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Global gene therapy has started, in recent years, domestic gene therapy companies have appeared frequently, what stage has domestic gene therapy developed? We studied the basic situation of more than 60 domestic gene therapy companies, and took stock one by one from the perspectives of company establishment, financing, technical layout, pipeline progress, etc.
, so as to roughly delineate the outline
of domestic gene therapy development.
Overview of establishment financing
Overview of establishment financingWe have inventoried all gene therapy companies that appear in domestic public information and news, and about 60 are active in the market
.
Looking back at the establishment and financing time of these companies, it is found that the number of funds raised increased significantly in 2020-2021 (Figure 1).
Figure 1 Overview of the establishment and financing of domestic gene therapy companies (Source: official websites and public information of each company, compiled by Fengshuo Venture Capital)
There are traces of all this
.
On the one hand, foreign gene therapy has made repeated breakthroughs
.
From 2016 to 2019, 3 gene therapy drugs
were launched.
At the same time, the frequent occurrence of transaction events in the field of gene therapy and the large-scale acquisition of gene therapy companies by international pharmaceutical companies also conveys the message
that the global gene therapy market is hot.
On the other hand, the domestic environment is gradually friendly
to gene therapy.
.
On the other hand, the domestic environment is gradually friendly
to gene therapy.
In the context of this, in 2018, the National Health Commission and others jointly issued the First Batch of Rare Disease Catalog, revealing the urgent clinical needs of rare diseases, and a considerable number of rare diseases are "available overseas and domestic without drugs"; In recent years, the breakthrough of 18A and innovative drugs has catalyzed the domestic pharmaceutical market, and the financing environment is good; At the same time, the general environment of the country
The 14th Five-Year Plan also increases support
for cell and gene therapy.
In addition, in the context of the industry, domestic CXOs, especially CDMO companies, have developed rapidly in recent years, laying the foundation
for the research and development and production of gene therapy.
There is demand, feasibility and funds, which has led to the establishment and financing of a large number of gene therapy companies
.
.
These corporate financing events are concentrated in 2020-2022, and the vast majority of companies have raised money in these three years, and some companies have raised two or more
rounds of financing in these three years.
But after that, will the heat still be there? In foreign countries, while gene therapy continues to make breakthroughs, there are also a considerable number of negative news: safety issues, failure to reach clinical endpoints, CMC problems, commercialization dilemmas.
.
.
The pioneers of gene therapy have encountered a number of problems
.
In China, the support and guidance policies for rare diseases and cell gene therapy are still being implemented; CXO is also growing, accumulating a considerable amount of experience in the development and production of cell gene therapy
.
The only thing that has changed is that the current pharmaceutical market continues to decline and has entered a "capital winter"
.
But the financing of gene therapy companies continues, and the number of financing events this year has reached a new high, after all, a large number of companies established in previous years always have to survive, and earlier companies have made some progress in their products and need funds to promote
.
And the number of newly established companies has plummeted this year, maybe everyone has calmed down? Calm is also a good thing, we can better reflect, to walk a better path
.
So let's analyze the existing companies and see which companies have taken a good path
over the years.
Over the years, the company has continued to raise funds, with more than 20 companies raising more than 100 million yuan and 7 companies exceeding 500 million
, 3 companies over 1 billion
.
Let's take a look at the top companies
that have disclosed their financing amounts.
Figure 2 Top companies in terms of financing amount (source: official websites and public information of each company, some companies have not disclosed financing status for the time being, compiled by Fengshuo Venture Capital)
After reading these companies, I probably have an impression, AAV, ophthalmology, CNS, cancer
.
These high-financing companies are mainly AAV platform companies, while all AAV technology companies have ophthalmology, followed by CNS
.
It can be seen that domestic funds prefer AAV gene therapy companies
.
Domestic investment appears to be stabilizing
.
Among the gene therapies currently on the market, two AAV gene therapies give a precedent for the success of AAV in the eyes and spinal cord, which brings relatively high certainty to the AAV track
.
In foreign countries, the gene therapy companies that have raised the most money in the primary market in recent years are new technology companies - mainly gene editing technology
.
