Mon.Nov 15, 2021

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Years later, a first-of-its-kind treatment shows the power, and limits, of gene therapy

Bio Pharma Dive

Luxturna, approved four years ago as the first gene therapy for an inherited disease in the U.S., is improving sight and quality of life for several of the patients who received it.

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It’s getting harder to launch a new drug to physicians

World of DTC Marketing

SUMMARY: Physicians are tired and overwhelmed with medical information on new drugs. They don’t have the time to sit in the office and search for more information, and they feel that pharma is giving them “too much” information. An exciting development presented itself. A client launched an Oncology drug based on extensive research with physicians, but the results were not that good during a post-launch evaluation.

Drugs 238
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Anticipating Aduhelm's costs, Medicare plans big jump in premiums

Bio Pharma Dive

While CMS is still weighing whether to authorize wide coverage of Biogen's pricey Alzheimer's drug, administrators are planning to build reserves in case costs skyrocket.

Drugs 306
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In the News: October 2021 Regulatory and Development Updates

Camargo

Each month, Camargo’s “In the News” series highlights important changes and advancements in the regulatory and development space and explores how those changes could impact your program. FDA Guidance Addresses Real-World Evidence Data Standards. The 21 st Century Cures Act generated a good deal of excitement and interest when it added a section called “ Utilizing Real World Evidence ” to the Food, Drug, and Cosmetic Act (Section 505F).

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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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Establishing an integrated evidence plan for medical affairs and beyond

Bio Pharma Dive

Over the life of a biopharmaceutical product, there are many situations when data from randomized controlled trials (RCTs) may not be sufficient to address stakeholder questions of value, safety and effectiveness.

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Pfizer, BioNTech and Zipline deliver COVID-19 vaccines by drone in Ghana

BioPharma Reporter

Pfizer and BioNTech have completed long-range drone deliveries of their mRNA COVID-19 vaccine: with partner Zipline claiming to be the first to deliver vaccines in this way at a national scale.

More Trending

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Vertex receives CHMP positive opinion for Kaftrio in children with cystic fibrosis

Pharma Times

Those eligible will have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene

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Smartphone-powered trial backs J&J’s Invokana for heart failure

pharmaphorum

Johnson & Johnson’s SGLT2 inhibitor Invokana has been shown to have a significant effect on heart failure symptoms in a clinical trial that relied entirely on remote monitoring of symptoms using a smartphone app. The real-world CHIEF-HF study is said to be the first fully “decentralised, mobile, indication-seeking clinical study” with no need for patients to visit clinic, according to J&J’s pharma division Janssen.

Trials 105
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Serotonin May Trigger Crohn’s Flare-Ups by Disrupting the Gut Microbiome

XTalks

Growing evidence continues to show fundamental links between the nervous system and the gut microbiome. Now, new research shows that the brain hormone serotonin may cause gut inflammation that could trigger flare-ups in Crohn’s disease. McMaster University researchers found that high levels of serotonin blocked the cellular process of autophagy in the gut, which cells use to destroy and remove their damaged cellular components.

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Legal requirement, ethical duty: How to develop compliant, useful lay summaries

pharmaphorum

New lay summary guidelines walk sponsors through their obligations under EU regulation No. 536/2014, due to come into force in February next year. Patient involvement is key, they say. The sooner patients are involved in developing and publishing lay summaries, which will soon be a legal requirement in the European Union, the better. That was among the key messages from speakers at a joint European Forum for Good Clinical Practice (EFGCP) and the European Federation of Pharmaceutical Industries

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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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NICE recommends cenobamate for treatment of adult epilepsy patients in UK

Pharma Times

Epilepsy affects over 600,000 people in the UK, which is approximately one in every 100 people

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All Mariah Wants for Christmas is for Fans to Try the Mariah Menu at McDonald’s

XTalks

Recently, big food companies have been making news for collaborating with celebrities to spread their flavors more widely. Some recent popular partnerships include Megan Thee Stallion working with Popeyes for the “Hottie Sauce” collaboration and McDonald’s partnering with BTS to create a different chicken nugget menu. Just in time for the Holidays, also known as Mariah Carey season, the singer is collaborating with McDonald’s to give customers a different menu item for fr

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Where COVID-19’s death grip slipped (briefly)

Scienmag

Geochemist Lex van Geen works at the intersection of public health and environmental risks. His research on natural arsenic contamination in groundwater has alerted the world community to this insidious danger over two decades. Credit: Kevin Krajick/Earth Institute Geochemist Lex van Geen works at the intersection of public health and environmental risks.

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Pfizer Lines Up Agreements for Global Access to COVID-19 Antiviral

BioSpace

Pfizer’s oral antiviral treatment for COVID-19 is making its way through regulatory hurdles. The company is working with global manufacturers to ensure a steady supply of the medication.

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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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Prions may channel RNA’s messages

Scienmag

HOUSTON – (Nov. 15, 2021) – Prions get mostly bad press, but they may be the keys to controlling protein synthesis in cells. Credit: Xinyu Gu/Rice University HOUSTON – (Nov. 15, 2021) – Prions get mostly bad press, but they may be the keys to controlling protein synthesis in cells. Prions, proteins that can misfold and aggregate, have been […].

