ADARx and Jinlan Gene Advance CGT Candidates ADX-324 and GC101 into Phase III Trials in May

In the biopharmaceutical field, cell and gene therapy (CGT) has become a highly focused area in recent years. In May 2025, two more CGT new drugs — ADX-324 and GC101 — have entered Phase III clinical trials.

ADX - 324

R&D Company: ADARx Pharmaceuticals, Inc.

ADARx Pharmaceuticals, Inc. is a biotechnology company focused on RNA-targeted therapies, dedicated to developing innovative treatment solutions to address unmet medical needs. The company has a professional R&D team with deep technical expertise and innovation capabilities in the field of RNA interference technology.

Indications: Hereditary Angioedema

Hereditary Angioedema: A Rare Autosomal Dominant Genetic Disorder

R&D Process

ADX-324 is a drug based on siRNA (small interfering RNA) technology, with its target being the KLKB1 gene. The siRNA technology achieves therapeutic purposes by specifically silencing the expression of the target gene and blocking the synthesis of related proteins. In earlier preclinical studies, ADX-324 demonstrated effective silencing of the KLKB1 gene, significantly reducing the production of related proteases and thereby lowering the risk of vascular edema. Subsequently, the drug successfully entered clinical trials, progressing from initial safety and tolerability tests to Phase III clinical trials, where its efficacy and safety in large patient populations are being validated. Phase III clinical trials will further evaluate the long-term effectiveness of ADX-324, the differences in effects at various doses, and the drug’s safety profile, providing critical data for subsequent regulatory approval and market launch.

GC101

R&D Company: Genecradle

Genecradle is an innovative enterprise in China that focuses on the field of gene therapy. Adhering to the concept of providing breakthrough treatment solutions for patients, the company continuously explores and advances in the research and development of gene therapy technology and clinical transformation. The company possesses an advanced gene therapy technology platform and a professional R&D team, with full-process R&D capabilities ranging from gene discovery, vector construction to clinical trials.

Indications: Type 2 Spinal Muscular Atrophy

Type 2 Spinal Muscular Atrophy (SMA) is a severe neuromuscular disorder that belongs to the category of autosomal recessive genetic diseases. Due to defects in the SMN1 gene, patients produce insufficient levels of survival motor neuron protein (SMN), leading to muscle atrophy and weakness. This significantly affects patients' motor function and quality of life, and can even be life-threatening. Currently, although there are some treatments available for spinal muscular atrophy, satisfactory therapeutic effects have not been achieved for all patients. The development of GC101 is expected to bring new hope to these patients.

R&D Process

GC101 is a gene therapy drug targeting the SMN1 gene. Its development concept involves introducing the normal SMN1 gene into patients to compensate for their own genetic defects, thereby increasing the expression of the SMN protein and improving patient symptoms. In the preclinical research stage, animal experiments confirmed that this therapy effectively raises SMN protein levels, enhancing muscle strength and motor function in animals. After entering clinical trials, from early small-scale safety studies to progressively larger-scale efficacy evaluations, GC101 has demonstrated certain therapeutic potential at every stage. The Phase III clinical trial will comprehensively assess GC101’s long-term efficacy, safety, and advantages compared to existing therapies, which is crucial in determining whether this drug can become an important treatment option for patients with Type 2 Spinal Muscular Atrophy.

Conclusion

ADX-324 and GC101 Enter Phase III Clinical Trials: A Significant Advancement in the Biopharmaceutical Field