Chaperone-enhanced platform for rescuing protein misfolding in neurodegenerative diseases
This technology is a gene therapy based on engineered chaperone protein variants that rescue toxic protein aggregation for the treatment of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and related neurodegenerative diseases.
Unmet Need: Targeted therapy for protein misfolding in neurodegeneration
Current treatments for neurodegenerative diseases like amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) offer only modest benefits and do not address the underlying causes of disease progression. A major pathological hallmark of these diseases is the misfolding and aggregation of RNA-binding proteins, yet existing therapies do not effectively prevent or reverse this aggregation. There is a critical need for targeted interventions that can mitigate protein misfolding and its toxic cellular effects, especially in a disease-specific and mutation-independent manner. Addressing this need could lead to more effective, disease-modifying treatments for a broad class of neurodegenerative conditions.
The Technology: Broad-acting chaperone gene therapy for neurodegenerative diseases
This technology uses engineered variants of the human chaperone protein DNAJB6 to reduce toxic aggregation of RNA-binding proteins implicated in neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). These variants enhance the intrinsic ability of DNAJB6 to modulate the solubility and phase behavior of aggregation-prone proteins, helping to maintain them in a less toxic, gel-like state. By doing so, the approach directly targets a key driver of disease pathology without altering the underlying genetic mutations.
This technology has been validated in vitro with yeast neurodegenerative disease models and human embryonic cell lines.
Applications:
- Gene therapy for neurodegenerative diseases
- Drug discovery and screening
- Cell-based disease modeling
- Proteostasis research
- Biomarker validation
Advantages:
- Broad applicability across neurodegenerative diseases
- Targets underlying neurodegenerative disease pathogenesis
- Mutation-independent therapeutic approach with broad applicability
- Compatible with gene therapy delivery platforms
Lead Inventor:
Patent Information:
Patent Pending (US20250205305)
Related Publications:
Tech Ventures Reference:
IR CU22082
Licensing Contact: Cynthia Lang