New Study Demonstrates Breakthrough with Retinal Progenitor Cell Therapy in a Preclinical Model of Photoreceptor Degeneration
05 Agosto 2024 - 5:00AM
Business Wire
University of California, Irvine and University of California,
Santa Barbara Researchers Report Groundbreaking Findings
Researchers from the Gavin Herbert Eye Institute and Sue &
Bill Gross Stem Cell Research Center at the University of
California, Irvine, in collaboration with the Neuroscience Research
Institute at the University of California, Santa Barbara, have
published a pioneering preclinical study demonstrating the
effectiveness of intravitreal injection of retinal progenitor cells
(RPCs) (jCell) in treating photoreceptor degeneration. This
research has been published on July 24 in the International Journal
of Molecular Sciences.
Photoreceptor dystrophies such as Retinitis Pigmentosa (RP) are
a major cause of blindness, affecting millions worldwide and
existing treatments are extremely limited. In RP, for example,
there is no FDA approved treatment for 99% of patients. The study
titled "Amelioration of photoreceptor degeneration by intravitreal
transplantation of retinal progenitor cells in rats" presents a
novel approach using RPCs to provide neuroprotection and functional
rescue of degenerating photoreceptors.
Key findings include:
- Innovative Treatment Approach: The research utilized
intravitreal injection of RPCs in a well-established animal model
of photoreceptor degeneration. The RPCs release neurotrophic
factors which are required to maintain the health and function of
retinal cells. This is a novel use of these cells and delivering
them to the vitreous cavity greatly simplifies the treatment
procedure versus more invasive alternatives.
- Therapeutic Benefits: The RPC treatment demonstrated
substantial preservation of both retinal structure and visual
function.
- Clinical Progress: Supported by this preclinical work in
rats, jCell has progressed through multiple clinical trials in
patients with retinitis pigmentosa (RP). jCyte held a successful
post-phase 2 Type B meeting with the FDA in January 2024 marking a
significant milestone towards a pivotal trial in RP.
Henry J. Klassen, MD, PhD, senior author and professor at the
Gavin Herbert Eye Institute, stated, "This study offers promising
proof-of-concept for using intravitreal injection of RPCs to treat
photoreceptor degeneration. Our findings underpin the clinical
development of jCell which has the potential to change the course
of the disease in retinal degenerations like RP."
The activities described were made possible by grant funding
from the California Institute of Regenerative Medicine (CIRM).
The publication of this study marks an important step forward in
the field of regenerative medicine and ophthalmology. Related
patents have been awarded in the US and internationally,
underscoring the innovative nature of this approach.
For more information, please contact: Dr. Henry Klassen Email:
hklassen@hs.uci.edu
About the Gavin Herbert Eye Institute
The Gavin Herbert Eye Institute is dedicated to advancing the
understanding and treatment of eye diseases through innovative
research and clinical care.
About the Neuroscience Research Institute
The Neuroscience Research Institute at the University of
California, Santa Barbara, focuses on understanding the fundamental
principles of brain and neural function through interdisciplinary
research.
About the California Institute for Regenerative Medicine
(CIRM)
At CIRM, we never forget that we were created by the people of
California to accelerate stem cell treatments to patients with
unmet medical needs, and act with a sense of urgency to succeed in
that mission. To meet this challenge, our team of highly trained
and experienced professionals actively partners with both academia
and industry in a hands-on, entrepreneurial environment to fast
track the development of today’s most promising stem cell
technologies.
With $5.5 billion in funding and more than 150 active stem cell
programs in our portfolio, CIRM is one of the world’s largest
institutions dedicated to helping people by bringing the future of
cellular medicine closer to reality.
About jCyte
jCyte, Inc. is a clinical-stage biotech company focused on
developing its first-in-class allogeneic cell therapy, jCell, for
retinitis pigmentosa (RP) and other retinal degenerative disorders.
The treatment has received FDA Regenerative Medicine Advanced
Therapy (RMAT) designation and is administered as a minimally
invasive intravitreal injection. Currently there are no FDA
approved therapies for over 99% of RP patients. The company is
pioneering a new era of regenerative therapies to address the
significant unmet medical needs of patients suffering from a broad
set of retinal degenerative diseases. For more information, visit
www.jcyte.com.
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