AlzeCure Pharma provides a scientific update on Alzheimer's disease and the Alzstatin project platform

AlzeCure Pharma | September 28, 2020

CEO Martin Jönsson and CSO Johan Sandin will, among other things, present the pharmaceutical platform Alzstatin and the research behind it. Within the Alzstatin platform, AlzeCure develops disease-modifying and preventive drugs for Alzheimer's disease that focus on reducing the production of toxic amyloid beta (Aβ) in the brain. Henrik Zetterberg, chief physician and professor at the Department of Neuroscience and Physiology at Sahlgrenska Academy and professor at University College of London (UCL), will participate and provide a deeper review of disease development in Alzheimer's and the amyloid hypothesis, as well as provide an update on developments in biomarkers and diagnostics in Alzheimer's.

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BUSINESS INSIGHTS, VIEWS AND ANALYSIS

Rancho BioSciences Welcomes BenevolentAI as Fifth Member of SCDS Consortium

Rancho BioSciences | February 02, 2023

On February 1, 2023, Rancho Biosciences, a leading provider of data sciences services, announced that BenevolentAI has joined the SCDS consortium as its fifth member. Rancho Biosciences launched its Single Cell Data Science (SCDS) pre-competitive consortium on February 26, 2022, with four Charter Members: Bristol Myers Squibb, Janssen Research & Development LLC, a subsidiary of Johnson & Johnson's Janssen Pharmaceutical Companies, Novartis, and Vesalius Therapeutics. The consortium's mission is to establish a uniform industry standard for creating and formatting single-cell datasets through a systematic effort to build data models and ensure that public data are curated consistently. Due to the evident impact of single-cell transcriptomics technology on drug discovery, pharmaceutical firms continue to adopt single-cell sequencing techniques at an exponential rate. The availability of ever-increasing volumes of single-cell datasets in the public domain enables pharmaceutical companies to vastly increase their universe of single-cell experiments over those generated internally. However, harnessing this enormous public data lake by locating, downloading, and curating single-cell data is difficult and time-consuming compared to the resources required for scientists to analyze data to derive value for biomedical research collectively. To date, Rancho has provided 83 analysis-ready datasets by using its extensive curation knowledge and expertise. Each dataset contains 79 columns of curated metadata that have been verified against a 4-entity data model developed by Rancho for SCDS. Based on member priorities, Rancho will continue to contribute a steady stream of harmonized single-cell datasets to the consortium. With this shared cost model, these may be supplied at a far higher throughput and much more cost-effectively than a single company could do. About Rancho BioSciences Founded in 2012, Rancho BioSciences is a global fee-for-service data curation firm that provides analysis and development services to clients, including pharma, foundations, government, and biotech. With a global team of qualified Ph.D. and Ph.D./MD scientists that provide high-quality work based on their expertise and domain knowledge in biology, diseases, and clinical data, it strives to find cures for diseases by serving its clients and saving lives through Data. It aims to be the world's leading provider of data services and harness domain skills and expertise to address complex problems.

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BUSINESS INSIGHTS, PHARMA TECH

Arrowhead Pharmaceuticals Announces $25 Million Milestone Payment from Amgen

Arrowhead and Amgen | December 21, 2022

Arrowhead Pharmaceuticals Inc. announced a $25M milestone payment from Amgen. This milestone was triggered by the first subject enrolled in Amgen’s Phase 3 trial of olpasiran. Arrowhead is further eligible to receive up to an additional $535 million in aggregate development, regulatory, and sales milestone payments from Amgen and Royalty Pharma plc. “We are pleased with the great progress on the clinical development of olpasiran, which was developed using Arrowhead’s proprietary TRiMTM technology. This is an important milestone for the program and for Arrowhead, as this is the second TRiMTM-enabled candidate to enter Phase 3 studies. Importantly, as our pipeline continues to advance expeditiously, we anticipate multiple Arrowhead therapies will also reach Phase 3 trials over the coming year.” Christopher Anzalone, Ph.D., Arrowhead’s president and CEO Olpasiran is a small interfering RNA originally developed by Arrowhead using its proprietary Targeted RNAi Molecule, or TRiM, platform and licensed to Amgen in 2016. It is designed to lower levels of lipoprotein(a) (Lp(a)), a genetically determined risk factor for cardiovascular disease. Phase 2 study results from the OCEAN(a)-DOSE study were presented at the American Heart Association Scientific Sessions 2022, where olpasiran demonstrated a significant and sustained reduction in Lp(a) levels over 36 weeks. These data were simultaneously published in the New England Journal of Medicine on November 6, 2022. About Lp(a) Lp(a) is genetically determined1-3 and a presumed independent risk factor for cardiovascular disease. Although an agreed upon threshold for elevated Lp(a) is not firmly established, approximately 20% of adults have Lp(a) >125 nmol/L (or approximately 50 mg/dL).1 Evidence has emerged from pathophysiological, epidemiologic, and genetic studies on the potential role of elevated Lp(a) in contributing to myocardial infarction, stroke, and peripheral arterial disease.3 About Arrowhead Pharmaceuticals Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.

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BUSINESS INSIGHTS, PHARMACY MARKET

Cellworks Announces Expansion Into Precision Pharma

Cellworks Group | January 13, 2023

Cellworks Group Inc., a pioneer in personalized therapy biosimulation, announced the introduction of its precision drug development business units. These units will use predictive biosimulation to shorten drug development time-to-market and revive previously researched but disapproved pharmaceutical assets. The two new business units will use Cellworks' computational biology model (CBM) and biosimulation platform to predict responses to pharmaceutical interventions in silico, streamlining the clinical trial by identifying suitable patients more quickly, thereby reducing the time and cost of developing successful pharmaceutical agents. Cellworks precision drug development: Pre-clinical stage Companion molecule development: The Cellworks platform can augment a drug's usability by finding companion molecules that improve performance when combined with the investigational agent. Biomarker development: Cellworks utilizes cell lines and historical patient data to uncover novel biomarkers for investigational agents. This may increase drug success rates by proactively identifying individuals who will respond to an agent. Indication expansion: By utilizing the Cellworks patient database of 100,000+ omics patient profiles and 1,800 oncology cell lines to examine the agent's effectiveness in different indications, the Cellworks platform can identify additional indications where an experimental treatment may be successful. Cellworks precision drug development: Clinical stage Drug revival and repurposing: The platform seeks to anticipate whether certain drugs cause a response, which may be used to identify whether shelved assets can be resurrected via suitable patient selection or the discovery of non-obvious reasons. Clinical trial biosimulation: Through biosimulation clinical trials, the Cellworks platform may anticipate humans' clinical response to pharmaceutical agents in weeks, yielding critical insight years ahead of a traditional clinical practice. Cellworks Platform and Computational Biology Model (CBM) The Cellworks platform biosimulates the impact of specific pharmacological molecules on a given patient or class of patients using the patient's genetic profile. To generate a customized or cohort-specific disease model, multi-omic data from a cohort or patient is fed into the Cellworks computational biology model (CBM). About Cellworks Group Cellworks is one of the leaders in precision medicine. The organization assists physicians and payers in navigating the most efficacious, cost-effective therapies for patients. Its primary therapeutic areas are oncology, immunology, dermatology, and infectious diseases. Its one-of-a-kind biosimulation platform is a unified representation of biological information gathered from disparate databases and used to find cures. The company has the world's strongest trans-disciplinary team of molecular biologists, cellular pathway modelers, and internet software technologists working towards a common goal, i.e., taking on significant diseases to improve patients' lives.

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