Sat.May 06, 2023 - Fri.May 12, 2023

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‘The luckiest of the unlucky’: A Duchenne gene therapy brings hope to families — and tests the FDA

Bio Pharma Dive

Patient advocates and doctors are anticipating an approval this month of a treatment they believe to be a breakthrough for a deadly disease. But it’s not clear how well the therapy really works, putting the FDA in a difficult position.

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BioNTech’s RNA vaccine sparks potential in pancreatic cancer

Pharmaceutical Technology

Results published in Nature for a personalised pancreatic cancer vaccine that uses neoantigens from patients’ tumours have lent further support to early positive signals. The vaccine, developed by BioNTech, led to half of the patients with pancreatic cancer in the Phase I trial remaining cancer-free 18 months later. In the study, 16 patients were treated with a vaccine called autogene cevumeran that contained a maximum of 20 neoantigens, alongside Roche’s anti-PD-L1 immunotherapy Tecentriq (atez

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Air pollution shown to worsen movement disorder after stroke

Medical Xpress

Air pollution has been shown to have a negative effect on the prognosis of ischemic stroke, or stroke caused by reduced blood flow to the brain, but the exact mechanism is unknown. A team of researchers recently conducted a study to determine whether or not increased inflammation of the brain, also known as neuroinflammation, is the main culprit.

Research 143
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Cronobacter Sakazakii: The Bacteria Behind the Baby Formula Shortage

XTalks

Cronobacter sakazakii , the bacteria linked to recent baby formula shortages and the Abbott infant formula recall, could soon join the federal disease watchlist. In June, the Council of State and Territorial Epidemiologists (CSTE) will vote to decide if it will officially recommend adding Cronobacter infections to the list of nationally notifiable diseases to the US Centers for Disease Control and Prevention (CDC).

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The New Age of Decentralized Clinical Trials

White paper that delves into the complex topic of Decentralized Clinical Trials and how to master them within the confines of FDA Regulations

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FDA panel narrowly backs approval of Sarepta’s Duchenne gene therapy

Bio Pharma Dive

Advisers voted 8-6 in favor of an accelerated approval despite uncertainty around the treatment’s benefit. The agency is set to make a final decision by May 29.

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The emergence of tranq amidst the fentanyl crisis

Pharmaceutical Technology

In February, the FDA issued an alert restricting the import of the veterinary sedative xylazine or tranq and the ingredients used to make the drug. This followed in the wake of a Drug Enforcement Agency (DEA) Joint Intelligence Report in October 2022, which stated that xylazine is widely available from Chinese suppliers on the internet. According to a June 2022 study, xylazine has been detected in the illicit drug supply in 36 US States, and the DEA has reported that approximately 30% of the f

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Why Webinar Accessibility is Important in the Global Market

XTalks

According to a report produced by the World Health Organization (WHO), around 1.3 billion people – or 16 percent of the world’s population – are living with significant disabilities. This makes it crucial that we adopt systems that make our webinars accessible to the greatest proportion of the population. Webinar hosts dedicate a lot of time and effort when creating webinar content, marketing it to potential attendees and practicing the delivery to ensure they convey their message to the audienc

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Cancer vaccine from BioNTech, Roche shows potential in small study

Bio Pharma Dive

The companies’ personalized shot generated tumor-specific T cells that researchers hope could help prevent pancreatic cancer from returning after surgery.

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Gilead wins patent battle with US government over HIV PrEP drugs

Pharmaceutical Technology

Gilead Sciences has emerged victorious in a legal battle with the US government over patents surrounding the HIV pre-exposure prophylaxis (PrEP) drugs Descovy and Truvada following a federal jury’s verdict on May 9. A jury in the Delaware District Court gave a favourable verdict to the Foster City, California-headquartered company, concluding that Gilead did not infringe any of the US government’s patents.

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Research reveals cancer-killing benefits of popular obesity treatment

Medical Xpress

Maynooth University's Kathleen Londsdale Institute for Human Health Research has just published research into the benefits of the popular obesity treatment drug, GLP-1.

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Roles and Responsibilities of Specialized Clinical Supply Experts

When selecting a clinical supply provider, consideration often focuses upon the manufacturing, packaging, storage and distribution capabilities available that will, at face-value, be sufficient to meet the needs of the sponsor and their trial. However, there are human-based and knowledge-driven factors that are often overlooked that go beyond these basic physical capabilities and are integral to the development and delivery of high performing clinical supply chains.

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Do patients have to pay for clinical trials?

Antidote

One of the questions most frequently asked by patients is, “Do I have to pay to participate in a clinical trial?” While patients typically will not incur expenses for taking part in a research study, in some instances, they may be responsible for copays and payments towards their deductible depending on their insurance plan.

