Bohdana Sokolova

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7 Biotechs Fighting Rare Diseases With Gene-Editing Tech

7 Biotechs Fighting Rare Diseases With Gene-Editing Tech

As of now, only 5% of so-called rare diseases (orphan diseases) have an FDA-approved treatment available to patients. Rare diseases are defined as those affecting fewer than 1 in 200.000 people with almost 80% of all rare diseases being of genetic nature.

With recent progress in gene-editing technologies, including breakthroughs like CRISPR/Cas9 and new therapy delivery technologies, there is a wave of innovative biotech companies entering the field of rare diseases equipped with new ways to address the problem. 

In this article, we would like to outline a list of notable companies targeting rare diseases with cutting-edge gene therapies.  


9 Notable AI-powered Biotech Companies Founded in 2021

9 Notable AI-powered Biotech Companies Founded in 2021

Ever since the early 2000s, Artificial Intelligence (AI) has become a useful tool in various fields, from game design and speech recognition to powering driving cars and military applications. No wonder that big pharma companies are striving to keep up with technological progress and implementing AI in their internal R&D and business workflows. Pharmaceutical giants are actively recruiting machine learning and AI experts: for instance, from January 2020 to June 2021 AstraZeneca had 676 job vacancies for AI tech specialists, being followed by Johnson & Johnson and Takeda Pharmaceuticals with 616 and 545 positions respectively. Another source of technological and artificial intelligence expertise comes from external vendors by collaborating with AI-powered startups and more established companies. The cumulative number of such collaborations has increased significantly over the 2016-2021 timeframe, from 29 to 266, and continues to grow. For instance, in 2022 Merck launched a collaboration with Absci Corporation in order to produce enzymes tailored to Merck’s biomanufacturing applications with help of artificial intelligence, and Janssen partnered with a French startup Aquemia aiming to predict the potency of small molecules for a given target based on quantum physics-based calculations for datasets selected by Janssen.

Below is a chart illustrating the increasing number of R&D collaborations involving "big pharma" and AI-driven drug discovery companies (mostly startups and scaleups).



The world of AI-driven startups in biotech is rapidly evolving, according to ‘The Landscape of Artificial Intelligence (AI) in Pharmaceutical R&D’ number of AI-driven companies in drug discovery and development increased from 45 in 2010 to 343 firms in 2021. Some companies in this list offer AI-driven computational tools and services for the key stages of a drug discovery pipeline — from finding consumables and reagents for chemical synthesis to clinical development and biomarker discovery. Others are building internal drug discovery pipelines using advanced AI-driven drug design platforms.

I this post I have summarized an alphabetically-ordered list of 9 biotech companies that were founded in 2021 and are developing innovative AI-powered solutions either for internal use, or as a part of service offering to clients.