Moderna’s personalized neoantigen mRNA vaccine recently achieved positive results in a Phase III trial as adjuvant treatment of melanoma, marking a major milestone in the clinical validation of this therapeutic approach. A year earlier, on April 24, 2025, RGL-270 Injection — China’s first personalized neoantigen mRNA vaccine designed to prevent postoperative recurrence of solid tumors — received implied approval of its Investigational New Drug (IND) application (Acceptance No. CXSL2500080). RGL-270 was independently developed by Shanghai Regenelead Therapies Co., Ltd. (Regenelead) and its wholly owned subsidiary, Guangzhou Revolead Therapies Co., Ltd. (Revolead).
This clearance not only placed Regenelead among China’s leading innovators in cancer immunotherapy, but also offered patients at high risk of postoperative recurrence an innovative solution, shifting the approach from reactive treatment to proactive prevention.
I. Urgent clinical need: postoperative recurrence remains the greatest challenge in solid tumor treatment
In recent years, neoadjuvant, adjuvant, and perioperative treatments — including chemotherapy, chemoradiotherapy, targeted therapy, endocrine therapy, and immunotherapy — have reduced the risk of recurrence or metastasis and improved recurrence-free and overall survival in patients with resectable solid tumors. However, the risk of recurrence or metastasis remains closely linked to multiple factors, including tumor type, stage, molecular characteristics, the extent of surgical resection, and the perioperative treatment received. Even after curative resection of a malignant solid tumor, patients may still harbor molecular residual disease (MRD) that is undetectable by imaging, which can lead to local recurrence or distant metastasis. Approximately 30%-55% of patients with non-small cell lung cancer (NSCLC) experience recurrence or metastasis after curative resection, resulting in treatment failure. Among patients with resectable esophageal cancer, postoperative recurrence rates range from 30.3% to 58.0%. Despite surgical resection, most patients with early-stage pancreatic cancer experience recurrence within one year, and the 5-year survival rate is only 12%-27%. Therefore, more advanced diagnostic and therapeutic strategies are urgently needed to meet this unmet clinical need.
RGL-270 was developed to address precisely this unmet need. As a personalized neoantigen mRNA vaccine, it aims to complement standard treatment with an approach tailored to the unique molecular features of each patient’s tumor.
II. Technological breakthrough: a dual-core “AI × Delivery” platform driving R&D innovation
Building on its expertise in artificial intelligence and drug delivery, Regenelead has established an integrated mRNA technology platform spanning target discovery, molecular design, safe and efficient delivery, and commercial-scale manufacturing. Its RGL-NeoAI bioinformatics platform uses AI algorithms to predict neoantigens with high precision, significantly improving the accuracy of target identification and providing a reliable basis for the development of cancer immunotherapies. Its mRNA-lipid nanoparticle (LNP) platform enables precise mRNA encapsulation and efficient delivery, substantially improving drug safety and efficacy.
Powered by these two core platforms, Regenelead has advanced multiple mRNA drug candidates across different therapeutic areas into clinical development, highlighting their clinical and commercial potential. Looking ahead, the company will remain committed to strengthening China’s domestic mRNA capabilities and accelerating the development of homegrown developed mRNA medicines.
1. RGL-NeoAI: pushing the boundaries of neoantigen prediction
As the technological foundation of the personalized cancer vaccine RGL-270, the company’s proprietary intelligent engine, RGL-NeoAI, uses a dual-model ensemble learning framework that integrates deep neural networks with XGBoost, offering an innovative solution to the challenges of accuracy and specificity in neoantigen prediction.
