Gene editing tech

CRISPR's historic first approval sets the gene-editing trend

In November, the UK's Medicines and Healthcare products Regulatory Agency authorized CRISPR's drug for sickle cell disease and beta-thalassemia. The FDA is set to decide on treating sickle cell disease by December 8th.

CRSP stock soared as a result of this announcement, driving a 62.54% rise in gene editing-related stocks, sparking market attention and investment interest.

The meteoric rise in

gene-editing

Gene-editing technology brings forth a new beacon of hope in tackling congenital diseases. It directly targets the root cause by rewriting the "code" within the human body. Acting as a kind of miraculous "scissors," it precisely cuts DNA at specific locations, enabling addition, removal, or modification of the genetic code. This therapeutic approach involves altering and correcting DNA sequences to disrupt the function of harmful or inhibitory gene actions.

1994

DNA double-strand breaks serve as the foundation for developing gene-editing technology.

1996

The first generation of gene-editing technology — ZFNs.

2009

Second-generation gene editing technology - TALENs

2012

Third-generation gene editing technology - CRISPR

2016

CRISPR technological innovation, the invention of base editor launched CRISPR clinical experiments

November,2023

The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has formally approved the CRISPR/Cas9 gene editing therapy Casgevy (exa-cel) for listing.

December,2023

The US FDA and Australia's EMA are currently in the approval process and are expected to grant market approval for Casgevy (exa-cel) by the end of this month.

*Source: Nature. They are for reference only and do not constitute financial advice.

CRISPR gene-editing market potential exceeds $36 billion

Gene editing technology has progressed through various stages, experiencing significant improvements in precision, efficiency, and usability. The recent UK regulatory approval has established CRISPR as the forefront technology in genetic editing. Renowned for its simplicity, efficiency, affordability, and versatility, CRISPR has spurred worldwide scientific and industrial progress.

Between 2021 and 2022, the global gene editing industry saw a rise in market size from $4.811 billion to $5.412 billion, marking an annual growth rate of 12.49%, according to data from Allied Market Research and Global Market Research. Projections indicate a soaring market size of $36.06 billion by 2030, forecasting a compound annual growth rate of 22.3%.

Wall Street analysts hold an optimistic outlook regarding the eventual approval of CRISPR by the US FDA. BMO analysts anticipate a potential 20% surge in CRISPR's stock price. Additionally, analysts at William Blair have granted "outperform" ratings for both CRISPR and Vertex, echoing similar sentiments expressed by Barclays and Citibank. This collective positive sentiment has intensified market attention and discussion.

Clinical applications

Cancer

CRISPR primarily focuses on editing cells for various cell therapies like CAR-T, TCR-T, NK, and TIL.
The global market size for cell therapy was $7.2 billion in 2021, with an expected Compound Annual Growth Rate (CAGR) of 20% by 2030.

Eye Diseases

The global market for ophthalmic drugs surpassed $37 billion in 2022.
Genetic editing therapy holds distinct advantages in treating eye diseases due to the eye's unique physiological structure.

Hemato-logical Diseases

Thalassemia had a global market of $3.5 billion in 2022, while sickle cell disease reached $2.6 billion in 2021.
These treatments are currently the most probable to be launched.
The track for CRISPR gene therapy is the most competitive in this field.

Other rare diseases

Worldwide, around 7,000 rare diseases have been identified, with over 80% attributed to single gene defects. Conversely, only a few hundred prevalent diseases have available therapeutic drugs.
The global market for drugs addressing rare diseases reached $135.1 billion in 2020.

*Source: PEdaily.cn, Investing.com and impact-i.com

Is the CRISPR tool a worthwhile investment?

｜Suppliers of materials and technology

equipment and raw material suppliers

Interpretation: In life sciences, TMO is a key supplier in gene editing. They provide top tools and tech for research and practical use. Hillhouse Capital's 60.67% increase in holdings in Q2 suggests a positive outlook.

Technology service

LZAGF interpretation：Lonza excels in biopharmaceuticals, known for strong production and tech capabilities. Their expertise in cell therapy and biopharma makes them crucial in gene editing therapy production, a key market player.

MKGAF interpretation：Merck KGaA is a vital tech supplier for gene editing, providing essential tools and reagents. Their product line includes key reagents and tools for cell culture, genomics, and disease research. They excel in technological supply, innovative R&D, and strong clinical partnerships.

｜Research and development (R&D) and manufacturing

Research and preclinical study institutions

CRSP interpretation: CRISPR Therapeutics leads in gene editing. Their joint venture with Vertex Pharmaceuticals for CRISPR-Cas9 therapy gained UK market approval, propelling an impressive 83.95% surge since November 1st.

VRTX interpretation: VRTX concentrates on researching rare disease treatments and partners with CRISPR Therapeutics for pending gene editing therapies. Its upcoming cystic fibrosis therapy holds potential for profitability. Projected growth in 2024 and 2025 indicates an accelerated phase for the company, backed by Cathie Wood's investment.

SGMO interpretation：Focused on developing and commercializing novel transcription factors for gene regulation and modification, this clinical-stage biopharmaceutical company emphasizes new therapeutic strategies addressing unmet medical needs, particularly in the research, development, and commercialization of DNA-binding proteins.

Technology platforms

EDIT interpretation：As one of the world's renowned three major gene editing therapy companies, it has propelled the development of numerous bedside drugs for rare diseases. It has established deep collaborative ties with pharmaceutical giants like Bayer and BlueRock, showcasing promising investment potential.

NTLA interpretation：One of the leading three gene editing companies, specializing in the development of unique and potentially curative therapies using CRISPR/Cas9-based systemic biological tools. Since November 1st, the stock price has surged by 28.62%.

BEAM interpretation：Dedicated to developing a novel class of precise gene medicines using their proprietary base editing technology, this company is poised to become a leader in the gene editing domain. They've partnered with industry leaders like Pfizer (NYSE: PFE) and Eli Lilly and Co (NYSE: LLY). Since November 1st, their stock has surged by 36.94%.

｜Industrial applications

Healthcare

Interpretation：Focused on applying gene editing technology to develop gene therapies for rare genetic diseases and cancer, this company's CAR-T therapy and sickle cell disease gene editing therapy are expected to release clinical trial data by the end of next year. Since November 1st, the stock price has surged by over 30%.

Agricultural application

CTVA interpretation：Corteva possesses robust research and innovative capabilities in the seed sector, enhancing crops through gene editing and genetics technologies. Their potential for growth lies in seed improvement, digital agriculture, and sustainable farming practices.

BAYRY interpretation：They focus on seed improvement using gene editing and related technologies, aiming to develop crop varieties that are resilient, high-yielding, and resistant to pests and diseases.

*Source：Official website、research、Tiger community. They are for reference only and do not constitute financial advice.

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