Patents Assigned to FUNDACIÓN PARA LA INVESTIGACIÓN MÉDICA APLICADA
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POLYNUCLEOTIDES ENCODING PORPHOBILINOGEN DEAMINASE FOR THE TREATMENT OF ACUTE INTERMITTENT PORPHYRIA
Publication number: 20230112986Abstract: The invention relates to mRNA therapy for the treatment of Acute Intermittent Porphyria (AIP). mRNAs for use in the invention, when administered in vivo, encode human porphobilinogen deaminase (PBGD), isoforms thereof, functional fragments thereof, and fusion proteins comprising PBGD. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to affect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of PBGD expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient PBGD activity in subjects, namely porphobilinogen and aminolevulinate (PBG and ALA).Type: ApplicationFiled: July 21, 2022Publication date: April 13, 2023Applicants: ModernaTX, Inc., Fundacion Para La Investigacion Medica AplicadaInventors: Paolo MARTINI, Stephen HOGE, Kerry BENENATO, Vladimir PRESNVAK, Lei JIANG, Iain MCFADYEN, Ellalahewage Sathyajith KUMARASINGHE, Antonio FONTANELLAS ROMA, Pedro BERRAONDO LOPEZ, Matias Antonio AVILA ZARAGOZA, Lin Tung GUEY, Staci SABNIS -
Patent number: 11560407Abstract: The present invention provides peptides of general formula (I) and salts thereof, wherein: R1 and R2, taken together, form a birradical linker; and R2? is hydrogen; or, alternatively, R1 is selected from hydrogen, —C(?O)—CH2—NH—C(?O)—(C1-C5)alkyl, and —C(?O)—(C1-C20)alkyl; one of R2 and R2? is hydrogen and the other is selected from —C(?O)NR3R4, and —C(?O)OH; and R3 and R4 are same or different and are selected from hydrogen and (C1-C10)alkyl. These peptides are highly efficient in binding and inhibiting FoxP3, being efficient in inhibiting and blocking Treg cell functionality, which make them useful in the treatment of cancer. The present invention also provides constructs comprising the peptide of formula (I) as well as combinations comprising the peptide of formula (I), the construct or both.Type: GrantFiled: April 20, 2020Date of Patent: January 24, 2023Assignee: FUNDACIÓN PARA LA INVESTIGACIÓN MÉDICA APLICADAInventors: Inés Noelia Casares Lagar, Juan José Lasarte Sagastibelza, Teresa Lozano Moreda, Julen Oyarzabal Santamarina, Maria Obdulia Rabal Gracia
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Patent number: 11390854Abstract: The present disclosure relates to human porphobilinogen deaminase derived proteins and polynucleotides and methods of using these proteins and polynucleotides.Type: GrantFiled: May 18, 2017Date of Patent: July 19, 2022Assignee: Fundacion Para la Investigacion Medica AplicadaInventors: Antonio Fontanellas Roma, Irantzu Serrano Mendioroz, Pedro Berraondo Lopez
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Publication number: 20220008559Abstract: The invention relates to nucleic acid constructs and gene therapy vectors that comprise an ATP7B variant for use in the treatment of conditions associated with a deficiency or dysfunction of Copper-transporting ATPase 2, and particularly of Wilson's disease. An AAV vector devised according to the invention significantly reduced urine Cu excretion, and liver Cu content in Wilson's disease mice treated with the vector, while ceruloplasmin activity was significantly restored. On the other hand, the administration of the vector resulted in the normalization of serum transaminases' levels and of liver histology, together with a marked reduction of the inflammatory infiltrate.Type: ApplicationFiled: September 24, 2021Publication date: January 13, 2022Applicant: FUNDACIÓN PARA LA INVESTIGACIÓN MÈDICA APLICADAInventors: Oihana MURILLO SAUCA, Gloria GONZÁLEZ ASEGUINOLAZA, Rubén HERNÁNDEZ ALCOCEBA
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Patent number: 11147887Abstract: The invention relates to nucleic acid constructs and gene therapy vectors that comprise an ATP7B variant for use in the treatment of conditions associated with a deficiency or dysfunction of Copper-transporting ATPase 2, and particularly of Wilson's disease. An AAV vector devised according to the invention significantly reduced urine Cu excretion, and liver Cu content in Wilson's disease mice treated with the vector, while ceruloplasmin activity was significantly restored. On the other hand, the administration of the vector resulted in the normalization of serum transaminases' levels and of liver histology, together with a marked reduction of the inflammatory infiltrate.Type: GrantFiled: December 17, 2015Date of Patent: October 19, 2021Assignee: FUNDACIÓN PARA LA INVESTIGACIÓN MÈDICA APLICADAInventors: Oihana Murillo Sauca, Gloria González Aseguinolaza, Rubén Hernández Alcoceba
