Roche and CombiGene sign an agreement to develop AAV gene therapy for the treatment of drug-resistant epilepsy

Recent popular reports from Yimaike ★Medical ClassApplication and prospects of CAR-T cell technology in the functional cure of AIDS EMedClub PR News/--Recently, Roche's gene therapy company Spark Therapeutics and Swedish gene therapy company CombiGene announced the signing of an exclusive cooperation and licensing agreement for the CG01 project, a project designed to treat drug-resistant focal epilepsy Research-based gene therapy
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According to the signed agreement, CombiGene provides Spark with an exclusive global license to develop, manufacture and commercialize CG01, and will be eligible for $328.
5 million (excluding royalties)
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Among them, an advance payment of 8.
5 million US dollars will be obtained when the agreement is signed, and 50 million US dollars will be obtained after reaching the preclinical and clinical milestones
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After the commercialization of CG01, CombiGene will be eligible to receive tiered royalties ranging from mid-single digits to low double-digits based on net sales
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Joseph La Barge, Chief Commercial Officer of Spark Therapeutics, said: “In terms of treating genetic diseases, Spark is constantly evaluating new opportunities to challenge the status quo.
Cooperation is an important part of our strategy
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” Spark Therapeutics chief scientist Dr.
Federico Mingozzi said: “Although many patients with epilepsy The response to current anti-epileptic treatment is good, but a large number of people need new treatment options
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Through the new cooperation and license agreement with CombiGene, we will jointly use our wealth of knowledge and experience in gene therapy to address the unmet needs of patients with drug-resistant focal epilepsy
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"Based on the research of AAV in the treatment of epilepsy.
Epilepsy is a major global medical disease that affects 0.
6% to 0.
8% of the world’s population
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In 2016, there were 5.
7 million patients with epilepsy diagnosed
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Although many epilepsy patients are currently anti-epileptic The response to treatment is good, but a considerable number of patients need new treatment plans
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It is estimated that about 47,000 new patients with drug-resistant focal epilepsy are added each year in the United States, the European Union 4 countries, the United Kingdom, Japan, and China
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Through this contract with CombiGene A new cooperation and licensing agreement, Spark will use its rich knowledge and experience in gene therapy to jointly solve the unmet medical needs of patients with drug-resistant focal epilepsy
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CG01 is a unique adenovirus-based (AAV) ) Gene therapy is aimed at directly expressing neuropeptide Y (NPY) and its receptor Y2 in the brain where epileptic seizures are initiated
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Previously, CG01 has successfully completed preclinical dose-response studies, proof-of-concept studies for chronic epilepsy, Pharmacokinetic research, learning and memory research, cell tropism research
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A proof-of-concept study of chronic epilepsy: A conceptual trial study of this drug showed that in human epileptic brain tissue (part of the brain that causes seizures), CG01 is successfully absorbed And express NPY and Y2
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Pharmacokinetic study: After using the drug, CG01 has obvious anti-epileptic effects through the use of magnetic resonance and EEG examinations.
At the same time, preclinical pharmacokinetic studies have shown that NPY and Y2 are The expression increased significantly and continued to increase within two weeks until it reached stability in three weeks, and remained stable in the subsequent 6 months of the study.
This result is undoubtedly encouraging
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At the same time, this also suggests that CG01 has the potential to provide expression for several years after being injected into human brain tissue
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The success of pre-clinical conceptual research provides guidance for subsequent research
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Learning and memory research: Does this drug affect people's cognitive function? Data from learning and memory studies show that the active substances NPY and Y2 encoded by CG01 have no obvious negative effects on cognitive function
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Cell tropism study: In the cell tropism study, it was confirmed that CG01 entered the hippocampus nerve cells and expressed completely as expected, but not in the glial cells
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The results of this study further confirmed that CG01 was as low as expected in brain cells
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The drug has shown considerable therapeutic potential in a series of verifications, and we look forward to later research data
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Roche has invested heavily in gene therapy in recent years, and the track of gene therapy has continued to flourish.
Roche is actively deploying gene therapy
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➤ On August 24, 2021, Shape Therapeutics, an RNA editing technology development company, reached a strategic research cooperation with Roche to develop AAV-based RNA editing gene therapy for neurological diseases and rare diseases
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Shape's RNA editing technology is based on research published by Dr.
Prashant Mali in Nature Methods in February 2019
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Studies have shown that using only the modified guide RNA, it can be combined with the natural protease editor (ADAR) and guide it to the RNA sequence, thereby repairing disease-related RNA gene mutations
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Recommended reading: Roche continues to increase gene therapy with US$3 billion, this time betting on RNA editingYimai Meng broke the news ➤ On September 7, 2021, Roche subsidiary Spark Therapeutics and NeuExcell Therapeutics announced a gene therapy cooperation agreement that will use Spark advanced AAV platform and NeuExcell nerve regeneration gene therapy platform to develop new gene therapy for Huntington's disease (HD)
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NeuExcell Therapeutics' technology is a new method of regenerating nerve tissue
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Its nerve regeneration gene therapy platform is based on transcription factor-mediated transdifferentiation technology, which aims to directly transdifferentiate endogenous glial cells into functional new neurons in situ
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Recommended reading: Regenerating new neurons! Roche and Xinrui reached a new gene therapy cooperationYimai Meng broke the news reference materials: 1.
https:// agreement-for-gene-therapy-candidate-cg01/