Duke and Seelos Therapeutics to Work on Parkinson’s Disease Gene Therapy

Seelos Therapeutics and Duke University have both signed a Sponsored Research Agreement, meaning they will work in tandem to evaluate and test SLS-004, a Parkinson’s disease treatment. Their research aims to test the safety and efficacy of the gene therapy, as well as study the LV-dCas9-DNMT3A virus’ ability to delay or even prevent Parkinson’s.

About Parkinson’s Disease (PD)

Parkinson’s disease is a progressive disorder that affects the central nervous system (CNS). It is characterized by its effect on movement through five different stages. As the disease progresses, severity increases.

  1. Stage one is characterized by subtle tremors on one side of the body.
  2. In stage two symptoms are more noticeable, with tremors and rigidity on both sides of the body.
  3. Stage three brings loss of balance and slowed movement.
  4. Stage four makes it impossible for one to live independently.
  5. Stage five is the most severe, as patients cannot stand or walk. Hallucinations and delusions are common symptoms of this stage.

Parkinson’s disease occurs due to the death of motor neurons, some of which produce dopamine. Dopamine is important in the transmittance of messages to the muscles from the brain, so the loss of dopamine results in the loss of motor functions. Abnormal brain activity occurs when these neurons are lost. Doctors do not know why these motor neurons die, but they do suspect a few factors that play a role, such as genetics, environmental factors like toxins, and Lewy bodies.

About the Agreement

Duke and Seelos will work together on a study of the safety and efficacy of SLS-004. They will do so through the use of mouse and other pre-clinical models.

Not only will SLS-004 be evaluated, but the two organizations will study a vector that is intended to suppress or even prevent Parkinson’s phenotypes. This virus, LV-dCas9-DNMT3A, has a unit that overexpresses and a unit that suppresses alpha-synuclein. This makes it able to inhibit the symptoms of Parkinson’s. If it finds success in trials it could offer a new treatment option for many PD patients.

About SLS-004

SLS-004 is a gene therapy that is intended to change the expression of the SNCA gene through modifying DNA-methylation. The changes that it makes will stop the overexpression of the gene by 30% in hiPSC-derived dopaminergic neurons.

This gene therapy, along with the new vector, have the chance to better the lives of many if they are successful in their trials.

Find the source article here.

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