Gene Therapy 1.0 Era: Spiral Advance

In 1953, Watson and Crick discovered the DNA double helix structure, which opened the era of molecular biology.

In 1963, the DNA double helix structure was published for 10 years, and Joshua Lederberg proposed the concept of gene exchange and gene optimization.

In 1972, Theodore Friedman proposed the hypothesis that gene therapy can be used for the treatment of human diseases.

In 1984, Cepko CL successfully designed a retroviral vector system that efficiently inserted foreign genes into mammalian chromosomes.

On September 14, 1990, the National Institutes of Health (NIH) approved the first clinical study of gene therapy. Four-year-old Ashanti DeSilva was the first to receive severe combined immunodeficiency due to adenosine deaminase deficiency (ADA- SCID) Gene Therapy. Since then, approximately 4,000 patients worldwide have participated in more than 500 clinical trials for gene therapy.

On September 13, 1999, 18-year-old Jesse Gelsinger died of multiple organ failure caused by adenoviral vector gene therapy, mainly due to the immune system's overreaction to adenoviral vectors. The FDA has suspended most clinical trials and re-evaluated the ethical and technical implementation of gene therapy.

The death of Jesse Gelsinger ended the era of radical leap in gene therapy, and the cold winter of the capital world forced researchers to abandon commercial temptation and return to academic and rational.

In August 2006, the US Science magazine disclosed that gene therapy successfully treated metastatic melanoma in two patients, and gene therapy began to gradually get out of the woods.

Gene Therapy 2.0 Era: Open in the first year

There are two reasons for the failure of treatment in the gene therapy 1.0 era. One is that the introduced recombinant gene is inserted into the wrong position of the genome, thereby activating the oncogene, and the second is because the carrier virus causes the patient's immune system to overreact, resulting in more Organ failure and brain death.

In the era of Gene Therapy 2.0, researchers used a new and highly efficient virus to transport new recombinant genes into cells, thus overcoming many problems in the 1.0 era. It mainly includes two gene therapies, which are represented by Glybera adeno-associated virus vector (AAV) for treating LPLD and Strimvelis retrovirus or Lentivirus vector for treating SCID, respectively. The so-called Cheng also virus, defeated the virus.

In October 2012, EMA approved uniQure's Glybera marketing approval, becoming the first approved gene therapy drug in the history of the European Union for gene therapy in adult patients with lipoprotein lipase deficiency (LPLD). Although Glybera's treatment costs as much as $1 million+, the launch of Glybera opens the spring of gene therapy.

In November 2013, the US FDA officially issued the "Guideance for Industry: Preclinical Assessment of Investigational Cellular and Gene Therapy Products", which replaced its 1998 Human Cell Therapy and Guidance for Human Somatic Cell Therapy and Gene Therapy.

In May 2016, EMA approved the launch of the gene therapy Strimvelis, jointly developed by GSK and Italian partners Telethon and OSR, for the treatment of severe combined immunodeficiency (ADA-SCID) caused by adenosine deaminase deficiency. According to the MIT Technology Review, Strimvelis's disposable therapy costs $665,000, a one-time lifetime cure and promises a refund.

In February 2017, the MIT Technology Review released the “Top 10 Global Breakthrough Technologies 2017” list, including: Gene Therapy 2.0. The approval of Glybera and Strimvelis marks the 50th anniversary of the development of gene therapy, and finally ushered in the spring of the second decade of the 21st century. Gene drugs become the third generation of treatment after chemical small molecule drugs and biomacromolecules. drug.

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