Eikonoklastes Therapeutics
Eikonoklastes Therapeutics is an early stage biopharmaceutical company focused on rapidly developing innovative medicines for neurodegenerative diseases, starting with ALS. They aim to create breakthrough treatments with potential to significantly improve patient lives and lifespans, leveraging a pipeline focused on diseases with high unmet medical needs. Their approach includes first-in-class gene therapy platforms and accelerated clinical development strategies.
Industries
Nr. of Employees
small (1-50)
Products
Neuron-targeted AAV-based gene therapy candidate for Caveolin-1 overexpression
A first-in-class AAV-based gene supplement therapy designed to overexpress Caveolin-1 in neurons to promote neuroprotection and improve neuromuscular signaling and function across neurodegenerative diseases; lead indication is amyotrophic lateral sclerosis with additional indications under preclinical evaluation.
Neuron-targeted AAV-based gene therapy candidate for Caveolin-1 overexpression
A first-in-class AAV-based gene supplement therapy designed to overexpress Caveolin-1 in neurons to promote neuroprotection and improve neuromuscular signaling and function across neurodegenerative diseases; lead indication is amyotrophic lateral sclerosis with additional indications under preclinical evaluation.
Services
Collaboration with CDMOs to perform contract development and manufacturing for AAV-based viral vectors to support clinical translation.
Development and optimization of manufacturing processes, analytical assays, and quality controls for gene therapy and antibody programs.
Collaboration with CDMOs to perform contract development and manufacturing for AAV-based viral vectors to support clinical translation.
Development and optimization of manufacturing processes, analytical assays, and quality controls for gene therapy and antibody programs.
Expertise Areas
- Gene therapy development for CNS indications
- Preclinical translational research in neurodegeneration
- CMC and process development for biologics and viral vectors
- AAV vector manufacturing and CDMO collaboration
Key Technologies
- AAV-mediated gene delivery (neuron-targeted capsids)
- Neuron-specific promoters for restricted transgene expression
- Intraspinal/subpial delivery techniques
- Gene overexpression (gene-supplement) therapeutics
News & Updates
Research on gene therapy augmenting neuroprotective pathways to mitigate neurodegeneration in Alzheimer’s disease models.
Announcement of the formation of a Scientific Advisory Board to advance the company’s gene therapy pipeline.
Partnership to advance Eikonoklastes’ AAV-based gene therapy, ET-101, into clinical trials for ALS.
The FDA granted Orphan Drug Designation for ET-101, a gene therapy for ALS.
Gene delivery approach that preserves motor neuron and neuromuscular junction morphology, delays disease onset, and extends survival in ALS mice.
Appointment of a new board member with experience in the industry.
Research on gene therapy augmenting neuroprotective pathways to mitigate neurodegeneration in Alzheimer’s disease models.
Announcement of the formation of a Scientific Advisory Board to advance the company’s gene therapy pipeline.
Partnership to advance Eikonoklastes’ AAV-based gene therapy, ET-101, into clinical trials for ALS.
The FDA granted Orphan Drug Designation for ET-101, a gene therapy for ALS.
Gene delivery approach that preserves motor neuron and neuromuscular junction morphology, delays disease onset, and extends survival in ALS mice.
Appointment of a new board member with experience in the industry.