30+ IIT Projects Delivered

1. Rett Syndrome (RTT) Gene Therapy IIT

Partners: The research team led by Prof. Zhou Wenhao (Guangzhou Women and Children’s Medical Center) + the team led by Prof. Qiu Zilong (School of Medicine, Shanghai Jiao Tong University)
Research scope: The first RTT gene therapy investigator-initiated clinical study in the central-southern region
Key achievements: After dosing, the first treated 6-year-old patient showed a significant improvement in motor skills and communication abilities
PackGene Role: Provide AAV vector CMC services to help accelerate the project from R&D to clinical transition

2. ALS (Amyotrophic Lateral Sclerosis) SNUG01 Gene Therapy IIT

Partners: SineuGene Therapeutics + the team led by Prof. Fan Dongsheng (The Third Hospital of Peking University)
Research scope: The global first AAV gene therapy targeting TRIM72
Key achievements: The IIT study demonstrated good safety and early efficacy signals; the project subsequently obtained U.S. FDA / NMPA IND approvals
PackGene Role: Provide AAV vector CDMO services (process development, GMP manufacturing, quality studies, etc.)

3. Gaucher Disease Type I Gene Therapy IIT

Partners: Lingyi Biotech + the team led by President Huang (Yinhe) from the First Affiliated Hospital, College of Medicine, Zhejiang University
Research scope: A study of LY-M001 injection for safety and preliminary efficacy in adult patients with Gaucher disease type I
Milestones: China’s first clinical study of a gene medicine treating Gaucher disease type I; dosing for the first patient was successfully completed
PackGene Role: Provide high-quality CMC services

4. Gene Therapy IIT for Retinitis Pigmentosa (GA001)

Partners: Jianda Kyushu + the team led by Dr. Zhang Xuxiang (Beijing Tiantan Hospital)
Research scope: Using an optogenetics platform to treat retinitis pigmentosa; evaluating safety and restoration of photoreception
PackGene Role: Provide GMP plasmids and AAV production

5. Late-Stage Retinitis Pigmentosa ZM-02 Gene Therapy IIT

Partners: ZM Healthcare + Prof. Wei Wenbin / Prof. Lu Laichun (Beijing Tongren Hospital)
Research scope: ZM-02 administered as a single dose via intravitreal injection for RP patients
Milestones: Received approval from the Ethics Committee of Capital Medical University and备案 with the National Health Commission (NHC)
PackGene Role: Provide GMP plasmids and AAV production

6. X-linked Retinoschisis JWK002 Gene Therapy IIT

Partners: Genevector + West China Hospital of Sichuan University
Research scope: An AAV gene therapy to restore RS1 protein expression
Key achievements: IIT results showed significant improvement in BCVA, reduced retinal thickness, and complete closure of the retinoschisis cavity
PackGene Role: AAV vector CDMO support (subsequently received NMPA IND approval)

7. Alzheimer’s Disease NXL-001 Gene Therapy IIT

Partners: NeuExcell Therapeutics + the team led by Prof. Shi Jiong (The First Affiliated Hospital of USTC / Anhui Provincial Hospital)
Research scope: The world’s first in-situ neural regeneration therapy for treating severe Alzheimer’s disease
Technology: Delivering the neuronal transcription factor NeuroD1 via an AAV vector to induce astrocytes to transdifferentiate into functional neurons
Key achievements: The first patient completed dosing and had a one-month safety follow-up with good recovery
PackGene Role: Provide end-to-end CDMO services from plasmid to AAV manufacturing

8. Wet Age-related Macular Degeneration (nAMD) XMVA09 Gene Therapy IIT

Partners: Starry Eyes Biotech
Research scope: Conduct the IIT clinical study at the First Affiliated Hospital of the University of Science and Technology of China, enrolling patients with wAMD
Testing support: PackGene Biotech provides a full suite of plasmid and AAV CMC services, including process development/optimization, GMP manufacturing, analytical method development and validation, quality studies, release testing, stability studies, and preparation of complete CMC documentation
Subsequent progress: IIT data supported the NMPA IND approval in March 2024

9. JWK001 Gene Therapy IIT

Partner: Genevector
Indication: Neovascular age-related macular degeneration (nAMD)
Research scope: Conduct an IIT clinical study; after dosing, the first subject achieved a 12-letter improvement in best-corrected visual acuity (BCVA) at 20 weeks
Testing support: PackGene Biotech provides one-stop analytical testing services, including method development and validation, release testing, stability testing, and characterization studies
Subsequent progress: IIT data supported the NMPA IND approval in February 2024

10. RRG001 Gene Therapy IIT

Partners: Dingxin Gene
Indication: Unpublished specific indication (ophthalmology / otology / metabolic field)
Research scope: The IIT clinical study achieved phased breakthroughs
Testing support: PackGene Biotech provides CDMO services
Subsequent progress: Obtained IND authorization