Major Breakthrough! Circular RNA Therapy Enters Clinical Development for the First Time, Focusing on Oral Disease Treatment

News Brief

Recently, RiboX Therapeutics Ltd., based in Princeton, New Jersey, has received a significant boost with the approval of the Investigational New Drug (IND) application for its RXRG001 by the US Food and Drug Administration (FDA) for the Phase I/IIa study, namely the SPRINX - 1 study. This milestone achievement in the medical field not only marks a crucial step forward for circular RNA therapies but also holds particular importance for research related to oral health.

RXRG001 is the world's first circular RNA therapy to have its IND application approved. It is primarily targeted at radiation-induced xerostomia (RIX) and hyposalivation, which are common oral problems among patients with head and neck cancer (HNC) after undergoing radiotherapy. With the continuously increasing incidence rate of HNC, it has become the sixth most common malignant tumor globally. Although radiotherapy can improve the survival rate of patients, it causes damage to the salivary glands and reduces saliva secretion, triggering a series of oral health problems such as dry mouth, and difficulty in chewing and swallowing, which seriously affect the patient's quality of life. Moreover, it has been 20 years since the FDA approved the last relevant treatment method. The existing treatment means are ineffective, short-lived in efficacy, and costly. Therefore, the development of new therapies is urgently needed.

As a proprietary product of the innovative circular RNA technology, RXRG001 possesses unique advantages. It is composed of circular RNA encoding human aquaporin 1 (hAQP1) encapsulated within lipid nanoparticles (LNP). Overexpression of hAQP1 to restore water permeability can increase saliva secretion and relieve the symptoms of dry mouth. Non-clinical studies have demonstrated that RXRG001 shows a favorable risk-benefit profile in animal models, with a single administration resulting in a significant increase in saliva flow that lasts for approximately four weeks.

About RIX

RIX is a common complication among patients with HNC during or after radiotherapy. When radiotherapy is administered to the head and neck regions such as the oral cavity, pharynx, larynx, and nasal cavity, the radiation will inevitably damage the salivary gland tissues, mainly the parotid glands, submandibular glands, and sublingual glands, thereby severely suppressing the saliva secretion function and subsequently triggering the symptom of dry mouth.

RIX seriously affects the patient's daily life quality. It not only causes numerous physical discomforts, such as insufficient nutritional intake due to difficulty in eating and sleep disturbance caused by oral pain but also imposes a significant psychological burden on the patients. For example, speech impairment may lead to restricted social interactions for the patients, giving rise to negative emotions like inferiority and anxiety.

Currently, the most common treatment methods for RIX are the use of artificial saliva substitutes and drugs that stimulate saliva secretion. Artificial saliva can mimic the composition and function of natural saliva, making patients feel that their mouths are relatively moist. However, applying or rinsing with artificial saliva can only temporarily relieve the discomfort of dry mouth to a certain extent. Although drugs that stimulate saliva secretion can increase the amount of saliva secreted, they may have certain side effects, such as sweating and gastrointestinal discomfort. Therefore, the existing treatment methods have obvious limitations and it is difficult to completely solve the problem fundamentally. RXRG001 may bring new hope for the treatment of RIX.

About Circular RNA

Circular RNA (circRNA) is a special type of non-coding RNA molecule with a closed circular structure. Compared with traditional linear RNA, it possesses unique stability. It is not easily degraded by exonucleases and can exist in cells for a long period, which provides a solid foundation for its use as a therapeutic vector or for exerting therapeutic effects.

As an emerging therapeutic strategy, circular RNA therapy faces two major challenges. The first is the issue of large-scale production. How to efficiently and cost-effectively produce sufficient quantities of high-quality circular RNA for clinical treatment is an urgent problem that needs to be solved. The second is the delivery challenge. How to ensure that circular RNA is not degraded in the complex oral environment (such as saliva, oral mucosa, etc.) and can smoothly enter the target cells remains a technical problem that urgently needs to be overcome.

The clinical approval of RXRG001 is a crucial milestone in the development of circular RNA therapy, indicating the tremendous potential of circular RNA therapy in future medical applications.

About Alta Stomatology

Alta Stomatology is a company that specializes in the research of oral diseases. We provide comprehensive services in the field of oral research, including disease detection, diagnosis, and drug development. In addition, we also offer a series of high-quality products required for oral disease research, such as cell culture media, detection kits, and antibodies.

Alta Stomatology will continue to keep an eye on the latest developments in the field of oral health and bring you the latest and most accurate relevant information. The breakthrough of RXRG001 has brought new hope for the treatment of oral diseases. We look forward to achieving good results in clinical trials and improving oral health conditions and the quality of life for patients who are suffering from RIX.

Reference Link

https://www.prnewswire.com/news-releases/ribox-therapeutics-announces-fda-clearance-for-ind-application-of-rxrg001-the-first-circular-rna-therapy-for-the-treatment-of-radiation-induced-xerostomia-and-hyposalivation-302287539.html