{"id":1072,"date":"2020-11-12T10:47:58","date_gmt":"2020-11-12T09:47:58","guid":{"rendered":"https:\/\/nervosavetx.com\/?page_id=1072"},"modified":"2024-03-12T18:17:18","modified_gmt":"2024-03-12T17:17:18","slug":"science","status":"publish","type":"page","link":"https:\/\/nervosavetx.com\/fr\/science\/","title":{"rendered":"Notre science"},"content":{"rendered":"
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OUR gene therapy platform<\/h2>\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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Unique Gene Therapy Platform<\/h2>\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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We developped an\u00a0gene therapy platform<\/strong> specific for peripheral nerves<\/strong>. AAV9 vectors locally delivered in nerves allows for the huge and<\/strong> specific transduction of myelinating Schwann cells in virtually any nerve<\/strong>. As these cells are not dividing and remain unchanged for very long, AAV also allows for very long term (>10 years expected) expression of a therapeutic biologic<\/strong> in nerves. This biologic includes peptides, polypeptides, mRNA, siRNA or CrisprCas9<\/strong>.\u00a0 Virtually any cellular and molecular defect occuring in myelinating Schwann cells can be corrected using this technology. \u00a0<\/p>\t\t\t\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t

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Novel validated delivery route for nerves<\/h2>\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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We adapted the standard delivery route for regional anesthesia<\/strong> to a novel gene therapt delivery route. Perineural injection<\/strong> currently used by anesthesiologists for local anesthesia is the perfect route for a safe and efficient local gene therapy<\/strong> in nerves. Therapeutic AAV vectors injected locally in nerves of legs and arms<\/strong> allows for motor and sensory nerve treatments in neuromuscular diseases<\/strong>. Local chronic or recurrent pain<\/strong> can be addressed by the local treatment of the relevant nerves<\/strong>.<\/p>\t\t\t\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t

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Lead program: Preventing CMT1A disease (>68,000 patients)\n<\/h2>\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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We primarily target patients close to the onset of the disease<\/strong>, as gene therapy is particularly efficient on this segment. The secondary target includes children patients at the onset also and all adult patients with mild symptoms to slow the disease down and to reverse symptoms thank to the regenerative capacities of peripheral nerves.<\/p>

Nervosave\u2019s unique local therapy is first-in-class for CMT pathology<\/strong> and provides the largest and most persistent benefits in the rat model of the disease. AAV vector\u00a0and local injection technologies are de-risked by FDA-approved AAV-based gene therapies <\/strong>and standard local anesthesia techniques<\/strong>.\u00a0<\/p>\t\t\t\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t

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Donn\u00e9es pr\u00e9cliniques<\/h2>\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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Nervosave\u2019s current preclinical data show that AAV\u00a0readily and specifically transduce target cells following a local injection in nerves (Gautier et al, 2021)<\/strong>. Vector biodistribution is mostly restricted to injected nerves. Studies also show very limited immune response. The treatment corrects molecular and cellular defects, maintains fast nerve conduction velocity (NCV) and prevents motor and sensory\u00a0symptoms in rats suffering from CMT1A<\/strong> (Gautier et al, 2021).<\/strong><\/p>

The safety and molecular efficacy have been confirmed \u00a0in monkeys<\/strong>. Translatability is being investigated in the sheep model<\/strong>, which has nerves\u00a0 similar to human nerves. Nervosave is actively working on the development of novel biomarkers<\/strong> to optimize clinical trials efficacy endpoints<\/p>\t\t\t\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t

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Une th\u00e9rapie g\u00e9nique de nouvelle g\u00e9n\u00e9ration pour les maladies CMT<\/h2>\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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Nervosave Therapeutics d\u00e9veloppe une nouvelle th\u00e9rapie g\u00e9nique bas\u00e9e sur l\u2019AAV pour l'ensemble des maladies CMT d\u00e9my\u00e9linisantes. La technologie exploite la d\u00e9couverte des m\u00e9canismes mol\u00e9culaires pr\u00e9coces se produisant lors de la d\u00e9g\u00e9n\u00e9rescence de la gaine de my\u00e9line. La th\u00e9rapie g\u00e9nique NVO-301 de Nervosave Therapeutics bloque la d\u00e9my\u00e9linisation<\/strong> dans les cellules de Schwann pour pr\u00e9venir la d\u00e9g\u00e9n\u00e9rescence de la gaine de my\u00e9line. Cette technologie a une application potentielle dans toutes les maladies de Charcot-Marie-Tooth d\u00e9my\u00e9linisantes, m\u00eame les plus rares.<\/p>\t\t\t\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t

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Compos\u00e9s anti-d\u00e9my\u00e9linisants r\u00e9volutionnaires pour pr\u00e9venir la d\u00e9my\u00e9linisation des nerfs p\u00e9riph\u00e9riques dans le DPN et le SGB<\/h2>\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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NVX-401 est la solution de Nervosave Therapeutics pour lutter contre la neuropathie p\u00e9riph\u00e9rique diab\u00e9tique et le syndrome de Guillain-Barr\u00e9.<\/strong> Cette solution \u00e0 base de peptides<\/strong> est id\u00e9ale pour traiter les maladies d\u00e9my\u00e9linisantes non g\u00e9n\u00e9tiques gr\u00e2ce \u00e0 une administration syst\u00e9mique locale, rapide et contr\u00f4l\u00e9e dans le temps.<\/p>

Cette approche syst\u00e9mique pour les neuropathies p\u00e9riph\u00e9riques communes est le compl\u00e9ment parfait \u00e0 la th\u00e9rapie g\u00e9nique locale que Nervosave Therapeutics d\u00e9veloppe pour les maladies g\u00e9n\u00e9tiques.<\/p>\t\t\t\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t