Gene therapy is subject to hand, Chinese scholars have a clinical trial of LHON gene treatment in ophthalmology

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Gene therapy is subject to hand, Chinese scholars have a clinical trial of LHON gene treatment in ophthalmology

2021-11-26 06:06:58 4 ℃

LHON is a matrix genetic mitochondrial disease, and patients are manifested as a double visual acuity. This disease was first found in 1871 in 1871 in 1871 in 1871. At present, there are approximately 6.75 million in global patients, including 126,000 in China. By gene sequencing detection, this disease is typically caused by mitochondrial DNA 11778 g> a bit mutation in the cell, because NADH dehydrogenase subunit-4 (ND4) is found. Since the mutation causes the mitochondrial biopsy chain to damage the mitochondrial biosphere, the lack of ATP, causing the patient to produce oxidative stress, resulting in apoptosis. At present, there is no effective therapy or cure means for LeBer's hereditary optic neuropathy.

In many people's impression, gene therapy is usually associated with cancer, rare diseases, etc. In fact, gene therapy has been "big and color" in the field of contemporary ophthalmology diseases, and it is expected to improve their vision and even see the light. On May 15, 2020, Boston, Massachusetts, Today's 23rd American Genes and Cell Treatment (ASGCT) WORDT WORK INFORMATION New Fusos Biotechnology Co., Ltd. (hereinafter referred to as "Newfus") (NFS-01, RAAV2-ND4) was released (NFS-01, RAAV2-ND4) in the treatment of LEBER's hereditary optic neuropathy (English abbreviation LHON) Clinical research data, the result is exciting.

In 2011, in Newfos, the leader of Professor Li Bin, who was the Ophthalmology of Tongji Medical College, Huazhong University of Science and Technology, and the world's first experimental NFS-01 (RAAV2-ND4) gene treatment for Leber's genetic optic neuropathy. Clinical Test (NCT01267422) launched in China. Scientists use recombinant adenophobia as carriers to deliver the correct human ND4 gene in injection to the patient's impaired optic gondroduce cells, repair the mitochondrial biosphere chain, to restore vitality and visual function of the primary cells. Among the 9 cases of G1178A (ND4) site mutation LHON patients, the visual acuity of 7 patients was significantly improved, and the best vision recovered to 0.8, and 9 patients had an average correct correction of 0.39Logmar (ie close alphabet four). Row improved). Eight patients completed the long-term follow-up of nearly 8 years, no serious adverse reactions, and six patient vision improvements have been maintained. This clinical research results were published on the "Ophthalmology" magazine in February 2020.

Professor Li Bin explained that the reason why gene therapy was "big exhibition" in the field of eye disease treatment, the reason for the eyes of the eye: the eyes are sealed spheres, drugs are only injected to partially eyeballs, which have little effect on the whole body and suitable for gene therapy. In addition, more than 220 genes related to ophthalmology, any genetic mutation may cause loss of vision and blindness. It is reported that the world's first drug for the treatment of ophrase defects is developed by the US Spark Therapeutics, and is used to treat LuxTurna, which is used to congenital Black (LCA2, RPE65 gene mutation), which was approved by the US FDA in December 2017. . This is regarded as an important milestone in human gene therapy. "Practice has proven that gene therapy with secure viral vectors to correct the nucleic acid drugs to correct genesis or expression of normal gene products is one of the most promising strategies that cause blind eye diseases." Professor Li Bin said.

Based on the positive results of pre-clinical research, Professor Li Bin completed the clinical trial of international gene therapy including 159 Leber's genetic optic neuropathy in China and Argentina during 2017 to 2018 (NCT03153293). This is also based on the public data showing the clinical trial of gene therapy for the global maximum sample size of this eye disease. In this American gene and the Cell Treatment Association 2020, the Chief Executive Officer of Newfus announced the latest developments in the clinical trial of this international gene treatment in the form of oral report, including 159 The 12-month tracking follow-up results of LEBER'S hereditary optic neuropathy patients. Dr. Newfus jointly founded in the form of the first gene therapy for the first gene in 2011 by the wall newspaper.

Dr. Lu Ying said: "The current clinical trial shows the potential of gene therapy in the field of Leber's genetic optic neuropathy, especially in long-term safety and efficacy, from China's enterprises - Newfus gene therapy platform He has been further verification. "He revealed that the company is actively preparation of recombinant human gland-related virus injection (NFS-01, RAAV2-ND4) domestic and foreign clinical trial registration declaration.

It is reported that the current global ophthalmic genetic treatment has gathered 10 well-known research and development enterprises. As the first ophthalmic disease gene therapy in China, Newfus is committed to developing the genetic therapy of ophthalmology and other hereditary diseases. In the field of R & D line, the company is advancing several clinical stages and clinical lines, including using gene therapy over a variety of ophthalmic diseases, such as diabetic retinal lesions, optic nerve injury, retinal pigmentation. In addition, the company is under construction of two technical platforms: ophthalmologic genetic treatment of research and development platform and gene therapy GMP production platform. Professor Li Bin revealed that the company plans to set up an ophthalmic gene therapy transformation center to use an animal experiment and cell experiments related to an ophthalmic disease drug research and development. "In the near future, domestic ophthalmology patients are expected to benefit from gene therapy." About AAV2-ND4

AAV2-ND4 officially entered the exploratory clinical trial phase in 2011. In 2011, three patients were incorporated in 2012, and six patients were introduced. Study that these 9 patients have improved their vision after 9 months and 36 months. In 2017, Newfour opened the clinical study of gene therapy in the world's sample size, further expanded the research scope and research depth of AAV2-ND4.

According to Li Bin, as of now, AAV2-ND4 completed the clinical study of 159 patients with LHON (including 10 Argentine patients). As far as the follow-up data for 6 months, there is no complications, and more than half of the patients have significantly improved. The main development of LHON gene therapy is mainly Professor GUY and GenSight, France, compared, AAV2-ND4 has the earliest and maximum sample quantity, also exhibits excellent validity and security data.

About Newfour Biotechnology Co., Ltd.

Newfus Biotechnology Co., Ltd. is the first company in China's first focused eye disease. The company is a clinical phase biotechnology company, which was created by Professor Li Bin (MD, PHD), which was founded in 2016, was found in 2016. Newfos is committed to developing the pathology of ophthalmology and other hereditary diseases. Currently, Newfus is advancing several clinical stages and a product line in the clinical stage. In April 2020, Newfus successfully completed the A-round financing of 130 million RMB. This round of financing was jointly governed by Fuxing Star Future Capital and Sequoia Capital China Fund. Newfos plans to provide leading gene therapy products for patients around the world, and will establish GMP workshops to provide clinical and commercial products to the world.