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Patent number: 11118238Abstract: The invention relates to a nucleic acid construct carrying ATP7B protein, an expression vector and a viral particle comprising the nucleic acid construct, and their use for treatment of Wilson's disease and other conditions caused by a deficiency or dysfunction of ATP7B protein. An AAV vector devised according to the invention significantly reduced urine Cu excretion, and liver Cu content in Wilson's disease mice treated with the vector, while ceruloplasmin activity was significantly restored. On the other hand, the administration of the vector resulted in the normalization of serum transaminases levels and of liver histology, together with a marked reduction of the inflammatory infiltrate.Type: GrantFiled: December 17, 2015Date of Patent: September 14, 2021Assignee: FUNDACIÓN PARA LA INVESTIGACIÓN MÈDICA APLICADAInventors: Oihana Murillo Sauca, Gloria González Aseguinolaza, Rubén Hernández Alcoceba
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Patent number: 10654896Abstract: The present invention provides peptides of general formula (I) and salts thereof, wherein: R1 and R2, taken together, form a birradical linker; and R2? is hydrogen; or, alternatively, R1 is selected from hydrogen, —C(?O)—CH2—NH—C(?O)—(C1-C5)alkyl, and —C(?O)—(C1-C20)alkyl; one of R2 and R2? is hydrogen and the other is selected from —C(?O)NR3R4, and —C(?O)OH; and R3 and R4 are same or different and are selected from hydrogen and (C1-C10)alkyl. These peptides are highly efficient in binding and inhibiting FoxP3, being efficient in inhibiting and blocking Treg cell functionality, which make them useful in the treatment of cancer. The present invention also provides constructs comprising the peptide of formula (I) as well as combinations comprising the peptide of formula (I), the construct or both.Type: GrantFiled: October 19, 2017Date of Patent: May 19, 2020Assignee: FUNDACIÓN PARA LA INVESTIGACIÓN MÉDICA APLICADAInventors: Inés Noelia Casares Lagar, Juan José Lasarte Sagastibelza, Teresa Lozano Moreda, Julen Oyarzabal Santamarina, Maria Obdulia Rabal Gracia
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Patent number: 10407423Abstract: It relates to the compounds of formula (I), or their pharmaceutically or veterinary acceptable salts, or their stereoisomers or mixtures thereof, wherein A, R1, R2, and R3 are as defined herein, which are inhibitors of one or more DNMTs selected from the group consisting of DNMT1, DNMT3A and DNMT3B. It also relates to pharmaceutical or veterinary compositions containing them, and to their use in medicine, in particular in the treatment and/or prevention of cancer, fibrosis and/or immunomodulation.Type: GrantFiled: November 15, 2016Date of Patent: September 10, 2019Assignee: FUNDACIÓN PARA LA INVESTIGACIÓN MÉDICA APLICADAInventors: Xabier Aguirre Ena, Julen Oyarzabal Santamarina, Felipe Prósper Cardoso, Maria Obdulia Rabal Gracia, Edurne San José Enériz, Juan Antonio Sánchez Arias
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Publication number: 20170356060Abstract: The invention relates to a nucleic acid construct carrying ATP7B protein, an expression vector and a viral particle comprising the nucleic acid construct, and their use for treatment of Wilson's disease and other conditions caused by a deficiency or dysfunction of ATP7B protein. An AAV vector devised according to the invention significantly reduced urine Cu excretion, and liver Cu content in Wilson's disease mice treated with the vector, while ceruloplasmin activity was significantly restored. On the other hand, the administration of the vector resulted in the normalization of serum transaminases levels and of liver histology, together with a marked reduction of the inflammatory infiltrate.Type: ApplicationFiled: December 17, 2015Publication date: December 14, 2017Applicant: FUNDACIÓN PARA LA INVESTIGACIÓN MÈDICA APLICADAInventors: Oihana MURILLO SAUCA, Gloria GONZÁLEZ ASEGUINOLAZA, Rubén HERNÁNDEZ ALCOCEBA
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Patent number: 9840500Abstract: It relates to the use of compounds of formula (I?), or their pharmaceutically or veterinary acceptable salts, or their stereoisomers or mixtures thereof, as anticancer agents and as agents for generating induced pluripotent stem cells. Compounds of formula (I?), wherein R2? is an alcoxy group, a hydrocarbon chain or a ring system, and R1, R3, and R4 are as defined herein, are dual inhibitors of histone methyltransferases and DNA methyltransferases. It also relates to the compounds of formula (I?), or their pharmaceutically or veterinary acceptable salts, or their stereoisomers or mixtures thereof, wherein R2? is phenyl or 5- to 6-membered heteroaromatic ring, both optionally fused to another rings (i.e., compounds of formula (I)). It also relates to pharmaceutical or veterinary compositions containing compounds of formula (I).Type: GrantFiled: March 30, 2015Date of Patent: December 12, 2017Assignee: FUNDACIÓN PARA LA INVESTIGACIÓN MÉDICA APLICADAInventors: Xabier Aguirre Ena, Julen Oyarzabal Santamarina, Felipe Prósper Cardoso, Maria Obdulia Rabal Gracia, Juan Roberto Rodríguez Madoz, Edurne San José Enériz
