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Early pipeline

RJK002

Also known as RJK-002, AAV-RJK002

Bottom line

Very early: small safety studies in China, with no published results. A company partner claimed lower nerve-damage markers in a few patients, but without a comparison group this means little, and most drugs at this stage do not reach approval.

Very early. This drug is in its first safety trial in people and has not been tested for whether it helps in ALS. Most drugs at this stage never reach approval. More about early drugs

RJK002 is a one-time gene therapy from a Shanghai company, given by spinal injection. It is meant to break up and prevent the protein clumps that build up in nerve cells in ALS. It has been given to small groups of people with ALS in China since 2023, and a 9-person registered Phase 1 trial in Beijing finished enrolling in 2025. No full results have been published.

Information checked 5 Nov 2026

Evidence: Results pending

Studies: 2 running

Trials are running, but there are no efficacy results yet.

How we decide the overall verdict: How to read this site.

At a glance

CompanyRJK Biopharma (Shanghai Rejukon Biopharm)Explanation and sources

Rejukon (瑞吉康) is a small private company based in Shanghai since 2022 that works on diseases caused by protein clumping, such as ALS and Parkinson's disease. RJK002 is its lead drug. Most public information comes from Chinese media and partner announcements rather than English-language company press releases.

Sources

  1. 瑞吉康：挑战神经退行性疾病世界难题 (Rejukon profile)Fengxian District Government, Shanghai · 18 Jul 2024 · News
  2. 瑞吉康渐冻症AAV基因治疗药物RJK002获临床试验默示许可 (RJK002 cleared for clinical trials in China)China National Radio (央广网) · 10 May 2024 · News
How it worksOne-time gene therapy meant to dissolve toxic protein clumpsAAV gene therapy targeting abnormal protein aggregation (target not disclosed)Explanation and sources

RJK002 uses an emptied-out virus (AAV) to deliver genetic instructions into nerve cells. The company says the resulting product can dissolve protein clumps that have already formed and stop new ones forming, restoring normal cell function. It has not published exactly which protein it acts on. The company reports strong survival gains in ALS animal models, but most treatments that work in animals do not work in people.

Sources

  1. 瑞吉康渐冻症AAV基因治疗药物RJK002获临床试验默示许可 (RJK002 cleared for clinical trials in China)China National Radio (央广网) · 10 May 2024 · News
  2. 瑞吉康：挑战神经退行性疾病世界难题 (Rejukon profile)Fengxian District Government, Shanghai · 18 Jul 2024 · News
StagePhase 1Phase 1 in people with ALS in China, 9 people · Dosing: people with alsExplanation and sources

China's drug regulator allowed a registered trial in May 2024, and the 9-person Phase 1 trial started at Peking University Third Hospital in September 2024; China's trial registry records the first participant enrolled on October 14, 2024. It stopped enrolling in late 2025. Before that, smaller doctor-led studies at Chinese hospitals had dosed their first patient in September 2023. The FDA granted orphan drug status in October 2023, which is not a decision on whether it works.

Sources

  1. Evaluate the Safety and Efficacy of Intrathecal Injection of RJK002 in Patients With ALS (NCT06493279)ClinicalTrials.gov · Trial registry
  2. CTR20243220 RJK002注射液 (China drug trial registry record, via YSCRO)YSCRO (mirror of China's CDE trial registry) · Trial registry
  3. 瑞吉康渐冻症AAV基因治疗药物RJK002获临床试验默示许可 (RJK002 cleared for clinical trials in China)China National Radio (央广网) · 10 May 2024 · News
  4. 瑞吉康：挑战神经退行性疾病世界难题 (Rejukon profile)Fengxian District Government, Shanghai · 18 Jul 2024 · News
  5. An IIT Clinical Study of a Single Intrathecal Injection of RJK002 in Patients With ALS (NCT06454682)ClinicalTrials.gov · Trial registry
Best reported effectSafety study; no published effect dataSafety; ALSFRS-R and NfL exploredExplanation and sources

No results have been published in a journal or at a major meeting. In September 2024 the company's manufacturing partner said early doctor-led studies showed the drug was safe and lowered the nerve-damage marker NfL by close to 60% after 24 weeks in some low- and middle-dose patients. This is a second-hand claim about a handful of patients with no comparison group, so it does not show the drug slows ALS.

This is the most favourable result reported. See all key studies for the full picture.

