How GentleGen leverages ASO to write a new chapter in precision therapy amid the oligonucleotide wave

Release time:2026-06-25

At the start of 2026, the oligonucleotide sector surged: Ribo Life Science surged 41.6% on its first day of listing, and China Biopharmaceutical acquired Hejia Biotech for 1.2 billion yuan...... A clear signal has emerged: oligonucleic acid drugs are being mass-produced as the "next-generation major drug."

Novartis's blockbuster small nucleotide drug Leqvio® has achieved accelerated growth beyond the U.S. region, reaching the blockbuster drug base by 2025, with sales revenue reaching $452 million in Q1 2026, a year-on-year increase of 76%.

The global oligonucleotide new drug market is expected to reach $7.1 billion by 2025, with total revenue exceeding $2.2 billion in Q1 2026, a year-on-year increase of about 45%. By 2025, sales of ASO drugs are expected to reach $3.1 billion, with their commercialization value already proven in practice.

ASO: Cutting off disease signals at the source

Among the many oligonucleotide technology routes, ASO (antisense oligonucleotides) is considered the "most experienced" member. By binding RNase H to degrade target mRNA and regulating pre-mRNA splicing methods, it blocks pathogenic protein synthesis at the mRNA level and targets that traditional drugs struggle to cover.

Among the 23 oligonucleotide drugs already marketed, 14 are ASO drugs. Nusinersen sodium, developed in collaboration between Ionis and Biogen, is a classic creation, with sales reaching $1.741 billion in 2023.

However, from target selection to final drug development, the synthesis design of ASOs—sequence specificity, chemical modification strategies, quality control—is the absolute core factor determining efficacy and safety.

Bepirovirsen: A milestone in the "functional cure" of hepatitis B

The most anticipated ASO product in 2026 will undoubtedly be GlaxoSmithKline's Bepirovirsen. In January, GSK announced that both of its pivotal Phase III clinical trials had met the primary endpoint—Bepirovirsen combined with existing therapies showed statistically and clinically significant "functional cure" rates.

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It is an ASO therapy with a triple mechanism of action, inhibiting viral DNA replication, lowering hepatitis B surface antigen levels in the blood, and activating the immune system to improve the chances of a lasting response.Currently, Bepirovirsen's marketing application has received priority review from the FDA, with the PDUFA date set for October 2026, and China's CDE has also accepted and included it in priority review.

In May, CP Tianqing and GSK reached an exclusive strategic cooperation to handle the import, distribution, and promotion of this product in mainland. Once approved, it will become the world's first drug to achieve a functional cure of chronic hepatitis B with only a limited course of treatment.

Empowering Industries: Junji Biotech's one-stop ASO platform

In the process from concept to clinical trial in ASO drugs, synthesis design, chemical modification, and process scale-up have always been core bottlenecks restricting R&D efficiency.

In GentleGen's deeply integrated platform, knock-down, splicing-controlled, and translation-controlled ASO can all achieve precise design—to address different targets and functional requirements, intelligent sequence design relies on a self-developed bioinformatics analysis platform to achieve cross-species conservation analysis, SNP scanning, and activity/off-target prediction.

For flexible synthesis and strict quality control, equipped with a high-throughput synthesis instrument, it can flexibly expand from PMOL level to gram-level production, with HPLC purity reaching over 95%. In terms of chemical modification and conjugation, it offers a rich library of modifications (such as PS, 2'-MOE, LNA, etc.) and conjugation services for GalNAc, peptides, antibodies, etc., providing key technical support for stability and targeting.

In addition, its independently developed GentFect® RNAi HTS transfection reagent is specially designed for large-scale ASO sequence screening, with transfection results superior to mainstream international products.

The application boundaries of oligonucleotide drugs are rapidly expanding. In addition to Bepirovirsen, Ionis's olezarsen for the treatment of hypertriglyceridemia is expected to submit a marketing application in 2026, while Haobo Pharma's unconjugated ASO drug AHB-137 has entered Phase 3 studies. Breakthroughs in extrahepatic delivery technology are expanding the reach of oligonucleotide drugs from the liver to muscle, CNS, lungs, and other fields. GentleGen's one-stop ASO platform has already paved a "highway" to the finish line. For detailed information about related services, please send an email to marketing@gentlegen.com.

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