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Publication number: 20170348435Abstract: The invention relates to nucleic acid constructs and gene therapy vectors that comprise an ATP7B variant for use in the treatment of conditions associated with a deficiency or dysfunction of Copper-transporting ATPase 2, and particularly of Wilson's disease. An AAV vector devised according to the invention significantly reduced urine Cu excretion, and liver Cu content in Wilson's disease mice treated with the vector, while ceruloplasmin activity was significantly restored. On the other hand, the administration of the vector resulted in the normalization of serum transaminases' levels and of liver histology, together with a marked reduction of the inflammatory infiltrate.Type: ApplicationFiled: December 17, 2015Publication date: December 7, 2017Applicant: FUNDACIÓN PARA LA INVESTIGACIÓN MÈDICA APLICADAInventors: Oihana MURILLO SAUCA, Gloria GONZÁLEZ ASEGUINOLAZA, Rubén HERNÁNDEZ ALCOCEBA
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Patent number: 9573956Abstract: It relates to certain compounds having a polycyclic structure and a hydroxamic acid moiety, wherein the polycyclic structure comprises at least three ring systems, wherein one ring system is a polycyclic ring system comprising from 2 to 4 rings; at least one ring is an aromatic ring; and wherein the structure comprises at least 3 nitrogen atoms and 1 oxygen atom. It also relates to a process for their preparation, as well as to pharmaceutical compositions containing them, and to their use in medicine, in particular in the treatment and/or prevention of neurological disorders coursing with a cognition deficit or impairment, or neurodegenerative diseases.Type: GrantFiled: February 27, 2014Date of Patent: February 21, 2017Assignee: FUNDACIÓN PARA LA INVESTIGACIÓN MÉDICA APLICADAInventors: María Del Mar Cuadrado Tejedor, Rafael Franco Fernández, Ana María García Osta, Julen Oyarzabal Santamarina, Maria Obdulia Rabal Gracia
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Patent number: 8771664Abstract: The present invention relates to compositions capable of promoting both the innate immune response as well as the adaptive immune response in a subject based on the jointly use of ApoA, interleukin 15 and the Sushi domain of the IL15 receptor alpha chain, as well as to the use of these compositions for the stimulation of the immune response in a patient and to therapeutic methods for the treatment of infectious and neoplastic diseases.Type: GrantFiled: December 10, 2010Date of Patent: July 8, 2014Assignees: Fundacion para la Investigacion Medica Aplicada, Research Center BorstelInventors: Pedro Berraondo Lopez, Jessica Fioravanti, Jose Medina Echeverz, Ignacio Javier Melero Bermejo, Maria del Carmen Ochoa Nieto, Francisco de Asis Palazon Garcia, Silvia Bulfone-Paus, Erwin Hans Duitmann
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Publication number: 20120244118Abstract: The present invention relates to compositions capable of promoting both the innate immune response as well as the adaptive immune response in a subject based on the jointly use of ApoA, interleukin 15 and the Sushi domain of the IL15 receptor alpha chain, as well as to the use of these compositions for the stimulation of the immune response in a patient and to therapeutic methods for the treatment of infectious and neoplastic diseases.Type: ApplicationFiled: December 12, 2010Publication date: September 27, 2012Applicants: FUNDACION PARA LA INVESTIGACION MEDICA APLICADA, RESEARCH CENTER BORSTELInventors: Pedro Berraondo Lopez, Jessica Fioravanti, Jose Medina Echeverz, Ignacio Javier Melero Bermejo, Maria del Carmen Ochoa Nieto, Francisco de Asis Palazon Garcia, Silvia Bulfone-Paus, Erwin Hans Duitmann
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Publication number: 20040224888Abstract: Use of cardiotrophin in liver diseases. The invention describes the increased expression of cardiotrophin (CT-1) during the process of hepatic regeneration coinciding with maximum proliferation of hepatocytes and the role of CT-1 as a stimulator of hepatic regeneration. Furthermore, it describes the hepatoprotective role of CT-1 in various models of acute liver damage.Type: ApplicationFiled: March 11, 2004Publication date: November 11, 2004Applicant: FUNDACION PARA LA INVESTIGACION MEDICA APLICADAInventors: Matilde Bustos De Abajo, Jesus Prieto Valtuena, Juan Jose Lasarte Sagastibelza, Elena Baixeras Llano