Background About ASO
Therapeutic oligonucleotides, including single stranded ASO and double stranded siRNA, 10–50 natural or modified nucleotides in length, is a versatile approach for treating a wide range of diseases. They exhibit numerous advantages, including:
- Feasible for a diverse range of disease targets.
- Improved efficacy.
- Greatly accelerate R&D process.
- Providing relative cost-effectiveness at large industrial scales.
- Offering a structured and enhanced safety profile.
However, Challenges like limited tissue specificity and poor cellular uptake makes extrahepatic delivery of oligonucleotides still problematic. Moreover, ASOs' potential in gene activation and allele specific knockdown remains largely untapped, hindering their application in a broader range of diseases.
- Target design challenges include the limitations of using a two-dimension mRNA structure prediction model, which cannot accurately represent the true mRNA structure, as well as the labor-intensive gene-walking method, which has a low success rate. Furthermore, the current approach is more applied to targeting and down-regulating mRNA, while upregulation of gene expression and allele specific knockdown is rarely achieved.
- Chemical design hurdles include the instability of oligonucleotides, making it difficult to control their half-life. Furthermore, the poor cell uptake and lack of tissue specificity greatly limit their effectiveness and impact the overall safety profile of these compounds.
About the Company
Shenzhen xNA Biotechnologies Co., Ltd. is an innovative biotech company specializing in the design and development of therapeutic antisense oligonucleotides (ASO). Founded in 2021, We focus on optimizing ASOs through two primary dimensions: delivery and expended mechanisms.
We make all the way through target sequence discovery, molecule design, chemical manufacture to qualitative analysis, breaking barriers between omics data and druggable target identification in translational medicine, to develop safe, robust, highly specific and organ enriched ASO drugs which transform treatment paradigms for conditions caused by various types of mutations.
What's Unique
- Naked, and Organ-Specific Targeting
Without the need for conjugation or coating, xNA's ASO can efficiently enrich in desired organs with IV or SC injection. We have successfully achieved targeting of various organs, such as the kidney, muscle, adipose tissue, liver, lung, CNS and etc.
- Allele-Specific Knockdown and Direct Activation
Expended ASO mechanisms, including universal knock-down (ASOi), allele specific knockdown, splice switching, and direct upregulation (ASOa).
- Broad-Spectrum Assets for Modern Diseases
Our assets are developed to tackle a variety of modern diseases, including obesity, muscle wasting, Alzheimer's disease, NAFLD, and etc. We also offer personalized medicine solutions.
Our Team
Shengpei Chen, MSc
Founder & CEO
Wanting Chen, PhD
Co-founder & COO
Xiaoyu Pan, PhD
Director of Preclinical Research
Yuxin Xie, PhD
Director of Computational Physics