25+ IND Projects

1. SNUG01 – Amyotrophic Lateral Sclerosis (ALS)

Partner: SineuGene Therapeutics
Indication: ALS

Regulatory Progress:

• March 2025: Received clinical trial clearance from the U.S. FDA
• August 2025: Received NMPA implied approval for clinical trial in China

Highlights: The world’s first AAV gene therapy targeting TRIM72, advancing an international multicenter clinical trial in China and the United States.

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

2. JWK001 Injection

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

Regulatory Progress:

• February 22, 2024: Received IND approval from China’s NMPA

Highlights: The first AAV gene therapy drug using a two-plasmid packaging system.

PackGene Role: Provided one-stop analytical testing services, including 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: Received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the U.S. FDA
• June 2025: Received IND approval from China’s NMPA

Highlights: China’s first registrational clinical trial of a gene therapy drug for X-linked retinoschisis.

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

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

Partner: StarryGene
Indication: Wet age-related macular degeneration (wAMD)

Regulatory Progress:

• March 2024: Approved by China’s NMPA CDE for the first indication
• August 2024: Approved by the CDE for a second indication

Highlights: The first gene therapy drug featuring both bispecific targets and an intravitreal injection capsid.

PackGene Services: A full suite of CMC services from plasmid to AAV, covering process development, GMP manufacturing, analytical method development and validation, quality studies, product release testing, stability studies, and CMC dossier preparation.

5. HG004 – RPE65 Mutation-Associated Retinal Disease

Partner: HuidaGene Therapeutics
Indication: RPE65 mutation-associated retinal disease

Regulatory Progress:

• Received IND approval from China’s NMPA

Highlights: An international multiregional and multicenter clinical trial program, with an initial effective dose significantly lower than that of the approved LUXTURNA product.

PackGene Role: Partner of the program; specific service details have not been disclosed.

6. LY-M001 – Type I Gaucher Disease

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

Regulatory Progress:

• October 2023: IND application accepted by China’s NMPA
• January 2024: Received NMPA implied approval in China
• January 2024: Received U.S. FDA IND clearance; IND No. 30210
• Previously received Orphan Drug Designation from the U.S. FDA

Highlights: An independently developed AAV gene therapy drug in China for Gaucher disease, with dosing of the first patient completed.

PackGene Services: Provided high-quality CMC services to accelerate clinical trial application.

7. RM-101 – Usher Syndrome

Partner: Reforgene Medicine
Indication: Usher syndrome associated with retinitis pigmentosa

Regulatory Progress:

• Received IND approval from the U.S. FDA
• October 2024: Received IND approval from China’s NMPA

Highlights: Targets USH2A gene mutations and uses gene regulation technology to induce retinal cells to produce functional protein.

PackGene Role: Strategic partner of the program; specific service details have not been disclosed.