Globenewswire | May 29, 2023
Monte Rosa Therapeutics, Inc. a clinical-stage biotechnology company developing novel molecular glue degrader (MGD)-based medicines, announced its second development candidate, MRT-6160, a novel, potent, and selective molecular glue degrader of VAV1. The Company plans to file an Investigational New Drug (IND) application for MRT-6160 in the first half of 2024 and to develop the molecule as a potential treatment for autoimmune diseases.
“MRT-6160 is a potent, orally bioavailable MGD designed to degrade VAV1, an important protein involved in the signaling pathways of T and B cells. Our in vitro studies have shown that MRT-6160 selectively degrades VAV1 without detectable effects on other proteins. By targeting VAV1, MRT-6160 attenuates multiple aspects of T- and B-cell function and inhibits disease progression in established in vivo models of autoimmunity,” said Owen Wallace, Ph.D., Chief Scientific Officer of Monte Rosa. “The underlying biology and our preclinical data both demonstrate that MRT-6160 acts as an immune modulator, which has the potential to avoid the broad immune suppression seen with other approaches. We look forward to progressing our clinical plan developed with the goal of providing early insights into safety, PK and PD, and proof of concept regarding differentiated effects on key immunomodulatory signaling pathways.”
“Our goal centers on pioneering therapeutically meaningful new drugs for patients with serious diseases. By addressing VAV1, a validated but previously undruggable target, we believe we've created a potentially groundbreaking therapy for patients suffering from a range of serious autoimmune conditions, particularly those involving both T- and B cell-mediated autoimmunity,” said Markus Warmuth, M.D., CEO of Monte Rosa. “MRT-6160 is expected to be our second MGD to enter clinical trials, showcasing the continued productivity of our QuEEN™ platform. We anticipate significant progress and milestones in our portfolio in the upcoming year, including initial clinical data from our GSPT1 MGD, MRT-2359, in the second half of this year and filing of an IND application for MRT-6160 in the first half of next year.”
About Monte Rosa
Monte Rosa Therapeutics is a biotechnology company developing novel molecular glue degrader (MGD) medicines for patients with serious diseases such as oncology, autoimmune and inflammatory diseases. MGDs are small molecule protein degraders that employ the body’s natural mechanisms to selectively eliminate therapeutically relevant proteins. The Company’s QuEEN™ (Quantitative and Engineered Elimination of Neosubstrates) platform enables it to rapidly identify protein targets and design highly selective degraders by combining diverse libraries of proprietary MGDs with in-house proteomics, structural biology, AI/machine learning, and computational chemistry capabilities.
PRNewswire | June 02, 2023
During this year's AAPS National biotechnology conference, Navigate BioPharma Services, Inc., a Novartis subsidiary and a leading provider of innovative biomarker solutions for drug development, announced the launch of its new multiplex digital PCR assays for comprehensive quantification of multiple chimeric antigen receptor (CAR) T cells, a type of cancer immunotherapy that involves genetically modifying T cells to recognize and destroy certain cancers. These assays are based on the Qiagen QIAcuity digital PCR System, which offers a powerful yet easy-to-use digital PCR experience through its unique nanoplate system.
The new multiplex digital PCR assay enables simultaneous measurement of the number, function and persistence of CAR-T cells in clinical blood and bone marrow samples, without the need for a standard curve. It can detect as low as 0.01% of CAR-T cells among normal T cells, with a turnaround time of less than two hours. These assays can measure up to five different CAR-T cell markers in a single reaction, using specific probes for different CAR targets, to quantitate multiple CAR-domains, or combinations of commonly used therapeutic targets for CAR-T therapies. The assay can help clinicians monitor response or safety events during clinical trials to enable the development of new therapies.
"CAR-T therapy is a promising and innovative approach to treat cancer, but it requires accurate and reliable quantification of CAR-T cells for successful drug development," said Dr. Nathan Riccitelli, Associate Director of Innovative Technologies at Navigate BioPharma Services, Inc. "Our new proprietary multiplex digital PCR assay provides a comprehensive and robust solution for measuring CAR-T cells in clinical samples with high sensitivity, specificity and reproducibility."
Navigate BioPharma Services, Inc. has developed this assay for use in sponsors-led clinical trials. If interested, contact them about how this assay can be used in upcoming clinical trial studies.
Navigate Biopharma Services, Inc.
Navigate BioPharma Services, Inc., an independently operating subsidiary within the Novartis group of companies, is a leading provider of innovative biomarker and specialty bioanalytic solutions for clinical development. Over the past decade, Navigate BioPharma has built and developed its exclusive library of assays, methodologies, and biomarker solutions. Their extensive expertise offers unique, accurate, reliable, and cost-effective solutions for its customers and partners.
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.