Drawing on large-scale global multi-omics data, and recognizing that HLA allele frequencies vary markedly across ethnic populations, Regenelead built an optimized, diverse dataset with broad coverage of HLA types. The AI engine was then specifically optimized and trained using an industry-leading pan-allele algorithm, enabling accurate neoantigen prediction across a wide range of HLA types. By analyzing each patient’s multi-omics data, RGL-NeoAI identifies tumor-specific mutations that may give rise to neoantigens. With this optimized dataset and advanced algorithm, the platform achieves a positive predictive value (PPV) of more than 60% at a recall of 40%, outperforming leading publicly available algorithms and ranking among the most competitive platforms internationally. Available clinical data show strong concordance between the platform’s predictions and experimental findings. Among patients who received the neoantigen vaccine, 100% developed antigen-specific immune responses. To ensure safety, each candidate peptide is screened against the world’s largest proteomics database to mitigate potential off-target risks at the source and protect patient safety.
2. The mRNA-LNP platform: sequence optimization and delivery innovation for efficient, low-immunogenicity and stable delivery
Regenelead has developed proprietary mRNA vector structures and an mRNA delivery system. For mRNA sequence design, the company combined bioinformatics with high-throughput screening to identify proprietary untranslated region (UTR) and poly(A) elements that support high expression efficiency. It also optimized codon usage, the 5’ cap structure, and nucleotide modifications to improve mRNA stability and expression, reduce unwanted immunogenicity, and enhance drug safety. For the delivery system, Regenelead leveraged its LNP platform to develop proprietary ionizable cationic lipids, LNP formulations, and drug products that enable efficient, safe mRNA delivery with good stability.
III. Synergy with immunotherapy: clinical data demonstrate potential value
Personalized cancer vaccines can activate and expand tumor-specific T cells, providing a strong rationale for exploring combinations with other immunotherapies. The efficacy of immunotherapy is associated with tumor mutational burden. By increasing neoantigen presentation, a vaccine can stimulate immune activity and improve T-cell infiltration, helping so-called “cold tumors” overcome the immunosuppressive tumor microenvironment and become responsive to immune attack. Therefore, personalized vaccines and other immunotherapies are expected to act synergistically and improve clinical outcomes.
In an ongoing investigator-initiated trial (NCT06156267), RGL-270 has demonstrated a favorable safety profile and preliminary efficacy. Among all eligible patients with resectable pancreatic cancer who received the investigational vaccine after surgery, no RGL-270-related grade ≥3 adverse events or serious adverse events (SAEs) were reported. Clinical data presented at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting showed that all patients remained recurrence-free through 18 months of follow-up and all developed immune responses. These data support the potential clinical value of RGL-270.
IV. Looking ahead: forging a new era of cancer treatment with innovation
Dr. Cheng Liao | Founder and Chief Executive Officer of Regenelead
“By integrating genomics, artificial intelligence, and mRNA technology, RGL-270 offers a tailored treatment approach that represents not only a technological breakthrough but also a concrete commitment to patient-centered care.
We focus not only on technological sophistication but also on the feasibility of clinical translation. RGL-270 is more than a product — it is also a platform. Its clinical advancement is not the finish line but the beginning of a new journey. Going forward, our team will work with leading oncologists in China and around the world to explore combinations of neoantigen vaccines with CAR-T, bispecific antibodies, and other therapies, with the goal of extending these benefits to a broader range of tumor types and disease stages.”
About Regenelead
Founded in August 2021, Regenelead is an innovative biopharmaceutical company focused on cell and gene therapy (CGT). The company has developed a proprietary, AI-powered R&D platform built on three core technologies: AAV, mRNA, and cell therapy. This platform is supported by end-to-end process development, manufacturing, and quality control capabilities for plasmids, viral vectors, mRNA, and cell therapies, delivering high yield, recovery, quality at low cost to accelerate drug development. Regenelead’s pipeline targets therapeutic areas with significant unmet medical needs and strong market potential, including neurology, ophthalmology, metabolic diseases, and oncology. Guided by its mission to safeguard human health, Regenelead continues to strengthen its core capabilities and pursue pioneering innovation, with the goal of enabling better, healthier lives for patients worldwide.