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Novel Gene Therapy Tested on First Human

Novel Gene Therapy Tested on First Human

Axovant Sciences, a Swiss pharmaceutical company, announced it has initiated treatment for the first human subject in a clinical study of AXO-Lenti-PD. The therapy, also known as OXB-102, is an investigational gene therapy that is believed to enable the expression of three critical enzymes required for making dopamine in the brain.1

People with Parkinson’s disease (PD) lose the ability to produce dopamine due to the damage or death of neurons in the substantia nigra area of the brain. Among the significant affects are motor symptoms including tremor, rigidity, and bradykinesia (slowness of movement).2 The new clinical trial is examining the ability of AXO-Lenti-PD to facilitate end-to-end synthesis of dopamine production.1

Axovant is working on developing innovative gene therapies for neurologic and neuromuscular diseases. Axovant is considering additional gene therapy assets for drugs that are ready for clinical studies or active in clinical stages in the coming year.3

What is gene therapy?

Gene therapy is a scientific approach to modifying the genetic material of living cells for therapeutic purposes. It works by inserting functional genes, enzymes, or other molecules that contain the required information sequence into specific cells with instructions to result in a change that has therapeutic effect. The gene acts as a drug.2,4

AXO-Lenti-PD therapy works by delivering three genes in vivo through a lentiviral vector. Lentiviral vectors in gene therapy are the method by which genes are inserted, modified, or deleted in organisms using a lentivirus. The lentivirus is primarily a research tool used to invade the targeted cells and change their expression for up to six months.5 It encodes these critical enzymes required for making dopamine synthesis in the brain. Preliminary research suggests that people receiving the treatment will benefit for many years following a single administration.

Study information

There are challenges to treating Parkinson’s, especially as the disease progresses. There are limited therapeutic options, especially for those who experience significant motor fluctuations. This clinical study will evaluate the safety and tolerability of AXO-Lenti-PD in people with PD. Information collected will include efficacy data that looks at measures of motor function in patients with Parkinson’s disease.

This trial expands upon the long-term safety and efficacy results from the phase I/II clinical trial of ProSavin®. That drug is a first-generation product from Oxford BioMedica that increases dopamine production. Axovant licensed this drug from Oxford BioMedica in June 2018.3

The new formulation modifies the payload configuration.1 Preclinical studies, which directly compared AXO-Lenti-PD to ProSavin, observed sustained benefits in patients for up to six years even though Parkinson’s is a progressive degenerative condition.

It is administered into the striatum, an area of the brain where dopamine is released in healthy people, by cells stemming from the substantia nigra. It is surgically accessible making gene targeting a feasible approach without disrupting other brain regions.2

The first study participant was treated at the Clinical Research Facility affiliated with the National Institute for Health Research (NIHR) and University College London Hospitals (UCLH). A woman in her 50s, with levodopa-responsive PD, she was diagnosed over 10 years ago. The treatment delivered the gene therapy on Wednesday, October 17, 2018.2

The initial report reveals that she experienced no surgical complications or side effects from the administration of the vector. She was discharged and able to return home on schedule.1 Axovant intends to study up to 30 people in this first trial and to report initial data in first half of 2019.1,2 Timing of new study participants is dependent on the Data Monitoring Committee (DMC), which requires a waiting period of 28 days to evaluate the initial process. After a second participant is dosed, Axovant will have to wait for another 28 days before determining if they can continue to dose additional participants.3

Future of AXO-Lenti-PD

Researchers believe genes that can increase the production of dopamine reduce the motor symptoms of Parkinson’s disease. This approach could have fewer side effects than traditional drug treatments by concentrating on the areas of the dopamine-depleted areas of the brain. The objective is to provide long term benefits for years following a single treatment.2,3

  1. Axovant Announces Dosing of First Patient in Clinical Study of AXO-Lenti-PD, a Novel Gene Therapy for Patients With Parkinson’s Disease. Available at: Accessed 10.28.18.
  2. Innovative gene therapy trial for Parkinson’s disease. Available at: Accessed on 10.28.18
  3. Lakusta, G. Axovant Doses First Patient in Parkinson’s Trial. Available at: Accessed 10.28.18.
  4. What is Gene Therapy? What are Lentiviral Vectors? Accessed 10.28.18.
  5. Lentiviral Vectors. Available at: Accessed 10.28.18.