Introduction to a Pivotal Gene Therapy Study

On October 30, 2023, a significant clinical trial commenced, focusing on LY-M001, a gene therapy drug for Gaucher Disease Type I. This trial is particularly noteworthy for its involvement of PackGene as the CMC (Chemistry, Manufacturing, and Controls) service provider, ensuring stringent quality and regulatory compliance in the drug’s production.


Study Details

The LY-M001 clinical trial is a prospective, single-center, open-label, single-arm interventional study. Its purpose is to evaluate the safety, initial efficacy, pharmacodynamic characteristics, immunogenicity, biodistribution, and viral shedding of a single intravenous infusion of LY-M001. PackGene’s role as the CMC service provider underscores the trial’s commitment to high-quality standards in gene therapy research.


Phases of the Study

The study is structured into several distinct phases:

  • Screening Period (Weeks -8 to Days -2): Pre-eligibility assessment.
    Baseline Period (Day -1): Establishment of health benchmarks before treatment.
  • Treatment and Safety Observation Period (Days 0 to 28): Administration of LY-M001 and monitoring for safety and immediate effects.
  • Short-Term Follow-Up Period (Weeks 5 to 38): Assessment of the treatment’s short-term impact.
  • Participants are required to complete all assessments for the End of Study (EOS) visit, regardless of whether they complete the study or withdraw early.
  • The initial dose group is set at 5.0 × 10^12 vg/kg, following a sentinel method for participant enrollment and observation.


Long-Term Follow-Up Study

This study also includes a long-term follow-up phase lasting a total of 5 years, essential for understanding the prolonged effects and safety of LY-M001.


Study Specifications

  • Study Type: Interventional (Clinical Trial)
  • Estimated Enrollment: 3 participants
  • Intervention Model: Single Group Assignment
  • Primary Purpose: Treatment
  • Official Title: Prospective, Single-center, Open-arm, Single-arm Study of Safety and Preliminary Efficacy of LY-M001 Injection
  • Start Date: October 30, 2023
  • Primary Completion Date: August 17, 2024
  • Study Completion Date: October 17, 2028


The Role of PackGene

PackGene’s involvement as the CMC service provider is a testament to the trial’s emphasis on quality and regulatory adherence in drug development. This collaboration is anticipated to play a crucial role in the successful execution of the trial and in potentially setting new benchmarks in gene therapy treatments for Gaucher Disease Type I.



This clinical trial, with PackGene’s support, represents a significant step in the advancement of gene therapy for Gaucher Disease Type I. The thorough approach to the study’s design and execution reflects an unwavering commitment to exploring innovative treatments in the field of medical science.

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About PackGene

PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha 293 cell AAV high-yield platform and the π-Omega plasmid high-yield platform. PackGene's mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.

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