Drugs / SNUG01
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 01 trial | ||||||
| Phase 0 | NCT06645197 | Oct 2024 → Apr 2025 overdue | amyotrophic lateral sclerosis | Peking University Third Hospital | Not yet recruiting | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT07169175 | Dec 2025 → Mar 2028 expected | amyotrophic lateral sclerosis | SineuGene Therapeutics Co., Ltd. | Not yet recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 7
| Date | Issuer | Release |
|---|---|---|
| 2025-08-11 | SineuGene Therapeutics Co., Ltd. | Regulatory Novel Gene Therapy for ALS cleared for global trials in China and the U.S. sineugene.com ↗
SineuGene Therapeutics Co., Ltd. (“SineuGene”), clinical-stage biotechnology company that mainly focuses on gene therapies for neurological disorders, announced today that China's National Medical Products Administration (NMPA) has cleared the Investigational New Drug (IND) application for SNUG01, a groundbreaking gene therapy product for amyotrophic lateral sclerosis (ALS). |
| 2025-06-26 | SineuGene Therapeutics Co., Ltd. | Regulatory SineuGene’s SNUG01 Receives FDA Orphan Drug Designation for ALS sineugene.com ↗
SineuGene Therapeutics, a clinical-stage biotechnology company focused on gene therapies for neurological diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its gene therapy, SNUG01, for the treatment of amyotrophic lateral sclerosis (ALS). |
| 2025-03-24 | SineuGene Therapeutics Co., Ltd. | Regulatory SineuGene Therapeutics Announces FDA IND Clearance for SNUG01, a First-in-Class TRIM72-Targeted Gene Therapy for ALS sineugene.com ↗
The clearance authorizes a global Phase I/IIa clinical trial designed to evaluate SNUG01’s safety, tolerability, and preliminary efficacy in adults with ALS through a dose-escalation and expansion study. |
| 2024-09-19 | SineuGene Therapeutics Co., Ltd. | Results SineuGene Reports Positive Clinical Data for ALS Gene Therapy SNUG01 sineugene.com ↗
SineuGene, a biotechnology company focused on gene therapies for neurological disorders, today announced positive clinical data from its investigator-initiated trial (IIT) of SNUG01, a potential gene therapy for Amyotrophic Lateral Sclerosis (ALS). |
| 2024-05-24 | SineuGene Therapeutics Co., Ltd. | Results China's First ALS Patient Treated with AAV Gene Therapy Completes One-Year Follow-Up with Stabilized Disease Progression sineugene.com ↗
近日，神济昌华自主研发的渐冻症（肌萎缩侧索硬化症，ALS）AAV基因治疗药物SNUG01首例同情用药受试者完成满1年随访，显示出了良好的安全性及疗效。 |
| 2024-01-26 | SineuGene Therapeutics Co., Ltd. | Results SNUG01 Latest Developments Covered by South China Morning Post and Other Hong Kong and Overseas Media sineugene.com ↗
Now a potential new drug being tested by a group of Chinese scientists and clinicians may offer a glimmer of hope, with promising results after it was used on a severely affected patient for six months. |
| 2023-09-11 | SineuGene Therapeutics Co., Ltd. | Results SineuGene Successfully Completes China's First AAV Gene Therapy for ALS Patient and Conducts 3-Month Follow-Up sineugene.com ↗
在疗效评估方面，患者疾病进展较前稳定，整体无显著再进展，肌电图结果、ALSFRS-R评分等主客观指标均有改善趋势，SG001基因表达在给药后有所上升。 |
All press releases naming this drug 15 releases
Evidence & citations 3 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | SNUG01 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06645197 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “SNUG01 is a gene therapy designed to deliver a protective protein gene” NCT06645197 ↗ |
| Route | Intrathecal | “delivered via intrathecal (IT) infusion” NCT06645197 ↗ |