- ASPIRE-FTD is now recruiting at The Ohio State University
Wexner Medical Center
- The study is evaluating AVB-101, an investigational gene
therapy designed to stop disease progression for people with
frontotemporal dementia with GRN mutations
AviadoBio, a pioneering gene therapy company dedicated to
developing and delivering potentially transformative medicines for
neurodegenerative disorders, today announced that its Phase 1/2
ASPIRE-FTD clinical trial is now open and recruiting patients in
the United States at The Ohio State University. ASPIRE-FTD is
evaluating AviadoBio’s investigational gene therapy, AVB-101, in
people with frontotemporal dementia (FTD) with progranulin (GRN)
gene mutations (FTD-GRN). The study is also recruiting in Europe
with study sites in the Netherlands, Poland, and Spain, with
additional sites expected to open in multiple countries.
This press release features multimedia. View
the full release here:
https://www.businesswire.com/news/home/20240701652660/en/
FTD is a devastating form of early-onset dementia that typically
leads to death within three to 10 years from diagnosis.1,2 People
with FTD commonly experience personality changes, behavioral
disturbance, loss of language, apathy, and reduced mobility.3 It is
a leading cause of dementia in people under the age of 65 and is
often misdiagnosed.4 People with FTD who have disease-causing GRN
mutations produce a reduced amount of progranulin protein. AVB-101
is a potential one-time therapy designed to stop disease
progression by delivering a functional copy of the GRN gene to
restore appropriate progranulin levels to affected areas of the
brain.
“People living with FTD-GRN have no available disease-modifying
treatments and the impact of this disorder is profound for both
patients and their families,” said neurosurgeon Dr. James “Brad”
Elder, professor of neurological surgery at The Ohio State
University Wexner Medical Center. “There is an urgent need for
collaboration between researchers, clinicians, patients, advocates,
and families to courageously explore new approaches for FTD,
including innovative and targeted delivery approaches. This study
will allow us to investigate how highly targeted GRN
supplementation could be a potential breakthrough treatment.”
“Although delivery of progranulin protein via gene therapy is
known to be possible, effective brain delivery remains a challenge
primarily due to the anatomy of the brain,” said David Cooper,
M.D., Chief Medical Officer of AviadoBio. “Our goal is to use
intrathalamic delivery to facilitate the biodistribution of PGRN
protein to areas of the cortex affected by FTD. The therapy has
already shown great promise in preclinical studies, and we now look
forward to taking this important next step in its clinical
development.”
AVB-101 is delivered as a one-time-only treatment using a
minimally invasive, stereotactic neurosurgical procedure directly
to the part of the brain called the thalamus. The thalamus has
extensive connections throughout the brain, including the frontal
and temporal lobes, which play a critical role in FTD. This
targeted delivery method aims to safely and effectively cross the
blood-brain barrier, delivering the investigational gene therapy
directly to the brain to restore GRN levels in the frontal and
temporal cortex where it is needed most, while at the same time
reducing the dose required and the potential systemic exposure.
“Thousands of people are diagnosed with FTD each year. The
relatively early onset of FTD and often strongly hereditary nature
can make it all the more challenging for patients and their
families,” said Lisa Deschamps, AviadoBio’s Chief Executive
Officer. “We are grateful to the patients and families with FTD-GRN
involved in ASPIRE-FTD, as well as The Ohio State University
researchers for their dedication to investigating AVB-101’s great
potential in stopping disease progression and offering hope to
patients and their loved ones.”
More information about the ASPIRE-FTD study can be found at
www.aspire-ftd.com or
https://clinicaltrials.gov/study/NCT06064890.
About ASPIRE-FTD
ASPIRE-FTD is an open-label, multi-center, Phase 1/2
dose-escalation study designed to evaluate the safety and
preliminary efficacy of AVB-101 in patients with FTD-GRN. In the
study, patients will receive a single administration of AVB-101
delivered as a one-time infusion into the thalamus via a
stereotactic neurosurgical procedure at expert neurosurgical
centers throughout Europe and the United States.
