Can CGT be cured for life with one treatment?

At present, the industry-recognized Cellular and Gene Therapy (CGT, Cellular and Gene Therapy) refers to the transfer of certain genetic material to specific target cells of a patient, and modifying the expression or expression of individual genes by means of gene addition, gene modification, gene silencing, etc.
The process of repairing abnormal genes and achieving the purpose of curing diseases
.
As a new field in the treatment of cancer and genetic diseases, CGT therapy is currently a very promising development direction in the field of biomedicine

One treatment is expected to be cured for life

Unlike most traditional chemical drugs and biological macromolecular drugs that target proteins, most CGT therapies directly target DNA, and treat diseases from the source by changing DNA, aiming at some serious and rare genetic diseases that are not cureable.
In the case that traditional medicine can only cure but cannot cure, CGT therapy has the potential to cure these diseases, and it is expected to achieve the purpose of one-time treatment and life-long recovery
.

Supervision and quality standards and regulations promote the development of the industry

In 2016, 21-year-old college student Wei Zexi passed away after receiving immunotherapy without regulatory approval.
This incident revealed the lack of strict government supervision in the early stage of the development of CGT therapy, and set off a domestic reflection on immunotherapy, which also prompted the Chinese government A 19-month ban was imposed on the research and clinical application of CGT therapy
.

After this incident, led by the National Health Commission and the National Medical Products Administration, the reform of the domestic policy side was fully launched
.
At the end of 2017, a priority review channel was opened for drugs for the treatment of rare diseases or life-threatening diseases.

The introduction of a series of domestic industry regulatory policies and treatment standards has gradually strengthened and improved the industry regulatory work in the field of CGT treatment, and also put forward specific and feasible technical requirements for the development of the industry.
This is consistent with the general trend of global CGT treatment supervision.
Promoted the development of the domestic CGT industry
.

Product form of CGT therapy

Specific to the product form, CGT therapy includes plasmid DNA, viral vectors, bacterial vectors, human gene editing technology, patient-derived cells, and gene therapy products
.

Source: public data compilation

The carrier is the most important part of CGT therapy

It is particularly noteworthy that the carrier is the most important link in cell gene therapy
.
Whether it is in vivo or in vitro therapy, vectors are indispensable: in in vivo therapy, it is necessary to deliver the therapeutic gene to the patient through a viral or non-viral vector; in in vitro therapy, the patient’s cells need to be in vitro Return after genetic modification

The development and production of CGT therapy mainly includes three links: plasmid production, viral vector production, and cell preparation
.
At the level of preparation and application technology, plasmid production technology has been developed for many years, the production process is very mature, and the difficulty is relatively small; at present, viral vectors are the mainstream vector for clinical trials of gene therapy products.
According to statistics, more than 2/3 of clinical trials have selected them.
Viruses are used as vectors, and the most widely used viral vectors include retroviruses and lentiviruses, adenoviruses, and adeno-associated viruses

According to rough statistics, plasmids and viral vectors account for half of the current CGT treatment field
.

Abundant reserves of products under research are waiting for the industry to break out

CGT therapy is gradually transforming from basic scientific research to industrial application, and it is also widely recognized and valued by the market
.

According to reports, in the future, a large number of cell and gene therapy research projects will begin to focus on entering the clinical stage.
It is expected that by 2026, there will be more than 8,000 cell therapy clinical pipelines and more than 3,000 gene therapy clinical pipelines.
The entire CGT industry will be in the future.
It is expected to achieve a ten-fold increase in the past 5 years
.
As early as 2019, the FDA issued a statement stating that in view of the surge in the number of CGT therapies entering the preclinical stage, the FDA expects that by 2025, 10 to 20 CGT products will be approved each year

The well-stocked pipeline of products under research is the current energy storage and the foundation for the prosperity and development of the entire industry in the future, waiting for the outbreak of the CGT treatment field