mRNA Industry Advances: Clinical Milestones and Regulatory Filings Highlight Sector Momentum

This Phase I clinical trial plans to enroll 210 healthy adult volunteers aged 18-45, with an expected completion in 2026.

① Arcturus: FDA Approves Phase 2 Clinical Trial Application for Inhaled Cystic Fibrosis mRNA Therapy

On September 3, 2024, Arcturus Therapeutics Holdings Inc. ("Company", "Arcturus", Nasdaq: ARCT) announced that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application for the Company’s product candidate ARCT-032 for the treatment of cystic fibrosis (CF), enabling the Company to initiate a Phase 2 multiple ascending dose study to evaluate the safety, tolerability, and efficacy of ARCT-032 in patients with cystic fibrosis.

②RuiZheng Gene: Non-viral Vector In Vivo Gene Editing Drug ART001 Receives U.S. FDA Clinical Trial Approval

ART001, a product independently developed by RuiZheng Gene (Suzhou) Co., Ltd. ("RuiZheng Gene") for transthyretin amyloidosis (ATTR), has recently received approval for its U.S. IND application, becoming the first in vivo gene editing drug based on non-viral vectors to obtain clinical trial authorization from the U.S. FDA in China.

ART001 is the first in vivo gene-editing drug based on a non-viral vector to enter human clinical trials (IIT) in China and is currently undergoing Phase 1 clinical trials in the country. With the recent approval of its FDA IND application in the United States, ART001 has become the only product in its class to receive clinical trial authorization in both China and the U.S.

③ YolTech Therapeutics: YOLT-203 Granted FDA Rare Pediatric Disease Designation (RPDD)

On September 4, 2024, YolTech Therapeutics announced that the FDA has granted Rare Pediatric Disease Designation (RPDD) to its investigational in vivo gene-editing drug YOLT-203 for the treatment of Primary Hyperoxaluria Type 1 (PH1). YOLT-203 utilizes YolTech Therapeutics' proprietary CRISPR/Cas gene-editing tool, YolCas12™, delivered via lipid nanoparticles (LNP). This marks the world's first application of an in vivo gene-editing drug for the treatment of PH1 and represents the first global clinical trial of in vivo gene editing for a pediatric rare disease.

④ AIM Vaccine: mRNA RSV and mRNA Shingles Vaccine Pre-IND Submission to FDA

On August 29, 2024, AmoyVac Biotech Co., Ltd. ("AmoyVac") announced that the group is actively promoting the development of its vaccine pipeline in accordance with its established corporate strategy. Leveraging the advantages of its mRNA technology platform and through continuous technological innovation, the company is rapidly advancing the research and development of its series of mRNA vaccine products. In August 2024, AmoyVac made its first Pre-IND application to the U.S. Food and Drug Administration (FDA) for its mRNA RSV (Respiratory Syncytial Virus) vaccine and mRNA Herpes Zoster vaccine.

⑤CDE: Notice on the Release of the "Technical Guidelines for Non-Clinical Studies of Preventive mRNA Vaccines (Draft for Comments)"

On August 29, 2024, the Center for Drug Evaluation of the National Medical Products Administration released a notice soliciting public comments on the "Technical Guidelines for Non-Clinical Studies of Preventive mRNA Vaccines (Draft for Comments)."

⑥CDE: Release of "Technical Guidelines for Clinical Trials of Vaccines (Revised Draft) (Draft for Comments)"

On September 3, 2024, the Center for Drug Evaluation of the National Medical Products Administration released a notice soliciting public opinions on the "Technical Guidelines for Vaccine Clinical Trials."

⑦CDE: Release of "Technical Guidelines for Clinical Trials of Vaccines for the Prevention of Monkeypox Virus (Draft for Comments)"

On August 28, 2024, the Center for Drug Evaluation of the National Medical Products Administration released a notice soliciting public comments on the "Guiding Principles for Clinical Trials of Vaccines for the Prevention of Monkeypox Virus (Draft for Comments)."

①【Cell】Monkeypox virus mRNA vaccine published in "Cell"! Clinical trial results expected to be announced next year

On September 4, 2024, the prestigious academic journal *Cell* published the latest research findings on Moderna’s mRNA candidate vaccine mRNA-1769 for the prevention of monkeypox. The data shows that mRNA-1769 significantly outperforms currently approved monkeypox vaccines in preventing severe disease after infection in monkeys and reducing viral load within the body. The mRNA-1769 vaccine is currently undergoing Phase 1/2 clinical trials in the UK, with 351 adults participating, and the results are expected to be announced by mid-2025.

②【Blood】New Advances in mRNA-LNP Engineered Platelet Therapy, Pieter Cullis Team Achieves Supraphysiological Plasma Transfection Concentrations

On August 27, 2024, the British Columbia University Blood Research Institute, in collaboration with Pieter Cullis' team, systematically optimized the mRNA-LNP platform again, enabling it to directly transfect and engineer platelets in donor plasma or plasma with Platelet Additive Solution (PAS) for clinical use. The related research, titled "Genetic Engineering of Transfusable Platelets with mRNA-Lipid Nanoparticles is Compatible with Blood Banking Practices," was published in the journal Blood.

③【PNAS】Mao Haiquan's team achieves size-controllable kinetic assembly of polycations/mRNA, confirming that larger particles have stronger affinity for immune cells

On August 27, 2024, the team of Mao Haiquan from Johns Hopkins University published a research paper titled "Supramolecular assembly of polycation/mRNA nanoparticles and in vivo monocyte programming" in the journal PNAS [4]. The team developed a controllable, reproducible, and scalable kinetic supramolecular assembly method for ultra-large poly(β-amino ester) (PBAE) mRNA nanoparticles (400-1000 nm), allowing the exploration of biological effects of mRNA nanoparticles beyond the conventional size (100 nm). The study found that larger nanoparticles have a stronger affinity for immune cells and can be used for targeted mRNA delivery to monocytes and macrophages. Using 400 nm nanoparticles enabled the safest and most effective transfection of monocytes after intravenous administration. This discovery is significant for the study of size design in mRNA delivery carriers.

④【Antiviral Research】Progress Made in Afana Biotech's Monkeypox mRNA Vaccine Development

On July 31, 2024, Professor Yucai Wang and Professor Chao Wang, co-founders of Afana Biotech, together with Sandra Chiu's team from the University of Science and Technology of China, published a research paper titled "An mpox quadrivalent mRNA vaccine protects mice from lethal vaccinia virus challenge" in the journal Antiviral Research.

Researchers have developed a multivalent mRNA candidate vaccine, MPXV-1103, which expresses the full-length B6, A35, A29, and M1 proteins in a single sequence with three flexible linkers (G4S1)3. Compared to a monovalent MPXV mRNA vaccine candidate or a quadrivalent mRNA vaccine mixture of four monovalent MPXV mRNA vaccines, MPXV-1103 elicited robust humoral responses and MPXV-specific T-cell responses, protecting mice from lethal vaccinia virus (VACV) challenge even at low doses, with no live virus detected in the nasal cavity or lungs. The study results show that the MPXV-1103 vaccine excels in high efficacy, safety, and simplified manufacturing processes, making it a highly promising candidate vaccine for MPXV infection.

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