Ultragenyx Pharmaceutical. has filed a patent for compositions and methods to reduce reverse packaging of cap and/or rep DNA sequences in the production of recombinant adeno-associated virus (rAAV). The patent also covers pharmaceutical compositions for gene therapy purposes. The claim specifically describes a recombinant nucleic acid construct with modified AAV Cap coding sequence. GlobalData’s report on Ultragenyx Pharmaceutical gives a 360-degree view of the company including its patenting strategy. Buy the report here.

Smarter leaders trust GlobalData


Data Insights Ultragenyx Pharmaceutical Inc - Company Profile

Buy the Report

Data Insights

The gold standard of business intelligence.

Find out more

According to GlobalData’s company profile on Ultragenyx Pharmaceutical, Adeno-associated virus vectors was a key innovation area identified from patents. Ultragenyx Pharmaceutical's grant share as of September 2023 was 24%. Grant share is based on the ratio of number of grants to total number of patents.

The patent is filed for a modified aav cap coding sequence

Source: United States Patent and Trademark Office (USPTO). Credit: Ultragenyx Pharmaceutical Inc

A recently filed patent (Publication Number: US20230235353A1) describes a recombinant nucleic acid construct that includes an AAV Rep coding sequence and an AAV Cap coding sequence. The AAV Cap coding sequence has been modified by inserting one or more heterologous excisable intron sequences in the VP3 region, with a total length of at least 1 kb. The AAV Cap coding sequence can encode a capsid protein of various serotypes, including AAV8, AAV9, AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV10, AAV11, AAV12, AAVrh10, AAVhu37, or an engineered variant.

The recombinant nucleic acid construct may also contain additional nucleic acid sequences such as a promoter, an AAV intron, and a coding sequence for a selectable marker. The heterologous excisable intron sequences in the construct include at least one splice donor and at least one splice acceptor site, with a total length of at least 1.5 kb or ranging from 1.0 kb to 5.0 kb. These intron sequences can be derived from genes encoding various proteins, including EIF2S1, COL1A2, SPARC, STATS, ENO1, PKM, ALDOA, YBX1, GNB2L1, RPS3, GNAS, FLNA, TFRC, PABPC1, UBA1, CANX, and LDHA.

The patent also covers a vector containing the recombinant nucleic acid construct and a host cell comprising the construct. The host cell can further contain a plasmid with adenoviral helper genes and a plasmid with a transgene coding sequence. The transgene coding sequence can be a native coding sequence or a codon-optimized coding sequence and may encode various proteins such as OTC, G6Pase, factor VIII, factor IX, ATP7B, PAH, and many others.

The patent also describes a method of producing a preparation of recombinant AAV (rAAV) by culturing the host cell under suitable conditions. The resulting rAAV preparation has reduced levels of cap DNA or rep DNA or both compared to a preparation produced using an unmodified AAV Cap coding sequence. The patent further covers the rAAV produced by this method and a pharmaceutical composition comprising the rAAV and a pharmaceutically acceptable carrier.

Overall, this patent describes a recombinant nucleic acid construct and related methods for producing rAAV with modified AAV Cap coding sequences. These modifications can enhance the production of rAAV and reduce the levels of unwanted DNA in the final product. The patent also covers the use of various serotypes and transgenes in the construct, providing potential applications in gene therapy and other fields.

To know more about GlobalData’s detailed insights on Ultragenyx Pharmaceutical, buy the report here.

Data Insights


The gold standard of business intelligence.

Blending expert knowledge with cutting-edge technology, GlobalData’s unrivalled proprietary data will enable you to decode what’s happening in your market. You can make better informed decisions and gain a future-proof advantage over your competitors.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData Patent Analytics tracks bibliographic data, legal events data, point in time patent ownerships, and backward and forward citations from global patenting offices. Textual analysis and official patent classifications are used to group patents into key thematic areas and link them to specific companies