AOC Wave Arrives: Samsung Biologics, WuXi Biologics, and Lonza Enter the Arena as First Drug Nears BLA

It is worth mentioning that,This is the first time Samsung Bioepis has officially listed AOC as a business area., which is not only a strategic shift for Samsung Bioepis, but also reflectsThe global biopharmaceutical industry is accelerating its leap into the era of "precise gene regulation."。

AOC and ADC are both based on antibody technology. Unlike ADC, which conjugates toxic drugs with antibodies to selectively kill cancer cells, AOC conjugates oligonucleotides.(siRNA or ASO, etc.)Conjugated with antibodies to regulate genes in specific cells. If ADC is likened to a "guided missile" that precisely targets cancer cells, then AOC can be seen as a "drone" that delivers gene therapy accurately to the target location.

At present, small nucleic acid drugs have become a hot spot in new drug research and development due to their gene silencing mechanism, but they are still limited by the inherent defects of delivery systems, making it difficult to overcome the extracellular targeting barriers. However, AOC technology, through...Antibody-Mediated Active Targeting Mechanism, equipping small nucleic acids with a "smart navigation system." It uses antibodies to precisely target specific tissues, delivering siRNA to lesions that traditional carriers cannot reach, while significantly improving the efficiency of small nucleic acid drugs entering cells.

At the same time, traditional RNA therapies have the limitation of potentially regulating genes in both diseased and normal cells, which may cause side effects. In contrast, AOCs possess precise targeting capabilities, binding only to cells that require gene regulation, effectively avoiding this issue and offering advantages that RNA therapies cannot match.

As the ADC field has already attracted dozens of pharmaceutical companies, with over 15 globally approved products and record-breaking license-out deals for China's innovative drugs, the competition surrounding "precise delivery of cytotoxic drugs" has long since reached a fever pitch. Meanwhile, AOC is emerging in the guise of a "gene-regulating drone."In another blue ocean, a quiet rise is underway.。

Compared to the mature paradigm of "toxic drug + linker + antibody" in the ADC field, the technical framework of AOC is still in the exploratory stage of "oligonucleotide + linker + antibody" — there are no commercialized products globally yet, and clinical pipelines are mainly concentrated in the rare disease field, which precisely constitutes its unique value洼地.

According to a report released by 360i Research, a global market research firm, the AOC market size is expected to grow at an average annual growth rate of 8.89%.Will reach 5.26 billion US dollars by 2030. Although there are no commercialized products yet, both CDMOs and pharmaceutical companies have already entered the field, gradually attracting a dual concentration of capital and technology.

In the CDMO field, in addition to Samsung Bioepis leveraging ADC technology to quickly gain an advantage, Lonza and WuXi Biologics have also established relevant businesses. The former has set up a dedicated AOC production line, while the latter is committed to providing "one-stop services" from drug design to production.

On the pharmaceutical company side, several innovative companies have entered the clinical sprint stage. The two companies that are about to take the lead are mainly Avidity Biosciences and Dyne Therapeutics.

Avidity is one of the leading companies in the AOC track, with its key pipeline including Del-zota.（DMD）、Del-desiran（DM1）、Del-brax（FSHD）。

Among them, Del-brax is the first to directly target the pathogenic gene double homeobox 4.（DUX4）To treat facioscapulohumeral muscular dystrophy（FSHD）A proprietary monoclonal antibody that binds to transferrin receptor 1（TfR1）Combined, and conjugated with siRNA targeting DUX4 mRNA, aiming to reduce the expression of DUX4 mRNA and DUX4 protein in the muscles of FSHD patients.

In June this year, Avidity announced the opening of the accelerated approval regulatory pathway in the United States for Del-brax, for the treatment of FSHD, andThe global confirmatory Phase 3 FORWARD™ study for this candidate pipeline has been initiated., aiming to support subsequent full approvals in the United States and other countries/regions worldwide. Results from previous Phase 1/2 clinical trials showed that FSHD patients receiving this therapy had DUX4-regulated genesConsistently down by more than 50%, and show a trend of muscle function improvement.

