China Renaissance Capital's 2021 Annual Review of the Medical and Life Technology Industry｜Pharmaceutical and Biotechnology

Globally, tumors, respiratory and infectious diseases, neurological diseases, and chronic diseases in the cardiovascular and metabolic fields are all diseases that threaten human health and put enormous pressure on the medical security system
.
Humans have never stopped to find and develop methods to overcome these diseases
.
With the expansion of policy support and the increase of R&D investment, we have reason to believe that the progress and breakthroughs in the sub-segment of medicine and biotechnology will make greater contributions to the diagnosis and treatment of the above-mentioned major diseases
.

In the content of the previous issue, the sharing of Huaxing Medical and the life science and technology team focused on the diagnosis and life science track
.
This issue will share the summary of the 2021 pharmaceutical and biotechnology track, and please look forward to the follow-up sharing in other fields
.


In 2021, the results of epidemic prevention and control have achieved initial results.
At the same time, new policies have been introduced to encourage innovation, and the total transaction volume and number of transactions in the pharmaceutical and biotechnology industries have both surpassed those in 2020
.
In particular, in the third quarter of 2021, the transaction value hit a new high since 2018, with a single-quarter disclosed transaction value exceeding $5.
4 billion
.

 
However, in addition to optimism, worries are looming: the secondary market breaks frequently, the rhythm of the primary market slows down, the market heats down, the concept hype exits, and the head effect becomes more prominent
.
This reminds us that future innovation must closely follow national policies and overall planning; progress is greater than valuation, indications are greater than targets, prevention is better than treatment, and integration of diagnosis and treatment; ultimately, innovation needs to be implemented through hard work
.

 









 
Keeping up with the overall plan of national policies and promoting key tracks
 
, the "14th Five-Year Plan" put forward new overall goals for the development direction of the medical industry.

In March 2021, the "14th Five-Year Plan for National Economic and Social Development and Outline of Vision 2035" was released
.
The 14th Five-Year Plan emphasizes the need to strengthen original and leading scientific and technological research, starting from the urgent and long-term needs of the country, and concentrate superior resources to tackle key core technologies such as emerging infectious diseases and biosafety risk prevention, medicine and medical equipment
.
Among them, sub-fields such as brain science and brain-like research, genes and biotechnology, clinical medicine and health are among them
.

 
Local governments have successively issued relevant support policies to implement the central guidance, and the benefits of cell therapy, gene editing, synthetic biology and other tracks have highlighted
that the local governments of Beijing, Shanghai, Jiangsu and other provinces and cities have actively responded to the national policies and actively deployed on relevant tracks.

.

 
On November 24, 2021, the Beijing Municipal Committee of the Communist Party of China and the Beijing Municipal People's Government issued the "Beijing International Science and Technology Innovation Center Construction Plan during the "14th Five-Year Plan" period, aiming to promote new antibody technology, gene editing, new cell therapy, stem cells and regeneration.
Research and development of cutting-edge biotechnology in key fields such as medicine
.

 
On November 29, the leading group of the Beijing Zhongguancun Demonstration Zone issued the "Development and Construction Plan for the Zhongguancun National Independent Innovation Demonstration Zone during the "14th Five-Year Plan" period", stating: "Support the development of brain science and brain-like research, gene editing, stem cells and regenerative medicine, Single-cell multi-omics, synthetic biotechnology, biological breeding and other life science and technology research
.
Strengthen the transfer and transformation services of achievements in colleges and universities, and in the field of biomedicine, use technologies such as medical device engineering platforms, biological sample banks, and biocompatibility evaluation platforms.
Platform role, building a pilot production platform for cell and gene therapy, biological drugs, recombinant protein drugs, an AI innovative drug incubation platform, etc.

