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.
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 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
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.