Last update 21 Nov 2024

Recombinant arylsulfatase A(Linno Pharmaceuticals)

Last update 21 Nov 2024

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Enzyme

Synonyms

ARSA, Arylsulfatase A(Linno Pharmaceuticals), Recombinant fusion protein composed of a recombinant human arylsulfatase A (rhARSA) fused with a variable domain of the heavy chain of heavy chain-only antibody fragment targeting human transferrin

+ [3]

Target

ASA

Mechanism

ASA replacements(Cerebroside sulfatase replacements)

Therapeutic Areas

Nervous System DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [1]

Active Indication

Leukodystrophy, Metachromatic

Inactive Indication-

Originator Organization

Linno Pharmaceuticals Inc.

Active Organization

Linno Pharmaceuticals Inc.

Inactive Organization-

Drug Highest PhasePreclinical

First Approval Date-

RegulationOrphan Drug (US)

100 Clinical Results associated with Recombinant arylsulfatase A(Linno Pharmaceuticals)

100 Translational Medicine associated with Recombinant arylsulfatase A(Linno Pharmaceuticals)

100 Patents (Medical) associated with Recombinant arylsulfatase A(Linno Pharmaceuticals)

Literatures (Medical) associated with Recombinant arylsulfatase A(Linno Pharmaceuticals)

01 Oct 2024·Journal of Clinical Ultrasound

Aberrant right subclavian artery in the absence of other prenatal ultrasound findings: Should we still be concerned?

Article

Author: Ayhan, Işıl ; Demirci, Oya ; Karaman, Ali ; Odacılar, Ali Şahap

AbstractObjectiveTo analyze the value of prenatal diagnostic genetic testing in cases with isolated aberrant right subclavian artery (ARSA).MethodsThis is a retrospective cohort study, conducted between January 2015–January 2022 in a fetal medicine center. Women who had an ultrasound scan and diagnosed with fetal ARSA were included. Ultrasonographic characteristics, genetic, obstetric, and neonatal outcomes were collected and analyzed.ResultsA total of 240 fetuses with ARSA were identified and included to the analysis. Eighty‐two of the group had isolated ARSA (34.2%, 82/240), 57 had additional soft markers (23.8%, 57/240) and 101 had additional major ultrasonographic abnormalities (42.1%, 101/240). Genetic results were available in 196 cases (81.7%, 196/240). Seventy‐four of isolated ARSA cases underwent genetic testing (90.2%, 74/82). A chromosomal abnormality was present in 60 cases; 54 (22.5%, 54/240) aneuploidies and 6 (2.5%, 6/240) copy number variants. Five (6.1%) of the isolated ARSA cases had chromosomal abnormalities. All of these five cases had prenatal genetic testing due to high‐risk aneuploidy screening fetuses who had ARSA with at least one additional anomaly had the highest chromosomal abnormality rate (38.6%, 39/101). Seventy‐seven of isolated ARSA cases were liveborn (93.9%, 77/82).ConclusionOur results supports the evidence from the literature that isolated ARSA confers a very low‐risk for aneuploidy, if the aneuploidy screening tests are low‐risk. Also, chromosomal microarray analysis did not yield any extra information in isolated ARSA.

26 Mar 2024·Blood Advances

Allogeneic hematopoietic cell transplantation for adult metachromatic leukodystrophy: a case series

Article

Author: Reuss, Kristina ; Riedel, Andreas ; Weissert, Nadine ; Lang, Peter J. ; Faul, Christoph ; Bethge, Wolfgang A. ; Hengel, Holger ; Schoels, Ludger ; Gröschel, Samuel ; Schröder, Jan C. ; Lengerke, Claudia

AbstractMetachromatic leukodystrophy (MLD) is a rare genetic disorder caused by pathogenic variants of the ARSA gene, leading to a deficiency of the arylsulfatase A enzyme (ARSA) and consecutive accumulation of galactosylceramide-3-0-sulfate in the nervous system. The condition leads to severe neurological deficits and subsequently results in profound intellectual and motoric disability. Especially, the adult form of MLD, which occurs in individuals aged >16 years, poses significant challenges for treating physicians because of the rarity of cases, limited therapeutic options, and different allogeneic hematopoietic cell transplantation (allo-HCT) protocols worldwide. Here, we report the results of allo-HCT treatment in 4 patients with a confirmed adult MLD diagnosis. Bone marrow or mobilized peripheral progenitor cells were infused after a reduced intensity conditioning regime consisting of fludarabine and treosulfan. In 3 patients, allo-HCT was followed by an infusion of mesenchymal cells to further consolidate ARSA production. We observed a good tolerability and an increase in ARSA levels up to normal range values in all patients. A full donor chimerism was detected in 3 patients within the first 12 months. In a 1-year follow-up, patients with complete donor chimerism showed a neurological stable condition. Only 1 patient with an increasing autologous chimerism showed neurological deterioration and a decline in ARSA levels in the first year. In summary, allo-HCT offers a therapeutic option for reconstituting ARSA enzyme levels in adult patients with MLD, with tolerable side effects.

24 May 2023·International journal of molecular sciences

Safety and Efficacy of Intravenous and Intrathecal Delivery of AAV9-Mediated ARSA in Minipigs.

Article

Author: Rizvanov, Albert ; Chulpanova, Daria ; Issa, Shaza ; Mukhamedshina, Yana ; Mullagulova, Aysilu ; Solovyeva, Valeriya ; Shaimardanova, Alisa ; Kostennikov, Alexander

Metachromatic leukodystrophy (MLD) is a hereditary neurodegenerative disease characterized by demyelination and motor and cognitive impairments due to deficiencies of the lysosomal enzyme arylsulfatase A (ARSA) or the saposin B activator protein (SapB). Current treatments are limited; however, gene therapy using adeno-associated virus (AAV) vectors for ARSA delivery has shown promising results. The main challenges for MLD gene therapy include optimizing the AAV dosage, selecting the most effective serotype, and determining the best route of administration for ARSA delivery into the central nervous system. This study aims to evaluate the safety and efficacy of AAV serotype 9 encoding ARSA (AAV9-ARSA) gene therapy when administered intravenously or intrathecally in minipigs, a large animal model with anatomical and physiological similarities to humans. By comparing these two administration methods, this study contributes to the understanding of how to improve the effectiveness of MLD gene therapy and offers valuable insights for future clinical applications.

100 Deals associated with Recombinant arylsulfatase A(Linno Pharmaceuticals)

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Leukodystrophy, Metachromatic	Preclinical	CN	Linno Pharmaceuticals Inc.	-

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Recombinant arylsulfatase A(Linno Pharmaceuticals)

Overview

Basic Info

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R&D Status

Clinical Result

Translational Medicine

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Core Patent

Clinical Trial

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