Patents by Inventor Maria Sol Rodriguez Pena

Maria Sol Rodriguez Pena has filed for patents to protect the following inventions. This listing includes patent applications that are pending as well as patents that have already been granted by the United States Patent and Trademark Office (USPTO).

  • Porphobilinogen deaminase gene therapy
    Patent number: 8697665
    Abstract: The present invention relates to nucleotide sequences coding for human porphobilinogen deaminase that are optimised for higher expression in mammalian cells. The invention further relates to DNA constructs comprising such optimised synthetic coding sequences for use in gene therapy of conditions caused by a deficiency in porphobilinogen deaminase, such as acute intermittent porphyria. Accordingly, the present invention relates to a nucleic acid or a nucleic acid construct comprising a nucleotide sequence coding for a human porphobilinogen deaminase, wherein at least 320 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 1 or wherein at least 305 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 3.
    Type: Grant
    Filed: September 29, 2009
    Date of Patent: April 15, 2014
    Assignees: Proyecto de Biomedicina CIMA S.L., Uniqure Biopharma B.V.
    Inventors: Antonio Fontanellas Romá, Gloria González Aseguinolaza, Maria Sol Rodriguez Pena, Maria Astrid Pañeda Rodriguez, Jaap Twisk, Jesús Maria Prieto Valtueña, Harald Petry, Sander Jan Hendrik Van Deventer
  • ALANINE-GLYOXYLATE AMINOTRANSFERASE THERAPEUTICS
    Publication number: 20120027745
    Abstract: The present invention relates to an alanine glyoxylate aminotransferase (AGXT) I340M therapeutic for use as a medicament or in a method of treatment, for example in the treatment of an AGXT-responsive condition. The AGXT I340M therapeutic is an AGXT I340M protein comprising an amino acid sequence, which, when optimally aligned with SEQ ID NO: 2, comprises a methionine at a position corresponding to position 340 in SEQ ID NO: 2, a nucleic acid molecule encoding such an AGXT I340M protein, or a virion of a viral gene therapy vector comprising such a nucleic acid molecule. The AGXT I340M therapeutic has a higher specific activity as compared to other AGXT alleles and may therefore be advantageously used in the treatment of primary hyperoxaluria type I.
    Type: Application
    Filed: February 1, 2010
    Publication date: February 2, 2012
    Inventors: Maria Sol Rodriguez Pena, Harald Petry, Jaap Twisk, Sander Jan Hendrik Van Deventer, Eduardo Carlos Salido Ruiz, Armando Torres Ramirez
  • PORPHOBILINOGEN DEAMINASE GENE THERAPY
    Publication number: 20110262399
    Abstract: The present invention relates to nucleotide sequences coding for human porphobilinogen deaminase that are optimised for higher expression in mammalian cells. The invention further relates to DNA constructs comprising such optimised synthetic coding sequences for use in gene therapy of conditions caused by a deficiency in porphobilinogen deaminase, such as acute intermittent porphyria. Accordingly, the present invention relates to a nucleic acid or a nucleic acid construct comprising a nucleotide sequence coding for a human porphobilinogen deaminase, wherein at least 320 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 1 or wherein at least 305 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 3.
    Type: Application
    Filed: September 29, 2009
    Publication date: October 27, 2011
    Applicants: Proyecto de Biomedicina CIMA S.L., Amsterdam Molecular Therapeutics (AMT) B.V.
    Inventors: Antonio Fontanellas Romá, Gloria González Aseguinolaza, Maria Sol Rodriguez Pena, Maria Astrid Pañeda Rodriguez, Jaap Twisk, Jesús Maria Prieto Valtueña, Harald Petry, Sander Jan Hendrik Van Deventer