For Rare Diseases

Introduction to Rare Diseases

Rare diseases are defined as those that affect fewer than 7, 4, or 5 in 10000 people in the USA, Japan, or EU, respectively. Individually, these diseases are rare, but overall, they affect about 475 million people, or approximately 6% of the global population^[1]. 80% of cases are caused by inherited genetic mutations, and the remaining 20% by infectious and autoimmune diseases. At present, there are about 7000 inherited rare diseases that have been identified, but 95% of them have no therapeutic methods. Rare diseases are often severely disabling, substantially affect life expectancy, and impair physical and mental abilities, reducing people's quality of life^[2].

Oligonucleotide Therapeutics for Rare Diseases

Oligonucleotide therapeutics were proposed based on the concepts of central dogma and RNA interference (RNAi). This type therapeutic has outstanding advantages in regulating genes and is therefore developed and used in the treatment of rare diseases. Up to now, fifteen oligonucleotide therapeutics including fomivirsen, pegaptanib, mipomersen, defibrotide, eteplirsen, nusinersen, inotersen, patisiran, volanesorsen, givosiran, golodirsen, viltolarsen, lumasiran, inclisiran and casimersen, have been approved in various countries, and most of them are used for rare diseases (Table 1)^[3].

Table 1. Approved oligonucleotide therapeutics

We are Developing Products for Rare Diseases

Our team consists of senior experts in the field of oligonucleotide therapeutics with many years of experience in rare disease drug discovery and development and the most cutting-edge technological capabilities in the industry. We are currently developing oligonucleotide therapeutics for the treatment of various rare diseases, and the following are a few examples of our products in development. If your research requires related technology, please contact us.

Note: FCS means familial chylomicronemia syndrome; HoFH means homozygous familial hypercholesterolemia.

Our products and services are for research use only and cannot be used for any clinical purposes.

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