"
 
On July 6, 2021, the General Office of the Shanghai Municipal People's Government issued the "Shanghai Advanced Manufacturing Industry Development Notice of the "14th Five-Year Plan"
.
The notice pointed out that it will focus on frontier biological fields such as brain science, gene editing, synthetic biology, cell therapy, stem cells and regenerative medicine, carry out major scientific and technological research, and promote equipment such as key raw materials, high-end raw and auxiliary materials, important pharmaceutical equipment and consumables, and precision scientific research instruments.
and material R&D innovation
.
On July 21, the General Office of the Shanghai Municipal People's Government officially released the "14th Five-Year Plan for the Development of Strategic Emerging Industries and Leading Industries in Shanghai".
Treatment and other high-end biological products; biotechnology services, promote artificial intelligence-assisted drug development, digital medical solutions and other technical integration applications and other related guidance plans
.

 
In September 2021, the General Office of the Jiangsu Provincial Government issued the "14th Five-Year Plan for Scientific and Technological Innovation of Jiangsu Province"
.
The "Plan" points out that it will focus on the development of therapeutic antibodies, new vaccines, nucleic acid drugs, recombinant protein and polypeptide drugs, genetic engineering drugs, cell therapy products, bacterial drugs and oncolytic viruses
.


In addition to Beijing, Shanghai, and Jiangsu, many provincial and prefecture-level governments, including Zhejiang, Guangzhou, Shenzhen, Tianjin, Dalian, Yantai, etc.
, have successively issued relevant pharmaceutical industry development guidance plans in 2021.
, gene editing, synthetic biology and other sub-track development resources are inclined, and the benefits are prominent
.

 
 
Implementing the integration of prevention and control of major diseases and diagnosis and treatment
 
Focusing on major disease areas such as CVRM and CNS
At the beginning of the "14th Five-Year Plan", we must focus on the national strategy and return to the essential needs of treatment
.
Among them, major disease areas such as tumors, respiratory and infectious diseases, neurological diseases, and chronic diseases in the cardiovascular and metabolic fields are the focus of national policy attention and industry efforts
.

 
Over the years, research on tumors and immune diseases has received a lot of capital support
.
The outbreak of the new crown epidemic has brought unprecedented attention to RSD (respiratory and infectious diseases)
.
Faced with the high complexity of CNS (neurological system diseases), many pharmaceutical companies have been hit hard in this field and lost confidence; but in June 2021, Aducanumab was approved for marketing amid controversy
.
Chronic disease CVRM (cardiovascular, renal and metabolic disease) has a large patient population and should also receive more attention
.

 
There are more than 500 million patients with cardiovascular disease in the world, and cardiovascular disease is still the main cause of death in the world; there are about 850 million people with kidney disease in the world; more than 460 million adults in the world suffer from diabetes, and the incidence of diabetes has reached 9.
3%
.
There are about 330 million patients with cardiovascular disease in China, and more than 5 million people die each year due to cardiovascular disease, which is the country with the highest cardiovascular mortality; the prevalence of chronic kidney disease is as high as 10.
8%, and the number of patients exceeds 100 million; 140 million, accounting for a relatively high proportion of the number of people with diabetes in the world
.
The global CVRM disease prevalence and disease burden are on the rise, and CVRM in China has also become a major public health problem
.

 
In the face of these major disease areas, Novartis, Roche, AstraZeneca, Eli Lilly and other large MNCs have begun to deploy CVRM (cardiovascular, renal and metabolic diseases), CNS (neurological system diseases), RSD (respiratory system diseases), etc.
, and expand diagnosis and treatment The integrated and next-generation treatment platform is worthy of benchmarking and reference by domestic enterprises
.
On July 15, 2019, the State Council issued the "Opinions of the State Council on Implementing the Healthy China Action" (hereinafter referred to as the "Opinions"
 
) .
"Prevention and control of major diseases" is the core area of ​​R&D investment in the healthcare industry.
The diagnosis and treatment of major diseases such as cardiovascular and cerebrovascular diseases, cancer, respiratory system, diabetes, and infectious diseases are the core diseases proposed in this "opinion" .
These five diseases are the most common diseases in my country, which have the most far-reaching impact on residents' health, cause the greatest pressure on society, and have the largest medical insurance expenditure.
They are also the main areas where my country's medical industry has continued to increase investment in research and development in recent years.



