Nervosave’s Gene Therapy
We developped an AAV-based gene therapy to allow correct myelination and long term maintenance of the myelin sheath. In order to prevent and correct demyelinating CMT diseases main symptoms, the therapeutic vector is injected locally in nerves of legs and arms through techniques established for regional anesthesia. Gene therapy is a safe and life-long cure.
Preventing CMT1A disease in affected young patients
We primarily target patients at the onset of the disease, as Nervosave’s 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 therapy is first-in-class for the 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 successful existing gene therapies and local anesthesia techniques.
Current preclinical data
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 of both technologies has been investigated in monkeys successfully.
Nervosave is actively working on the development of novel biomarkers to optimize clinical trials efficacy endpoints
Next generation gene therapy for CMT diseases
Nervosave develops a novel AAV-based gene therapy for demyelinating CMT diseases. The technology exploits the discovery of early molecular events occuring during myelin sheath degeneration. Nervosave’s NVO-301 gene therapy blocks Schwann cell demyelination to prevent myelin sheath degeneration in all demyelinating Charcot-Marie-Tooth diseases.
Groundbreaking anti-demyelinating compounds to prevent peripheral nerve demyelination in DPN and GBS
NVX-401 is Nervosave’s solution to fight Diabetic Peripheral Neuropathy and Guillain-Barré syndrome. This peptide-based solution is ideal to treat non-genetic demyelinating diseases through a quick and timely controlled systemic delivery.
This systemic approach for general diseases is the perfect add-on to the local-delivery gene therapy that Nervosave develops for genetic diseases.