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In addition to clearing Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy, the FDA also granted an early OK to Bluebird bio’s sickle cell treatment Lyfgenia.
A new sickle cell disease therapy developed by CRISPR Therapeutics and Vertex Pharmaceuticals is now approved in the U.S. This is the story of how it came to be.
In the rapidly advancing field of cell therapies, Dr Jason Bock has emerged as a leader, known for his innovative approach to optimising the development process. With over 25 years of experience in therapeutics, Bock has played a pivotal role in shaping the future of cell therapies, particularly through his work at CTMC.
A gene editing therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics can mute sickle cell disease’s most damaging symptoms. Yet treatment may not be as simple as its dramatic benefit makes it seem.
The decision stands in contrast to the agency’s plans for a would-be rival gene editing treatment from Vertex Pharmaceuticals and CRISPR Therapeutics that is also under a regulatory review.
Artificial intelligence (AI) has revolutionised many industries, yet its adoption in pharmaceutical drug development has been notably slower. Looking ahead, 2025 could represent a major turning point for the pharmaceutical sector.
Knowing a patients subtype or phenotype can be used to guide obesity treatments of all kinds, including drug therapy, devices, bariatric surgery and even diet and lifestyle interventions, Bagnall explains.
Longer follow-up results from a mid-stage trial indicate the company’s treatment for a genetic cause of dwarfism may be competitive to an approved therapy from BioMarin Pharmaceutical.
Deal Structuring Deal structuring in pharmaceutical M&A requires a balance of financial, legal, and strategic considerations. Intellectual Property Valuation Valuing intellectual property (IP) assets is a critical component of pharmaceutical M&A, as these assets often represent a significant portion of a company's value.
Bayer and Broad Institute extend cancer therapy research collaboration By Corie Lok November 2, 2023 Breadcrumb Home Bayer and Broad Institute extend cancer therapy research collaboration Long-standing industry-academia collaboration has already resulted in three clinical oncology candidates.
In the vast realm of pharmaceutical research and development, there’s a fascinating intersection between ancient wisdom and modern science. “Pharmacognosy is the bridge between traditional medicine and modern pharmaceutical science, offering a treasure trove of potential new drugs waiting to be discovered.”
The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. In some cases, cell therapy can be curative for the patient.
As a result, biopharma and biotech companies working to bring their drug pipelines to the market require deep expertise from trusted partners to help deliver critical therapies for their patients. According to the United Nation’s 2024 World Population Prospects data, the global population of those 65 years old and over will grow from 0.8
This month features many of the big reveals from the ACS fall 2023 first disclosures including Nurix’s BTK degrader and RAPT’s CCR4 autoimmune therapy. selective inhibitor developed by Vertex Pharmaceuticals for acute pain in Phase III clinical trials. Several of the molecules making the August list are in Ph.
Historically, what has limited investor interest in funding neurological and psychiatric therapies in development? Investors have historically taken a ‘guilty until proven innocent’ approach to investing in neuroscience therapies. We were able to license a molecule from a multi-national pharmaceutical company.
Nasir discusses how hubs help patients initiate and adhere to drug therapies. Today’s guest post comes from Nasir Ali, Chief Product Officer at CareMetx. Based on CareMetx research, he outlines the key factors that drug manufacturers value in their hub partners.
Avacta has appointed Neil Bell, a highly experienced pharmaceutical and biotech industry executive, to lead the ongoing transition to a clinical stage biotech developing innovative cancer therapies
New Solutions for Aseptic Filling for Gene Therapy In the development of gene therapies, it is more important than ever to stay on top of the latest containment and aseptic filling solutions.
West Pharmaceutical Services (West) and Cytiva have teamed up to offer innovative and versatile fill finish solutions to efficiently meet the unique demands of advanced pharmaceutical applications. In this webinar, you will learn about: The challenges and risks related to the packaging and filling of high-value drug products.
1] [2] It was developed by Vertex Pharmaceuticals , [5] and was approved for medical use in the United States in January 2025. [2] The application received Breakthrough Therapy , Fast Track and Priority Review designations by the FDA. The FDA granted approval of Journavx to Vertex Pharmaceuticals Incorporated. under nitrogen.
Liver Fibrosis : Targeted therapies using ADCs could help reduce liver fibrosis and prevent the progression to cirrhosis. Expedited programs like the FDA’s Breakthrough Therapy Designation and Fast Track can shorten development timelines and reduce risk. CDMOs are crucial.
The use of biomarkers has transformed the pharmaceutical industry by allowing researchers to develop therapies that target specific pathways in diseases. These can be proteins, genes, or other molecules that signal an abnormal or normal process in the body.