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Discovery of 17 Genes Implicated in Obesity Could Lead to Breakthrough Treatments

BioSpace

Scientists from the University of Virginia recently revealed the discovery of 17 total genes directly implicated in the development of obesity.

Gene 106
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Combining pressure, electrochemistry to synthesize superhydrides

Scienmag

A new study featured in Proceedings of the National Academy of Sciences offers a potential alternate approach that combines pressure and electrochemistry to stabilize superhydrides at moderate, perhaps even close to ordinary, pressures. Superhydrides are a materials system where temperature superconductivity has been achieved, but only at very high pressures.

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AcelRx to Buy Lowell Therapeutics; Kala Expands Pipeline with Combangio Acquisition

BioSpace

AcelRx Pharmaceuticals and Kala Pharmaceuticals both shared acquisition news on Monday. Here’s a closer look at each of the acquisition announcements.

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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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Ludwig Lausanne team devises a method to selectively grow tumor-targeting T cells for cancer therapy

Scienmag

A Ludwig Cancer Research study has devised a highly efficient method to generate large numbers of immune cells specifically engineered to recognize neoantigens—small fragments of randomly mutated proteins that are typically unique to a patient’s cancer—and destroy the tumors that express them. Credit: Ludwig Cancer Research A Ludwig Cancer Research study has devised a highly […].

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ACELYRIN Scores $250 Million to Progress Innovative Antibody Mimetic

BioSpace

Los Angeles-based ACELYRIN, Inc. announced a quarter-million dollars in Series B financing Tuesday morning, along with a splashy licensing deal.

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UBC researchers train computers to predict the next designer drugs

Scienmag

UBC researchers have trained computers to predict the next designer drugs before they are even on the market, technology that could save lives. Credit: Photo by Raimond Klavins on Unsplash UBC researchers have trained computers to predict the next designer drugs before they are even on the market, technology that could save lives. Law enforcement […].

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End-of-Year Restructuring Means Layoffs for These Companies

BioSpace

It’s not unusual for biopharma companies to end the year with a restructuring that includes job cuts. Here are some of these companies.

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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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This light-powered catalyst mimics photosynthesis

Scienmag

CAMBRIDGE, MA — By mimicking photosynthesis, the light-driven process that plants use to produce sugars, MIT researchers have designed a new type of photocatalyst that can absorb light and use it to drive a variety of chemical reactions. Credit: MIT CAMBRIDGE, MA — By mimicking photosynthesis, the light-driven process that plants use to produce sugars, […].

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COVID-19 Vaccines Not Linked to Menstrual Changes; Possible Link Between SSRIs and Survival

BioSpace

Earlier studies suggest a correlation between COVID-19 vaccines and menstrual changes. A new study says otherwise.

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Study adds evidence that altered fat metabolism, enzyme, plays key role in Lou Gehrig’s disease

Scienmag

EMBARGOED TILL MONDAY, NOV. 15, AT 11 A.M. Credit: Dr. Hojae Lee EMBARGOED TILL MONDAY, NOV. 15, AT 11 A.M. A new study using genetically engineered mice and human cell and tissue samples has added to evidence that higher levels of inflammatory chemicals involved in fat metabolism occur in people with amyotrophic lateral sclerosis (ALS), […].

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JCVI advises extending COVID-19 booster programme

Pharma Times

All adults aged 40- to 49-years-old to be offered an mRNA vaccine booster dose

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The New Clinical Trial Supply Chain: Resilient, Flexible, and Patient-Centric

The global landscape of clinical trials is rapidly changing as studies become more complex. An increasing number of sponsors are seeking enhanced flexibility in their supply chains to address a variety of clinical supply challenges, including patient demand and reducing delays. Demand-led supply and direct-to-patient distribution are next-generation solutions that are helping to meet these growing needs, allowing for more streamlined processes and patient-centric studies.

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Exploring psychological resiliency of older adults with diabetes

Scienmag

PHILADELPHIA (NOVEMBER 15, 2021) – Studies suggest that exposure to the COVID-19 pandemic has been associated with a variety of different mental health consequences including reports of depression, loneliness, and insomnia. People who are more than 65 years of age and those with underlying medical conditions such as type 2 diabetes and obesity are particularly […].

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Nasal Vaccine Starts Trial to Protect Against Alzheimer's

BioSpace

?Scientists from Brigham and Women's Hospital are ready to start a clinical trial that will evaluate the use of an intranasal vaccine to prevent Alzheimer's disease.

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How the secret world of soil microbes helps keep carbon in the ground

Scienmag

AMHERST, Mass. – The largest terrestrial carbon sink on earth is the planet’s soil. One of the fears that many scientists have is that a warming planet will liberate significant portions of the soil’s carbon, turning it into carbon dioxide (CO2) gas, and so further accelerate the pace of planetary warming. One of the key […].

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Cell and Gene Therapy Catapult and Deep Science Ventures collaborate to overcome barriers in ATMPs

BioPharma Reporter

The Cell and Gene Therapy Catapult and Deep Science Ventures have formed a partnership aimed at creating innovative approaches to address barriers in the Advanced Therapy Medicinal Products (ATMP) industry.

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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.