27 patents
Utility
Vectors with Modified Initiation Codon for the Translation of AAV-REP78 Useful for Production of Aav
28 Dec 23
The present invention relates nucleic acid constructs for the production of recombinant parvoviral (e.g. adeno-associated viral) vectors in insect cells, to insect cells comprising such constructs and to methods wherein the cells are used to produce recombinant parvoviral virions.
Wilhelmus Theodorus Johannes Maria Christiaan Hermens, Saskia Jacoba Petronella Haast, Dennis Johan Biesmans, Andrew Christian Bakker
Filed: 7 Mar 23
Utility
Dual Bifunctional Vectors for Aav Production
25 May 23
The present invention relates novel combinations of nucleic acid constructs for the production of recombinant parvoviral gene therapy vectors.
David Johannes Francois DU PLESSIS, ANGGAKUSUMA, Sebastiaan Menno BOSMA, Jacek LUBELSKI
Filed: 20 Sep 22
Utility
Rnai Induced Huntingtin Gene Suppression
20 Apr 23
The present invention provides for a double stranded RNA comprising a first RNA sequence and a second RNA sequence wherein the first and second RNA sequence are substantially complementary, wherein the first RNA sequence has a sequence length of at least 19 nucleotides and is substantially complementary to SEQ ID NO. 1.
Pavlina Stefanova KONSTANTINOVA, Jana MINARIKOVÁ
Filed: 24 May 22
Utility
Means and Methods for Aav Gene Therapy In Humans
15 Dec 22
The present invention relates to means and method for AAV based gene therapies in humans.
Bart Antonius NIJMEIJER, Valerie FERREIRA
Filed: 30 Jun 22
Utility
Aav Vectors Produced by Insect Cells Comprising REP52 and REP78 Coding Sequences with Differential Codon Biases
11 Aug 22
The present invention relates to production of proteins in insect cells whereby repeated coding sequences are used in baculoviral vectors.
Andrew Christian BAKKER, Wilhelmus Theodorus Johannes Maria Christiaan HERMENS
Filed: 15 Apr 22
Utility
Factor IX Polypeptide Mutant, Its Uses and a Method for Its Production
24 Mar 22
Disclosed are a modified FIX (Factor IX) polypeptide comprising a leucine, cysteine, aspartic acid, glutamic acid, histidine, lysine, asparagine, glutamine or tyrosine in position 338; pharmaceutical preparations containing said modified FIX polypeptide; a nucleotide sequence coding for the modified FIX polypeptide; and a method for producing the modified FIX polypeptide.
Paolo SIMIONI
Filed: 8 Dec 21
Utility
Liver-specific Viral Promoters and Methods of Using the Same
10 Mar 22
The present invention relates to promoters that function specifically or preferentially in the liver.
Jacek LUBELSKI, David Johannes Francois DU PLESSIS, Ying Poi LIU, Olivier TER BRAKE, Juan Manuel IGLESIAS GONZALEZ, Ross FRASER, Michael ROBERTS
Filed: 14 May 21
Utility
Companion Diagnostic to Monitor the Effects of Gene Therapy
27 Jan 22
The invention relates to the field of gene therapy.
Astrid VALLES-SANCHEZ, Pavlina Stefanova KONSTANTINOVA
Filed: 18 May 21
Utility
Rnai Induced Reduction of ATAXIN-3 for the Treatment of Spinocerebellar Ataxia Type 3
13 Jan 22
The current invention relates to gene therapy approaches for the treatment of SCA3, in particular RNAi based gene therapy approaches utilizing a total knockdown approach.
Melvin Maurice EVERS, Pavlina Stefanova KONSTANTINOVA, Raygene Michaël MARTIER
Filed: 13 May 21
Utility
Factor IX Polypeptide Mutant, Its Uses and a Method for Its Production
6 Jan 22
Disclosed are a modified FIX (Factor IX) polypeptide comprising a leucine, cysteine, aspartic acid, glutamic acid, histidine, lysine, asparagine, glutamine or tyrosine in position 338; pharmaceutical preparations containing said modified FIX polypeptide; a nucleotide sequence coding for the modified FIX polypeptide; and a method for producing the modified FIX polypeptide.
