INDUSTRIAL IMPACT, MEDICAL
Accesswire | March 24, 2023
Moderna, Inc. a biotechnology company pioneering messenger RNA therapeutics and vaccines, and Generation Bio Co. a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, today announced that the two companies have entered into a strategic collaboration to combine Moderna's biological and technical expertise with core technologies of Generation Bio's non-viral genetic medicine platform. The collaboration aims to expand the application of each company's platform by developing novel nucleic acid therapeutics, including those capable of reaching immune cells, to accelerate their respective pipelines of non-viral genetic medicines.
"Moderna continues to invest in innovative technology to enable us to develop a breadth of transformative medicines for patients," said Rose Loughlin, Ph.D., Moderna's Senior Vice President for Research and Early Development. "Through this collaboration, which builds on Generation Bio's non-viral genetic medicines platform, we have the potential to target immune cells with diverse nucleic acid cargos and the liver for gene replacement. We are excited to have Generation Bio as our partner as we continue to broaden our therapeutic pipeline and extend the potential benefit of nucleic acid therapeutics to more patients."
"Non-viral DNA therapeutics may offer durable, redosable, titratable genetic medicines to patients suffering from rare and prevalent diseases on a global scale," said Phillip Samayoa, Ph.D., Chief Strategy Officer of Generation Bio. "This collaboration represents a foundational investment in our platform science, both deepening our pipeline of rare and prevalent liver disease programs beyond hemophilia A and accelerating our work to reach outside of the liver with nucleic acid therapies. We are thrilled to collaborate with Moderna to extend genetic medicines to new tissues and cell types through the joint development of novel targeting for our stealth ctLNPs to reach immune cells."
About the Collaboration
Under the terms of the agreement, Moderna may advance two immune cell programs, each of which may use a jointly developed ctLNP to deliver ceDNA. In addition, Moderna may advance two liver programs, each of which may use a liver-targeted ctLNP developed by Generation Bio to deliver ceDNA. Moderna retains an option to license a third program for either immune cells or the liver.
Generation Bio will receive a $40 million upfront cash payment and a $36 million equity investment issued at a premium over recent share prices. Moderna will fund all collaboration work, including a research pre-payment. Generation Bio is also eligible for future development, regulatory and commercial milestone payments, as well as royalties on global net sales of liver-targeted and immune cell-targeted products commercialized under the agreement. The agreement additionally provides Moderna with the right, subject to certain terms and conditions, to purchase additional shares of common stock in connection with a future equity financing by Generation Bio.
Further, Moderna and Generation Bio will both leverage collaboration research to continue to advance in vivo immune cell targeting as a new class of genetic medicines, with downstream economics on products utilizing such technology. Generation Bio is eligible to receive certain exclusivity fees as well as potential development and regulatory milestones and royalties on products that Moderna advances using ctLNP technology developed under the collaboration.
About Moderna
In over 10 years since its inception, Moderna has transformed from a research-stage company advancing programs in the field of messenger RNA (mRNA), to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across seven modalities, a broad intellectual property portfolio in areas including mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant that allows for rapid clinical and commercial production at scale. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has allowed for the pursuit of both groundbreaking science and rapid scaling of manufacturing. Most recently, Moderna's capabilities have come together to allow the authorized use and approval of one of the earliest and most effective vaccines against the COVID-19 pandemic.
Moderna's mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, and has allowed the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases. Moderna has been named a top biopharmaceutical employer by Science for the past eight years.
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MEDTECH, MEDICAL
Businesswire | May 23, 2023
Twist Bioscience Corporation a company enabling customers to succeed through its offering of high-quality synthetic DNA using its silicon platform, today announced the launch of a portfolio of RNA sequencing tools, which includes the Twist RNA Exome, Twist RNA Library Prep Kit and the Twist Ribosomal RNA (rRNA) & Hemoglobin (Globin) Depletion Kit, as well as custom target enrichment capabilities for RNA and whole transcriptome sequencing. This comprehensive suite of RNA tools can enable targeted or whole transcriptome research across fields including precision medicine, biomarker discovery and immuno-oncology research.
“Twist’s RNA sequencing portfolio builds on our established NGS capabilities in genomic sequencing and methylation detection and expands into gene expression analysis, opening a new and significant market for Twist,” said Emily M. Leproust, Ph.D., CEO and co-founder of Twist Bioscience. “This offering allows our customers to leverage Twist’s leading target enrichment platform to discover and resolve low abundance RNA transcripts in the human transcriptome. Our RNA sequencing workflows are designed specifically for challenging samples. By enriching relevant transcripts, we can enable researchers to get high-quality data from a range of sample types, including damaged RNA.”
Unlike the genome, which is relatively static, the transcriptome changes over time in response to varying disease states and drug therapies. Gene expression levels also differ across tissues and cell types. To fully understand this dynamic quality of RNA, researchers often sequence RNA samples from the same source at multiple time points, incurring additional costs and resource burdens. Each component of the Twist RNA sequencing portfolio is purpose-built to maximize data quality, minimize workflow inefficiency, decrease wasted sequencing reads, and support comprehensive transcriptomic profiling across even low-quality and low-input samples common in oncology.
