Oraxion Therapeutics is a drug discovery company focused on rare and orphan diseases.
A rare disease is defined as one that affects less than 1 in 2000 patients. There are an estimated 7000 distinct rare diseases known with a global population of ~350 million patients. USA is expected to have ~30 million patients, while India is expected to have ~70 million patients suffering from rare diseases.
Fewer than 5% of these diseases have therapeutic alternatives available. More than 80% of these diseases have a genetic origin while almost 50% of them primarily affect children.
Oraxion aims to develop therapeutics for these rare diseases with a high unmet medical need. We are leveraging our technology platform to develop novel therapeutic modalities for debilitating rare diseases with the goal of creating a novel therapeutic intervention that can improve the quality of life for patients significantly.
Of the 7000 distinct rare diseases, greater than 95% of them do not have a single clinically approved treatment. For many rare diseases basic knowledge, such as, cause of the disease, pathophysiology, semiology, natural course of the disease and epidemiological data is limited or worse, missing. This significantly hampers the ability to both diagnose and treat these diseases. Rare diseases also have a huge socio-economic burden on society due to the direct and indirect costs incurred by the patients and their families which in turn affects the health system.
The global rare disease market is fast approaching the USD 100 Billion mark and there are close to 600 orphan drugs in various stages of clinical development currently. Several academic groups, government organizations, patient support groups, and biotech and pharmaceutical companies are coming together to address the unmet needs of these rare diseases. With awareness of rare diseases and their biology increasing, novel therapies for previously incurable or untreatable diseases will be developed.
To bring affordable healthcare solutions for rare and orphan diseases with high unmet medical needs
To leverage the potential of bionanotechnology and drug delivery for the development of novel and affordable therapeutic alternatives for patients with rare diseases.
To achieve the desired therapeutic effect while avoiding off-target effects, a drug or biopharmaceutical needs to have target specificity, an optimal pharmacokinetic profile and clearance profile.
Several delivery systems have been studied and developed for achieving these characteristics and facilitating the efficient delivery of the drugs or bioactives. However most of these systems cannot be translated clinically due to a lack of robustness, difficulty in production owing to high costs, inefficient unpackaging and release of drug, inherent toxicity and immunogenecity.
We have designed our technology platform as a way to address these issues and thereby enable the development and translation of therapeutics for rare diseases with a high unmet medical need.
We design platform technologies based on “first principles thinking” to address unmet medical needs. Novel therapeutic solutions are developed by combining materials chemistry and discovery biology. Our agnostic approach to drug discovery allows us to employ diverse technologies for the development and delivery of novel drugs for specific disease targets with a well understood underlying pathophysiological mechanism.
At Oraxion, we are developing a class of polymer drugs that target diseases where aberrant cholesterol and lipid accumulation is implicated as one of the key causes. We are advancing a diverse pipeline covering a broad range of rare diseases with our partners who have extensive expertise in disease biology.
Niemann-Pick Type C Disorder
Focal Segmental Glomerulosclerosis
We would like to dedicate this milestone in memory of our recently departed friend and colleague Soniya Johny who was instrumental in bringing this work to fruition.