Clinical applications for bio-eye gene therapy in Newforth were accepted

Source: Pharmaceutical Mission, January 19, China's State Drug Administration Drug Review Center (CDE) announced that Newforth Bio-Ophthalmology Gene Therapy NR082 Eye Injection (NFS-01 Project) was declared clinical trials and accepted.
, NR082 was previously qualified by the FDA for the treatment of Leber's hereditary optic neuropathy (LHON) caused by the ND4 mutation.
screenshot source: NR082 is the first drug candidate developed by Newforth Bio, after completing clinical trials of the global large sample LHON gene therapy.
In this project, scientists used recombinated adeno-related viruses as vectors to deliver the correct human ND4 gene through glass cavity injections to the patient's damaged optic nerve cells, repair mitochondrial biorespiration chains, and re-in viable and visual function of optic nerve cells.
as early as 2011, Newforth Bio launched a clinical trial of experimental NR082 gene therapy in China.
results showed that in 9 patients with ND4-bit mutation LHON who participated in the trial, 7 patients had significantly improved their vision, with the optimal vision restored to 0.8 and the average optimal corrective vision improvement of 0.39logMAR (i.e., an improvement close to the four lines of the alphabet).
8 patients completed nearly 8 years of long-term follow-up, no serious adverse reactions.
based on positive results from previous studies, NR082 completed international gene therapy clinical trials in China and Argentina, including 159 LHON patients, between 2017 and 2018.
According to an earlier press release issued by Newforth Bio, gene therapy is "greatly developed" in the field of eye disease treatment because of the particularity of the eye: the eye is a closed sphere, the drug only injects local eyeballs, the effect on the whole body is small, suitable for gene therapy.
addition, there are more than 220 ophthalmology-related genes, and any one gene mutation can cause vision loss and blindness.
practice has proved that gene therapy, which uses safe viral vectors to deliver nucleic acid drugs to correct genetic abnormalities or express normal gene products, is one of the promising strategies for treating blind eye diseases.
LHON is a rare maternal genetic mitochondrial genetic disease, manifested in retinal neurocellular degeneration, leading to irreversible vision loss and even blindness.
genetic sequencing tests have found that the disease is usually caused by mutations in mitochondrial DNA 11778G-A site within cells, which are caused by NADH dehydrogenase sub-base-4 (ND4).
the mutation caused damage to the mitochondrial biological respiratory chain, resulting in ATP deficiency, causing oxidative stress in the patient's optic nerve cells, leading to apoptosis.
, there is no effective treatment or cure for LHON clinically, and the clinical unfinished need is obvious.
that the clinical development of Newforth BioNR082 in China is progressing smoothly and that innovative therapies will be brought to patients at an early time.
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