Updated: Oct 20
Medically reviewed by ChinaCureLink Team
Table of Content
Introduction
Patients researching inherited retinal dystrophy caused by RPE65 mutations may encounter RPE65 gene therapy as a treatment option. In China, this therapy is available through the supervised Boao Lecheng pilot-zone pathway as part of the second batch of approved projects. This guide explains what the treatment is, who may be considered, its estimated cost, and what international patients should know before pursuing it.
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What Is RPE65 Gene Therapy?
RPE65 gene therapy is a gene-replacement approach for inherited retinal dystrophy associated with mutations in both copies of the RPE65 gene. The treatment uses an AAV vector to deliver functional copies of the RPE65 gene to retinal pigment epithelial cells.
The purpose of the therapy is to introduce functional genetic material into the affected retinal cells. Eligibility depends on both the patient's genetic diagnosis and the condition of the remaining retinal tissue.
The treatment has a moderate-to-high regulatory risk classification, and its availability through the Boao Lecheng pathway should not be interpreted as a guarantee of individual treatment benefit or outcome.
Is RPE65 Gene Therapy Available in China?
Yes. The therapy is included in the second batch of approved Boao Lecheng projects in the Hainan Boao Lecheng International Medical Tourism Pilot Zone.
This is a specific pilot-zone access pathway rather than nationwide approval for the therapy across China. Treatment is provided within the designated medical-access framework and remains subject to medical assessment and eligibility review.
International patients considering this option should confirm current treatment availability, assessment requirements, and scheduling directly with the hospital before making travel arrangements.
Where Is the Treatment Offered?
The treatment is offered at Shanghai Jiao Tong University Affiliated Ruijin Hospital Hainan Hospital (Hainan Boao Research Hospital) in Hainan.
Technical support is provided by Langxin Qi-Sheng (Suzhou) Biotechnology Co., Ltd. The treatment uses an AAV vector to deliver functional RPE65 gene copies to retinal pigment epithelial cells.
Who May Be Eligible?
Eligibility requires genetic testing confirming double-allele RPE65 mutations. The retina must also retain sufficient surviving cells for the treatment to be considered. An ophthalmologist evaluates the retinal condition, and eligibility is confirmed through a face-to-face medical assessment.
These criteria do not determine whether an individual patient qualifies. Final eligibility is determined by the treating medical team after a face-to-face consultation and review of the patient's medical records.
Patients researching inherited retinal dystrophy may encounter RPE65 gene therapy as a treatment option. Inherited retinal dystrophy gene therapy in China is currently available through the supervised Boao Lecheng pilot-zone pathway as part of the second batch of approved projects. This guide explains what the treatment is, who may be considered, its estimated cost, and what international patients should know before pursuing it.
How Much Does It Cost?
The official approved price is ¥480,000 (approximately US$71,400) per eye. The USD figure uses a reference exchange rate of about ¥6.72 per US$1; actual currency-conversion costs may vary.
The stated price is for the treatment per eye. It does not necessarily include consultation, diagnostic testing, hospitalization, translation or coordination services, accommodation, or international travel. Confirm the final price and full scope of included services directly with the hospital before treatment.
Why International Patients May Consider Inherited retinal Dystrophy Gene Therapy China
For some international patients with genetically confirmed RPE65-associated inherited retinal dystrophy, China's supervised pilot-zone pathway may provide a treatment-access route to discuss with specialists.
The key considerations are genetic eligibility, the amount of surviving retinal tissue, the treatment's regulatory status, cost, and the practical requirements of receiving care in Hainan. Patients should evaluate these factors with qualified ophthalmology and medical teams rather than treating pilot-zone availability as evidence of a guaranteed outcome.
How ChinaCureLink Can Help Patients Access This Treatment
We help international patients explore available treatment options and facilitate communication with appropriate medical specialists. Patients should discuss potential benefits, risks, eligibility, and alternatives directly with qualified physicians. ChinaCureLink does not represent or endorse the treatment technology, manufacturer, or hospital.
Case review: We collect relevant medical history and inherited retinal dystrophy-management records to help determine whether a case is appropriate for physician review.
Hospital coordination: We help connect patients with the treating team at Shanghai Jiao Tong University Affiliated Ruijin Hospital Hainan Hospital (Hainan Boao Research Hospital) in China for an initial assessment.
Online consultation before travel: Patients can discuss potential eligibility, the procedure, and expected next steps with the medical team remotely before committing to international travel.
Coordination support: Depending on the case, this can include translation assistance, appointment scheduling, and logistics coordination.
Considering This Treatment in China?
Before planning travel, speak with a qualified specialist to determine whether the treatment is appropriate for your condition and whether you meet the medical eligibility requirements. ChinaCureLink can help international patients arrange a specialist review and explore the treatment pathway in China. [Request a Medical Review / Speak With a Specialist]
Get a Second Opinion Before Fly to China
For suitable cases, ChinaCureLink can then coordinate an online consultation with the specialist to discuss the technical treatment details directly. The purpose is to help the patient understand which treatment option and which specialist center may be most appropriate before making an international treatment decision.
What Patients Say About ChinaCureLink & Medebound HEALTH
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Frequently Asked Questions
Is RPE65 gene therapy available in China?
Yes. It is available through the Boao Lecheng pilot-zone pathway as part of the second batch of approved projects, at Shanghai Jiao Tong University Affiliated Ruijin Hospital Hainan Hospital.
How much does RPE65 gene therapy cost in China?
The official approved price is approximately US$71,400, per eye. Confirm the final price and included services with the hospital.
Who may qualify for RPE65 gene therapy?
Patients need genetic confirmation of double-allele RPE65 mutations and sufficient surviving retinal cells. An ophthalmologist must assess the patient's retinal condition and eligibility.
Can international patients receive RPE65 gene therapy in China?
International patients may explore access through the Hainan Boao Lecheng pathway, subject to medical assessment, eligibility, and the hospital's current treatment arrangements.
About ChinaCureLink
ChinaCureLink helps patients across the world access the best cancer treatment at China's top hospitals, without the delays, language barriers, and administrative confusion that typically come with seeking care abroad.
We connect patients directly with China's top cancer hospitals, ensuring that from the first case submission through to treatment and follow-up, every step is guided, translated, and coordinated by a team experienced in cross-border patient care.
ChinaCureLink is proudly affiliated with Medebound HEALTH — founded by US physicians, with over 10 years of experience and more than 3,000 patients served worldwide, Medebound HEALTH is recognized by Forbes and it is one of the most trusted patient cross-border concierge service company across North America and the Asia Pacific.
Disclaimer
This article is for informational purposes only and is not medical or financial advice. Treatment eligibility, protocols, and costs vary by individual case — confirm specifics with a coordinator and treating physician before making any decisions.