In the early years, when the gene editing company was not yet listed, the AAV platform company was hot, and with the development of gene editing technology in recent years, gene editing was sought after, the technology was updated from generation to generation, and the amount of financing reached a new high
.
In addition, the top foreign listed gene therapy companies in terms of market value and the top gainers since listing are gene editing companies
.
The different preferences of domestic and foreign investors for these two types of companies stem from a more central question - how strong this company is
.
The source of gene editing technology and the core underlying patents are in the hands of some foreign companies and academic institutions, China's current gene editing companies started late, the number is not much, the existing research and development strength and foreign similar companies still have a certain gap, it is difficult to attract the attention of investors
.
Imagine if there were Zhang Feng and David in China
Liu's ordinary characters or they come to China to incubate a company, presumably the capital will rush in
.
.
Although domestic gene editing companies are relatively unpopular, we should see the future trend of gene therapy: gene editing will play an increasingly important role
in gene therapy.
in gene therapy.
In addition, one of these financing TOP companies is noteworthy - Rui Hongdi Pharmaceutical, which is a subsidiary of
Hengrui Pharmaceutical.
In addition to Hengrui, there are Kanghong Pharmaceutical-subsidiary Hongji Biotechnology, Huada Group - subsidiary Hemu Gene, Huahai Pharmaceutical-subsidiary Seselqing Biotechnology, domestic large pharmaceutical companies have begun to establish subsidiaries to layout this emerging market
.
by establishing subsidiaries.
Specific track conditions
Specific track conditionsEstablishment, financing, domestic gene therapy companies bloom in a hundred flowers, involving a variety of technologies, multiple fields, which tracks should they be divided into?
We categorized companies into three categories based on their leading pipelines, focusing on AAV technology, gene editing technology, and others (Figure 3).
Figure 3 Situation of domestic gene therapy companies (Source: official websites and public information of each company, compiled by Fengshuo Venture Capital)
There are about 32 companies focusing on AAV technology, most of which are deployed in multiple disease fields at the same time, and more fields are ophthalmology and CNS; There are about 17 companies focusing on gene editing technology, and general in vivo therapy and in vitro therapy will be deployed, the most concentrated disease area is blood diseases, and some companies are involved in cancer, covering the scope of
traditional gene therapy.
The reason for this classification is that the specific problems encountered by these types of companies in research and development, the types of diseases that can be applied, and the development strategies are therefore different
.
So how are each of these tracks going? What are the first areas to make breakthroughs? We further analyze the clinical-stage pipeline
.
Clinical-stage pipeline
Clinical-stage pipelineIn the clinical stage, 12 companies have entered the clinical stage, with a total of 16 pipelines, all of which are in the very early stages
except for Newforth.
The following are the details of the clinical stage and IND application acceptance pipeline:
Figure 4 Pipeline of domestic clinical/IND application stage gene therapy companies (Source: official websites and public information of each company, compiled by Fengshuo Venture Capital)
Specifically, the pipeline is aimed at the disease field, and combined with the technology and field layout of domestic companies summarized above, we find that there are many companies in the field of ophthalmology, and there are many pipelines for ocular gene therapy to enter the clinic; There are many companies that have deployed CNS and gene editing technologies, but there are currently fewer
drugs in these two fields that have entered the clinic.
Perhaps it is a late start, or it may be difficult, these two segments are relatively blank, so there are relatively many opportunities
.
But AAV eye therapy is not just a bone
to gnaw.
Although many multinational pharmaceutical companies such as Novartis, Biogen, Regeneron and AbbVie have made frequent moves in the field of ocular gene therapy, they have acquired companies and pipelines one after another, and kept buying, buying, buying
.
But after that, Biogen's eye gene therapy failed last year; Adverum, an eye gene therapy company
Biotechnologies trials frustrated, layoffs and restructuring; Even Novartis has recently been rumored to be considering selling its ophthalmic business, but the company has just acquired 3 AAV-eye gene therapy companies
in recent years.
