Rejuvenate Bio Receives $4M in Funding from the California Institute for Regenerative Medicine (CIRM)
July 01 2024 - 8:00AM
Business Wire
- Funding will advance development of Rejuvenate’s one-time gene
therapy RJB-0402 for the treatment of desmoplakin gene variant
arrhythmogenic cardiomyopathy
Rejuvenate Bio, announced today that the California Institute
for Regenerative Medicine (CIRM) has awarded the company a $4M
grant to complete IND-enabling activities for the development of
RJB-0402 for the treatment of desmoplakin gene variant
arrhythmogenic cardiomyopathy (DSP ACM).
RJB-0402 is a novel systemically administered investigational
AAV8 gene therapy that drives liver specific expression of FGF21
protein, targeting multiple key pathological drivers of DSP ACM.
Since the DSP gene is too large for gene replacement therapy, novel
strategies are needed to treat DSP ACM. FGF21 has known salutary
effects on cardiac dysfunction as well as ventricular arrhythmias,
adipogenesis, inflammation, and fibrosis, all of which are DSP ACM
hallmarks. Furthermore, clinical efficacy of RJB-0402 in DSP ACM
may form the basis for the treatment in other forms of ACM.
“DSP ACM is a rare, severe, life-threatening, and debilitating
disease that typically manifests in young adults as a high risk of
life-threatening ventricular arrhythmias, sudden cardiac death, and
progression to heart failure,” said Dan Oliver, CEO &
Co-Founder, Rejuvenate Bio. “There is currently no disease
modifying therapy for patients with this disease, and our gene
therapy RJB-0402 addresses a significant unmet medical need.”
“The funding and strategic support from CIRM will accelerate
development of our gene therapy candidate RJB-0402 into clinical
trials for DSP ACM patients in desperate need of new therapies,”
said Noah Davidsohn, Ph.D., Chief Scientific Officer &
Co-Founder, Rejuvenate Bio. “We are confident that RJB-0402, a
one-time gene therapy, could be a transformative regenerative
medicine and the first disease modifying therapy to address the
unmet medical need of DSP ACM patients, and the recent funding from
CIRM will enable us to initiate our first in human clinical
trial.”
Nonclinical proof-of-concept studies in the ACM mouse model
showed that RJB-0402 significantly improved cardiac structure,
function, and markedly reduced the arrhythmia burden, normalizing
premature ventricular contractions (PVCs) to wild type levels. The
clinical relevance of these efficacy signals provides a scientific
rationale for further development as a therapeutic with the
potential to meaningfully impact patients’ survival, symptoms, and
quality of life.
“Our goal is to move the most promising research forward,” said
Dr. Abla Creasey, Vice President of Therapeutics Development at the
California Institute for Regenerative Medicine (CIRM). “A one-time
gene therapy treatment for patients with this rare cardiac disease
DSP ACM, would have significant impact for patients with this
degenerative disorder. We look forward to supporting Rejuvenate Bio
in bringing this regenerative therapy to patients with this
degenerative disease.”
About Desmoplakin Arrhythmogenic Cardiomyopathy (DSP
ACM)
DSP ACM is a severe, life-threatening, and debilitating, rare
autosomal recessive disease that results in ventricular arrhythmias
that are often life threatening and progressive cardiac dysfunction
caused by fibrofatty replacement of ventricular myocardium which
can lead to heart failure for which no disease modifying therapies
exist. While more than 10 different genes have been implicated in
this heterogeneous disease, the majority involve one or more
variants in desmosomal genes which are key components of the
cardiac intercalated disc that are essential for the electrical and
mechanical coupling of cardiomyocytes. These multiprotein complexes
work together to provide cardiac mechanical integrity and strength
as well as electrical communication and synchronization. While the
exact prevalence of DSP ACM is unknown, it is estimated to affect
an estimated 30,000-50,000 people in the United States.
About Rejuvenate Bio
Rejuvenate Bio is a biotechnology company dedicated to
developing novel gene therapies for chronic age-related diseases.
Rejuvenate Bio has built a gene therapy pipeline with huge
potential in chronic disease by utilizing clinically validated gene
targets and a delivery approach that ensures well tolerated,
durable expression. Founded on scientific research developed at the
Wyss Institute at Harvard Medical School, Rejuvenate Bio has
developed groundbreaking therapies to treat chronic age-related
disease in both humans and animals. The company is headquartered in
San Diego, CA. For more information, visit
www.rejuvenatebio.com.
About 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 the world’s largest institution
dedicated to helping people by bringing the future of cellular
medicine closer to reality.
For more information go to www.cirm.ca.gov.
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