Flagship Pioneering conceives, creates, resources, and grows first-in-category ventures to transform human health and sustainability. The company accelerates Flagship Pioneering’s advances in biology, engineering, medicine, energy, and beyond. The firm harnesses science and entrepreneurialism to envision alternative futures, beginning with seemingly unreasonable propositions and navigating to transformational outcomes through an evolutionary methodology. Flagship Pioneering combines innovative capacity, entrepreneurial leadership, and professional capital management in a single enterprise that systematically produces first-in-category companies.

Flagship Pioneering

Vesalius Therapeutics

Vesalius Therapeutics is focused on redefining the understanding and treatment of the diseases that drive ninety percent of human illness.

<div>Vesalius Therapeutics is an AI-powered biotechnology company leveraging its Continuum Discovery platform, which uses AI and machine learning to analyze human data, genetics, and proprietary experimental models for developing new therapies that can potentially treat more than 90% of human illnesses</div>

Artificial intelligence is identifying and testing new therapies faster than ever.

AI Drug Discovery

<div>
 
 Vesalius Therapeutics is an AI-powered biotechnology company leveraging its Continuum Discovery platform, which uses AI and machine learning to analyze human data, genetics, and proprietary experimental models for developing new therapies that can potentially treat more than 90% of human illnesses
 
 
 The platform identifies the diseases that stem from a common diagnosis but are in fact different in terms of symptoms, biological roots, and patient groups. It breaks down and classifies the diseases accordingly, enabling the company to develop drugs based on the specific disease profiles.
 
 
 The company is currently developing its internal clinical pipeline and expects to advance multiple programs forward by 2023/24. It is also in the process of entering into industry partnerships with pharmaceutical companies and drug developers.&nbsp;
 
 
 Funding and financials
 
 
 The company 
 <a href="https://sp-edge.com/updates/8871">
 emerged from stealth
 </a>
 in 
 <a href="https://sp-edge.com/updates/8871">
 March 2022
 </a>
 with an investment of USD 75 million from its founder Flagship Pioneering. The funds were allocated to fuel the company&rsquo;s R&amp;D efforts, and grow its workforce by 200 people by 2024.
 
</div>

<div>
 
 e-Therapeutics is a UK-based company specializing in drug discovery and development. It leverages computational biology to expedite the drug discovery process, particularly in RNA interference (RNAi) medicines. The company's approach integrates network analysis, machine learning, AI, data mining, and optimization into its proprietary databases and computational tools to identify and develop therapies.&nbsp;
 
 
 The company utilizes two platforms: 1) The HepNet Computational Platform, which is an in-silico (performed via computer simulation) laboratory that allows users to conduct hypotheses, phenotypic screening, and the identification and prioritization of novel therapeutic targets, and 2) the GalOmic RNAi Platform that enables highly specific silencing of gene expression and specializes in the development of RNAi therapies.
 
 
 Further, it has four drug candidates in the pipeline, namely 1) ETX-312 to treat metabolic dysfunction-associated steatohepatitis (MASH), 2) ETX-407 to treat dry age-related macular degeneration (dAMD), 3) ETX-148 to treat pan-haemophilia, and 4) ETX-291 to treat a broad spectrum of cardiometabolic diseases.
 
 
 Key customers and partnerships
 
 
 The company&rsquo;s customers include several pharmaceutical companies to develop therapies using its proprietary platforms. These include partnerships with 
 <a href="https://www.etherapeutics.co.uk/news-and-media/press-releases/e-therapeutics-signs-research-collaboration-agreement-novo-nordisk/">
 Novo Nordisk
 </a>
 (December 2018), 
 <a href="https://www.etherapeutics.co.uk/news-and-media/press-releases/collaboration-galapagos-discover-new-therapeutic-strategies-treatment-idiopathic-pulmonary-fibrosis-and-other-fibrotic-condition/">
 Galapagos
 </a>
 (June 2020), and 
 <a href="https://www.etherapeutics.co.uk/news-and-media/press-releases/immuno-oncology-research-collaboration-iteos-therapeutics/">
 iTeos Therapeutics 
 </a>
 (April 2022). It is also working with another undisclosed top-five pharma company in a specific area of neurodegeneration.&nbsp;
 
 
 Further in May 2018, the company partnered with C4X Discovery to identify new pathophysiological mechanisms in Parkinson&rsquo;s disease.&nbsp;
 
 
 Funding and financials
 
 
 In 2007, the company was listed on the Alternative Investment Market, the London Stock Exchange&rsquo;s public market for small and medium-sized growth companies, under the ticker symbol &ldquo;ETX&rdquo;. Further in 
 <a href="https://sp-edge.com/updates/13462">
 September 2022
 </a>
 , the company raised GBP 13.5 million (USD 15.1 million) from funds managed by institutional investor M&amp;G Investment Management Limited via the subscription of new ordinary shares. The funds were expected to be used for the company's growth initiatives.
 
