Gene therapy industry may become the key development direction of the biopharmaceutical industry in the future after antibody drugs, China's industrial innovation activity is increasing, supporting service system is constantly improving, but at the same time the core link of the upstream industrial chain card neck phenomenon is still serious, need to break through.
1. The development status of gene therapy industry chain China's gene therapy industry development time is relatively short, but the development speed, enterprise innovation activity continues to strengthen, the integrity of the overall industrial chain is not high, the current product is concentrated in the field of midstream drug research and development innovation, the core of the upper reaches of the industrial chain is not strong control.
1 Core link of the gene therapy industry chain Source: Firestone creates a breakthrough based on public information on the upstream single point of gene therapy, but the overall competitiveness is not strong.
China's gene therapy upstream industrial chain innovation is weak, although Shanghai and Yuan, Beijing Wuga and other gene therapy carrier research and development enterprises to achieve innovation breakthroughs, but in most of the upstream core raw materials and components in the field of foreign neck phenomenon is serious, such as media, disposable biological reactors and other core links of most of the market is still monopolized by foreign countries. Table
2 Key raw materials and core suppliers of China's pharmaceutical and health industry sources: Firestone creation according to public information finishing to culture base as an example, basic culture base, serum-free culture base raw materials are mainly amino acids, vitamins, etc., the requirements for medicinal levels, domestic related raw materials in terms of purity is difficult to meet the requirements, mainly imported (such as Sigma, etc.).
the import cycle is relatively long, raw materials need to be stocked for 3-6 months, greatly extending the research and development cycle.
At present, domestic culture base is still in its infancy, research and development and production enterprises basically exist in the relatively mature Yangtze River Delta region of biopharmaceitical research and development, Beijing Tianguangshi Biotechnology Co., Ltd. in serum cell culture base products have a certain market.
3 Domestic medium key enterprises source: Firestone creation according to public information finishing mid-stream, product approval process is relatively lagging behind.
statistics, China's current gene therapy field accumulated 489 enterprises, the vast majority of concentrated in the field of drug research and development.
China's gene therapy in the field of drug research and development enterprises in recent years continue to achieve breakthroughs, such as the field of cart Nanjing Legend, Koji Biology, Boshengji, Yi miao Shenzhou, etc. ;
at the same time for drug research and development of large-scale production capacity, although the current accelerated layout of enterprises, but the overall market space will take time to fully release.
, the domestic gene therapy industry trends one: the government has become an important entry, the construction of industrial ecology, service industry innovation.
recent years, the layout of innovative service systems in the field of cell/gene therapy has accelerated in various parts of China, and industrial service systems in Beijing, Jinan, Shenzhen, Sichuan and other places have been gradually improved.
November 16, 2020, Jinan Gene Editing Industry Base Project officially started, the project is the first in the country to lay out the gene editing industry, committed to promoting gene editing as a revolutionary technology in the source innovation, crop improvement and molecular testing and other fields of industrialization.
project planning and construction land of 80 acres, planning and construction of a total construction area of 95,200 square meters, including 10,000 square meters of all-light intelligent greenhouse, 10,000 square meters of rapid breeding plant plant and technology platform experimental building, business incubation base.
September 28, 2020, the country's first cell therapy sharing industry support platform settled in Jinan, cell therapy technology is one of the most promising areas of the biopharmaceutical industry.
platform for the country's first set of shared laboratories, shared CDMO workshop, shared clinical center as one of the industrial support system.
Industrial Park will be completed in 2022 and put into operation in 2023.
Cell Industrial Park with CDMO (drug research and development outsourcing services) as the core, focusing on CAR-T, CAR-NK and other frontier areas of immunocellular therapy, stem cell therapy, gene therapy and regenerative medicine, and strive to promote the treatment of tumor, autoimmune, metabolic, neurodegenerative and other diseases of the treatment technology and drug research and application.
July 31, 2020, the Center for Cell and Gene Therapy Innovation landed in Changping, Beijing.
The center, created by the Tsinghua Institute of Industrial Development in Beijing in collaboration with a number of leading international research institutions, is expected to be completed in May 2021 and will serve more than 10 cell and gene therapy technology companies to conduct research and development of innovative drugs and clinical trials.
to build a key technology research, core process research and development, pilot production research and development support system in line with the international biopharmaceutical GMP specifications, and foster incubation and innovation drug start-ups.
On June 14, 2020, gene editing small pig production and research collaborative innovation Neijiang base was awarded in the capital, Northwest Agricultural and For forgross University of Science and Technology School of Animal Science and Technology, Sichuan Agricultural University Animal Genetic Breeding Research Institute to gene editing small pig production and research collaborative innovation Neijiang base licensing.
the base total investment of 15 million yuan, covers an area of about 100 acres, the first phase of the project about 10 acres has been put into use, now has the largest number of genetic modification of small pigs, 10 kinds of genetic modification of pigs a total of more than 200 head.
December 6, 2019, the "Rozman Bay Biopharmaceutical Base" initiated by the Shenzhen Rozman International Institute of Translational Medicine was officially opened in Longhua District.
base focuses on the field of gene therapy, in Longhua to build a set of "research and development," "pilot production," "preclinical research," "clinical research" in one of the professional park, to build a highly professional, innovative drug research and development and incubation of the entire industrial chain park and base.
park and base around the entire industrial chain of nodes, with large-scale equipment sharing center, SPF experimental animal center, preclinical pharmacological CRO, experimental pathology platform, gene therapy virus vector production CDMO, clinical approval ATD declaration CRO at home and abroad, clinical research CRO at home and abroad, clinical PK/PD center laboratory.
