still much to learn and refine, these early results are encouraging and provide a solid foundation for further research. We are cautiously optimistic that with continued investigation, we can develop effective therapies that may one day benefit patients suffering from conditions like 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 transgenic ARTE10 mice treated with brain-specific inducible OSKM vectors, YouthBio observed reduced amyloid beta levels and a lower brain plaque burden in pivotal areas such as the hippocampus, and improved cognitive performance in 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 outcomes in preventing Alzheimer's symptoms through brain-specific partial reprogramming in a 5xFAD mouse model. Their study, "Expansion of the Neocortex and Protection from Neurodegeneration by In Vivo Transient Reprogramming," corroborates YouthBio's positive findings.

"Our positive Alzheimer's data, along with the independent positive results by Altos Labs and its collaborators, reinforces our confidence in partial reprogramming's ability to provide a novel treatment paradigm for neurodegenerative conditions," said Deigin. "Our next key milestone is to bring our Alzheimer's therapy to clinical trials, and we are fully committed to achieving this goal."

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Clinical Trials and Beyond
YouthBio aims to advance its Alzheimer's disease therapy to clinical trials within three years. During this period, 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.

Concurrently, YouthBio will continue its discovery program, led by CSO Dr. João Pedro de Magalhães, to identify superior rejuvenating factors beyond the Yamanaka factors for use in subsequent generations of its tissue-specific gene therapies. The company will also conduct several proof-of-concept animal studies in other CNS indications and continue its progeria program.

In their progeria program, YouthBio was granted a Rare Pediatric Disease Designation by 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 for treating progeria. The PRV can be redeemed for an expedited priority review of any subsequent marketing application or it can be sold. Historical PRV transaction values have exceeded $100mm, reaching as high as $350mm.

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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 targeted 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 primary program.