Science
A significant challenge has prevented genetic medicines from reaching their full potential: Delivery. The genetic medicine delivery methods available today limit the potential benefit to patients.
NanoGalaxy® Platform
At GenEdit, we are overcoming this limitation with our proprietary technology, NanoGalaxy®. Our NanoGalaxy platform contains a library spanning thousands of unique non-viral, non-lipid, polymer nanoparticles. Using our iterative screening process, we are able to identify polymer nanoparticles that can deliver the needed genetic medicine payload to the needed tissue to treat disease.
Our combinatorial library of hydrophilic polymers is designed to create nanoparticles with diverse attributes. Polymer backbones with diverse side chains are combined with diversity structures to yield hundreds of thousands of structurally distinct polymers. Nanoparticles created from these polymers can be further diversified by size and other properties.
Our iterative screening process identifies tissue-selective polymers. We utilize in vitro primary cell screening, in vivo assays for functional delivery of payloads, and computational methods to identify key characteristics that impart delivery selectivity.
In addition to tissue-selectivity, GenEdit’s polymer nanoparticles possess other attributes that make them uniquely suited for delivery of genetic medicine therapies. The ability to carry whichever therapeutic payload modality is needed, and to dose multiple times, expands the range of diseases that can be treated and patients that can benefit. With NanoGalaxy, we are now on a trajectory to develop therapies for the patients who need them most.
Payload Flexibility
Ability to Redose
Ease of Manufacture
Tissue Selectivity
NanoGalaxy nanoparticles deliver genetic medicines to specific tissues in the body where they can provide therapeutic benefit. This increases efficacy and reduces potential safety liabilities.
With NanoGalaxy, we use a systematic screening process to identify tissue-selective nanoparticles. Hundreds of chemically distinct polymer nanoparticles are screened in primary human cells and in vivo to identify key structure-activity relationships for tissue selectivity. Through rounds of optimization, we select the optimal polymers for delivery to the specific organ and cell type needed.
Ability to Redose
Unlike viral delivery systems, patients are unlikely to have pre-existing immunity to GenEdit’s nanoparticles, making treatment available to a much larger number of people. The low immunogenicity of our nanoparticles shows promise in enabling redosing for patients in need of multiple doses to achieve the desired therapeutic benefit.
Pre-existing immunity can prevent 30-50% of patients from being treated with therapies utilizing viral delivery systems. GenEdit has not observed an immune response or loss of payload function upon single or multiple administrations in vivo of our polymer nanoparticles. The low risk of vector-mediated immunogenicity with GenEdit’s nanoparticles should support a more predictable treatment outcome.
Payload Flexibility
Genetic medicines provide therapeutic benefit through a range of desired actions. From adding, silencing or deleting a gene to correcting a mutation, GenEdit’s delivery system can carry the needed therapeutic payload to its needed site of action.
Our polymer nanoparticles can encapsulate a number of diverse payloads, including nucleic acids (for example, mRNA, ASOs or siRNA), therapeutic proteins and CRISPRs. Our nanoparticles can carry the large nucleic acids and enzymes required for gene editing. Our functional payloads range from 20bp siRNA to 10kbp constructs.
Ease of Manufacture
Our chemical synthesis manufacturing process for our polymers is designed for batch-to-batch purity and reproducibility while also being scalable to support future commercialization. Our polymer nanoparticles can be lyophilized for long-term stability and reduction in cold-chain storage and shipping logistics.
Our polymers are water-soluble and can produce nanoparticles containing a broad spectrum of genetic medicine payloads. This formulation of polymer nanoparticles in aqueous solution enables encapsulation of not only nucleic acids but also ribonucleoprotein payloads without denaturation. Our process allows us to capture the economies of scale to lower overall manufacturing costs.
Focus Areas
NanoGalaxy creates a new universe of possibilities for curative genetic medicines. We are focusing our internal research on the treatment of diseases for which our delivery technology can enable therapeutic options that otherwise may not be possible. We are currently developing a pipeline of therapeutic candidates that target the immune and nervous systems to treat a range of diseases with high unmet medical need.
Publications and Presentations
The first-generation of our platform demonstrated efficient preclinical gene editing in muscle for Duchenne muscular dystrophy (Nature Biomedical Engineering, 2017) and the brain for Fragile X syndrome (Nature Biomedical Engineering, 2018). In addition, the polymer library has also enabled the delivery of Cpf1 (Cas12a), a next-generation CRISPR, in muscle (Nature Communications, 2018).