Can therapeutics be delivered through genetic transfer? - Whitepaper

White Paper

Conventional monoclonal antibody therapy depends on external manufacture and repeated administration. Cold-chain distribution, dosing burden, limited tissue penetration. These are properties of how the antibody is delivered, not of the antibody itself.

AAV-mediated antibody delivery changes where production happens. The vector carries the coding sequence, the patient’s cells express it, and a single administration can sustain antibody production for months to years.

Preclinical studies show sustained expression across multiple disease models. Early clinical programs in ophthalmology and HIV show durable expression in humans from a single administration.

The engineering is where the work is. Packaging capacity forces real choices about antibody format. Capsid and promoter shape where and how strongly the antibody is produced, with biodistribution dependent on species, route, dose, and vector design. Pre-existing anti-capsid immunity limits who can be dosed at all. Re-dosing is constrained once the immune system has seen the capsid. And persistent expression complicates titration and reversibility in ways a repeatedly administered biologic does not.

This paper covers the rationale, the design decisions, the current evidence, and the problems that remain open. Download it for the full review, including antibody format comparisons, cassette design schematics, capsid selection, and the current preclinical and clinical landscape.

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