AmPro Therapeutics Advances Multiple Aptamer-Based Drug Pipelines with Lead Candidate Receiving FDA Orphan Drug Designation

In the history of pharmaceutical development, monoclonal antibody drugs (hereinafter referred to as “mAbs”) have been one of the most important therapeutic modalities over the past few decades. Since the FDA approved the first mAb in 1986, antibody-based therapies have accounted for nearly one-fifth of all new drug approvals by the FDA. Meanwhile, small-molecule drugs with broad-spectrum antitumor properties have also constituted half of the approved drugs.

However, compared to these two,Professor Zhang Ge of Hong Kong Baptist University appears to favor nucleic acid aptamer drugs.“ The inherent sequence diversity of aptamers endows them with unique properties distinct from those of small-molecule drugs and monoclonal antibodies. In clinical applications, compared with monoclonal antibodies, nucleic acid aptamers exhibit superior tissue penetration and lower immunogenicity due to their low molecular weight, and their selection cycle is significantly shorter. Compared with small-molecule drugs, aptamers demonstrate higher selectivity and specificity,” stated Professor Zhang Ge.

Recognizing the numerous advantages of aptamer-based drugs, Professor Zhang Ge and his student, Dr. He Yixin, co-founded Anpei Therapeutics, which is dedicated to the development of innovative aptamer therapeutics.The Anpei therapeutic team told VCBeat that the team is combining aptamers with drugs, and even integrating them with siRNA technology, to expand the potential of aptamer-based therapeutics for a broader range of indications.。

In the field of drug development, time serves as a dual test for innovation. To accelerate the development of aptamer-based therapeutics, Anpei Therapeutics must first address three key challenges: first, how to efficiently screen and identify high-affinity aptamers; second, how to extend the elimination half-life of aptamers; and third, how to enhance the binding affinity between aptamers and their protein molecular targets. To this end,Anpei Therapeutics has built three major technology platforms, covering the entire spectrum from aptamer discovery to long-acting design and high-affinity optimization.

First, leveraging an AI-powered high-throughput screening platform, Anpei Therapeutics employs advanced algorithms to deeply mine massive SELEX datasets, accurately identifying and generating potential high-affinity aptamer sequences. This approach significantly narrows the scope of subsequent experimental validation, effectively reducing R&D costs and time consumption.

In the design of long-acting aptamer drugs, Anpei Therapeutics has innovatively adopted a dual-molecule modification strategy. By screening for small-molecule compounds that bind tightly to serum albumin, the company has successfully extended the half-life of aptamers in vivo. Experimental data from Anpei Therapeutics indicate that,This strategy extends the half-life of the sclerostin aptamer in rats to 12 days, which is four times longer than that of conventional PEGylated agents and monoclonal antibodies, thereby significantly enhancing the drug’s sustained efficacy and patient treatment adherence.。

“For aptamers to be developed into drugs, they must possess high affinity,” Zhang Ge particularly emphasized. To this end, Anpei Therapeutics has developed an advanced affinity modification platform technology. In the early stage, the team screened hydrophobic molecules modified at the C5 position of deoxyuridine (dU) that exhibit high affinity for target proteins, and applied them to the pre-SELEX phase to screen for aptamers with high affinity. To further mimic the high-affinity, high-specificity interactions between antibodies and antigens, the team directly introduced amino acid side chains onto the aptamers during the post-SELEX phase while preserving the intact chemical structure of the aptamer bases. From the pre-SELEX to the post-SELEX phase, the team underwent three generations of iterative improvements in affinity modification technologies,The improvement in the affinity of sclerostin aptamers for the sclerostin protein has become increasingly significant, with their affinity being nearly 10 times that of monoclonal antibodies targeting the same antigen.

Following the successful innovation of three major technology platforms,Anpei Therapeutics has officially launched three self-developed pipelines and two collaborative R&D projects, dedicated to applying innovative nucleic acid aptamer drugs to multiple indications.

As the flagship product of Anpei Therapeutics’ self-developed pipeline, APC001 is a sclerostin aptamer inhibitor that brings new hope to patients with osteoporosis, hypophosphatemic rickets, and osteogenesis imperfecta. Notably,Its novel molecules for the treatment of osteogenesis imperfecta and hypophosphatemic rickets have received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the U.S. FDA. The product is expected to simultaneously submit Investigational New Drug (IND) applications in both China and the United States in the third quarter of 2025.

The second pipeline targets anti-fibrotic diseases, with the team selecting Duchenne Muscular Dystrophy (DMD) as the initial indication. Anpei Therapeutics has already identified specific target-specific aptamer molecules and preliminarily validated their efficacy in animal studies. Furthermore, to comprehensively evaluate the drug’s effects, Zhang Ge is leading the team in conducting efficacy studies on pulmonary fibrosis, aiming to determine the optimal direction for indication development.

In its more exploratory third in-house pipeline, Anpei Therapeutics has developed an innovative drug targeting refractory diseases such as triple-negative breast cancer. This therapeutic employs an aptamer-based PROTAC modality to target the intracellular degradation of sclerostin. The candidate molecule has completed molecular optimization and demonstrated preliminary efficacy in animal models. The next steps will focus on evaluating key parameters, including drug distribution and cellular uptake efficiency, to prepare for subsequent Investigational New Drug (IND) application.

To broaden the boundaries of R&D, Anpei Therapeutics has formed powerful alliances in its collaborative pipeline. InAnPei Therapeutics is collaborating with a startup specializing in siRNA therapeutics to develop an extrahepatic delivery system for a novel-target siRNA drug against multiple myeloma, as part of its exploration into the research and development of aptamer-drug conjugates and aptamer-siRNA therapeutics with extrahepatic delivery systems.It is reported that the team successfully screened aptamers targeting tumor cells and efficiently conjugated them with siRNA, achieving effective binding to and endocytosis via the cell membrane, while demonstrating favorable therapeutic efficacy in mouse models.

As a university-incubated enterprise, Anpei’s therapeutic team features clear and complementary division of labor.

He Yixin introduced to VCBeat, “The university team focuses on in-depth research into molecular mechanisms and target pathways, laying a solid theoretical foundation for drug development.” On the other hand, the company team emphasizes industrialization and translation. Once a target is confirmed, the company team quickly takes over, conducting aptamer screening, optimization of candidate molecules, and various druggability evaluations.

When discussing the selection of the next indication or pipeline, He Yixin emphasized the company’s prudent and precise strategy. He pointed out that, against the backdrop of the current capital winter, Anpei Therapeutics will not blindly expand to pursue a broad pipeline layout; instead, it will make well-considered decisions based on the availability of its own resources.Currently, Anpei’s therapeutic strategy prioritizes targets that are unique in their molecular mechanisms and remain difficult to address effectively with existing drug modalities.

In terms of financial planning, He Yixin revealed thatThe team is actively preparing for fundraising. The proceeds will be primarily allocated to internal processes and activities related to the Investigational New Drug (IND) application, with a portion also dedicated to further enhancing team capabilities, such as strengthening in-house expertise in clinical regulatory affairs.

As a key strategic partner of Anpei Therapeutics, Chen Yiqun, Partner at Shangjun Investment, also stated to VCBeat that Anpei Therapeutics is currently in a critical phase of accelerated development. The company’s primary objective is to fully leverage this financing opportunity to provide strong momentum for its leapfrog growth. In addition to financial support, Shangjun Investment plans to offer comprehensive assistance to Anpei Therapeutics in brand building and promotional strategies, thereby enhancing its market visibility and influence.