Rgenta Therapeutics Launches with $20 Million Seed Investment

BioSpace | April 02, 2020

Rgenta Therapeutics, a new biotechnology company focusing on developing RNA-targeting medicines for historically undruggable disease-relevant targets, announced today that it closed a $20 Million seed investment, co-led by Boehringer Ingelheim Venture Fund and Matrix Partners China. Rounding out the series seed syndicate are two additional investors, Kaitai Capital and Legend Star Fund. The company has assembled a world-class team of small-molecule drug developers and an exceptional Scientific Advisory Board in the fields of genomics and RNA biology.

Spotlight

Interesting to note, China holds the top spot for patents while the U.S. is dominant in biotech discoveries.

Spotlight

Interesting to note, China holds the top spot for patents while the U.S. is dominant in biotech discoveries.

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AI

Labcorp and PathAI Expand their Partnership to Accelerate Use of AI-Powered Pathology

PathAI, Labcorp | March 09, 2021

PathAI, a global supplier of artificial intelligence-powered (AI-powered) innovation for use in pathology research, and Labcorp, a main global life sciences company, today declared the expanding of their essential organization in the field of AI-powered pathology. The coordinated effort expands on Labcorp's past investment in PathAI and the companies' cooperation on a progression of projects, remembering the deployment of PathAI algorithms for clinical trials oversaw by Labcorp Drug Development. A critical focal point of the extended joint effort will be to empower, in planned clinical trials of cancer and different diseases, the seamless deployment of PathAI's algorithms in the expansive arrangement of projects oversaw by Labcorp Drug Development. These PathAI algorithms are created utilizing Good Clinical Laboratory Practice (GCLP) rules and cycle controls and are bolted and validated as fit for reason; they can likewise be additionally reached out to companion diagnostic (CDx) gadget development and commercialization. They can be sent in review or imminent clinical trials to evaluate tissue-based biomarkers and can uphold patient stratification and selection. The algorithms have numerous potential extra uses where robust deployment and information assortment are required. Labcorp and PathAI plan to investigate connecting the utilization of AI-powered algorithms from translational research into a clinical laboratory setting. “We are very impressed with the vision that Labcorp has for the future of AI-powered pathology in drug development and diagnostics, and we look forward to joining forces to enable our bio-pharma partners to obtain the most accurate, standardized, and data-rich set of pathology readouts from clinical trials to help advance drug development,” said PathAI CEO Andy Beck. “After exploring successful initial projects with Labcorp, we are excited to leverage the scale of its network to grow the reach of digital and computational pathology with the goal of ultimately improving patient outcomes.” “It has been great working with PathAI to expand computational pathology applications in oncology and other diseases that have often been resistant to digitization,” said Paul Kirchgraber, M.D., CEO, Labcorp Drug Development. “This collaboration will provide our bio-pharma partners a differentiated understanding of relevant patient characteristics through applying leading AI-driven algorithms to support clinical trials from novel biomarker development through patient stratification and companion diagnostic development.” About PathAI PathAI is a leading provider of AI-powered research tools and services for pathology. PathAI’s platform promises substantial improvements to the accuracy of diagnosis and the efficacy of treatment of diseases like cancer, leveraging modern approaches in machine and deep learning. Based in Boston, PathAI works with leading life sciences companies and researchers to advance precision medicine. About Labcorp Labcorp is a leading global life sciences company that provides vital information to help doctors, hospitals, pharmaceutical companies, researchers, and patients make clear and confident decisions. Through our unparalleled diagnostics and drug development capabilities, we provide insights and accelerate innovations to improve health and improve lives. With more than 70,000 employees, we serve clients in more than 100 countries. Labcorp reported revenue of $14 billion in FY2020.

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CELL AND GENE THERAPY

Graphite Bio Enrolls First Patient in Phase 1/2 Clinical Trial of GPH101 for Sickle Cell Disease