Del-zota is a treatment for Duchenne muscular dystrophy targeting exon 44 skipping therapy.（DMD44）AOC drugs. The topline data from the Phase 1/2 EXPLORE44 clinical trial, released in March this year, showed exon 44 skipping efficiency in patients from both dose groups who received treatment.Significantly Increase by 40%. The production of anti-dystrophy protein significantly increased, with an average increase to 25% of normal levels, and a maximum reaching 58% of normal levels. Notably,Avidity Plans to Submit BLA Application to FDA by End of This Year。

Another candidate, Del-desiran, is composed of a full-length monoclonal antibody targeting TfR1, a linker, and DMPK mRNA. Its indication is DM1. Currently, this candidate pipelinePhase 3 clinical study is ongoing。

Dyne is also a leading company in the AOC track, with a wide range of candidate pipelines established for indications including DM1, DMD, FSHD, Pompe disease, etc., showing a similar indication layout to Avidity.

Its core pipeline, DYNE-101, consists of a TfR1 antibody conjugated with an ASO, designed to reduce pathogenic DMPK mRNA levels and correct the genetic defect in DM1. The latest data from the Phase 1/2 clinical trial ACHIEVE was announced in January this year.（6.8 mg/kg）Show that at the 6th month, the myotonia symptoms in the DYNE-101 group were improved.vHOT time decreased by 2.9 seconds, while the placebo group increased by 0.4 seconds.。

In June, following a Type C meeting with the FDA, the primary clinical endpoint that formed the basis for the accelerated approval was changed to CASI-22.(Evaluation of Splicing Correction Effect), replaced with vHOT(Evaluation of improvement in myotonia symptoms), leading to the announcement of a delay in the launch plan for AOC drug DYNE-101.BLA to be submitted to FDA by the end of next year。

DYNE-251 consists of a phosphorodiamidate morpholino oligomer（PMO）Conjugated with a fragment antibody binding to TfR1, aimed at targeted muscle tissue delivery and promoting exon skipping within the cell nucleus for the treatment of DMD mutations suitable for exon 51 skipping. Currently, this therapy is in the Phase 1/2 clinical research stage.

In March 2025, Dyne Therapeutics announced breakthrough progress in the 1/2 phase DELIVER trial of its innovative therapy DYNE-251 for DMD. Data showed that DMD boys who received treatment experienced significant improvement in motor function after one year of treatment, with dystrophin levels increasing to 3.71% of normal levels, and up to 8.72% after adjustment for muscle content, far exceeding the existing therapy Exondys 51.

In addition, Tallac Therapeutics’ TAC-001 and ALTA-002 are currently in the clinical stage. The key candidate pipelines of Avidity and Dyne are on the verge of market entry — the AOC track is no longer just a concept on the technological blueprint. These Phase 3 clinical candidates are becoming the first knock on the door of commercialization.

The value of AOC lies not only in breaking through the bottleneck of extrhepatic delivery of small nucleic acid drugs but also in reconstructing the therapeutic logic of "antibody targeting + gene regulation." The next 2-3 years may become the breakthrough year for AOC, which is worth looking forward to.

Editor-in-Chief | Xun

Proofread by Xun

References:

1.https://biz.chosun.com/en/en-science/2025/06/19/AT7ZSWFPXBG4HB7HKLUWE3WESM/

2.Official Website of the Company

Exciting Live Broadcast Preview

Press and hold to scan the QR code and participate immediately ↓

Recommendation 1

June 26, 14:00-15:00

Transformation and Application of High-efficiency, Low-toxicity Cell Preservation Solutions

Statement:This article aims to convey industry development information and explore the frontier progress of biopharmaceuticals. The content of the article represents the author's viewpoint, and does not represent the position of Eureka Biomedical, nor does it constitute any value judgment, investment advice, or medical guidance. If necessary, please consult a professional for investment or visit a regular hospital for medical advice.

Copyright Statement:Welcome to share the article on your personal Moments. Unauthorized reprinting by media or organizations in any form to other platforms is prohibited. For reprinting, please leave a message below the article to obtain authorization.

Cover image source: internet. If there is any copyright infringement, please contact us for removal.

DianDian "Share”、“Like" and "In View", charge me up a bit~"