Published: August 2021
Considering its potential to intricately alter genetic DNA, the CRISPR technology is being widely used to develop viable therapeutics for the treatment of rare and complex genetic disorders, which are currently considered untreatable
With over 115 candidates under development for various indications, the future of this niche market seems promising. The year 2020 was particularly eventful, with Emmanuelle Charpentier and Jennifer Doudna receiving the Nobel Prize for their work on the CRISPR technology and several other important milestones, such as the approval of CRISPR based diagnostics for COVID-19, discovery of novel Cas proteins, and the progression of promising leads based on this technology, in clinical trials. Promising trial results have garnered the attention of notable investors in the healthcare sector and the current focus of research in this field is on developing advanced delivery vehicles for CRISPR based therapeutics.
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115+ CRISPR based therapeutics are currently being evaluated in different phases of development
Nearly 80% of the therapeutic candidates are in discovery / preclinical stage, while the rest (20%) are under clinical evaluation. It is worth noting that no CRISPR based therapeutics have progressed beyond phase I/II trials. Interestingly, most of product candidates in the clinical stage are ex vivo therapeutics, whereas, several of the early stage leads are in vivo therapeutics.
Several technical advances have been made to facilitate the development of viable CRISPR based therapeutic leads
Over 50% of product candidates are being developed using the CRISPR/Cas9 platform. Certain companies are evaluating novel Cas proteins, including Cas3 and Cas12a, in their therapy development programs, some of which have advanced to clinical trials. Recently, the use of dCas proteins to modulate CRISPR and single base editing technologies, have contributed to improve the safety of this gene editing technology.
More than half of the companies engaged in this domain are based in North America
The developer landscape is dominated by start-ups (established after 2012, having 1-200 employees), which represent around 71% of total number of stakeholders in the industry. Several start-ups based in the US, are university spin-offs and have licenses for foundational IP from their parent research institutes. It is important to highlight that more than 12 big pharma companies are also involved in CRISPR based therapy development.
Over USD 5 billion has been invested by both private and public investors, since 2014
USD 3.4 billion was raised through public investments, representing more than 60% of the total capital raised, till February 2021. The other popular financing instrument in this field was venture capital funding, which accounts for 34% of the total number of instances and USD 1.8 billion worth of capital raised in the given time period.
More than 2,700 patents have been filed / published related to the CRISPR technology, as of December 2020
Of these, around 78% were patent applications, while the remaining (12%) were granted patents. Around 22% of the total number of applications were related to CRISPR based therapeutics, while the rest were associated with related proteins / nucleases, diagnostic applications and delivery systems.
Partnership activity in this market has grown at a CAGR of 21.1%, during 2015-2021
One of the key drivers of such deals was technology licensing to support R&D and product development efforts, representing more than 65% of the agreements mentioned in the report. Interestingly, of all the licensing agreements inked, 30% involved technologies facilitating the delivery of CRISPR system components into cells.
North America is anticipated to capture over 58% of the market share by 2030
Since the current product landscape primarily features early-stage candidates, the first batch of approved therapies is expected to enter the market by 2024. Owing to the personalized nature of such therapies and a high price tag, initial adoption is expected to be slow. However, with the launch of in vivo therapeutics, the demand is anticipated to grow at a relatively faster pace.
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The USD 2.2 billion (by 2030) financial opportunity within the CRISPR therapeutics market has been analyzed across the following segments:
The report also features inputs from eminent industry stakeholders, according to whom, the therapeutic potential of the CRISPR technology will soon be realized across multiple application areas, including xenotransplantation, in the foreseen future. The report includes detailed transcripts of discussion held with prominent industry representatives.
The research includes profiles of key players (listed below); each profile features a brief overview of company, its proprietary gene editing technology, pipeline details, recent developments (including collaborations and expansions) and an informed future outlook.
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