{
  "abstract": "We report a neonate with IOPD who had high pre-existing anti-AAV9 antibody titers, which precluded eligibility for AAV9-mediated gene therapy (GC301). Preconditioning with a single dose of the IgG-degrading enzyme GC801 (0.2 mg/kg) safely reduced serum IgG from 6.25 g/L to a nadir of 0.72 g/L within 5 days. This pharmacological window facilitated successful GC301 vector transduction, driving a robust increase in GAA activity to 19.38 mmol/L/16h by day 84. Crucially, no adverse events beyond the underlying IOPD were observed. Our findings support the potential utility of GC801 as an agent to overcome pre-existing humoral barriers in neonatal gene therapy candidates.",
  "abstract_shown": true,
  "affiliations": [
    "Genecradle Therapeutics"
  ],
  "article_type": "PUBLISHAHEADOFPRINT",
  "arxiv": null,
  "author_total": 17,
  "authors": [
    {
      "name": "J. Chen"
    },
    {
      "name": "W. Ma"
    },
    {
      "name": "H. Qi"
    },
    {
      "name": "X. Ma"
    },
    {
      "name": "Y. He"
    },
    {
      "name": "L. He"
    },
    {
      "name": "Y. Ke"
    },
    {
      "name": "L. Zhuang"
    },
    {
      "name": "X. Jiang"
    },
    {
      "name": "J. Wang"
    },
    {
      "name": "J. Xu"
    },
    {
      "name": "Y. Xia"
    },
    {
      "name": "J. Zhou"
    },
    {
      "name": "Y. Wang"
    },
    {
      "name": "X. Dong"
    },
    {
      "affs": [
        0
      ],
      "name": "X. Wu"
    },
    {
      "name": "Z. Feng"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/9pmysa35cafnm.bib",
  "cited_by": 0,
  "doi": "10.64898/2026.09.23.26363499",
  "fulltext_url": "https://www.medrxiv.org/content/10.64898/2026.09.23.26363499v1.full",
  "id": "9pmysa35cafnm",
  "issns": [],
  "landing_url": "https://www.medrxiv.org/content/10.64898/2026.09.23.26363499v1",
  "language": null,
  "licence": "CC-BY",
  "licence_label": "CC BY",
  "licence_source": "article",
  "licence_url": "https://creativecommons.org/licenses/by/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "medrxiv",
      "genetic and genomic medicine"
    ],
    [
      "openalex",
      "2737|Lysosomal Storage Disorders Research"
    ]
  ],
  "openalex": "W7214793446",
  "pdf_url": "https://www.medrxiv.org/content/10.64898/2026.09.23.26363499v1.full.pdf",
  "pmcid": null,
  "pmid": null,
  "published": "2026-09-23",
  "publisher": null,
  "ris": "https://research.draconitic.com/paper/9pmysa35cafnm.ris",
  "sources": [
    {
      "first_seen": "2026-10-30T12:19:37Z",
      "native_id": "10.64898/2026.09.23.26363499",
      "source": "medrxiv",
      "url": "https://www.medrxiv.org/content/10.64898/2026.09.23.26363499v1"
    }
  ],
  "subjects": [
    {
      "field": "medicine",
      "origin": "medrxiv:genetic and genomic medicine",
      "subfield": "human-genetics"
    }
  ],
  "title": "GC801-enabled AAV gene therapy in a neonate with Pompe disease",
  "title_text": "GC801-enabled AAV gene therapy in a neonate with Pompe disease",
  "type": "preprint",
  "updated_at": "2026-10-30T12:19:37Z",
  "url": "https://research.draconitic.com/paper/9pmysa35cafnm",
  "venue": "medRxiv",
  "versions": []
}