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FDA staff skeptical of Sarepta’s Duchenne gene therapy, documents show

Bio Pharma Dive

Sarepta has not provided “unambiguous evidence” its treatment can help patients, agency scientists wrote in documents released ahead of a crucial Friday advisory committee meeting.

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Synlogic’s SYNB1934 receives orphan drug status from US FDA

Pharmaceutical Technology

Synlogic has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for SYNB1934 to treat phenylketonuria (PKU), a rare inherited metabolic disease. The orally administered, non-systemically absorbed drug candidate SYNB1934 has been designed for reducing blood phenylalanine (Phe) levels in PKU patients. SYNB1934 consumes Phe in the gastrointestinal (GI) tract by leveraging genetic engineering of the drug or drug-carrying capsule, probiotic escherichia coli (E coli)

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New method enables detection of plasma biomarkers cheaply and at scale

Medical Xpress

Blood plasma is collected from people routinely during clinical care and for research. It is potentially a rich source of protein biomarkers for diagnostic and prognostic purposes, for measuring response to treatment, and for revealing disease biology. Yet identifying such biomarkers in plasma with proteomics, our best available tool, has been a challenge: 99% of plasma consists of everyday proteins like albumin, globulins, and coagulants that essentially crowd out the needle-in-a-haystack prote

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How Machine Learning Drives Clinical Trial Efficiency

Clinical trial data management is increasingly challenging as studies grow in complexity. Quickly accessing and analyzing study data is vital for assessing trial progress and patient safety. In this paper, we explore real-time data access and analysis for proactive study management. We investigate using adverse event (AE) data to monitor safety and discuss a clinical analytics platform that supports collaboration and data review workflows.

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Change is Inevitable – Plan Ahead: An Assessment of FDA’s Draft Guidance on Predetermined Change Control Plans for Artificial Intelligence/Machine Learning-Enabled Device Software Functions

FDA Law Blog

By Lisa M. Baumhardt, Senior Medical Device Regulation Expert & Philip Won & Gail H. Javitt — FDA recently published a long-awaited draft guidance aimed at reducing the need for prior FDA authorization of modifications to artificial intelligence/machine learning (AI/ML)-enabled device software functions (ML-DSFs). The draft guidance, “Marketing Submission Recommendations for a Predetermined Change Control Plan for Artificial Intelligence/Machine Learning (AI/ML)-Enabled Device Software

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Alzheimer’s doctors see promise, limits in Lilly’s latest drug data

Bio Pharma Dive

While some see the new donanemab results as adding to a “watershed moment” in Alzheimer’s research, others say they reinforce the limitations of so-called anti-amyloid therapies.

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Ginkgo and Boehringer partner to develop hard-to-treat disease therapies

Pharmaceutical Technology

Ginkgo Bioworks and Boehringer Ingelheim have collaborated to develop breakthrough therapies for hard-to-treat diseases. The companies will use the natural product discovery capabilities of Ginkgo Bioworks to expedite the discovery and development of new therapeutic molecules to address diseases with high unmet patient needs. They will also use the metagenomic sequence database of Ginkgo Bioworks which comprises more than three terabases of sequence data and over two billion protein sequences fr

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Researchers solve mystery of how statins improve blood vessel health

Medical Xpress

Using new genetic tools to study statins in human cells and mice, Stanford Medicine researchers and collaborators have uncovered how the cholesterol-lowering drugs protect the cells that line blood vessels.

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What the FDA's New Dosage Guidance Means for the Future of Clinical Research

Speaker: Dr. Ben Locwin - Biopharmaceutical Executive & Healthcare Futurist

What will the future hold for clinical research? A recent draft from the FDA provides valuable insight. In "Optimizing the Dosage of Human Prescription Drugs and Biological Products for the Treatment of Oncologic Diseases," the FDA notes that "targeted therapies demonstrate different dose-response relationships compared to cytotoxic chemotherapy, such that doses below the Maximum Tolerated Dose (MTD) may have similar efficacy to the MTD but with fewer toxicities.

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GSK and Magic Johnson Team Up For RSV Awareness

XTalks

Just ahead of having nabbed a historic approval for its respiratory syncytial virus (RSV) vaccine, GlaxoSmithKline (GSK) launched ad campaigns to promote RSV awareness, particularly among older adults. This includes the ‘Cut Short by RSV’ and ‘Sideline RSV’ campaigns launched in April and March, respectively. For its ‘Sideline RSV’ campaign, GSK teamed up with NBA legend Earvin ‘Magic’ Johnson to help raise awareness among communities about the risk RSV could pose to older adults.

