30+ IIT Projects Delivered

PackGene Biotech, a leading gene therapy CRO & CDMO in China, has successfully delivered dozens of investigator-initiated clinical study (IIT) programs, supporting end-to-end CMC services from early proof of concept to clinical translation.

1. Gene Therapy IIT for Rett Syndrome (RTT)

Partner: Professor Wenhao Zhou’s team at Guangzhou Women and Children’s Medical Center + Professor Zilong Qiu’s team at Shanghai Jiao Tong University School of Medicine
Research Focus: The first investigator-initiated clinical study of gene therapy for RTT in Central and South China
Outcomes: The first 6-year-old pediatric patient showed significant improvements in motor skills and communication ability after dosing
PackGene Role: Provided AAV vector CMC services, supporting the program’s transition from R&D to clinical translation

2. SNUG01 Gene Therapy IIT for ALS

Partner: SineuGene Therapeutics + Professor Dongsheng Fan’s team at Peking University Third Hospital
Research Focus: The world’s first AAV gene therapy targeting TRIM72
Outcomes: IIT data demonstrated a favorable safety profile and early efficacy signals, supporting subsequent FDA and NMPA IND approvals
PackGene Role: Provided AAV vector CDMO services, including process development, GMP manufacturing, quality studies, and more

3. Gene Therapy IIT for Type I Gaucher Disease

Partner: Lingyi Biotech + President He Huang’s team at The First Affiliated Hospital, Zhejiang University School of Medicine
Research Focus: Safety and preliminary efficacy of LY-M001 Injection in adult patients with Type I Gaucher disease
Milestone: China’s first clinical study of a gene therapy drug for Type I Gaucher disease, with successful dosing of the first patient
PackGene Role: Provided high-quality CMC services

4. GA001 Gene Therapy IIT for Retinitis Pigmentosa

Partner: GenAns Biotech + Director Xuxiang Zhang of Beijing Tiantan Hospital
Research Focus: Evaluation of the safety and photosensitivity restoration potential of an optogenetics-based technology platform for the treatment of retinitis pigmentosa
PackGene Role: Provided GMP plasmid and AAV manufacturing

5. ZM-02 Gene Therapy IIT for Advanced Retinitis Pigmentosa

Partner: Zhongmou Therapeutics + Professor Wenbin Wei / Professor Laichun Lu of Beijing Tongren Hospital
Research Focus: Single-dose intravitreal administration of ZM-02 for the treatment of patients with RP
Milestone: Approved by the Ethics Committee of Capital Medical University and filed with the National Health Commission
PackGene Role: Provided GMP plasmid and AAV manufacturing

6. JWK002 Gene Therapy IIT for X-Linked Retinoschisis

Partner: Genevector + West China Hospital, Sichuan University
Research Focus: AAV gene therapy designed to restore RS1 protein expression
Outcomes: IIT data showed significant improvement in best-corrected visual acuity (BCVA), reduction in retinal thickness, and complete closure of retinoschisis cavities
PackGene Role: Provided AAV vector CDMO support; the program subsequently received NMPA IND approval

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

Partner: NeuExcell Therapeutics + Professor Jiong Shi’s team at The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China / Anhui Provincial Hospital
Research Focus: The world’s first in situ neural regeneration therapy for severe Alzheimer’s disease
Technology: AAV vector-mediated delivery of the neural transcription factor NeuroD1 to induce the transdifferentiation of astrocytes into functional neurons
Outcomes: The first patient completed dosing and one-month safety follow-up, with a favorable recovery
PackGene Role: Provided one-stop CDMO services from plasmid to AAV manufacturing

8. XMVA09 Gene Therapy IIT for Wet Age-Related Macular Degeneration (wAMD)

Partner: StarryGene
Research Focus: IIT clinical study conducted at The First Affiliated Hospital of USTC, enrolling patients with wAMD
CMC Support: PackGene provided a full suite of CMC services from plasmid to AAV, including process development and optimization, GMP manufacturing, analytical method development and validation, quality studies, release testing, stability studies, and complete CMC dossier preparation
Subsequent Progress: IIT data supported NMPA IND approval in March 2024

9. JWK001 Gene Therapy IIT

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

10. RRG001 Gene Therapy IIT

Partner: Refreshgene
Indication: Specific indication not publicly disclosed; the program focuses on ophthalmology, otology, and/or metabolic disease areas
Research Focus: The IIT clinical study achieved interim progress
Analytical Support: PackGene provided CDMO services
Subsequent Progress: Received IND clearance