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TECHNOLOGY

In gene-based therapies, delivery is differentiating

THE OPPORTUNITY

+First generation gene therapies have created significant value via delivery to only a few tissue and cell types.

Targeted delivery
to new tissues

A major opportunity as the existing approaches have various tradeoffs related to potency, tolerability and formulation.

Despite mixed results in aerosol delivery of mRNA,
the field continues to push forward with LNPs

Problems with existing solutions.


PARAMETER

RESULT

PARAMETERRESULTTHERAPEUTIC IMPLICATIONS
Potency
Low
Requires high doses
Immunogenicity
High
Drives dose-limiting toxicity
Formulation
Complex
Formulate for each payload
Aerosol Stability
Fragile
Degradation during delivery
Lung Deposition
Airways
Limits disease targets

Biodegradable
polymers: next-generation gene
delivery vehicles

The tunable chemical structure of polymers enables design of tailor-made particles that can be engineered to reduce cytotoxicity, optimize cargo capacity, and increase affinity for specific tissue or cell types. The potency and flexibility of these nanoparticles makes them an ideal fit for addressing the challenges of aerosol delivery.

+Tether’s polymers are differentiated from LNP-based approaches to lung delivery

Optimized for lung delivery, our proprietary class of polymers are a next-generation delivery solution based on novel chemistry developed at Georgia Tech and Emory University. Tether’s proprietary polymer formulations address the bottlenecks facing first-generation approaches, and outperform leading LNPs in delivery to the lungs.

1/4

Potency

Protein expression orders of magnitude higher than previously studied LNPs

2/4

Tolerability

No signs of inflammation or toxicity exhibited in rodent and NHPs deliveries

3/4

Translatable Delivery

Demonstrated expression across the airways and lungs of six different species

4/4

Payload-agnostic Chemistry

Capable of carrying nucleic acid payloads of various sizes, including mixed payloads

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