Last update 01 Nov 2024

LTGT-06

Last update 01 Nov 2024

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

AAV based gene therapy

Synonyms

Recombinant adeno-associated virus serotype 9(Kunming Hope of Health Hospital), recombinant adeno-associated virus vector serotype-9 carrying the human ATP7A transgene (Lantu Biopharma)

+ [1]

Target

ATP7A

Mechanism

ATP7A modulators(ATPase copper transporting alpha modulators), Gene transference

Therapeutic Areas

Nervous System DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [2]

Active Indication

Menkes Kinky Hair Syndrome

Inactive Indication-

Originator Organization

Lantu Biopharma Inc.

Active Organization

Lantu Biopharma Inc.

Inactive Organization

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China

Drug Highest PhasePreclinical

First Approval Date-

RegulationOrphan Drug (US)

Clinical Trials associated with LTGT-06

NCT05507996 / TerminatedEarly Phase 1IIT

An Investigator-initiated Trial Evaluating the Efficacy and Safety of Recombinant Adeno-associated Virus Administration in Patients With Menkes Syndrome

It is a single-center, open, single-arm, non-randomized investigator-initiated trial evaluating the efficacy and safety of recombinant adeno-associated virus administration for patients with Menkes syndrome.

Start Date18 Aug 2022

Sponsor / Collaborator

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China

100 Clinical Results associated with LTGT-06

100 Translational Medicine associated with LTGT-06

100 Patents (Medical) associated with LTGT-06

Literatures (Medical) associated with LTGT-06

01 Nov 2021·Cell Biology InternationalQ4 · BIOLOGY

MiR‐26a‐5p alleviates cardiac hypertrophy and dysfunction via targeting ADAM17

Q4 · BIOLOGY

Article

Author: Li, Hao ; Shi, Hongtao ; Han, Qinghua ; Zhang, Yanan ; Xue, Honghong ; Zhang, Fan

AbstractCardiac hypertrophy has been a high prevalence rate throughout the world. It has posed a big threat to public health due to limited therapeutic approaches. Previous studies showed that pathological cardiac hypertrophy was associated with autophagy, microRNAs (miRNA), and other signaling pathways, while the molecular mechanisms remain incompletely characterized. In this study, we used thoracic aortic constriction (TAC)‐induced mice and angiotensin‐II (Ang‐II)‐induced H9C2 cell line as cardiac hypertrophy model to investigate the role of miR‐26a‐5p in cardiac hypertrophy. We found that miR‐26a‐5p was downregulated in cardiac hypertrophy mice. Overexpression of miR‐26a‐5p by type 9 recombinant adeno‐associated virus (rAAV9) reversed the heart hypertrophic manifestations. The phenotypes were also promoted by miR‐26a‐5p inhibitor in Ang‐II‐induced H9C2 cells. Through miRNA profile analysis and dual‐luciferase reporter assay, ADAM17 was identified as a direct target of miR‐26a‐5p. Restored expression of ADAM17 disrupted the effect of miR‐26a‐5p on cardiac hypertrophy. To sum up, these results indicated that miR‐26a‐5p played an inhibitory role in cardiac hypertrophy and dysfunction via targeting ADAM17. The miR‐26a‐5p‐ADAM17‐cardiac hypertrophy axis provided special insight and a new molecular mechanism for a better understanding of cardiac hypertrophy disease, as well as the diagnostic and therapeutic practice.

01 Aug 2018·Human Gene Therapy Methods

Systemic Gene Delivery by Single-Dose Intracardiac Administration of scAAV2/9 and scAAV2/rh10 Variants in Newborn Rats

Article

Author: Dehay, Benjamin ; Dovero, Sandra ; Bourdenx, Mathieu ; Chansel-Debordeaux, Lucie ; Canron, Marie-Helene ; Jimenez, Clement ; Bezard, Erwan ; Dutheil, Nathalie

Recombinant adeno-associated virus serotype 9 (rAAV2/9) and pseudotype rhesus-10 (rAAV2/rh10) are used for gene delivery, especially into the central nervous system. Both serotypes cross the blood-brain barrier and mediate stable long-term transduction in dividing and nondividing cells. Among possible routes of administration, intracardiac injection holds the potential for widespread vector diffusion associated with a relatively simple approach. In this study adopting the intracardiac route, we compare the cell-specific tropism and transfection efficacy of a panel of engineered rAAV2/9 and rAAV2/rh10 vectors encoding the enhanced green fluorescent protein. We observed transduction in the brain and peripherally, with a predominant neuronal tropism while the various serotypes achieved different expression patterns.

01 Feb 2018·Journal of Molecular MedicineQ2 · MEDICINE

A20 prevents obesity-induced development of cardiac dysfunction

Q2 · MEDICINE

Article

Author: Wang, Yan ; Huang, Dan ; Liang, Minglu ; Xu, Wenjing ; Wang, Cheng ; Chen, Long ; Huang, Kai ; Fu, Qin ; Zhang, Fengxiao

Obesity and an increased free fatty acid (FFA) level are tightly linked, leading to aberrant oxidative stress, inflammation, apoptosis, and progression to cardiovascular disorders. A20 is a ubiquitin-modifying enzyme that plays a significant role in the negative regulation of inflammatory response. Here, we study the role of A20 in obesity-induced heart injury and explore the underlying mechanisms. A20 expression was first increased in mouse hearts after 4 weeks of a high-fat diet (HFD) and then was gradually decreased in the following 20 weeks. Cardiac-specific supplementation with A20 via recombinant adeno-associated virus subtype 9 (rAAV9) could reverse myocardial dysfunction, hypertrophy and fibrosis in mice exposed to 24 weeks of HFD, along with reduced cardiac apoptosis and inflammation. The beneficial actions of A20 were closely associated with its ability to repress TAK1 activation and the downstream inhibition of P38, JNK1/2, and the NF-κB pathway. TAK1 over-expression could efficiently retard the above-mentioned positive effects of A20. Therefore, our data uncovered a novel function of A20 in obesity-induced heart injury and presented a therapeutic approach for the treatment of obesity-related cardiovascular disorders.KEY MESSAGESA20 expression is downregulated in obesity-related hearts. A20 ameliorates HFD-induced lipid accumulation, ROS, inflammation, apoptosis, hypertrophy, fibrosis, and cardiac dysfunction. A20 represses TAK1 activation and the downstream inhibition of P38, JNK1/2, and the NF-κB pathway. TAK1 overexpression retards the beneficial effects of A20.

100 Deals associated with LTGT-06

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Menkes Kinky Hair Syndrome	Preclinical	CN	920th Hospital of Joint Logistics Support Force of People's Liberation Army of China	18 Aug 2022

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LTGT-06

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