It seems that the development of AAV eye therapy is not so easy
.
to gnaw.
From the perspective of clinical progress, the fastest progressing company is Newforth, and the clinical trial of NR082 (AAV delivery ND4 gene) for the treatment of Leber hereditary optic neuropathy caused by ND4 mutation has entered clinical phase
III.
In addition to Newforth's pipeline, the other 15 pipelines and 9 indications are in a very early stage, and have been approved in 2021-2022, which is similar to the pipeline progress of the indication, and there is not much gap
in time for the time being.
Looking at the world, domestic self-developed drugs lag behind a lot
.
Most of the domestic pipelines follow foreign countries, and the foreign progress of gene therapy with the same indication and target is in clinical phase III and beyond
.
In fact, this situation is not a bad thing, not all drugs have to compete for global FIC, must pursue speed, in the gene therapy is still in the pioneer period, FIC drugs face many unknown risks, competing for BIC is also a good way
.
The existing clinical data of these foreign drugs with the same target are positive, and the domestic pipelines have also done IIT trials for preliminary verification, and we can maintain an optimistic attitude
towards these pipelines first.
.
Not all drugs have to compete for global FIC, must pursue speed, in the pioneer period of gene therapy, FIC drugs face many unknown risks, competing for BIC is also a good way
.
And from the perspective of the total number of pipelines, whether global or domestic, gene therapy belongs to the blue ocean, and the competition for the same target is not too fierce
.
Latecomers also have the opportunity to break through, as long as they can be successfully approved, they can get a lot of market share
.
Therefore, many companies submit applications
to the FDA and EMA at the same time as filing IND in China.
.
On behalf of the company
On behalf of the companyFinally, we list representative companies
of various domestic tracks.
AAV – Faith Medicine, Newforth
AAV – Faith Medicine, NewforthFounded in 2018, Chairman Professor Xiao Xiao has been engaged in the field of recombinant adeno-associated virus (AAV) gene vectors and gene therapy for more than 30 years, and was a tenured distinguished professor of gene therapy at the University of North Carolina, and a co-founder
of Askbio, Bamboo 。 Faith Pharma integrates product development, production and clinical application of gene therapy, and has dozens of world-leading patented technologies, including the world's leading HEK293 cell suspension serum-free culture process and full-chromatography large-scale purification process, and has established the first commercialization production platform
for clinical-grade AAV gene therapy drugs in China.
Its hemophilia AAV gene therapy drug is the first intravenous rare disease gene therapy in China and even Asia, and entered clinical phase
I last year.
Newforth was founded in 2016, and the founder Professor Li Bin led the team to carry out the world's first LHON gene therapy exploratory clinical study
.
With the help of the mature AAV gene therapy technology platform, Newforth has established a rich product pipeline, including more than 10 research projects
for various ophthalmic diseases such as optic nerve injury diseases and vascular retinopathy.
At present, the latest pipeline has entered clinical phase III, which is the most advanced gene therapy drug
in China.
Gene editing—Boyajiin
Gene editing—BoyajiinFounded in 2015, Boya Gein focuses on the transformation of gene editing technology and is committed to developing innovative therapies
for hard-to-cure genetic diseases and cancers.
It has created four major platforms based on gene editing technology: in vitro therapy-hematopoietic stem cell platform and universal CAR-T platform; In vivo therapy – RNA base editing platform; High-throughput genomic screening—a new drug discovery platform
.
At present, the fastest pipeline (treatment of β-thalassemia) entered clinical phase I last year and is the most advanced drug
in domestic gene editing therapy.
epilogue
In the past few years from 2019 to 2021, the domestic environment is very friendly to gene therapy, and before that, the number of companies was few and they could not get any money
.
Riding the east wind, domestic gene therapy companies followed the trend, took root and blossomed, and gave birth to the first batch of gene therapy pipelines
in China.
The current competitive environment is relatively relaxed, gene therapy companies have cash reserves, and the next stage is a very good growth stage
.
Optimistically, it is expected that the drugs of domestic gene therapy companies will make breakthroughs in the next few years and usher in new growth points
.
