Innovation Insights: Target validation for genetic targets

Innovation Insights: Target validation for genetic targets

Summary

Target validation is a crucial step in the drug discovery process, especially when it comes to genetic targets. This process involves confirming that a specific gene or protein is indeed involved in the disease pathway and is a viable target for therapeutic intervention. In this topic, we will explore the various methods and approaches used for target validation, including genetic knockout studies, RNA interference, and small molecule inhibitors. We will also discuss the challenges and limitations of target validation, as well as the importance of robust experimental design and data interpretation. By understanding the principles of target validation for genetic targets, researchers can increase the likelihood of developing successful and effective therapies for a wide range of diseases.

Genetic target validation is a critical step in the drug discovery process, confirming the potential of a target candidate as a therapy. In terms of key innovation areas, genetic target validation is considered a medium-impact innovation in the near term due to the availability of multiple products and solutions in this field.

Patent analysis shows a growing trend in genetic target validation, although the pace is slower compared to other innovations. Interestingly, universities and research institutions dominate the patent landscape, accounting for close to 50% of all patents filed in recent years. China-based companies also show dominance in patent trends, with 60% of all patents filed at the China patent office.

Competition in the field is focused on genomic analysis and organ/tissue-based techniques, with more companies now exploring genetic manipulation-based techniques. Leading patent filers include BGI Biotech and Illumina, with patents in genomic analysis and high throughput screening methods. Chinese companies like Biocytogen, GemPharma, and Surexam have technology cycle times lower than the industry average. Among big pharma companies, Alnylam, Regeneron, Vertex, and Johnson & Johnson have patents related to target validation.

In the startup ecosystem, companies like Emulate, Bi/Ond Solutions, and Insphero are leading the way in developing organoid/organ-on-a-chip models for target validation studies. Over 40 startups are active in this space, with Leapfrog Bio being the latest entry with a patent on Crispr screens for oncology targets. Startups are particularly focused on organ/tissue-based models and genetic manipulation techniques.

More than 200 universities are involved in genetic target validation research, with Stanford University and the Chinese Academy of Sciences leading the way.

In terms of deals related to genetic target validation, there have been 14,172 deals totaling US$11.4 billion. The United States has been a major player in this space, with a significant Grants deal worth $7.8 billion. The highest deal volume was seen in 2023 with 3,569 deals, while the highest deal value was recorded in 2020 at $4.1 billion. The slight decline in deals in 2021, followed by a modest increase in 2022, may indicate market adjustments and strategic consolidations. The United States leads in terms of deal amount and volume since 2020.

Key Highlights

• Target validation for genetic targets report is a high-impact innovation area in the Pharma industry, with a growing number of patents being filed and a focus on new indications.
• Big pharma companies like BGI biotech dominate the patent activity in Target Validation for genetic targets with a dip in new companies filing patents in recent years.
• Competitive intelligence shows that BGI biotech and illumina are among the leaders with strong innovation portfolios in Target Validation for genetic targets.
• Startups like surexam and Emulate are leading in terms of innovation impact and focus in the Target Validation for genetic targets.

• Innovation Insights: innovation examples by each use cases segment of various sectors to present key trends.
• Key player: This represents a sample list of key players in each use case highlighted in the report.
• Startups: This represents a sample list of emerging starups in each use case highlighted in the report.
• University: This represents a sample list of leading universities in each use case highlighted in the report.

• Gain a deeper understanding of the critical role of Target validation in drug discovery.
• This report provides comprehensive insights into the various methods used for validating genetic targets, including genetic knockout studies, RNA interference, and small molecule inhibitors.
• It highlights the challenges and limitations of target validation while emphasizing the importance of robust experimental design and data interpretation. With valuable data on the latest trends and best practices, this report equips researchers and decision-makers with the tools needed to increase the success rate of developing effective therapies for a range of diseases.
• Stay ahead of the curve and make informed decisions in the fast-evolving field of genetic target validation.