Amsterdam Molecular Therapeutics B.v.

Uniqure IP B.v.

Masashi Urabe

Wilhelmus Theodorus Johannes Maria Christiaan Hermens

Saskia Jacoba Petronella Haast

Keiya Ozawa

Wilhelmus Theodorus Johannes Maria Christian Hermens

Wilhelmus TJMC Hermens

The present invention relates to the production of adeno-associated viral vectors in insect cells. The insect cells therefore comprise a first nucleotide sequence encoding the adeno-associated virus (AAV) capsid proteins, whereby the initiation codon for translation of the AAV VP1 capsid protein is a non-ATG, suboptimal initiation codon. The insect cell further comprises a second nucleotide sequence comprising at least one AAV inverted terminal repeat (ITR) nucleotide sequence; a third nucleotide sequence comprising a Rep52 or a Rep40 coding sequence operably linked to expression control sequences for expression in an insect cell; and, a fourth nucleotide sequence comprising a Rep78 or a Rep68 coding sequence operably linked to expression control sequences for expression in an insect cell. The invention further relates to adeno-associated viral vectors with an altered ratio of the viral capsid proteins that provides improved infectivity of the viral particles.

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Tochigi, Japan

Masashi Urabe