AviadoBio
AviadoBio is dedicated to transforming lives through innovative gene therapies targeting neurodegenerative diseases such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). With a neuroanatomy-led approach and proprietary platforms, the company aims to maximize therapeutic potential by delivering gene therapies directly to the central nervous system, potentially halting or reversing neurodegenerative diseases.
Industries
Nr. of Employees
small (1-50)
Products
AVB-101 (AAV-based gene supplementation therapy)
An investigational AAV-based, single-administration gene supplementation therapy designed to deliver a functional copy of a deficient CNS gene via targeted intrathalamic infusion to restore protein levels in brain tissue.
vMiX™ platform
An adeno-associated virus (AAV) vectorized gene silencing platform expressing proprietary microRNAs capable of down-regulating target mRNA levels for potential application in ALS and FTD.
vMiX™ RNA silencing system
Proprietary platform for RNA silencing to enable precise, durable therapeutic expression across complex neural systems.
AAV-miR-SOD1
Gene silencing therapy candidate for SOD1-associated amyotrophic lateral sclerosis.
AAV-miR-C9orf72
Gene silencing therapy candidate for C9orf72-associated ALS and FTD.
AAV-miR-ATXN2
Gene silencing therapy candidate for ALS and FTD.
AVB-101 (AAV-based gene supplementation therapy)
An investigational AAV-based, single-administration gene supplementation therapy designed to deliver a functional copy of a deficient CNS gene via targeted intrathalamic infusion to restore protein levels in brain tissue.
vMiX™ platform
An adeno-associated virus (AAV) vectorized gene silencing platform expressing proprietary microRNAs capable of down-regulating target mRNA levels for potential application in ALS and FTD.
vMiX™ RNA silencing system
Proprietary platform for RNA silencing to enable precise, durable therapeutic expression across complex neural systems.
AAV-miR-SOD1
Gene silencing therapy candidate for SOD1-associated amyotrophic lateral sclerosis.
AAV-miR-C9orf72
Gene silencing therapy candidate for C9orf72-associated ALS and FTD.
AAV-miR-ATXN2
Gene silencing therapy candidate for ALS and FTD.
Services
Design and conduct of early-phase open-label clinical studies for investigational CNS gene therapies, including collaboration with neurology and neurosurgery sites.
In vitro assay development and in vivo efficacy/toxicity testing using disease-relevant transgenic animal models and established potency readouts.
Development and refinement of neurosurgical procedures and delivery technologies to achieve targeted CNS biodistribution (e.g., intrathalamic infusion, spinal/subpial delivery).
Development of analytical methods (HPLC) and quantitative potency assays (RT-ddPCR) for characterization, quality control and release testing of AAV vector products.
Design and conduct of early-phase open-label clinical studies for investigational CNS gene therapies, including collaboration with neurology and neurosurgery sites.
In vitro assay development and in vivo efficacy/toxicity testing using disease-relevant transgenic animal models and established potency readouts.
Development and refinement of neurosurgical procedures and delivery technologies to achieve targeted CNS biodistribution (e.g., intrathalamic infusion, spinal/subpial delivery).
Development of analytical methods (HPLC) and quantitative potency assays (RT-ddPCR) for characterization, quality control and release testing of AAV vector products.
Expertise Areas
- CNS gene therapy development
- AAV vector design and engineering
- RNA interference therapeutics (miRNA/RNAi)
- Neuroanatomy-led delivery strategies
Key Technologies
- Adeno-associated virus (AAV) vectors
- AAV9-based constructs
- AAV-delivered RNA interference (miRNA/RNAi)
- Intrathalamic infusion delivery
News & Updates
AvidoBio announced the initiation of a Phase 1/2 clinical trial in the UK to evaluate its gene therapy AVB-101 for frontotemporal dementia (FTD).
The company announced the completion of the second cohort in its ongoing clinical trial evaluating AVB-101 for FTD with GRN mutations.
AvidoBio announced the initiation of a Phase 1/2 clinical trial in the UK to evaluate its gene therapy AVB-101 for frontotemporal dementia (FTD).
The company announced the completion of the second cohort in its ongoing clinical trial evaluating AVB-101 for FTD with GRN mutations.