Continuously improve patient symptoms Parkinson's gene therapy long-term clinical positive

On September 11th Neurocrine Biosciences and Voyager Therapeutics jointly announced that the gene therapy NBIb-1817 (VY-AADC), developed jointly by the two companies, has shown positive long-term results in Phase 1b clinical trials for patients with Parkinson's disease.
of the 15 patients with advanced Parkinson's disease who received one gene therapy treatment, 14 still experienced improvements in disease classification after three years of treatment.
NBIb-1817 not only continuously improves the patient's motor function, but also reduces the patient's need for oral drugs.
the first patient to be given the gene therapy, which has completed a critical clinical trial in 2018, but patient registrations for this critical clinical trial were temporarily suspended due to the impact of the new crown outbreak.
clinical trial will restart patient registration later this year, according to a press release.
is the second most common neurodegenerative disease after Alzheimer's disease.
the Parkinson's Foundation estimates that there are more than 10 million patients worldwide.
the disease is caused by the absence of neurons that produce the neurotransmitter dopamine, substantia nigra.
currently, L-Doba remains the standard treatment for the disease.
Although lysodabba significantly improves the patient's motor symptoms in the early stages of the disease, as the disease progresses, the patient's response to treatment becomes worse and sometimes medication does not work optimally, a period known as the shutdown period ("off" time).
even during the open period ("on" time) during which the drug has an effect, patients may develop dyskinesia (dyskinesia) characterized by involuntary abnormal muscle movements.
Therapeutics is a biotechnology company dedicated to developing gene therapies to treat neurological diseases.
NBIb-1817 is a genetically modified gene therapy that uses AAV2 virus vectors to carry the expression of the human aromatic L-amino acid dehydrase (AADC).
the characteristics of Parkinson's disease, in addition to the death of black neurons that synthesize dopamine, the level of AADC required to synthesize dopamine in the brain is also declining.
NBIb-1817 is designed to help improve the patient's motor symptoms by expressing AADC enzymes in brain cells, helping to convert lysodab into dopamine.
this innovative gene therapy has been recognized by the FDA as an advanced therapy for regenerative medicine.
Biosciences, a company, called Newerocrine Biosciences, in January 2019 agreed to a $1.7 billion partnership with Voyager to develop a number of gene therapies, including NBIb-1817. the goal of
NBIb-1817 gene therapy is to "turn back the clock" and restore symptoms in patients with advanced Parkinson's disease to the level of early patients (Photo Source: Reference 3)) In a Phase 1b open-label clinical trial called PD-1101, 15 patients with late-stage Parkinson's disease received a treatment of NBIb-1817 (divided into three groups and treated with different doses of gene).
results released by the New York Times showed that after three years of treatment, the patient's closing period decreased by an average of 0.15-1.91 hours (baseline 4.28-4.93 hours).
the length of the open period without movement disorders increased by an average of 0.26-2.23 hours (baseline 10.32-10.46 hours).
, the demand for oral drugs was reduced in patients receiving higher doses of gene therapy in Queue 2 and Queue 3 patients.
the average daily oral dose of L-Doba was reduced by 322.0 and 441.2 mg/day (baselines 1507.0 and 1477.0 mg/day).
of motor function in patients assessed by clinicians also improved continuously after 3 years.
"The results are very encouraging because the motor function of people with Parkinson's disease usually declines over a three-year period," said lead researcher Dr. Chad Christine, a professor of neurology at the University of California, San Francisco.
" References: . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Retrieved September 12, 2020, from Voyager Therapeutics 2018 Annual Report. Retrieved September 12, 2020, from Voyager Therapeutics 10K form. Retrieved September 12, 2020, from original title: One treatment, 3 years after the continuous improvement of patient symptoms, Parkinson's disease gene therapy long-term clinical results positive attention to the "Drug Mingkang" WeChat public number