Neurophoenix is a preclinical stage biotech company advancing its proprietary PTEN inactivation platform to unlock neuron repair in optic neuropathies and several other neuronal diseases. The Neurophoenix platform is based on the evolution-optimized mechanism from Rabies virus to unlock neuron repair. The well characterized molecular mechanism is based on the highly specific disruption of the protein-protein interaction between the so far “undruggable” neuron survival inhibitor PTEN and the kinase MAST2. Proprietary synthetic biology solutions permit to deliver the active amino-acid sequence through different modalities including engineered cell-penetrating molecules as well as gene therapy approaches. This allows to restore neuron repair in optic neuropathies such as non-arteritic anterior ischemic optic neuropathy (NAION) and Glaucoma, as well as motoneuron disease and other neuronal pathologies. The lead asset is a polypeptide of less than 150 amino-acids able to protect and repair degenerating neurons of the optical nerve in acute and chronic conditions. Animal POC has been established and the program is poised to enter the first clinical trial in 2024, with a proof of concept read-out expected in 2026 in NAION disease. NAION is an orphan disease with an incidence of 2.5-10 / 100'000 among individuals over 50, leading to partial or total vision loss in one eye in 60-70% of patients. Currently there is no approved treatment for NAION. In parallel, Neurophoenix is also working on an in vivo proof of concept of an AAV-based gene therapy for neurodegenerative conditions, starting with amyotrophic lateral sclerosis (ALS). In vivo POC is expected in Q4 2022.