The Twist RNA sequencing portfolio provides end-to-end workflows that can be used to study total RNA and measure the whole transcriptome, protein coding sequences of messenger RNA (mRNA) or custom targets. These products can also be integrated into customers’ existing workflows. Twist RNA sequencing tools enable minimal hands-on time and increase the scale of samples sequenced through utilizing automation. They are compatible with a wide range of sample inputs, including difficult or low-quality samples. RNA panel designs can also now incorporate Twist’s new exon-aware design algorithm, which reduces bias in detection and can provide an important tool for researchers studying rare gene transcripts that drive tumor biology.
Twist Targeted RNA Sequencing Workflow
Twist target enrichment for RNA leverages the performance, efficiency and sensitivity of Twist target enrichment for sequencing of RNA transcripts of interest across samples and species. It can be used to enrich relevant transcripts with the RNA Exome or in custom RNA panels.
Twist RNA Exome
The Twist RNA Exome with Twist’s exon-aware design algorithm enables targeted sequencing of the protein coding regions of the human transcriptome without bias toward specific combinations of exons. The Twist RNA Exome covers up to 99.9% of protein coding sequencing in Gencode and RefSeq databases. This coverage, along with the exon-aware design approach, could enable researchers to capture important transcripts including those in low input and degraded samples from formalin fixed paraffin embedded (FFPE) sections.
Twist Whole Transcriptome Sequencing Workflow
Twist Whole Transcriptome Sequencing can be used to discover novel transcripts, including low expressing genes and novel isoforms. It can be used with a variety of samples including whole blood, fresh and frozen tissue samples and low quality samples, such as FFPE samples. The Twist rRNA & Globin Depletion Kit can be used with this workflow to deplete rRNA and hemoglobin targets so that researchers can study the rest of the transcriptome. The full workflow can be completed in less than five hours with minimal hands-on time.
About Twist Bioscience Corporation
Twist Bioscience is a leading and rapidly growing synthetic biology and genomics company that has developed a disruptive DNA synthesis platform to industrialize the engineering of biology. The core of the platform is a proprietary technology that pioneers a new method of manufacturing synthetic DNA by “writing” DNA on a silicon chip. Twist is leveraging its unique technology to manufacture a broad range of synthetic DNA-based products, including synthetic genes, tools for next-generation sequencing (NGS) preparation, and antibody libraries for drug discovery and development. Twist is also pursuing longer-term opportunities in digital data storage in DNA and biologics drug discovery. Twist makes products for use across many industries including healthcare, industrial chemicals, agriculture and academic research.
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INDUSTRIAL IMPACT, MEDICAL
Synthego | March 17, 2023
Synthego Corp., a leading genome engineering solutions provider, announced today the launch of its service partner ecosystem, CRISPR Discovery Partners, designed to accelerate drug discovery by providing a seamless research workflow from CRISPR editing to phenotypic data and downstream services. The ecosystem brings together high-value partners including Arctoris, BrainXell, Curia, PhenoVista, and Pluristyx to offer a coordinated solution for CRISPR-driven drug discovery.
The CRISPR Discovery Partner ecosystem creates an integrated workflow starting with Synthego's CRISPR Engineered Cells through functional readouts, providing researchers with a faster, more efficient, and more reliable way to move their discoveries forward. Researchers can access the full range of genome engineering services through Synthego, as well as downstream services including small molecule screening, cell-based assays, high content imaging, iPSC differentiation, and multi-omics through partners. The result is a streamlined drug discovery process that can help accelerate the development of life-saving treatments.
Synthego's CRISPR Discovery Partners include
Arctoris, a tech-enabled drug discovery service company, combines wet lab automation and machine learning with a world-class team of experienced drug discovery researchers to enable the accelerated development of small molecule, biologics, and cell therapy assets.
BrainXell provides differentiation services to generate high-purity, iPSC-derived human neurons, astrocytes, and microglia for research use with a focus on drug discovery. BrainXell also offers fibroblast reprogramming and select downstream assay services.
Curia, a global contract research, development and manufacturing organization (CDMO), offers products and services from discovery through manufacturing to help customers transform R&D results into real-world impact.
PhenoVista, a San Diego-based CRO, specializes in modern, human, cell-based in vitro assay development and analysis. Internationally recognized for developing and implementing both fully bespoke projects and pre-validated Ready-2-Go assay services, this award-winning research partner leverages high-content imaging to enable life scientists to accelerate their discovery of novel therapeutics and improve global health.
Pluristyx offers expertise and tools to support and accelerate the development and commercialization of revolutionary therapeutics to treat serious human diseases by providing unique products, consulting, and contract services, including cell line expansion and banking.
About Synthego
Synthego is a genome engineering company that enables the acceleration of life science research and development in the pursuit of improved human health. The company leverages machine learning, automation, and gene editing to build platforms and products for science at scale. With its foundations in engineering disciplines, the company's platform technologies vertically integrate proprietary hardware, software, bioinformatics, chemistries, and molecular biology to advance basic research, target validation, and clinical trials. With its technologies cited in hundreds of peer-reviewed publications and utilized by thousands of commercial and academic researchers and therapeutic drug developers, Synthego is at the forefront of innovation enabling the next generation of medicines by delivering genome editing at an unprecedented scale.
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