OUR gene therapy platform
Unique Gene Therapy Platform
We developped an gene therapy platform specific for peripheral nerves. AAV9 vectors locally delivered in nerves allows for the huge and specific transduction of myelinating Schwann cells in virtually any nerve. 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 in nerves. This biologic includes peptides, polypeptides, mRNA, siRNA or CrisprCas9. Virtually any cellular and molecular defect occuring in myelinating Schwann cells can be corrected using this technology.
Novel validated delivery route for nerves
We adapted the standard delivery route for regional anesthesia to a novel gene therapt delivery route. Perineural injection currently used by anesthesiologists for local anesthesia is the perfect route for a safe and efficient local gene therapy in nerves. Therapeutic AAV vectors injected locally in nerves of legs and arms allows for motor and sensory nerve treatments in neuromuscular diseases. Local chronic or recurrent pain can be addressed by the local treatment of the relevant nerves.
Lead program: Preventing CMT1A disease (>68,000 patients)
We primarily target patients close to the onset of the disease, 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.
Nervosave’s unique local therapy is first-in-class for CMT pathology and provides the largest and most persistent benefits in the rat model of the disease. AAV vector and local injection technologies are de-risked by FDA-approved AAV-based gene therapies and standard local anesthesia techniques.
Données précliniques
Nervosave’s current preclinical data show that AAV readily and specifically transduce target cells following a local injection in nerves (Gautier et al, 2021). 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 symptoms in rats suffering from CMT1A (Gautier et al, 2021).
The safety and molecular efficacy have been confirmed in monkeys. Translatability is being investigated in the sheep model, which has nerves similar to human nerves. Nervosave is actively working on the development of novel biomarkers to optimize clinical trials efficacy endpoints
Une thérapie génique de nouvelle génération pour les maladies CMT
Nervosave Therapeutics développe une nouvelle thérapie génique basée sur l’AAV pour l'ensemble des maladies CMT démyélinisantes. La technologie exploite la découverte des mécanismes moléculaires précoces se produisant lors de la dégénérescence de la gaine de myéline. La thérapie génique NVO-301 de Nervosave Therapeutics bloque la démyélinisation dans les cellules de Schwann pour prévenir la dégénérescence de la gaine de myéline. Cette technologie a une application potentielle dans toutes les maladies de Charcot-Marie-Tooth démyélinisantes, même les plus rares.
Composés anti-démyélinisants révolutionnaires pour prévenir la démyélinisation des nerfs périphériques dans le DPN et le SGB
NVX-401 est la solution de Nervosave Therapeutics pour lutter contre la neuropathie périphérique diabétique et le syndrome de Guillain-Barré. Cette solution à base de peptides est idéale pour traiter les maladies démyélinisantes non génétiques grâce à une administration systémique locale, rapide et contrôlée dans le temps.
Cette approche systémique pour les neuropathies périphériques communes est le complément parfait à la thérapie génique locale que Nervosave Therapeutics développe pour les maladies génétiques.