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On September 8, 2022, Gatehouse Bio ("Gatehouse") announced that it will further expand its cooperation with AstraZeneca to develop the treatment of Ejection Fraction Retention (HFpEF) RNA therapy for heart failure . The collaboration will advance the development of compounds designed by Gatehouse’s AI-Powered platform, combining AstraZeneca’s scientific expertise and therapeutic experience in the RNA field with Gatehouse’s ability to design disease-modified small RNA (sRNA) compounds, leverage Undeveloped small non-coding regions in the genome accelerate the development of a wide range of novel therapies.
Gatehouse is a biotechnology company that uses AI to identify the characteristics and molecular pathways of sRNA to elucidate disease mechanisms. The sRNAlytics platform is able to analyze millions of sRNA characteristics to identify the following mutations: (1) disease classification; (2) stratified topics; (3) predict disease drivers and thus design RNA therapies. oligonucleotide , which targets sRNA mutations, is a new class of RNA therapeutics that can deliver drugs to specific tissues through the use of lipid nanoparticles and conjugates, achieving unparalleled accuracy.
In addition, the company discovered common mutations across disease indications through semantic knowledge graphs, and developed new drugs that were used in combination with small molecules, protacs, and mabs to treat multiple indications.
Gatehouse is developing oligonucleotide therapy, focusing on therapeutic areas such as inflammation, fibrosis and neurodegenerative diseases to correct disease-driven mutations in the sRNA gene.
Gatehouse's R&D pipeline
In December 2019, Gatehouse announced that it would cooperate with AstraZeneca to explore new targets in the field of respiratory diseases and cardiovascular diseases, and use Gatehouse Bio's AI empowerment platform for target screening and identification. HFpEF is considered to be the largest unmet need in cardiovascular medicine. This cooperation further deepens cooperation in the field of cardiovascular diseases, especially the heart failure field of HFpEF. According to Frost & Sullivan data, the number of heart failure patients worldwide was about 29.7 million in 2019, and it is expected to reach 38.7 million in 2030, with a CAGR of 2.5%. Among them, the ejection fraction reduction (HFrEF) and HFpEF account for the highest proportion, respectively 54% and 24%. At present, there are few HFpEF approved and under development, and there are still a large number of unmet clinical needs.
The number of patients with heart failure increases every year (unit: millions)
About sRNA and oligonucleotide drugs
sRNA is a class of 50~500 nucleotide , which is transcribed in the genome of the cell but does not encode proteins. a class of RNA molecules. They come in 2 forms: Most of the sRNAs studied are from non-coding regions located between two encoding protein genes, and a small portion of sRNAs are cut from the head or tail non-translated regions of mRNA. The main mode of action of sRNA is to bind to the target mRNA through complementary pairing, inhibit or promote the translation of the target mRNA, or accelerate or slow down the degradation of the target mRNA.
Currently, there are many types of oligonucleotide drugs under development. The most researched include antisense oligonucleotides (ASO), small interfering ribonucleic acid (siRNA), microribonucleic acid (microRNA, miRNA), nucleic acid aptamers (aptamer) and others, according to Frost & Sullivan data, the global market size of oligonucleotide drugs has increased from US$10 million in 2016 to US$3.25 billion in 2021, with an annual compound growth rate of up to 217.8%. The commercial success, further promoting the research and development of oligonucleotide drugs, especially indication drugs targeting a larger patient population, will further drive the rapid development of the market.