 


.
This "opinion" once again clarifies the importance attached to these major diseases, encourages investment in related fields of diagnosis and treatment, and guides the core areas of the development of the healthcare industry programmatically
.

 
With the development of genomics, proteomics and other technologies, human beings have entered the era of precision medicine, and the prevention, diagnosis, precise treatment and rehabilitation of CVRM and other diseases have become a national strategy
.
Especially for single-gene inherited cardiovascular diseases with clear etiology, early prevention, screening and intervention of the disease should be actively carried out, and a precise health management system integrating diagnosis and treatment should be established
.



Single-point technological breakthroughs
 
Small molecule and macromolecule new technologies emerge in an endless stream, and new products return to the essence of clinical needs.

In the field of small molecules , breakthroughs have been made to challenge "undruggable" targets through subversive design and mechanism of action
.
For example, targeted protein degradation (TPD) is an emerging strategy for drug development that only requires small molecules (not necessarily functional) that can specifically bind to the target protein and degrade ubiquitinase or lysosome The combination of pathways can theoretically degrade these "undruggable targets" (mainly including transcription factors, proteins that only play a skeleton function, etc.
), which has great medicinal potential
.

 
In 2021, TPD technology represented by protein-targeted degradation chimera (PROTAC), molecular glue (molecular glue) and lysosome degradation will receive unprecedented attention, and major pharmaceutical companies have begun to deploy
.
In July, PROTAC therapy star company Arvinas announced that it had reached an agreement with Pfizer of more than 2 billion US dollars to advance the research and development of the potential "first-in-class" therapy ARV-471; in August, Bayer announced that it planned to acquire the new protein degradation therapy for US $ 2 billion.
Vividion Therapeutics, Eli Lilly and Lycia Therapeutics, which develop innovative protein degradation technology, also reached a cooperation of about US$1.
6 billion; in November, Novartis and Dunad Therapeutics reached a research and development cooperation agreement of more than US$1.
3 billion to develop a new generation of oral targeted protein degradation therapy
.


The field of macromolecules is at a turning point , and the global antibody drug market will exceed US$200 billion for the first time in 2021
.
Through intensive mechanism research, differentiated molecular design, clinical innovation exploration, target combination and combination therapy to return to clinical value, expand the field of "untreatable" diseases
.
The research and development of new biological drugs is not a single molecule, but a set of technologies, with "drug" as a specific form of expression; this drug can be a protein macromolecule, or mRNA or DNA delivered by cells or carriers, starting from the target.
role
.


New approaches and technologies such as multispecific antibodies, ADCs and nanobodies, cell and gene therapy, next-generation delivery technologies, etc.
, can expand into more “untreatable” disease areas
.
  
 
Cell and gene therapy is on the fast track, bringing new hope to incurable diseases.

Although cell and gene therapy-related products account for a relatively small proportion of the entire biological industry at this stage, they are the fastest growing in the global biopharmaceutical market in recent years.
one of the fields
.
2021 is the first year of cell therapy in China.
Two CD19-targeting CAR-T cell therapies from Fosun Kite and WuXi Junuo have been approved for marketing one after another, breaking China's gap in this therapeutic field.

.
Seven drugs based on CAR-T therapy have been approved in hematological tumors worldwide, establishing the status of CAR-T therapy in the treatment of various hematological malignancies; similarly, CAR-T therapy also exists in the field of solid tumor treatment.
There is a huge clinical demand, and clinical projects for the treatment of solid tumors with CAR-T targets such as Claudin 18.
2, GPC3, MSLN, HER2, and EGFR are widely carried out
.