Graphite Bio | November 26, 2021

Graphite Bio, Inc. a clinical-stage, next-generation gene editing company focused on therapies that harness targeted gene integration to treat or cure serious diseases, announced that the first patient has been enrolled in the company’s Phase 1/2 clinical trial of GPH101, an investigational gene-edited autologous hematopoietic stem cell therapy designed to directly correct the genetic mutation that causes sickle cell disease. The company expects to treat the first patient with GPH101 in the first half of 2022, with initial proof-of-concept data anticipated by the end of 2022. “GPH101 is the first investigational therapy to enter clinical development that uses our next-generation gene editing platform technology to directly correct the mutation in the beta-globin gene that causes sickle cell disease. Using our gene correction approach, we have demonstrated in preclinical studies an ability to decrease the production of harmful sickle hemoglobin and restore the expression of normal adult hemoglobin. This approach has the potential to restore normal physiology and is viewed as the gold standard for curing sickle cell disease. We are thrilled that our first patient is now enrolled in our CEDAR clinical trial, and we look forward to evaluating GPH101’s potential as we continue to advance its development with urgency in hopes of delivering a curative therapy to the sickle cell community.” Josh Lehrer, M.Phil., M.D., chief executive officer at Graphite Bio The CEDAR trial is an open-label, multi-center Phase 1/2 clinical trial of GPH101 designed to evaluate the safety, engraftment success, gene correction rates, total hemoglobin, as well as other clinical and exploratory endpoints and pharmacodynamics of GPH101 in patients with severe SCD. The trial will enroll approximately 15 adult and adolescent participants at up to five clinical trial sites in the United States. Graphite Bio will present information about the CEDAR trial at the 63rd American Society of Hematology (ASH) Annual Meeting & Exposition, being held virtually and in Atlanta December 11-14. The company’s poster presentation will take place on Saturday, Dec. 11, at 5:30-7:30 p.m. ET. About Sickle Cell Disease (SCD) SCD is a serious, life-threatening inherited blood disorder that affects approximately 100,000 people in the United States and millions of people around the world, making it the most prevalent monogenic disease worldwide. SCD is caused by a single mutation in the beta-globin gene that leads red blood cells to become misshapen, resulting in anemia, blood flow blockages, intense pain, increased risk of stroke and organ damage, and reduced life span of approximately 20-30 years. Despite advancements in treatment and care, progressive organ damage continues to cause early mortality and severe morbidity, highlighting the need for curative therapies. About GPH101 GPH101 is an investigational next-generation gene-edited autologous hematopoietic stem cell (HSC) therapy designed to directly correct the genetic mutation that causes sickle cell disease (SCD). GPH101 is the first investigational therapy to use a highly differentiated gene correction approach that seeks to efficiently and precisely correct the mutation in the beta-globin gene to decrease sickle hemoglobin (HbS) production and restore normal adult hemoglobin (HbA) expression, thereby potentially curing SCD. Graphite Bio is evaluating GPH101 in the CEDAR trial, an open-label, multi-center Phase 1/2 clinical trial designed to assess the safety, engraftment success, gene correction rates, total hemoglobin, as well as other clinical and exploratory endpoints and pharmacodynamics in patients with severe SCD. About Graphite Bio Graphite Bio is a clinical-stage, next-generation gene editing company harnessing high efficiency targeted gene integration to develop a new class of therapies to potentially cure a wide range of serious and life-threatening diseases. Graphite Bio is pioneering a precision gene editing approach that could enable a variety of applications to transform human health through its potential to achieve one of medicine’s most elusive goals: to precisely “find & replace” any gene in the genome. Graphite Bio’s platform allows it to precisely correct mutations, replace entire disease-causing genes with normal genes or insert new genes into predetermined, safe locations. The company was co-founded by academic pioneers in the fields of gene editing and gene therapy, including Maria Grazia Roncarolo, M.D., and Matthew Porteus, M.D., Ph.D.

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MEDICAL

SOPHiA GENETICS Launches New Solution to Advance Chronic Lymphocytic Leukemia Care, in Collaboration with IDIBAPS

biotech, biotechnology, cell, gene, tissue | June 10, 2022

SOPHiA GENETICS (Nasdaq: SOPH), the Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS) of Barcelona, and the Spanish Diagnóstica Longwood today announced from the European Hematology Association Congress in Vienna that they have combined their expertise to develop a new Chronic Lymphocytic Leukemia (CLL) solution to advance CLL care. This collaboration will unify the wide variety of current guideline recommendations into one single application aiming to improve CLL characterization and CLL research practices in Iberia. SOPHiA GENETICS has already facilitated the analysis of one million genomic profiles through the SOPHiA DDM™ Platform, including a significant amount of onco-hematological-related diseases such as acute myeloid leukemia and lymphomas. The Platform computes a wide array of genomic variants needed to continually hone machine learning algorithms designed to accurately detect rare and challenging cases. The growing number of users of the SOPHiA DDM™ community can share and access insights by pulling the relevant signals detected from the noise, ultimately saving time and helping to make better-informed decisions. The new CLL solution allows for progress of Chronic Lymphocytic Leukemia research using genomic analysis. This could lead to better detection of the disease, which accounts for 25-30% of all leukemia cases in Western countries and affects yearly more than 100,000 people globally[1], and ultimately improve patient care. Thanks to the SOPHiA DDM™ Platform and IDIBAPS, hematopathologists can now access guidelines for the mutational status of TP53, immunoglobulin (IG) gene rearrangements and their somatic hypermutation status, while benefitting from the identification of 23 CLL-specific genes for SNVs, InDels and CNVs including NOTCH1, SF3B1, ATM, IGLV3-21, BTK, PLCG2, BCL2, del13q14, and trisomy 12, all in one single NGS workflow. The new CLL solution has already been used in routine in Spain, with further opportunities throughout the country, and in Latin America through Diagnóstica Longwood's distribution channels. We are proud to work together with some of the most renowned experts in the field of Chronic Lymphocytic Leukemia research around the world. This partnership is a great example of how combining advanced data analytics with emerging CLL strategies can move the possibilities of data-driven medicine forward." Lara Hashimoto, Chief Business Officer at SOPHiA GENETICS. It is a great satisfaction to see how thanks to the partnership with SOPHiA GENETICS and Diagnóstica Longwood we will be able to transfer into clinical practice the relevant information generated for so many years in the research of CLL using a simple and robust assay." Elías Campo, principal investigator and director of IDIBAPS. About SOPHiA GENETICS SOPHiA GENETICS (Nasdaq: SOPH) is a healthcare technology company dedicated to establishing the practice of data-driven medicine as the standard of care and for life sciences research. It is the creator of the SOPHiA DDM™ Platform, a cloud-based SaaS platform capable of analyzing data and generating insights from complex multimodal data sets and different diagnostic modalities. The SOPHiA DDM™ Platform and related solutions, products and services are currently used by more than 790 hospital, laboratory, and biopharma institutions globally. About IDIBAPS The August Pi i Sunyer Biomedical Research Institute (IDIBAPS) is a biomedical research center of excellence that addresses the most common diseases in our environment. It is a public consortium comprising the Catalan Government (Generalitat de Catalunya), the Hospital Clínic Barcelona, the Faculty of Medicine and Health Sciences at the University of Barcelona and the CSIC Institute of Biomedical Research of Barcelona. About 1,500 professionals are organized in a hundred research groups. IDIBAPS undertakes translational research. It focuses on ensuring that the questions that arise at the patient's bedside have a response in the laboratory, and that advances made in the laboratory are promptly applied to patients. With over 1,200 articles published annually, it is the leading biomedical research center in Spain.

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