The line is coming, witnessing the industrialization of cell and gene therapy

2021 is a year when cell and gene therapy (Cell and Gene Therapy) shines
.
In China, two CAR-T therapies have received historic approval; overseas, in vivo CRISPR gene editing has also achieved the first positive clinical trial results; looking at the world, more innovative cell and gene therapies are expected to be launched this year Approved

.

From 0 to 1, from 1 to N
.
The continuous advent of cell and gene therapies means that humans have gradually begun to master such powerful tools to compensate for the random mistakes made by nature when encoding life forms

.

The era of rewriting the blueprint of life has come
.

01.
Cooperation-all for the patient

01.

"Our original intention is to save more lives, and all our efforts come from this
.
"

——Mr.
Liu Bizuo, Chief Administrative Officer and Chief Financial Officer of Sibiman Biotechnology

Image source: 123RF

At the beginning of his career in the cell and gene therapy industry, Mr.
Liu Bizuo keenly paid attention to the key factors affecting the development of the industry
.
"Before the test of the disease,'time is life', the industry must try its best to shorten the production time and quickly deliver the treatment to the patient

.

Although the new generation of therapy has great potential in terms of efficacy, it is especially full of opportunities for diseases that have been difficult to break through in the past

.

Fortunately, at the moment, the industry is working towards a more efficient model
.
In the development of a new generation of therapy, the CTDMO (Contract Testing Research and Development Organization) platform has become an important force for industrial cooperation and innovation

.

The combination of cell and gene therapy companies + CTDMO enables R&D companies to maintain a relatively lightweight operation and management, and focus more on the development and innovation of core products, while large-scale testing and part of the production links can be handed over to more professional CTDMO partners to form industrialization advantages, improve efficiency, reduce costs, and improve patient accessibility
.

"I hope that in the near future, the new generation of therapies, including CAR-T, can truly become affordable conventional treatment options for the majority of patients
.
" Mr.

Liu Bizuo said, "We are willing to continue to work with outstanding R&D partners in the industry to accelerate The research and development process of innovative therapies to save more patients and families

02.

Craftsmanship-quality comes from design

02.

——Mr.
Gao Yang, Senior Vice President and Chief Strategy Officer of Bangyao Biotechnology

Image source: 123RF

Due to the characteristics of high demand for individualization, rapid technological iteration, and complex mechanism of action, the production and development processes of cell and gene therapies are diverse and complex, and the regulatory systems of various countries are constantly adjusted and reformed
.

Like many peers, Bangyao Bio, the cell and gene therapy company that recently won the "2021 China Bio-Innovative Drug Most Growth Annual Award", chose to devote more resources to product research and development, and expand and commercialize the clinical stage.
The production is completed by professional CTDMO
.

Mr.
Gao Yang, Senior Vice President and Chief Strategy Officer of Bangyao Biotech, said, “Safety and effectiveness are the core issues facing the current cell and gene therapy development process, and the design of the later process plan determines the scale-up and process of the product.
The key factor for rapid commercialization
.
"

At present, cell and gene therapy process control adopts the concept of QbD (Quality by Design) for short.
Through thorough knowledge and research of the process in the early stage, the source and extent of risks in the quality control process can be accurately grasped
.

For this reason, choosing a professional CTDMO partner is very prudent for the majority of cell and gene therapy companies
.

Mr.
Gao Yang said: "IP protection, professional capabilities, and global operations and declarations are important considerations when we choose CTDMO partners
.
With mature technology and experience, CTDMO is more professional in quality control and can help us Reduce R&D risks and get through the entire process efficiently and accurately

.

03.
Development-the dream of helping R&D

03.

"The CTDMO empowerment platform further reduces the barriers to research and development, making the dream of cell and gene therapy research and development no longer out of reach
.
"

——Dr.
He Chunyan, founder and CEO of New Bud Gene

Image source: 123RF

The safety and effectiveness of the new generation of therapy not only require the concept of QbD, but also rely on a unique evaluation system for testing and characterization
.
The quality control requirements of individualized treatment are very high.
At present, only a few CTDMOs in the world have broken the barrier of full-process testing
.