August 17, 2018, China Gene Group and Luohu Hospital Group announced that they will build China's first gene and cell therapy hospital.
, such as cerebral palsy, GM2 neuroglucoside lipid disease and other rare diseases, as well as Alzheimer's disease, leukemia and other diseases, will hopefully be treated in the hospital through gene and cell technology.
hospital will also actively explore a three-way partnership with the Massachusetts Memorial Hospital.
China Gene Group will fully invest about 2 billion yuan, the location of Shenzhen Bugi Li Lang, construction area of about 150,000 m2, from the start of construction to accept rare disease treatment appointment registration.
trend two: the speed of enterprise layout, gene therapy into antibody drugs after the next enterprise must compete.
years of technological accumulation, innovation began to appear.
November 2, 2020, "Ruifeng Bio" has recently completed nearly 100 million yuan A round of financing.
is a preclinical, high-tech company dedicated to the development of new drugs with gene editing technology at its core, and has extensive experience in areas including editorial system innovation, new mouse and large mammalian modeling, and multi-layered drug ability assessment.
October 18, 2020, Boya announced that China's State Drug Administration's Drug Review Center (CDE) had accepted its clinical trial application for ET-01, a gene-editing therapy product for transfusion-dependent β thalassemia.
September 25, 2020, it is understood that "Ino micro" has recently completed 10 million U.S. dollars B-plus round of financing.
this round of financing is funded exclusively by Shanghai Pharmaceutical Investment and managed by SIIC Capital.
August 11, 2020, Ino micro and Shanghai Pharmaceuticals announced clinical cooperation and exclusive licensing agreements for the MVR-T3011 in-tumor lysovirus project.
this round of funding will be used to deepen cooperation in the MVR-T3011 in-tumor project.
On September 17, 2020, Kanghong Pharmaceuticals said it would build a gene therapy platform to seek treatment for major diseases, and that the company has plans and technical reserves to work deep in the field of gene therapy, and will build a gene therapy platform with independent intellectual property rights to find treatment options for major diseases with medical needs;
April 28, 2020, Soryuan Bio announced the acquisition of Tocagen (TOCA. US) Gene Therapy Platform and the world's first anti-tumor drug, including gene therapy platform (retrovirus replication vector platform, RRV) and the global interest in all related product pipelines (except for one product Greater China equity).
trend three: the domestic innovation drug industrialization ability in the new technology is constantly breaking through the test, and strive to break through the industry bottleneck, gene therapy CDMO outbreak.
November 6, 2020, Kingsley's CDMO business unit, Kingsley Vibrant BioGMP Production Center, opened its "Innovative Cooperation Model Accelerated Antibody Drug Development and Investment Forum" in Nanjing, Jiangsu Province.
The platform includes Nanjing Biopharmaceian Research and Development and Production Center and Kingsley Zhenjiang Commercial Production Center, with a total area of about 210,000m2, after the production of antibody drug part of the cumulative production capacity will reach 47600L, the production of antibody GMP production center, to provide clinical phase I and clinical phase II GMP production services, its overall workshop construction and capacity to undertake in the industry-leading level.
November 1, 2020, the Boten BioSuzhou Gene and Cell Therapy CDMO platform was launched, with a total area of about 16,000 square meters, to further improve the research and development and commercial production capacity of gene and cell therapy processes.
October 25, 2020, focusing on AAV carrier packaging and gene therapy CDMO overall solution provider Pixin Bio announced the completion of a new round of B-plus financing, financing will be mainly used for the construction of GMP-level large-scale AAV production base, expansion of GMP production plant, increase production capacity, promote AAV carrier new technology, new process development, accelerate the process of industrialization of gene therapy at home and abroad.
September 23, 2020, Heyuan Bio completed over 300 million yuan C round of financing, has started nearly 80,000 square meters of gene and cell therapy CDMO industrial base project, the annual production capacity is expected to exceed 1 billion yuan.
July 24, 2020, Auscon completed a 450 million yuan B round of financing to promote the expansion of cell culture base and CDMO sector capacity, led by ZHONGC Jiatai (a fund owned by ZHONGC Capital) and Jinshi Investment, CCB International, Wenshi Investment and Shanghai Huaxu He investment.
financing will be mainly used for the construction of serum cell culture base haimen base project and CDMO plate capacity expansion, will assist the current undertaking and planned to undertake a number of BLA/NDA production phase projects in Auscon landing and industrialization process, while ensuring that Auscon meets the CDMO needs for different product types (including therapeutic biologics, preventive biological products, cell therapy and gene therapy drugs, etc.) projects.
September 2019, Yiming Cell's first preparation base was officially opened, and several orders for gene drugs have been completed and delivered, one of which is about to be declared clinically.
business has basically covered the entire research and development process of gene drugs, including from the initial help customers to build vectors, build bacterial libraries, develop product processes, the production of clinical grades of prosites /lyrovirus/ AAV vectors, stability research to reporting data writing services.
Knot With more and more technical experts, capital entry, cell/gene therapy sub-industry is about to usher in a flowery development trend, local governments are also in the layout of cell/gene therapy industry, but also in the corresponding links of industrial platform construction and layout, but at the same time we also see in the upstream link of the industrial chain supervision, policy support there are many unresolved issues and unresolved policies.
With the deepening of the industry, capital support, government policies will also enter the deep water area of cell/gene therapy, and only in this way can enterprises, scientists, governments, parks, capital innovation service ecology and virtuous circle be formed.
author | Miao Pioneer