25+ IND Projects

1. SNUG01 – Amyotrophic Lateral Sclerosis (ALS)

Partner: SineuGene Therapeutics
Indication: ALS (amyotrophic lateral sclerosis / Lou Gehrig’s disease)

Regulatory Progress:

• March 2025: Granted approval for an Investigational New Drug (IND) clinical trial application by the U.S. FDA
• August 2025: Received China NMPA “implied approval” for an IND clinical trial

Highlights: Global’s first AAV gene therapy targeting TRIM72; ongoing multi-center international clinical trials in both the U.S. and China.

PackGene Services: Plasmid and AAV process development and optimization, GMP manufacturing, analytical method development and validation, quality studies, product release testing, stability studies, and preparation of complete CMC documentation.

2. JWK001 Injection

Partner: Genevector
Indication: Neovascular age-related macular degeneration (nAMD)

Regulatory Progress:

• February 22, 2024: Received China NMPA IND approval

Highlights: The first AAV gene medicine adopting a two-plasmid packaging system.

PackGene Role: Provides one-stop analytical testing services, including analytical method development and validation, release testing, stability testing, and characterization studies.

3. JWK002 – X-linked Retinoschisis

Partner: Genevector
Indication: X-linked retinoschisis (XLRS)

Regulatory Progress:

• 2024: Granted U.S. FDA Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD)
• June 2025: Received China NMPA IND approval

Highlights: China’s first gene therapy medicine registered for a clinical trial targeting X-linked retinoschisis.

PackGene Services: One-stop analytical testing services, including analytical method development and validation, release testing, stability testing, and characterization studies.

4. XMVA09 – Wet Age-related Macular Degeneration (wAMD)

Partner: Starry Eyes Biotech
Indication: Wet age-related macular degeneration (wAMD)

Regulatory Progress:

• March 2024: Received approval from China NMPA CDE (China Center for Drug Evaluation) (first indication)
• August 2024: Approval from CDE for a second indication

Highlights: A gene therapy product combining dual-specificity targets and a capsid for intravitreal injection.

PackGene Services: Full set CMC services for plasmid and AAV, covering process development, GMP manufacturing, analytical method development and validation, quality studies, product release testing, stability studies, and preparation of CMC documentation.

5. HG004 – Retinal Diseases Associated with RPE65 Mutations

Partner: HuiDa Gene
Indication: Retinal diseases associated with RPE65 gene mutations

Regulatory Progress: Received China NMPA IND approval
Highlights: International multi-regional, multi-center clinical trial; the starting effective dose is far lower than that of the already approved LUXTURNA product.
PackGene Role: Partner (specific service scope not detailed).

6. LY-M001 – Gaucher Disease Type I

Partner: Lingyi Biotech
Indication: Gaucher disease types I and III

Regulatory Progress:

• October 2023: China NMPA IND application accepted for review
• January 2024: Received implied approval from China NMPA (默示许可)
• January 2024: Received U.S. FDA IND clearance (IND No.: 30210)

Earlier: Received U.S. FDA Orphan Drug Designation
Highlights: China-developed and China-owned AAV gene therapy for Gaucher disease; first patient dosing completed.
PackGene Services: High-quality CMC services to accelerate clinical trial application.

7. RM-101 – Usher Syndrome

Partner: Ruifeng Biotech
Indication: Usher syndrome (associated with retinal degeneration)

Regulatory Progress:

• Received U.S. FDA IND approval
• October 2024: Received China NMPA IND approval

Highlights: For USH2A gene mutation, using gene regulation technology to induce retinal cells to produce functional proteins.
PackGene Role: Strategic partner (specific service scope not detailed).