Sources

  1. 宜明生物祝贺瑞吉康渐冻症药物RJK002启动Ⅰ期临床试验 (Phase 1 launch, from manufacturing partner)Yiming Biologics · 26 Sept 2024 · Company press release
Who it's forPeople with ALS within 2 years of first symptomsAll ALS · Single spinal injection (one time)Explanation and sources

The Phase 1 trial takes adults with sporadic or familial ALS whose symptoms began no more than 2 years ago, with an ALSFRS-R score of at least 30 and breathing capacity of at least 70%. The drug is given once by spinal injection.

Sources

  1. Evaluate the Safety and Efficacy of Intrathecal Injection of RJK002 in Patients With ALS (NCT06493279)ClinicalTrials.gov · Trial registry
What's nextSafety results from the 9-person Phase 1 trialExpected November 2026Explanation and sources

The company has not announced a date for results. The trial registry estimates the main data will be collected by November 30, 2026, with follow-up running to 2030.

Sources

  1. Evaluate the Safety and Efficacy of Intrathecal Injection of RJK002 in Patients With ALS (NCT06493279)ClinicalTrials.gov · Trial registry
Can I join?Not recruiting; enrolment closed in ChinaExplanation and sources

Both registered studies are listed as active but no longer recruiting. The company said in 2024 it was preparing an application to run trials in the US, but we found no sign that one has been filed. We found no expanded access programme.

Sources

  1. Evaluate the Safety and Efficacy of Intrathecal Injection of RJK002 in Patients With ALS (NCT06493279)ClinicalTrials.gov · Trial registry
  2. An IIT Clinical Study of a Single Intrathecal Injection of RJK002 in Patients With ALS (NCT06454682)ClinicalTrials.gov · Trial registry
  3. 瑞吉康：挑战神经退行性疾病世界难题 (Rejukon profile)Fengxian District Government, Shanghai · 18 Jul 2024 · News

Signal: Silent

Signal measures how often, and how reliably, RJK Biopharma (Shanghai Rejukon Biopharm) tells the public about this drug. It says nothing about whether the drug works. A quiet company isn't necessarily a bad sign, but regular, on-time updates help patients plan.

Last company updateNone found0 / 40
Company updates in the last 12 months00 / 35
Promises keptNot enough history yet (neutral score)12.5 / 25
Signal score13 / 100 · Silent

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

Timeline

6 events, newest first. Company updates are marked; independent news and regulator decisions are shown too.

2025

  1. Trial registryClinicalTrials.gov

    Phase 1 enrolment closed

    The registry record was updated to active, not recruiting, with main results now expected by November 2026.

2024

  1. NewsYiming Biologics

    Phase 1 trial launched in Beijing

    Rejukon's manufacturing partner announced the Phase 1 kick-off at Peking University Third Hospital and claimed early NfL drops in a few patients from the doctor-led studies.

  2. NewsFengxian District Government

    Local government profiles Rejukon

    A Shanghai district government article said the first doctor-led study dosed its first patient in September 2023 and that the FDA granted orphan drug status in October 2023.

  3. Trial registryClinicalTrials.gov

    Phase 1 trial registered

    The 9-person Phase 1 trial at Peking University Third Hospital was posted on ClinicalTrials.gov.

  4. Trial registryClinicalTrials.gov

    Doctor-led study registered

    A 9-person doctor-led study of RJK002 at a Fuzhou hospital, which began in 2023, was posted on ClinicalTrials.gov.

  5. NewsChina National Radio

    China clears RJK002 for registered trials

    Chinese state media reported that China's drug regulator had allowed clinical trials of RJK002 in ALS, and that doctor-led studies were already under way.

All sources for this page

  1. 瑞吉康渐冻症AAV基因治疗药物RJK002获临床试验默示许可 (RJK002 cleared for clinical trials in China)China National Radio (央广网) · 10 May 2024 · News
  2. 瑞吉康：挑战神经退行性疾病世界难题 (Rejukon profile)Fengxian District Government, Shanghai · 18 Jul 2024 · News
  3. 宜明生物祝贺瑞吉康渐冻症药物RJK002启动Ⅰ期临床试验 (Phase 1 launch, from manufacturing partner)Yiming Biologics · 26 Sept 2024 · Company press release
  4. CTR20243220 RJK002注射液 (China drug trial registry record, via YSCRO)YSCRO (mirror of China's CDE trial registry) · Trial registry
  5. Evaluate the Safety and Efficacy of Intrathecal Injection of RJK002 in Patients With ALS (NCT06493279)ClinicalTrials.gov · Trial registry
  6. An IIT Clinical Study of a Single Intrathecal Injection of RJK002 in Patients With ALS (NCT06454682)ClinicalTrials.gov · Trial registry