To date, there are less than 30 known surface proteins that form the basis of all approved cancer-targeted therapies of all modalities, including antibody-drug conjugates (ADCs), T-cell engagers, CAR T-cells and radiopharmaceuticals. These could be targets for antibody-drug conjugates (ADCs), bi-specific antibodies, or other therapies.
Pharmaceutical researchers, in particular, have a keen interest in understanding the unfolding dynamics of this market. Inspired by its success, pharmaceutical companies are pursuing the development of drugs with similar therapeutic profiles, aiming to expand treatment options.
If you're a pharmaceutical researcher or drug developer, understanding these breakthroughs is critical to staying at the forefront of innovation. You’ll discover real-world examples, such as the transformation of KRAS from an undruggable target to a success story in targeted therapy.
BioPharma Dive: Pharmaceutical Research on mRNA and CAR T-Cell Therapy in Cancer — Pioneering the Future of Oncology aasimakopoulos Mon, 10/21/2024 - 13:18 Category News Tags Clinical Trials Preclinical Research Weight 1
Environmental Impacts of Traditional Pharmaceutical Production Traditional pharmaceutical manufacturing is complex, with each phase contributing to environmental degradation. The extraction and synthesis of active pharmaceutical ingredients (API) utilize vast amounts of water and energy.
This review aims to provide information on cutting-edge Nano particulate-based technologies and Nano therapies for colorectal cancer. The field of cancer detection and therapy has undergone a revolution with the development of nanotechnology for targeted drug delivery.
My Attendance at the 2024 Boston Society Cell and Gene Therapy Conference pmjackson Tue, 06/11/2024 - 20:49 , via Wikimedia Commons" data-entity-type="file" data-entity-uuid="c7a7fa8b-b2fe-4d84-a75e-d1ba3a4e2caf" src="[link] width="742" height="249" loading="lazy" /> Ecm85, CC BY-SA 3.0
A new shift is occurring in the pharmaceutical industry, leading to a rapidly expanding field known as digital therapeutics (DTx). DTx interventions include sensor-equipped wearable devices, remote patient monitoring tools, and virtual reality platforms integrated with conventional pharmaceutical treatments.
Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.
Exploring the transformative impact of large language models (LLMs) in molecular biology and drug development, discussing potential areas of applications and breakthroughs in personalized therapies. While enhancing molecular biology aspects, we also address ethical concerns ensuring responsible application of these models.
billion to bring a new therapy to market. In 2019 alone, research and development spending totaled $186 billion for the global pharmaceutical industry. Between 2009 and 2018, U.S.
By directly interfering with the eIF4F complex, eFFECTOR’s STRIs provide a promising avenue for disrupting the core mechanisms of cancer, offering the potential to improve patient outcomes and overcome the limitations of traditional cancer therapies. Prior to Anadys Pharmaceuticals, Inc., Prior to Anadys Pharmaceuticals, Inc.,
By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.
Adam discusses how patients seeking to access specialty therapies can benefit from a digital intake approach. He then explains three ways that specialty pharmaceutical companies can use digital intake strategies to speed patient enrollment. Today’s guest post comes from Adam Stotts, SVP of Customer Success at AssistRx.
Contrary to popular belief, ageing is not caused by just random wear and tear of our bodies over time but is instead caused by a discrete set of biological mechanisms that we now better understand and can target with therapies. a commercial stage pharmaceutical company. acquired by Teva Pharmaceuticals in 2014.
From Data to Therapy: Transforming Cancer Treatment One of the most exciting aspects of RADR ® is its potential to transform real-world outcomes for patients. The company plans to continue expanding the capabilities of RADR ® , with a significant focus on immuno-oncology, combination therapy prediction, and the development of ADCs.
This article incorporates text from this source, which is in the public domain. ^ “Qfitlia approved as the first therapy in the US to treat hemophilia A or B with or without inhibitors” Sanofi (Press release). 28 March 2025. Retrieved 29 March 2025. World Health Organization (2016). hdl : 10665/331046.
Designation is supported by Phase II efficacy and safety data that will be presented at ATS 2022 Boehringer Ingelheim plans to study this novel investigational therapy in patients with progressive fibrosing interstitial lung diseases. senior vice president, Medicine and Regulatory Affairs, Boehringer Ingelheim Pharmaceuticals, Inc. “BI
We embark on a fascinating journey through the realms of cell and gene therapy, CRISPR technology, cancer research, and the latest advancements in screening and imaging. Prepare to be captivated by the groundbreaking discoveries and innovative approaches revolutionising the field of healthcare. But it doesn’t stop there.
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