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AVROBIO Inc (AVRO)

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  • May 18, 2022, 12:30 UTC

(10% Negative) AVROBIO, INC. (AVRO) Announces Delay in trial Trials for Pompe disease Due to Pandemic-Related Challenges, Patient Enrollment Issues, Regulatory Process, Safety Review, Efficacy Assessment

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  • News bot Oct. 1, 2025, 9:09 p.m.

    📋 AVROBIO, INC. (AVRO) - Clinical Trial Update

    Filing Date: 2022-05-18

    Accepted: 2022-05-18 08:30:41

    Event Type: Clinical Trial Update

    Event Details:

    AVROBIO Inc (AVRO) Announces Clinical Trial Update AVROBIO Inc (AVRO) provided an update on its clinical development programs. Clinical Development Highlights:
    • Drug Program: trial, uptake
    • Diseases/Conditions: Pompe disease, a mouse model of infantile onset Pompe disease
    • Clinical Stage: clinical trial
    • Update Type: Trial Timeline Adjustment
    • Primary Factors: Pandemic-Related Challenges, Patient Enrollment, Regulatory Process
    • Collaboration: the American Society of Gene
      • Targeting (GILT)-tag which consists of a short peptide sequence linked to the therapeutic protein and is designed to enhance uptake in targeted tissues. “Pompe disease is a progressive, life-limiting neuromuscular disorder caused by the accumulation of lysosomal glycogen in cardiac and skeletal muscle as well as the CNS. As glycogen accumulates, patients experience severe and progressive myopathy, leading to muscle weakness, loss of motor function and ultimately cardiorespiratory failure,” said AVROBIO Chief Medical Officer, Essra Ridha, M.D., MRCP, FFPM. “We believe there is an urgent need for new treatment options that can address the systemic impact of Pompe disease and our preclinical data suggest that one dose of HSC-based gene therapy can potentially normalize glycogen levels in key tissues and halt or potentially reverse the head-to-toe impact of Pompe disease.” Data collected from six gene therapy study groups in mice show: • Stable engraftment up to eight months after gene therapy, with median vector copy numbers (VCN) between 1.9 and 3.6 per diploid genome (n=9-13 per group) in bone marrow among all study groups. • Significant GAA enzyme activity measured in the bone marrow eight months after gene therapy. The supraphysiological GAA enzyme activity levels in bone marrow ranged from a median 300 to 534 nmol/h/mg (p value <0.005, n=9-13 per group), compared to ± 4-6 nmol/h/mg in normal mice. • Glucose levels in mice infused with genetically modified HSCs were similar to those of controls. Significant reductions of glycogen observed in key tissues, including: • >99% reduction of glycogen in the heart, with reversal of cardiac hypertrophy (thickening of the heart muscle) and normalization of the left ventricular mass index, observed seven months post gene therapy • >97% reduction of glycogen in the diaphragm and >85% reduction in the quadriceps. Significant improvements in gait and wire hang functions after gene therapy were also observed seven months post gene therapy • Additionally, glycogen was reduced >95% in the brain and >99% in the spinal cord, improving locomotor function and demonstrating that modified HSCs crossed the blood-brain barrier where their offspring produced functional protein • Importantly, the data show a typical lentiviral vector (the vector used by AVROBIO to deliver the therapeutic gene to the HSC) preference to integrate into genes, with no indications of proto-oncogene selection or clonal dominance, reinforcing the safety of the vector. Additionally, new data from a separate study show that human HSCs were efficiently transduced, producing robust quantities of transgene product and GAA enzyme activity. “These data show a substantial reduction of substrate in key tissues, muscle and CNS, reduced tissue pathology and correction on a functional level, which are all very relevant for potential future translation into Pompe patients,” said Niek P. van Til, Ph.D., assistant professor, Child Neurology, Amsterdam University Medical Centers and consultant to AVROBIO, who led the research and will present it at ASGCT this afternoon. AVROBIO plans to engage with regulatory agencies on the clinical development strategy for AVR-RD-03 in 2022

    💼 Business Developments:

    • Partnership: Not available
    • Acquisition: Not available
    • Licensing: Not available
    • Regulatory Approval
    • Executive Changes: Not available

    Structured Data:

    • Company Name: AVROBIO Inc
    • CIK: 0001681087
    • Ticker Symbol: AVRO
    • Period End Date: 2022-05-18
    • Document Type: 8-K

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