</div>

eTherapeutics

<div>
 
 HiberCell is a biotechnology company that develops novel therapies focused on oncology. It entered the AI drug discovery space when it acquired AI genomics analysis company Genuity Science (and its subsidiaries in Iceland and Ireland) in August 2021.&nbsp;
 
 
 Genuity Science&rsquo;s solutions include end-to-end therapeutic discovery and development services such as building custom disease datasets, discovering and prioritizing targets, identifying biomarkers, and optimizing clinical trials. Genuity Science leverages its AI-powered platform to manage, process, and analyze genomic data. HiberCell intends to use Genuity Science&rsquo;s AI/ML capabilities to advance its oncology pipeline of five programs, of which one has reached the Phase 2 clinical trial stage.
 
 
 As of March 2024, the company&rsquo;s pipeline included three drug candidates, namely: 1) HC-7366, targeting the general control nonderepressible 2 (GCN2) kinase as part of the integrated stress response (ISR) signaling pathway; 2) HC-5404, aimed at inhibiting the PKR-like endoplasmic reticulum kinase (PERK) involved in the unfolded protein response (UPR) signaling pathway; and 3) odetiglucan, a therapy targeting tumor-immune interactions within the tumor microenvironment​.&nbsp;
 
 
 Key customers and partnerships
 
 
 In December 2023, HiberCell and Merck &amp; Co partnered to conduct a Phase Ib trial to evaluate the combination therapy of belzutifan and HC-7366 for treating clear cell renal cell carcinoma. Further, in January 2021, the company partnered with Biodesix to develop an enzyme-linked immunosorbent assay as a companion diagnostic test.
 
 
 Funding and financials
 
 
 In 
 <a href="https://hibercell.com/news/hibercell-closes-67-4-million-series-b-financing-and-secures-30-million-debt-facility-from-hercules-capital/">
 May 2021
 </a>
 , HiberCell completed a Series B financing round, raising USD 67.4 million, and secured an additional USD 30 million in debt financing from Hercules Capital. The funds were expected to be used for clinical advancement of its novel therapeutic candidates. 
 
</div>

HiberCell

<div>
 
 Arbor Biotechnologies specializes in gene editing to develop novel therapeutics for a variety of indications, starting with liver-related diseases. It operates an AI-powered biodiscovery platform to discover novel CRISPR enzymes, which are sequences of repeating genetic code. To do this, its platform deploys computational search techniques, genome sequencing, gene synthesis, and high-throughput screening. This can eventually be turned into drugs or even applied to agriculture and other industrial uses. In 2018, the company announced the discovery of Cas13d, a new type of gene editing enzyme that helps to alter RNA to treat genetic diseases.&nbsp;
 
 
 As of February 2024, the company had five internal drug discovery programs, of which one was in the IND-enabling stage. It also has multiple collaborative programs under development focused on oncology and various other therapeutic areas.
 
 
 Key customers and partnerships
 
 
 The company has a long-standing partnership with Vertex Pharmaceuticals since
 <a href="https://www.businesswire.com/news/home/20190103005194/en/Vertex-and-Arbor-Biotechnologies-Establish-Collaboration-to-Discover-Novel-Proteins-to-Advance-Discovery-of-Gene-Editing-Therapies">
 January 2019
 </a>
 , which focused on developing gene-editing treatments for cystic fibrosis and four other diseases. The alliance was further extended in 
 <a href="https://www.biospace.com/article/vertex-and-arbor-strike-a-1-2-billion-deal-for-gene-editing-programs/">
 August 2021
 </a>
 and 
 <a href="https://sp-edge.com/updates/15741">
 January 2023
 </a>
 .&nbsp;
 
