• Technology
A leading method for gene editing in the CNS
Evox Therapeutics is advancing a transformative approach to treating genetically-driven neurodegenerative diseases by combining state-of-the-art genome editing technologies with the natural biological capabilities of exosomes.
CRISPR-Cas genome editing is a groundbreaking technology that enables modification of specific genetic sequences underlying disease pathology. However, applying this modality to severe CNS disorders has been challenging due to the liver-centric limitations of LNPs and the safety concerns associated with viral vectors.
Evox’s gene editing medicines are designed to address these challenges by offering safer, more effective and redosable genetic medicines. Through our ExoEdit® platform, we are expanding genetic medicine from a largely liver-specific modality to a drug class capable of engaging the most complex organ in the body.
ExoEdit®
Evox has developed ExoEdit®, a proprietary genome-editing technology that harnesses the natural delivery power of exosomes, tiny vesicles naturally produced and used by cells as transport vehicles, to precisely deliver CRISPR-based editing tools into specific organs and cells.
Our technological leadership over the past decade has established exosome-based genetic medicines as a premier modality with a potentially differentiated safety, potency, manufacturability and versatility profile.
Safety
- Because of their natural origin, exosomes exhibit low immunogenicity and toxicity. This makes them well-suited for targeting the brain, which is especially vulnerable to the toxicity risks of viral vectors as well as risks of inflammatory and immune reactions from LNPs
- We have demonstrated the safety of exosomes at therapeutic doses, adding to the strong safety profile established by over fifty in-human clinical trials
- Our Cas9 RNP genetic editors degrade quickly, greatly reducing the chances of off-target activity. In contrast, the activity of viral-based gene editors may persist for months or years, which increases the risk of off-target editing
Potency
- By concentrating high amounts of genome editing modalities, such as CRISPR/Cas9, into exosomes, we have increased the potency of our therapeutic. Combined with broad cell tropism in the CNS and efficient neuronal penetration, this potency leads to 10X gene editing efficiency compared to leading non-viral platforms in mouse models
- Our editors introduce permanent genetic changes that can potentially eliminate the target protein within a cell. In contrast, RNA-targeting approaches such as RNAi or antisense oligonucleotides (ASOs) only transiently reduce protein levels
Manufacturability
- ExoEdit® therapeutics are manufactured using a conventional and standardized process closely resembling that of antibodies
Versatility
- The modular design of Evox’s gene editing platform readily integrates a wide range of genome-editing technologies. And while our primary focus is the CNS, our platform has also been substantially validated in other tissues. Together, these strengths provide the flexibility to meet evolving therapeutic needs and unlock potential opportunities across multiple disease areas