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Horizon Discovery and Fulcrum Therapeutics Form Collaboration for Novel CRISPR-Based Target Discovery in Genetic Diseases

Exploration into the development of next-generation therapies

8 Sept 2016
Finn Price
Administrator / Office Personnel

Industry news

Horizon Discovery Group plc have announced a new collaboration with Fulcrum Therapeutics Inc., a US-based firm founded in July 2016 by Third Rock Ventures. The collaboration will utilize Horizon’s world-leading CRISPR-based screening platform to identify novel targets for regulating gene expression. The programme will initially focus on genetic diseases where no effective treatment options currently exist.

Horizon’s CRISPR-based screening platform, sophisticated bioinformatics, and cell line libraries provide a novel and highly efficient way to examine the regulation of genes and their role in disease. In this collaboration, Horizon will apply its platform and extensive know-how to identify novel gene regulation targets for further exploration by Fulcrum for the development of next-generation therapies.

The work will be engaged under a Full-time Equivalent (FTE) plus consumables model in a programme expected to run between five and seven months. Fulcrum has chosen Horizon as its preferred CRISPR-based screening platform service provider, and there is potential for further screens to identify targets for additional rare diseases after this initial programme.

Dr. Darrin Disley, Chief Executive Officer, Horizon Discovery commented, “CRISPR-based screening promises to be a powerful tool for the identification of novel targets and the collaboration with Fulcrum demonstrates the potential for this technology to be used in areas outside of oncology.” He continued: “This partnership confirms Horizon’s role as a preferred partner not only for established biotechnology and pharmaceutical companies, but also for start-ups looking for long-term collaborations rather than having to build in-house capabilities.”

Robert Gould, Ph.D. President and Chief Executive Officer, Fulcrum Therapeutics, commented, “Horizon’s reputation in gene editing and its applications, including CRISPR-based screening, is unparalleled. It was natural for Fulcrum to turn to Horizon as a long-term partner. With their deep scientific expertise, and with a broad IP portfolio underpinning their work, we are confident in a productive and exciting collaboration.”

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Cell Lines Stem Cells and Primary CellsPrimary cell cultures, established cell lines and stem cells are vital for <i>in vitro</i> and <i>ex vivo</i> experimentation. High-quality cells, optimized for your applications, alongside optimized cell substrates, growth medium and supplements, are critical for experimental success. Explore a range of cells suitable for your applications, including isogenic cell lines, competent cells, induced pluripotent stem cell (iPSC)-derived cell lines, fungal/bacterial/mammalian cell lines, stem cells and cancer cell lines. Find the best cells for your research in our peer-reviewed product directory: compare products, check customer reviews and receive pricing direct from manufacturers.Chem / BioinformaticsCheminformatics and bioinformatics are computational techniques used in chemistry and biology, respectively, for data acquisition, processing and storage. Cheminformatics focuses on compound information, whereas bioinformatics is mainly applied to analysis and modeling of genomics, genetic and sequencing information. Hardware and software is available for data acquisition, analysis, management and storage.DNA SequencingDNA sequencing, such as sanger sequencing, is a biological technique that determines the precise order of nucleotide bases in a fragment or template of DNA. DNA sequencers and genetic analyzers are based on capillary electrophoresis, where labeled DNA fragments are electrophoretically separated by size as they migrate through a polymer. Find the best DNA sequencing products, including DNA sequencing kits, genomic libraries and genetic identity kits in our peer-reviewed product directory: compare products, check customer reviews and receive pricing direct from manufacturers.Gene Expression and Molecular CloningMolecular cloning is a set of techniques that utilizes vectors to transfer recombinant DNA into host cells and is an essential tool for investigating the expression of genes and proteins in bacterial or mammalian cells. A variety of vectors optimized for gene cloning and expression in a range of host organisms are available, alongside competent cells for genetic replication. Here, you can explore a range of molecular tools, high-quality genomic and cDNA libraries, premade clones, transformation and transfection reagents and mutagenesis or gene expression detection assays and expression arrays. Find the best gene expression and molecular cloning products in our peer-reviewed product directory: compare products, check customer reviews and receive pricing direct from manufacturers.Next Generation SequencingNext-generation sequencing (NGS), also known as whole-genome sequencing, high-throughput sequencing and massive parallel sequencing, produces and analyses thousands to millions of nucleotide sequences at once. Sequencing systems operate via varying technologies depending on the manufacturer, including sequencing by synthesis, ligation, pyrosequencing, ion semiconductor and single-molecule real-time sequencing. For NGS, library preparation is paramount to successful sequencing. In this section, explore a range of library preparation kits, from targeted, amplicon-based or hybridization-based kits including epigenomic, transcriptomic and genomic workflows to fragmentation kits. Find the best next-generation sequencing products in our peer-reviewed product directory: compare products, check customer reviews and receive pricing direct from manufacturers.BioinformaticsBioinformatics uses computational models and methodologies to study biological processes and to understand biological data.DNADeoxyribonucleic Acid (DNA) is the main component of chromosomes and the carrier of genetic information of living organisms. Find out here about PCR, NGS, ChIP-Seq, gel imaging, and many other techniques which can be used for the analysis of DNA.SequencingSequencing refers to determining the order of nucleotides in a DNA or RNA molecule. This process is essential in genomics, diagnostics, and evolutionary biology. Techniques like next-generation sequencing (NGS) and Sanger sequencing provide high-throughput and precise data for analyzing genomes and transcriptomes. Browse our peer-reviewed product directory to find the best sequencing tools and reagents, compare products, check reviews, and get pricing directly from manufacturers.CollaborationTherapeuticsTherapeutics are treatments designed to alleviate or cure diseases. These include pharmaceuticals, biologics, and gene therapies, which work by targeting specific disease mechanisms. Advances in personalized medicine and biologics offer new hope for patients with conditions that were previously difficult to treat. Browse our peer-reviewed product directory to find therapeutic solutions for various diseases; compare products, check user reviews, and get pricing directly from manufacturers.Gene RegulationCRISPRCRISPR technology enables precise editing of genes, allowing scientists to modify DNA at specific locations. This revolutionary tool is used in genetic research, drug development, and gene therapy. CRISPR has applications in agriculture, disease treatment, and creating genetically modified organisms (GMOs). Explore CRISPR solutions in our peer-reviewed product directory; compare products, check reviews, and get pricing directly from manufacturers.

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Horizon Discovery and Fulcrum Therapeutics Form Collaboration for Novel CRISPR-Based Target Discovery in Genetic Diseases