GM1 gangliosidosis is a rare and fatal pediatric disease characterized by the toxic buildup of GM1 gangliosides due to a defect in the GLB1 gene leading to impaired β-galactosidase (β-gal) enzyme activity. This buildup of gangliosides results in neurodegeneration, cognitive impairment, paralysis and life expectancy shortened to two to four years of age in the severe forms.

GM1 Gangliosidosis

AXO-AAV-GM1

AXO-AAV-GM1 is an investigational gene therapy for GM1 gangliosidosis that aims to restore β-galactosidase (β-gal) function by introducing a functional copy of the GLB1 gene through an adeno-associated viral (AAV) vector

ROA

AXO-AAV-GM1 delivers GLB1 intravenously with the goal of treating systemic manifestations of the disease, including bone and cardiac abnormalities. The IV administration is easier on young patients compared to alternate routes of administration

Stage

We are currently enrolling patients in our Safety & Efficacy Part A study of our Phase 1/2 clinical trial. We expect to enroll ~5 patients by Q1 2020 with data expected in Mid-2020. Learn more at https://clinicaltrials.gov/ct2/show/NCT03952637 

Tay-Sachs and Sandhoff diseases are a set of rare and fatal pediatric neurodegenerative genetic disorders caused by defects in the HEXA (leading to Tay-Sachs disease) and HEXB (leading to Sandhoff disease) genes that encode the two subunits of the β-hexosaminidase A (Hex A) enzyme. These genetic defects lead to the toxic accumulation of gangliosides, resulting in neurodegeneration and life expectancy shortened to two to four years of age.

Tay-Sachs and Sandhoff Diseases

AXO-AAV-GM2

AXO-AAV-GM2 is an investigational gene therapy for GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff diseases) that aims to restore β-hexosaminidase A (Hex A) function by introducing a functional copy of the HEXA and HEXB genes via two, co-administered AAVrh8 vectors

ROA

AXO-AAV-GM2 is administered via dual intrathalamic and intrathecal routes which allow for transduction across the entire neuraxis. This is a key determinant of efficacy in animal models across affected tissues including the spinal cord and brain. 

Stage

Once we have an IND filed and approved by the FDA in 2H 2019, we will begin enrolling ~7 patients in our Dose Ranging Part A study of our Phase 1/2 clinical trial

Parkinson's disease is characterized by multiple symptoms such as muscle rigidity, slowing of movement, and resting tremor. It is a progressive neurodegenerative disorder resulting from decreased levels of dopamine in the brain region responsible for movement and other functions. Parkinson’s disease affects approximately 7-10 million patients worldwide.​

Parkinson's Disease

AXO-Lenti-PD

AXO-Lenti-PD is an investigational gene therapy for Parkinson’s disease that delivers three genes via a single lentiviral vector to encode a set of critical enzymes required for dopamine synthesis, with the goal of reducing variability and restoring steady levels of dopamine in the brain. The gene therapy aims to provide patient benefit for years following a single administration.

ROA

AXO-Lenti-PD is a one-time gene therapy delivered directly into the putamen, a neurosurgical procedure that does not require the use of intraoperative MRI

Stage

We are currently completing dosing of 4-6 patients in Cohort 2 of our Dose Escalation Part A of our SUNRISE-PD Phase 2 clinical trial. We expect to provide 3-month data from the second dose cohort in Q4 2019

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