 
From the perspective of technological iteration and accessibility, universal cell therapy is an inevitable trend in future development
.
The fastest-growing UCART19 therapy was jointly developed by Servier and Allogene, but was stopped by the FDA in October 2021 due to some safety risks in clinical trials, which also revealed that behind the high potential is extremely high development difficulty
.
In February 2021, Cytovia and Cellectis entered into a strategic research and development cooperation of up to US$760 million to develop iPSC NK and CAR-NK cells based on TALEN gene editing
.
In December 2021, the CTA101 UCAR-T (universal CAR-T) cell injection product independently developed by Beiheng Bio was accepted for clinical trials.
significant
.


Since 2018, the number and scale of private placement financing of immune cell therapy companies in China have continued to grow, with the number of financings rising from 11 in 2018 to 40 in 2021, and the scale of financing has exceeded US$1.
1 billion, with many incidents attracting attention large financing
.
Since 2020, domestic cell therapy companies have begun to enter the secondary market
.

 
Follow the national policy and overall plan to promote key tracks

.
The "14th Five-Year Plan" has put forward new overall goals for the development of the medical industry.



Local governments have successively issued relevant support policies to implement the central guidance.
Cell therapy, gene editing, synthetic biology and other tracks The positive highlights the













implementation of the prevention and control of major diseases and the integration of diagnosis and treatment

, focusing on major disease areas such as CVRM and CNS








The prevention and control of major diseases and the integration of diagnosis and treatment are the core areas of the development of the healthcare industry.








Single-point technological breakthroughs Small molecules and macromolecules

are emerging in an endless stream.
New products are returning to the essence of clinical needs
.
Gene therapy on the fast track, bringing new hope to incurable diseases













 
In the field of gene therapy, products are moving from the laboratory to the ward, and research and development and production have also entered the fast lane
.
Almost all of the top 20 pharmaceutical companies in Europe and the United States have entered the field of gene therapy through mergers and acquisitions or cooperation, and the entry of international pharmaceutical giants has become a booster for the development of the industry
.
In clinical practice, the efficacy and safety of gene therapy have been verified in several types of genetic diseases
.

 
Novartis' Zolgensma for the treatment of spinal muscular atrophy has sales of $634 million in the first half of 2021, a year-on-year increase of 69%, and is expected to enter the "$1 billion club"
.
In November 2021, the FDA granted priority review status to Bluebird Bio's β-thalassemia gene therapy Zynteglo Biologics License Application (BLA), making it the next gene therapy to receive FDA approval
.
In the field of diseases, gene therapy has also expanded from the treatment of genetic diseases to cardiovascular diseases (such as Tenaya Therapeutics, XyloCor Therapeutics, Rejuvenate Bio), metabolic diseases (such as Kriya Therapeutics, Verve Therapeutics) and infectious diseases (such as Beam Therapeutics), Gene therapy will become an indispensable form of medicine
.

 
The RNA platform has developed unprecedentedly, and the era of RNA therapy will finally come.

In 2021, RNA therapy is booming.
There are more than 600 RNA therapies in the development stage around the world.
Just looking at mRNA-based vaccines and therapies, the market size will grow at a CAGR of 16.
8%
.
Cooperation and transactions in the industry, as well as the continuous increase in capital markets, have accelerated the new wave of RNA therapy development
.

 
In August, Sanofi announced the acquisition of Translate Bio, which focuses on mRNA vaccine development, for $3.
2 billion; in the same month, Abogen Biosciences announced the completion of more than $700 million in Series C financing to support the clinical trials of its potential COVID-19 vaccine Develop and expand the pipeline of other vaccines and therapeutic candidates, refreshing the single pre-IPO financing amount of Chinese biopharmaceutical companies
.
In October, Hexion announced a $1.
3 billion collaboration with Silence Therapeutics to develop siRNA drugs targeting three targets, and a $450 million collaboration with OliX Pharmaceuticals to develop asymmetric small interferences for key targeted indications RNA therapy; in November, Novo Nordisk announced the acquisition of Dicerna Pharmaceuticals, which focuses on RNAi drug development, for US$3.
3 billion, which is expected to apply RNAi technology to fields such as diabetes, obesity, cardiovascular disease and NASH; in December, domestic RNA therapy ranked first The shares of Sirnaomics landed in the capital market
.