 
 Other partnerships included a collaboration with 
 <a href="https://www.prnewswire.com/news-releases/arbor-biotechnologies-announces-collaboration-with-ginkgo-bioworks-to-advance-the-discovery-and-development-of-precision-gene-editors-302011991.html">
 Ginkgo Bioworks
 </a>
 (December 2023) to leverage Ginkgo's R&amp;D services to further optimize Arbor's novel precision editors, a non-exclusive licensing agreement with 
 <a href="https://www.globenewswire.com/news-release/2022/02/08/2380680/0/en/EdiGene-and-Arbor-Biotechnologies-Announce-Worldwide-Non-exclusive-License-Agreement-to-Advance-Ex-Vivo-Engineered-Cell-Therapy-Programs.html">
 EdiGene
 </a>
 (February 2022) to leverage Arbor&rsquo;s platform for ex vivo oncology therapies, a strategic research and non-exclusive licensing agreement with 
 <a href="https://investors.tcr2.com/news-releases/news-release-details/tcr2-therapeutics-and-arbor-biotechnologies-establish">
 TCR2 Therapeutics
 </a>
 (January 2022) to develop allogeneic TRuC T-cell therapies using Arbor&rsquo;s CRISPR technology, and a license agreement with 
 <a href="https://www.lonza.com/news/2020-12-23-14-00">
 Lonza
 </a>
 (December 2020) to use Arbor&rsquo;s gene-editing technology for Lonza&rsquo;s bioproduction products and services.
 
 
 Funding and financials
 
 
 In its latest Series B funding round in November 2021, which was led by Ally Bridge Group, TCG Crossover, and Temasek, Arbor Biotechnologies raised USD 215 million. The proceeds of the round were earmarked to fuel the company&rsquo;s clinical development pipeline.
 
</div>

Arbor Biotechnologies

<div>
 
 Based in the UK, Arctoris provides an automated drug discovery platform, offering fully validated research and development (R&amp;D) processes under an Experiments-as-a-Service model. The company works with biotechnology companies, AI drug discovery companies, pharma and academic institutions enabling these institutions to run experiments in Arctoris&rsquo;s platform and provides on-demand access to a broad scale of datasets for AI drug discovery companies to train their algorithms.
 
</div>

Arctoris

<div>
 
 Frontier Medicines leverages AI/ML technologies to develop treatments for cancer-causing proteins that are dismissed by traditional pharma companies as undruggable.&nbsp;
 
 
 The company has a proprietary chemoproteomic platform which entails an approach based on the interaction of small-molecule compounds and proteins to screen a large number of targets and combine them with a protein database that contains more than 150,000 novel binding pockets (hotspots). This enables it to access 90% of the human proteome that were not previously targeted due to lack of hotspots. The platform&rsquo;s AI/ML engine incorporates a library of diverse compounds, a hotspot map that serves as the starting point for understanding the druggability of a potential therapeutic target, and lead design and generation.&nbsp;
 
 
 The company focuses on oncology and immunology target and, as of February 2024, it had five wholly-owned therapeutic programs and three AbbVie-partnered programs in its pipeline. All programs were in their discovery stage, except the company&rsquo;s lead candidate, FMC-376 (activated and inactivated forms), focused on treating different cancer types, including non-small cell lung cancer (NSCLC), colorectal carcinoma (CRC), and pancreatic ductal adenocarcinoma (PDAC), which is in the IND-enabling stage.
 
 
 Key customers and partnerships
 
 
 The company entered a USD 100 million partnership with 
 <a href="https://news.abbvie.com/2020-12-02-AbbVie-and-Frontier-Medicines-Establish-Global-Partnership-to-Discover-and-Develop-Novel-Therapies-and-E3-Degraders-Against-Difficult-to-Drug-Targets">
 AbbVie
 </a>
 In December 2020 to develop small-molecule therapeutics against hard-to-drug protein targets including around 600 E3 ligases (the enzymes that drive the ubiquitination and degradation of proteins.&nbsp;
 
 
 Funding and financials
 
 
 In July 2021, the company raised USD 88.5 million in a 
 <a href="https://sp-edge.com/updates/4347">
 Series B
 </a>
 funding round co-led by Woodline Partners LP and RA Capital Management, with equal participation from Deerfield Management Company. The proceeds were earmarked for advancing the company&rsquo;s drug pipeline and expanding operations in Boston, where its new facility was to complement the company&rsquo;s headquarters in California. 
 
</div>

Frontier Medicines

Companies leverage GenAI to support drug discovery through accelerated drug designing

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Offers AI/ML-based solutions to accelerate drug discovery and development

Biotechnology companies that have integrated AI as a core component of their in-house drug development process. They typically have a few in-house drug development programs, and also collaborate with pharma companies to develop certain drugs.

Vesalius Therapeutics

AI Drug Discovery

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Vesalius Therapeutics launches with USD 75 million investment

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