Advances in “transient conjugation” (TransCon) technology for treating rare endocrine diseases

doi:10.5246/jcps.2026.01.001

Abstract

Abstract:

Genetic hypoparathyroidism (HP), achondroplasia (ACH), and primary growth hormone deficiency (GHD) are listed as rare diseases in the second List of Rare Diseases in China in 2023. Numerous studies have explored optimal therapies for certain rare endocrine diseases, and the development of long-acting therapeutic agents has been considered a key strategy for improving treatment outcomes, especially given the challenges associated with daily subcutaneous injections. However, limited attention has been given to the potential of "transient conjugation" (TransCon) technology, a platform designed to convert drugs into prodrug forms, thereby extending their half-lives and reducing dosing frequency, which demonstrates promise as a more convenient treatment option for these conditions. This is the first study to review the research progress of TransCon technology in the treatment of HP, ACH, and GHD, focusing on its pharmacokinetic properties, efficacy, safety, tolerability, and patient-reported outcomes in comparison with conventional therapies, in order to provide a reference for formulation development and clinical management of these rare endocrine diseases.

Key words: TransCon, Prodrug, Long-acting, Rare endocrine diseases

Supporting:

Qingyang Liu, Tianyu Xu, Jiayi Du, Yan Tang. Advances in “transient conjugation” (TransCon) technology for treating rare endocrine diseases[J]. Journal of Chinese Pharmaceutical Sciences, 2026, 35(1): 1-15.

Figures/Tables 4

References 46

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