RNAi technology can regulate the expression of extracellular and intracellular proteins by directly targeting the base pairs of mRNA and other targeted RNAs in the cytoplasm, preventing the production of disease-related proteins, and promoting targeted degradation, thereby forming an effective targeted gene silence
.
Alnylam Pharmaceuticals, a pioneer in RNAi therapy, received the world's first RNAi drug ONPATTRO® (patisiran) in 2018, the second Givlaari® (givosiran) and the third OXLUMO® (lumasiran) RNAi drugs were also approved in 2019 and 2020.
batch
.
Alnylam is currently advancing RNAi therapy into more disease areas, including genetic diseases, cardiometabolic diseases, infectious diseases, central nervous system diseases and eye diseases
.


RNA-based therapeutics are showing great application potential, not only to expand the target range of drugs, but also to personalize the design of drugs to bring effective targeted therapy to currently incurable diseases
.
At present, RNA therapy has expanded from mainly targeting rare genetic diseases to more common clinical indications, such as cancer, cardiovascular diseases, and neurological diseases.
The era of RNA therapy will eventually come
.

 
Next-generation drug delivery technologies enable the development of more products
Drug delivery technologies enable the development of many products, from small and macromolecules, to cell and gene therapy, that rely on delivery technologies
.
With the continuous advancement of small molecules and antibody modification, the development of antibody-drug conjugates (ADCs), which combine antibodies with cytotoxic small molecules, can be administered in a highly targeted manner while providing synergistic Immunomodulatory function
.
Oligonucleotide drugs have the great advantage of programmed design and have been widely used in the research of targeted gene therapy.
Currently, 8 antisense oligonucleotides (ASO) and 4 small interfering RNA (siRNA) drugs have been launched on the market.
, multiple candidate therapies are in Phase 3 clinical trials, primarily for genetically defined rare diseases
.
With the gradual maturity of stem cell, gene editing and other technologies, stem cell-derived engineered red blood cell technology has developed rapidly.
By reprogramming hematopoietic stem cells in vitro, in conjunction with gene editing technology, the target gene encoding the drug is introduced into hematopoietic stem cells, followed by culture, Differentiate and mature so that drug-loaded erythrocytes can be produced directly
.
New therapeutic approaches present additional challenges, particularly with regard to stability (proteins and peptides), intracellular delivery requirements (nucleic acids), and manufacturing and scalability (cell therapy)
.


AI and synthetic biology empower the development of life science and technology.

"AI+Medical" brings more possibilities for healthy life and medical services, and "AI+Pharmaceutical" optimizes the process and method of traditional pharmaceuticals
.
Many international pharmaceutical companies have cooperated with AI pharmaceuticals, and domestic Internet giants are also scrambling to deploy the AI ​​pharmaceutical industry
.
At this stage, the most important value of AI pharmacy is to reduce the cost of evaluation and the number of iterations in the later stage through lower cost and high-quality investment in the early stage; while the core of AI pharmacy is not only to improve efficiency, but also to improve the success rate in the clinical stage
.
In the long run, AI is an indispensable and important element in the research and development of new drugs in the future
.

 
Synthetic biology has entered a period of rapid development in recent years, and its core idea is to transform life (cells, etc.
) or build a set of life systems from the source through engineering methods
.
Synthetic biology can not only use prokaryotic cells to regulate effector molecules in response to environmental stimuli, but also equip eukaryotic cells as chassis cells with biosensors to detect disease targets while activating downstream signaling pathways
.
At present, synthetic biology has been increasingly used in medical and health, agriculture, chemicals, energy, consumer goods, food and beverages and other fields.
The manufacturing capabilities of complex organisms have expanded the boundaries of biological applications and also leveraged huge economic value.

