Rumi Scientific

Rumi Scientific is a research-based company dedicated to developing medications and novel treatments for human genetic illnesses of the central nervous system. It has created an interesting human organoid-based platform that may be used for drug discovery and high-throughput screening in a variety of situations. Rumi Scientific has developed a platform that replicates human development in vitro by combining genetic models of human diseases introduced by gene editing with multi-cellular self-organization to construct defined diseased embryonic tissues. This provides a basis for drug screening using artificial intelligence. By focusing on synthetic human embryology in both normal and disease contexts, the company aims to unlock new doors toward treatments for many genetic disorders that still have no cures.

Rumi Scientific is a biopharmaceutical company focused on developing therapies for rare and neurodegenerative diseases, utilizing its proprietary high-throughput organoid phenotypic screening platform. Their lead program is an orally available bromodomain-containing protein nine (BRD9) inhibitor currently in lead optimization for the treatment of Huntington's disease (HD).

Rumi Scientific Announces Partnership with X-Chem for Development of Lead Candidate in Huntington’s Disease

Partnership aims to advance disease modifying therapy for Huntington’s disease

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

AI Drug Discovery

RUMI Scientific announces Receipt of Two-Year Phase II SBIR

Rumi Scientific is a biopharmaceutical company focused on developing therapies for rare and neurodegenerative diseases, utilizing its proprietary high-throughput organoid phenotypic screening platform. Their lead program is an orally available bromodomain-containing protein nine (BRD9) inhibitor currently in lead optimization for the treatment of Huntington's disease (HD).
Through extensive research, Rumi has identified BRD9, an epigenetic reader, as a novel therapeutic target for HD with the potential to modify the disease's progression. The company has successfully validated this target in the R6/2 model of HD and in cellular models that replicate various aspects of the HD phenotype. Notably, their studies have demonstrated that lowering BRD9 levels can restore striatal volume, a significant finding indicating disease-modifying potential.
Rumi Scientific's proprietary platform enables the production of synthetic human tissues, providing more predictive data and potentially leading to a safer and faster clinical trial process. The company was founded in 2016 and licensed foundational technology from The Rockefeller University, developed by co-founders Ali H. Brivanlou, Ph.D., and Eric D. Siggia, Ph.D.
In December 2023, Rumi Scientific announced a partnership with X-Chem, a leading provider of innovative solutions in early-stage drug discovery. Through this collaboration, X-Chem will leverage its expertise in screening, computational chemistry, machine learning, and specialized synthesis to develop a lead candidate BRD9 inhibitor for the treatment of HD.

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 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. 
 
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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.
 
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Arbor Biotechnologies

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’s platform and provides on-demand access to a broad scale of datasets for AI drug discovery companies to train their algorithms.

Arctoris

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 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. 
 
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Frontier Medicines

<div>
 
 Verge Genomics is a biotechnology company engaged in developing therapeutics for neurological diseases using human genomics and AI to accelerate drug discovery. The main target areas are Amyotrophic Lateral Sclerosis (ALS), Parkinson&rsquo;s Disease (PD), and Frontotemporal Dementia (FTD).&nbsp;
 
 
 It has built a proprietary all-human platform (unlike standard clinical trials that rely on animal data), called CONVERGE, that uses data sets from brain tissue samples of deceased patients with ALS and PD. While most drugs target only one gene at a time, the company claims its technology can identify hundreds of genes that cause disease and develop drugs that target all the genes at the same time. The company uses AI technology to optimize the drug discovery process as a whole.
 
 
 As of March 2024, Verge had one drug candidate in the clinical stage (Phase I) and 10 drug candidates in the discovery stage.&nbsp;
 
 
 Key customers and partnerships
 
 
 The company has partnerships with various tissue banks (such as Harvard University and University of Miami Miller School of Medicine) and several educational institutes and foundations (such as Target ALS, Answer ALS) that enabled its drug discovery platform.&nbsp;
 
 
 In addition, the company&rsquo;s customers include several pharmaceutical companies, such as 1) 
 <a href="https://www.vergegenomics.com/news-blog/verge-genomics-announces-research-collaboration-with-the-sheffield-institute-for-translational-neuroscience">
 Sheffield Institute for Translational Neuroscience (SITraN)
 </a>
 to accelerate the validation of novel drug targets and screening of drug compounds for Parkinson's disease (February 2021), 2) 
 <a href="https://sp-edge.com/updates/4146">
 Eli Lilly
 </a>
 to research and develop novel therapies to treat ALS (July 2021), 3) 
 <a href="https://sp-edge.com/updates/21707">
 Alexion
 </a>
 to identify novel drug targets for rare neurodegenerative and neuromuscular diseases (September 2023), and 4) 
 <a href="https://www.vergegenomics.com/news-blog/verge-genomics-and-the-pritzker-neuropsychiatric-disorders-research-consortium-form-cutting-edge-schizophrenia-research-collaboration">
 Pritzker Neuropsychiatric Disorders Research Consortium
 </a>
 to analyze data from schizophrenic patient&rsquo;s brain tissue with Verge&rsquo;s technology (October 2023).
 
 
 Funding and financials
 
 
 In December 2021, Verge raised USD 98 million in an oversubscribed 
 <a href="https://sp-edge.com/updates/7426">
 Series A
 </a>
 funding round led by BlackRock. The funds were used to scale up its pre-clinical and clinical candidate programs in the pipeline and enhance its lead therapeutics through clinical testing. 
 
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Verge Genomics

SaaS (Software-as-a-Service) companies providing their ML platform to other pharma companies for drug discovery services such as target selection, validation and lead optimization.

SaaS (Software-as-a-Service) companies providing their ML platform to other pharma companies to design and run preclinical experiments.

SaaS (Software-as-a-Service) companies providing their ML platform to other pharma companies to identify biomarkers (measurable biological signs used to determine disease progression) and predict drug response.

SaaS (Software-as-a-Service) companies aggregating and analyzing biomedical data with the help of AI to generate insights across the drug development pipeline.

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.

Includes companies that use GenAI as a core part of their product offering for drug discovery & development

Companies leverage GenAI to support drug discovery through accelerated drug designing

Companies offer AI/ML-based SaaS products to accelerate the discovery and development of novel therapeutics

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

Rumi Scientific

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