About AVB-101
An investigational gene therapy, AVB-101 contains a correct
(non-mutated) version of the GRN gene. It is designed to restore
levels of progranulin in the brain, potentially slowing or stopping
the progression of FTD-GRN. AVB-101 will be delivered as a one-time
infusion directly into the brain via a minimally invasive surgical
procedure, performed by a study neurosurgeon at a specialist
neurosurgical center.
About Frontotemporal Dementia (FTD) and FTD with GRN
Mutations (FTD-GRN)
FTD is a devastating form of early-onset dementia that typically
leads to death within seven to 13 years of symptom onset and three
to 10 years from diagnosis.1,2 People with FTD commonly experience
personality changes, behavioral disturbance, loss of language,
apathy, and reduced mobility.3
FTD is a leading cause of dementia in people under the age of
654 with an estimated prevalence at any one time of up to 4.6 cases
per 1,000 people.5 FTD typically strikes younger than Alzheimer’s
disease and the majority of FTD cases occur between 45 and 68 years
of age.6,7 Given the early onset, FTD can have a substantially
greater impact on work, family, and finances than Alzheimer’s
disease.8 Genetic FTD cases account for about one-third of cases
and are associated with autosomal dominant mutations in three
genes, including the GRN (progranulin) gene.9 Approximately 11,000
people in the U.S. and EU5 are living with FTD-GRN with
approximately 2,200 new cases per year.10,11 Some FTD cases may be
misidentified, and diagnostic delay is common. As disease
education, genetics knowledge, and research and treatment options
grow, these numbers are expected to increase.
About AviadoBio
At AviadoBio, we are relentlessly chasing cures by translating
groundbreaking science and precision delivery into life-changing
medicines for people living with frontotemporal dementia (FTD) and
amyotrophic lateral sclerosis (ALS). With our deep understanding of
the brain and suite of proprietary gene therapy platforms and
delivery technologies, AviadoBio is working to overcome the
challenges of delivering the right drug to the right place. Its
innovative, neuroanatomy-led approach is designed to maximize the
therapeutic potential of gene therapy to halt or potentially
reverse neurodegenerative diseases. AviadoBio was founded on
pioneering research from King’s College London and the UK Dementia
Research Institute and has a leadership team with extensive gene
therapy development, delivery, and commercialization experience
which uniquely positions the company for success in bringing
transformative medicines to patients.
AviadoBio’s investors include New Enterprise Associates (NEA),
Monograph Capital, F-Prime Capital, Johnson & Johnson
Innovation – JJDC, Inc. (JJDC), SV Health Investor’s Dementia
Discovery Fund (DDF), Advent Life Sciences, EQT Life Sciences
(Dementia Fund), and LifeArc.
For more information, please visit www.aviadobio.com and follow
us on X @AviadoBio and LinkedIn at AviadoBio.
- Onyike CU. Neuroepidemiology. 2011;37:166–167
- Riedl L et al. Neuropsychiatr Dis Treat. 2014;10:297–310
- Pressman P and Miller BL. Biol Psychiatry.
2014;75(7):574–581
- Hendriks S, Peetoom K, Bakker C, et al. Global Prevalence of
Young-Onset Dementia: A Systematic Review and Meta-analysis. JAMA
Neurol. 2021;78(9):1080–1090. doi:10.1001/jamaneurol.2021.216
- Hogan DB et al. Can J Neurol Sci. 2016;43 (Suppl
1):S96–S109
- Moore KM et al. Lancet Neurol. 19(2):145–156
- Kansal K et al. Dement Geriatr Cogn Disord.
2016;41:109–122
- Galvin JE et al. Neurology. 89(20):2049–2056
- Young JJ et al. Ther Adv Psychopharmacol. 2018;8(1):33–48
- Onyike CU and Diehl-Schmid J. Int Rev Psychiatry.
2013;25(2):130–137
- Kuang, L., et. al. Frontotemporal dementia non-sense mutation
of progranulin rescued by aminoglycosides. Human Molecular Genetics
2020;29(4):624-634
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240701652660/en/
Media Contact: Farah Speer SVP, Head of Communications
and External Relations Fspeer@aviadobio.com +1-312-543-2881