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Gilead acquires San Diego startup for early-stage cancer, immune drugs

Bio Pharma Dive

The buyout of privately held XinThera continues a string of recent acquisitions of smaller biotechs by Gilead and hands it a group of drug prospects that could begin clinical testing later this year.

Drugs 269
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Researchers develop CRISPR-based drug candidate for microbiome targeting

Pharmaceutical Technology

The Technical University of Denmark (DTU) has announced that an international scientific team has developed a CRISPR-based drug candidate that targets E coli directly and leaves the microbiome intact. Blood cancer patients with E coli infection are at risk of bacteria infecting the bloodstream. An E coli infection can be fatal in these cases, with a 15-20% mortality rate.

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T cells can activate themselves to fight tumors

Medical Xpress

When you need a bit of motivation, it often has to come from within. New research suggests cancer-fighting immune cells have found a way to do just that.

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The New Age of Decentralized Clinical Trials

This new white paper defines and details the impact of Decentralized Clinical Trials on the Pharmaceutical industry and how the impact can be measured along with steps companies can take to ensure adoption.

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Cellular Origins partners with ScaleReady to automate cell therapy manufacturing

BioPharma Reporter

Cellular Origins has partnered with ScaleReady, to automate portions of its cell and gene therapies (CGT) manufacturing workflow using a robotics system for sterile liquid transfer.

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EQRx resets strategy, abandoning plans to disrupt drug pricing

Bio Pharma Dive

Launched in 2020 with a "radical" vision, EQRx ran into roadblocks that stymied its efforts to develop new medicines and undercut competitors on price.

Drugs 282
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Takeda reports 12.8% rise in FY2022 revenue

Pharmaceutical Technology

Takeda has reported a 12.8% increase in its reported revenue to $29.96bn (Y4,027bn) during the fiscal year 2022 (FY2022) compared to that reported in FY2021. At a constant exchange rate (CER), the company’s core revenue grew by 3.5% compared to the previous year. In the FY2022 ending 31 March 2023, the company also reported a 6.4% rise in its operating profit to $3.65bn (Y490.5bn) compared to 2021.

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A potential pathway to improved stroke recovery

Medical Xpress

Ischemic stroke, caused by a blockage of blood flow to the brain, is a common cause of death and disability. Treatments are urgently needed to improve patient outcomes, because recovery currently depends largely on the timely injection of a blood clot-dissolving drug. Priorities for therapy include limiting inflammation at the ischemic site and rebuilding neuronal connections damaged by the stroke.

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Accelerating Clinical Supply Through Integrated Drug Development

As the development pipeline for new drugs continues to grow, biopharmaceutical companies are re-evaluating how to best manage and balance resources across an increasing number of development projects and complex clinical trials. There are two approaches that can be used to speed a drug from development to clinic faster: timeline compression and parallel processing, but only one that considers the benefits of integrating clinical supply into the overall drug development process.

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Garden Lites, Sweet Loren’s and Other Mother-Owned Food Brands to Support This Mother’s Day

XTalks

Mother’s Day is a time to celebrate and honor the mothers in our lives who have nurtured us and helped shape who we are today. One way to show appreciation is to support mother-owned food brands that have made a mark in the food industry. The mothers behind brands like Garden Lites and Sweet Loren’s have been able to turn their passion for food into successful businesses, creating innovative products and driving growth in the industry.

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Recursion to acquire two Canadian drug discovery startups

Bio Pharma Dive

The company agreed to buy Cyclica and Valence after winnowing down a list of more than 100 potential takeover targets, its CEO told BioPharma Dive.

Drugs 294
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Editas continues to trend efficacy signals with sickle cell disease gene therapy

Pharmaceutical Technology

Editas Medicine will release new efficacy and safety results of its gene therapy EDIT-301 in severe sickle disease as part of an oral presentation at the European Hematology Association’s (EHA) Hybrid Congress. The EHA Hybrid Congress will take place on June 8-11 in Frankfurt, Germany. Last month, the US Food and Drug Administration (FDA) granted the treatment an orphan drug designation for sickle cell disease.

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Gene-editing technique could speed up study of cancer mutations

Medical Xpress

Genomic studies of cancer patients have revealed thousands of mutations linked to tumor development. However, for the vast majority of those mutations, researchers are unsure of how they contribute to cancer because there's no easy way to study them in animal models.

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Deliver Fast, Flexible Clinical Trial Insights with Spotfire

Clinical research has entered a new era, one that requires real-time analytics and visualization to allow trial leaders to work collaboratively and to develop, at the click of a mouse, deep insights that enable proactive study management. Learn how Revvity Signals helps drug developers deliver clinical trial data insights in real-time using a fast and flexible data and analytics platform to empower data-driven decision-making.