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 Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

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