DELIVERING THE FUTURE OF GENE THERAPY
At AviadoBio, our mission is to transform the lives of people living with devastating neurodegenerative diseases such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS).
Our clinical development program is built on the research of our co-founders and world-leading scientists. We work with an extensive network of leading neurologists, neurosurgeons and disease experts to evaluate our investigational gene therapies.
INVESTIGATIONAL GENE THERAPY AVB-101
AVB-101 is an investigational, adeno-associated virus-based (AAV) gene supplementation therapy for FTD patients with disease-causing mutations in the GRN gene. Designed as a one-time therapy to potentially halt disease progression, it delivers a functional copy of the GRN gene to restore progranulin levels in the brain. It is delivered by an intrathalamic infusion for targeted biodistribution with the aim to limit the treatment to only the brain itself, where it is needed. AVB-101 has been granted orphan designation by the US FDA and European Commission.
A Phase 1/2 open-label clinical study to evaluate the safety and preliminary efficacy of AVB-101 in people with a genetic sub-type of frontotemporal dementia (FTD-GRN)
WHO CAN PARTICIPATE?
- People between the ages of 30 and 75, diagnosed with FTD and a confirmed GRN mutation will be included in this study.
- To help support you, a caregiver/study partner should accompany you to all study visits, including surgery.
- There are other criteria that determine eligibility, which may be discussed with a healthcare provider.
- Further details about this study can be found at clinicaltrials.gov
ASPIRE-FTD is currently enrolling participants at the following sites. For more information and trial site contact details please visit clinicaltrials.gov
In the coming months we will be updating this list as more sites become active.
-Wielospecjalistyczna Poradnia Lekarska SYNAPSIS
-Mazowiecki Szpital Brodnowski Sp z.o.o
-Hospital Clinic Barcelona
Valencia, Spain 46026
-Hospital Universitari i Politecnic La Fe
What is FTD?
Frontotemporal dementia (FTD) is a brain disease that affects adults, with an average age of onset between 50 and 60. It is one of the most common forms of dementia below the age of 65. It is so called because it affects the frontal and temporal areas of the brain. Typical symptoms include changes in behaviour and impairment with language. There are currently no disease-modifying therapies for the treatment of FTD. Find out more about FTD here.
What is a progranulin mutation?
This is a mutation in the ‘GRN’ gene. Genes are instructions that tell the body’s cells what to do. Sometimes, genes contain errors called mutations.
Mutated gene Gene with no mutation
The GRN gene tells the body to make progranulin, which is a protein that plays an important role in the healthy functioning of cells in the brain. When there is mutation in the GRN gene, the body may not make enough progranulin, which can lead to cell death in the brain and the symptoms of FTD.
What is the ASPIRE-FTD Study?
ASPIRE-FTD is a clinical study to research the effects of AVB-101, an experimental gene therapy designed to restore levels of progranulin in the brain, potentially slowing or stopping the progression of FTD-GRN. ASPIRE-FTD is the first study of AVB-101 in humans. It aims to evaluate the safety of AVB-101 and measure its effects on progranulin levels and the symptoms of FTD.
Who can participate in the study?
You may be eligible to participate if you meet the following criteria*:
- 30-75 years old
- Diagnosed with FTD-GRN (this will be confirmed with a genetic test)
- Have a caregiver who is able to support you (including attending study visits) for the duration of the study (5 years and 3 months)
*Other criteria will also apply.
What are clinical research studies?
Clinical research studies help scientists and doctors investigate whether a new drug or treatment is safe and effective. Before a doctor can prescribe a new drug or treatment, it must go through several phases of clinical research and obtain a license from national authorities. Clinical research is only possible with the help of people who participate in research studies. All study participation is voluntary and if you decide to join a study, you are free to leave at any time.
What is the study treatment?
The study treatment in this clinical research study is an experimental gene therapy called AVB-101, which 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 dose’ directly into the brain via a minimally invasive surgical procedure, performed by a study neurosurgeon at a specialist neurosurgical center.
What does study participation involve?
“Being relentless means always striving for the best implementation of our plans and ensuring we deliver as individuals, and as a company as whole. We know that patients have limited options and time. Even when the path may not be clear, we navigate a way forward by being bold and tenacious in our decision making.”
David Cooper, MD
Chief Medical Officer
David Cooper, MD
Chief Medical Officer