Paolo SIMIONI
Filed: 10 Sep 21
Utility
METHOD AND MEANS TO DELIVER miRNA TO TARGET CELLS
2 Dec 21
The invention relates to the field of gene therapy.
Astrid VALLES-SANCHEZ, Pavlina Stefanova KONSTANTINOVA, Sander Jan Hendrik VAN DEVENTER, Marina Sogorb GONZÁLEZ
Filed: 11 May 21
Utility
Immunoadsorption
18 Nov 21
Upon administration of rAAV vectors the humoral immune response (neutralizing antibodies) is the first barrier that needs to be overcome.
Valerie FERREIRA
Filed: 3 Jun 21
Utility
Dna Impurities In a Composition Comprising a Parvoviral Virion
28 Oct 21
The current invention relates to nucleic acid impurities in a composition comprising a parvoviral vector.
Jacek LUBELSKI, Wilhelmus Theodorus Johannes Maria Christiaan HERMENS
Filed: 6 May 21
Utility
Rnai Induced C9ORF72 Suppression for the Treatment of Als/ftd
28 Oct 21
The present invention provides for specific target RNA sequences that can in particular be applied in RNAi based gene therapy approaches for the treatment of ALS and/or FTD.
Raygene Michaël MARTIER, Pavlina Stefanova KONSTANTINOVA
Filed: 9 Mar 21
Utility
Factor IX Polypeptide Mutant, Its Uses and a Method for Its Production
5 Aug 21
Disclosed are a modified FIX (Factor IX) polypeptide comprising a leucine, cysteine, aspartic acid, glutamic acid, histidine, lysine, asparagine, glutamine or tyrosine in position 338; pharmaceutical preparations containing said modified FIX polypeptide; a nucleotide sequence coding for the modified FIX polypeptide; and a method for producing the modified FIX polypeptide.
Paolo SIMIONI
Filed: 26 Mar 21
Utility
Further Improved Aav Vectors Produced In Insect Cells
22 Jul 21
The present invention relates to the production of adeno-associated viral vectors in insect cells.
Jacek LUBELSKI, Sebastiaan Bosma, Harald Peter Albert Petry, Wilhelmus Theodorus Johannes Maria Christ Hermens
Filed: 5 Nov 20
Utility
Factor IX Polypeptide Mutant, Its Uses and a Method for Its Production
15 Jul 21
Disclosed are a modified FIX (Factor IX) polypeptide comprising a leucine, cysteine, aspartic acid, glutamic acid, histidine, lysine, asparagine, glutamine or tyrosine in position 338; pharmaceutical preparations containing said modified FIX polypeptide; a nucleotide sequence coding for the modified FIX polypeptide; and a method for producing the modified FIX polypeptide.
Paolo SIMIONI
Filed: 26 Mar 21
Utility
Vectors with Modified Initiation Codon for the Translation of AAV-REP78 Useful for Production of Aav
20 May 21
The present invention relates nucleic acid constructs for the production of recombinant parvoviral (e.g. adeno-associated viral) vectors in insect cells, to insect cells comprising such constructs and to methods wherein the cells are used to produce recombinant parvoviral virions.
Wilhelmus Johannes Theodorus Maria Christiaan Hermens, Saskia Jacoba Petronella Haast, Dennis Johan Biesmans, Andrew Christian Bakker
Filed: 14 Dec 20
Utility
Methods of Improving Adeno-associated Viral Transduction
18 Nov 20
Described herein are saturating agents, AAV gene therapy vectors, and therapeutic agents, as well as methods and kits comprising the same.
Valerie FERREIRA
Filed: 8 Jun 20
Utility
RNAi Induced Huntingtin Gene Suppression
28 Oct 20
The present invention provides for a double stranded RNA comprising a first RNA sequence and a second RNA sequence wherein the first and second RNA sequence are substantially complementary, wherein the first RNA sequence has a sequence length of at least 19 nucleotides and is substantially complementary to SEQ ID NO. 1.
Pavlina Stefanova KONSTANTINOVA, Jana MINIARIKOVÁ
Filed: 14 Jul 20