As WuXi AppTec’s CTDMO platform focusing on cell and gene therapy, WuXi Biologics integrates the development, testing, manufacturing and commercialization of cell therapy, gene therapy and other high-end therapies, providing gene vectors and cell therapy products from R&D Full service to commercial production
.

Regarding the key role of testing capabilities in overall research and development, Dr.
Chen Qi, general manager of WuXi Biosciences China, has profound industry insights: "The development and application of standard methods for pharmacodynamic testing has always been a difficult point in the marketing of new high-end therapeutic drugs.

.
"

All testing and development, biosafety, virus removal, and final product release can be implemented on the platform "one-stop", which will not only greatly improve the efficiency of R&D and process amplification and optimization, but also reduce the sample consumption rate and cost.
The instability caused by the secondary transfer is more conducive to perfecting the risk points of each link of the process amplification and helping developers to greatly shorten the entire listing cycle
.

Limited capacity is another important challenge facing the cell and gene therapy industry
.
WuXi Biosciences actively deploys and efficiently promotes the globalization of production capacity.
It has 4 R&D and production bases in China, the United States and the United Kingdom, with a total construction area of ​​approximately 80,000 square meters
.
In 2020, WuXi Biologics expanded its cell and gene therapy production base in Philadelphia, USA, which tripled its production capacity
.
The Shanghai Lingang Base, which has been put into use recently, is equipped with more than 200 independent suites and 6 complete carrier and cell production lines, including an independent dosing center and GMP-level aseptic filling isolators.
It is expected to provide carrier and cell therapy services.
Provide customers with greater flexibility and faster delivery time
.

The Shanghai Lingang Base, which has been put into use recently, is equipped with more than 200 independent suites and 6 complete carrier and cell production lines, including an independent dosing center and GMP-level aseptic filling isolators.
It is expected to provide carrier and cell therapy services.
Provide customers with greater flexibility and faster delivery time
.

▲ Recently, drug-based Ming-sheng Shanghai Lingang new technology research and development, testing and production center was officially put into operation
.

▲ Recently, drug-based Ming-sheng Shanghai Lingang new technology research and development, testing and production center was officially put into operation
.

While steadily advancing its global production capacity layout, WuXi Biosciences has further expanded its technological capabilities in cell and gene therapy platforms
.
For example, in the AAV production process, WuXi Biosciences is committed to the development of an efficient adeno-associated virus vector production platform.
The TESSA-AAV technology platform under development realizes the de-plasmidization production process and can efficiently remove the production process.
The by-products greatly increase the concentration of the final product and effectively achieve mass batch production , breaking through the industry’s original capacity and technical barriers, and accelerating the development of the industry
.

The TESSA-AAV technology platform under development realizes the de-plasmid production process, and can efficiently remove the by-products produced during the production process, greatly increasing the concentration of the final product and effectively realizing mass batch production.

Dr.
Chunyan He, CEO of New Bud, said: “Next year, New Bud's proprietary intellectual property rights of Duchenne Muscular Dystrophy exon 50 skipping gene therapy product GEN6050 will complete pharmaceutical research and apply for clinical approval.
We will continue to deepen cooperation with WuXi Biotech to make full use of WuXi's high-quality CTDMO service platform accelerates the process of registration of clinical trials, realizes the drug market as soon as possible, and meets the urgent needs of patients around the world
.
"

WuXi's professional regulatory and registration service elite team can provide global regulatory registration and application services for cell and gene therapy development entering the clinical stage, helping developers to accelerate the globalization process of product development
.

04.
The law is approaching and the prospects are promising

04.

According to ASGCT (American Society of Gene and Cell Therapy, American Society of Gene and Cell Therapy) data, by the end of 2020, there have been more than 1,300 clinical trials of gene therapy under development worldwide
.
It is expected that the global cell and gene therapy industry will enter a harvest period in the next ten years
.

Looking forward to the future, Dr.
Zhang Youxiang, CEO of WuXi Biosciences, is full of confidence in the development of the industry: "I believe that in the near future, industrial innovations and breakthroughs will bring significant progress in the manufacturing process of cell and gene therapy, and the manufacturing of new generation therapies.
It will become more and more standardized, industrialized, and large-scale, with stable processes and controllable costs.
It will become the preferred treatment for cancer and rare diseases.
It is also expected to join hands with chemical and biological drugs to overcome more unknown diseases
.
"