A Munich-based biotech venture called Secarna Pharmaceuticals GmbH & Co. KG (“Secarna”) focusing on the discovery and development of next generation antisense oligonucleotide (ASO) therapies to address challenging or previously undruggable targets via its LNAPlus™ platform reports it entered into a collaboration with The First Affiliated Hospital at Guangzhou Sun Yat-sen University (SYSU) for the development of a treatment of prophylactic for SARS-CoV-2 viral infections. The German company will deploy the LNAplusTM platform to develop antisense oligonucleotides (ASOs), which specifically suppress the expression of a certain host factor that is essential for the viral infection cycle.
TrialSite News offers a brief breakdown of this new partnership targeting COVID-19.
What are ASO therapies?
With the advent of the genetic revolution came the identification and classification of a numerous neurologic disease genes. With great hopes of curing diseases investment poured into ventures that sought to track down mutated protein and place into a known cellular pathway translated into robust new therapies. As reported in Antisense Oligonucleotides a Primer, this promise “has remained largely unfulfilled.” But now great new opportunity for therapy development materialized with the ability to target the disease gene or its encoded messenger (RNAs) (mRNAs). The use of antisense oligonucleotides represents one of the approaches to target expression of RNA.
They are DNA- or RNA-like substances, synthetic versions of those that are able to potentially have or have demonstrated impact in a range of disease areas. An emerging therapeutic modality, it can be thought of as a blend of biological and small molecule as they are short synthetic structures that are very much like nucleic acids—sort of a gene-based modality so now developers can target very specific genes within the cell to treat disease. Can this be translated into a drug? Yes, this is a trend moving forward. The emergence of ASO brings a new and important treatment modality—a treatment option that didn’t exist prior.
The successes on the development of antisense oligonucleotide (ASO) therapeutics for spinal muscular atrophy and Duchenne muscular dystrophy predict a robust future for ASOs in medicine, as suggested in Antisense Oligonucleotides a Primer. The outlook for the utilization of ASOs for neurodegenerative diseases looks strong as the existing pipeline for the development of ASO therapies includes spinocerebellar ataxias, Huntington disease, Alzheimer’s disease, amyotrophic lateral sclerosis, Parkinson’s disease, and others.
A potential advantage ASO has over other small molecule approaches “is that acquisition of the target sequence provides immediate knowledge of putative complimentary oligonucleotide therapeutics” as reported in Antisense Oligonucleotides a Primer.
Has their specific approach been successfully tested before?
Yes. The feasibility of Secarna’s concept has been already demonstrated in 2019—the company employed its proprietary platform (LNAplus™) to develop ASOs targeting the host factor NPC1 to efficiently reduce Ebola virus infection in vitro.
Is the approach currently being evaluated?
Yes. Prof. Claus Bachert at the University of Ghent in Belgium and the First Affiliated Hospital at Guangzhou Sun Yat-sen University (SYSU) as discussed in this announcement.
Why is Secarna’s approach to target host factors superior to others when considering the direct targeting of the SARs-CoV-2 virus?
Most viruses are known to evade targeted therapies by mutation over time. This risk can be avoided by targeting host factors. As different viruses can depend on a common host factor, a therapy such as this one, according to the sponsor, could potentially serve as a treatment against existing and future challenges.
According to Jonas Renz, Managing Director and Co-Founder of Secarna Pharmaceuticals, “We are very excited to be working together with Guangzhou’s Sun Yat-sen University and leveraging our proprietary technology to develop LNAplus-based ASOs to potentially tackle SARS-CoV-2 infections. By targeting a host factor essential to the viral infection cycle, we hope to present the infection of cells and viral spread.”
What are more details involving LNAplus?
A proprietary drug development platform—actually in the form of a proprietary third-generation antisense oligonucleotide (ASO) platform—LNAplus, it encompasses all aspects of drug discovery and pre-clinical development, enabling the company to discover novel antisense-based therapies for challenging or currently undruggable targets. The Secarna platform and ASOs have previously been validated by numerous in-house projects as well as several academic and industry collaborations.
The company has over 15 development programs with promising targets in indications such as immuno0oncology, immunology, ophthalmology, viral-neurodegenerative-and cardiometabolic diseases, where antisense-based approaches have clear benefits compared to other therapeutic modalities. The company positions itself as the leading independent European antisense drug discovery and development company.
Secarna Pharmaceuticals is the next-generation antisense oligonucleotide (ASO) company with multiple innovative antisense therapies in various stages of pre-clinical development in the areas of immuno-oncology, immunology, ophthalmology, as well as viral-neurodegenerative and cardiometabolic diseases. Founded in 2015, the company is privately held. They have approximately 10 to 20 employees.