Dr. Walter Schmidt, CEO
What if we could trick the human body into curing itself? While the idea seems far-fetched, an Austria-based startup ACCANIS—founded by a team of highly-qualified visionary scientists—is working on making this a reality. The firm is on a mission to introduce specially designed messenger RNA (mRNA) into the body to help cure diseases.
As one of nature’s core building blocks, mRNA is responsible for translating genetic code into functional proteins, providing the blueprint for each cell within the body. Abnormal gene expression, caused by a mutation in a DNA sequence, can result in the transcription of defective instructions. The translation of defective instructions by the cell can lead to protein defects, which are the underlying cause of genetic diseases. By engineering mRNA to carry the right information, it is possible to deliver the right message to the cells to produce everything from disease-fighting proteins to functional antibodies. ACCANIS builds on this same concept and develops innovative therapeutics for the treatment of common, localised diseases using mRNA. Dr. Walter Schmidt, CEO of the firm, says, “Our innovative technology takes advantage of the natural mechanism that already happens in the body to enable uniquely-effective, on-demand therapeutics.”
Historically, despite their advantages, mRNAs were considered to be highly unstable and difficult to manipulate to be efficiently used as therapeutics. However, researchers recently realised that by making chemical modifications on the nucleotide building blocks that form the mRNA or by using certain formulations, these limitations can be overcome, and protein translation can be improved. While several advanced companies have succeeded in obtaining preclinical proof-of-concept in animal models, the challenge now lies in making the technology work on humans.
We tap into the most foundational way the body works, aiming to deliver instruction manuals for our cells to churn out proteins to reverse all kinds of diseases
This is where ACCANIS is proving its mettle. The company is en route to creating a breakthrough in mRNA therapy with its proof-of-concept in human skin explants and in pigs in vivo already achieved.
ACCANIS develops tailor-made mRNAs by repurposing and reformatting validated targets. ACCANIS´ therapeutic program builds mRNAs that encode for proteins which are known to be effective. Dr. Schmidt adds, “Our approach is simple. We tap into the most foundational way the body works, aiming to deliver instruction manuals for our cells to churn out proteins to reverse all kinds of diseases without stimulating an immune response or causing unwanted side-effects.”
Unlike other peers in the industry, ACCANIS focuses explicitly on the skin. Since the skin is easy to analyse and is readily accessible, the effect of the introduced mRNA can be evaluated with minimal effort. Using this approach, the firm tested different in vitro-transcribed Interferon alpha (IFN-α) mRNA constructs in human skin tissue explant and ‘living’ pigs. The lead for the treatment of actinic keratosis and non-melanoma skin cancer was identified, which expressed bioactive amounts of the protein leading to a significant expression of the respective responder genes. Similar tests were performed with FGF2 mRNA constructs for skin rejuvenation and the lead here is also already identified. “We are seeing it work. The mRNA induces the responder genes several fold above background, which then drives the synthesis of specific proteins that, at the end of the day, should make the skin look fresh and young again,” informs Dr. Schmidt.
ACCANIS doesn’t plan to stop there. The company is currently examining the efficacy of its mRNA technology by conducting clinical trials to generate human proof-of-concept data. Once it is successful, ACCANIS will continue to expand the use of its mRNA technology for further indications.
With the first of many mRNA therapies in the clinic, proving their safety, physiologic activity, and potential for efficacy in patients, the path forward for ACCANIS looks promising.