SEATTLE, Aug. 6, 2024
/PRNewswire/ -- 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."
"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."
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
to receive an expedited priority review for any subsequent
marketing application or sold. Historical PRV transaction values
have exceeded $100mm, reaching as high as $350mm.
***
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.
Contact:
Yuri Deigin
yuri@youthbiotx.com
Photo(s):
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SOURCE YouthBio Therapeutics Inc.