.

 
Looking forward
to the beginning of 2022, in the context of the country's continuous introduction of policies to encourage industrial innovation and development, the pharmaceutical industry is accelerating its entry into the "fast lane" of transformation and upgrading with lightning speed
.
In addition, with the accelerating process of internationalization and the prominent trend of differentiation and substitution of homogeneity, the pharmaceutical and biotechnology industries are facing both challenges and new opportunities
.

 
 
Disclaimer
 
This report is provided for your informational purposes only and is not intended or deemed to be an invitation to sell or purchase or subscribe for securities or an invitation to any particular person
.
The investments mentioned in this report may not be available in certain regions
.
Any investments mentioned in this report may involve considerable risk, some investments may not be readily available and may not be suitable for all investors
.
The value of investments or the income derived therefrom mentioned in this report may fluctuate due to foreign exchange rates
.
Past performance is not indicative of future performance
.
This report does not take into account any investor's investment objectives, financial situation or special needs
.
Investors should not rely solely on this report, but should use their own judgment to make investment decisions
.
Investors should seek professional advice before making any investment actions based on the recommendations of this report
.

 
The information contained in this report has been prepared by China Renaissance Group ("China Renaissance")
.
The sources of the information contained in this report are believed by China Renaissance to be reliable
.
The insights, analyses, forecasts, inferences and expectations contained in this report are based on such reliable data and are merely opinions
.
China Renaissance, its holding company and/or its subsidiaries and/or related individuals cannot guarantee the accuracy or completeness of this report
.
The information, opinions and projections contained in this report reflect the judgment of China Renaissance as of the date on which this report was originally issued and are subject to change without notice
.
China Renaissance, its holding company and/or its subsidiaries and/or related individuals shall not be liable for any direct or indirect or related losses suffered by any person due to the use of the materials in this report
.

 
This report is fully protected by copyright and material
.
No one may reproduce, distribute or publish this report for any purpose unless authorized by China Renaissance
.
China Renaissance Capital reserves all rights
.

 Unprecedented




development of the RNA platform, the era of RNA therapy is finally coming.
Next -








generation drug delivery technology enables the development of more products.



AI and synthetic biology empowers life science and technology development




prospects or be deemed an invitation to sell or buy or subscribe for securities or an invitation to any particular person .
The investments mentioned in this report may not be available in some regions




 


.
Any investments mentioned in this report may involve considerable risk, some investments may not be readily available and may not be suitable for all investors
.
The value of investments or the income derived therefrom mentioned in this report may fluctuate due to foreign exchange rates
.
Past performance is not indicative of future performance
.
This report does not take into account any investor's investment objectives, financial situation or special needs
.
Investors should not rely solely on this report, but should use their own judgment to make investment decisions
.
Investors should seek professional advice before making any investment actions based on the recommendations of this report
.

 
The information contained in this report has been prepared by China Renaissance Group ("China Renaissance")
.
The sources of the information contained in this report are believed by China Renaissance to be reliable
.
The insights, analyses, forecasts, inferences and expectations contained in this report are based on such reliable data and are merely opinions
.
China Renaissance, its holding company and/or its subsidiaries and/or related individuals cannot guarantee the accuracy or completeness of this report
.
The information, opinions and projections contained in this report reflect the judgment of China Renaissance as of the date on which this report was originally issued and are subject to change without notice
.
China Renaissance, its holding company and/or its subsidiaries and/or related individuals shall not be liable for any direct or indirect or related losses suffered by any person due to the use of the materials in this report
.

 
This report is fully protected by copyright and material
.
No one may reproduce, distribute or publish this report for any purpose unless authorized by China Renaissance
.
China Renaissance Capital reserves all rights
.

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