Tue.Jun 21, 2022

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Mixed results for PTC Duchenne drug put spotlight on EU approval

Bio Pharma Dive

A confirmatory study of PTC Therapeutics’ muscular dystrophy treatment Translarna missed its main goal, although the company highlighted the trial’s overall positive results.

Drugs 306
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AZ preps FDA filing for $3.6bn amyloidosis drug eplontersen

pharmaphorum

Armed with new phase 3 data, AstraZeneca is preparing to file for regulatory approval of eplontersen, an antisense drug for one of the complications of the disease transthyretin amyloidosis (ATTR) it licensed from Ionis in a $3.6 billion deal last year. Eplontersen – previously known as IONIS-TTR-LRX – is designed to switch off the production of transthyretin (TTR), a protein which builds up in the disease to toxic levels causing heart problems (cardiomyopathy) and nerve damage (polyneuropathy).

Drugs 111
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Ionis, AstraZeneca claim success for competitor to Alnylam rare disease drug

Bio Pharma Dive

A medicine at the center of a multibillion-dollar alliance between the two companies passed a major test in transthyretin amyloidosis. Full study details were not disclosed, however.

Medicine 285
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University of Strathclyde develops 3D breast cancer screening tool

Pharma Times

Pre-clinical screening technology could increase cancer test numbers by 50 times

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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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Pfizer takes stake in Lyme disease partner ahead of key study

Bio Pharma Dive

Valneva will receive $95 million from Pfizer to support development of a vaccine candidate as the French company’s COVID-19 work sputters.

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CO2COA is Mars’ Latest Animal-Free, Planet-Friendly Chocolate Bar

XTalks

Mars recently announced the launch of CO2COA , an animal-free, lactose-free and earth-friendly chocolate bar, in the US. Partnering with Perfect Day to swap regular milk for animal-free dairy, CO2COA promises to deliver the same taste and texture of traditional chocolate, but without the downsides of factory farming cattle. Chocolate lovers, rejoice!

Protein 98

More Trending

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IP is no barrier to COVID vaccine access, says industry

pharmaphorum

While proponents argue waiving IP on COVID-19 vaccines would better enable low- and middle-income countries to inoculate their populations, the pharma industry has called the proposals “unnecessary and harmful to innovation”. Proposals to waive COVID-19 product patents have been described as “political posturing” and an “answer to a problem that does not exist”.

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A biotech startup launches with $38M to develop a cystic fibrosis gene therapy

Bio Pharma Dive

Carbon Biosciences believes its technology, which is based on parvoviruses, could allow for larger gene delivery and repeat dosing.

Gene 200
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Rensselaer researchers make virus-fighting face masks

Scienmag

Rensselaer Polytechnic Institute researchers have developed an accessible way to make N95 face masks not only effective barriers to germs, but on-contact germ killers. The antiviral, antibacterial masks can potentially be worn longer, causing less plastic waste as the masks do not need to be replaced as frequently. Credit: Gregi69 Rensselaer Polytechnic Institute researchers have […].

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Boost your copay program's visibility before it's too late

Bio Pharma Dive

Give your copay program the gift of awareness.

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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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Designable spatially coherent wideband radiation and frequency conversion

Scienmag

Components of a typical laser include gain medium, laser pumping energy and resonant optical cavity. The output frequency of most lasers is fixed or within a small range, and it is difficult to generate lasers of some frequencies by common ways. Regarding some practical applications, optical frequency conversion technology which can convert the laser into […].

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Diversity Makes Clinical Trial Recruitment Easier and Outcomes Better

BioSpace

Decentralized clinical trials may cost more upfront, but can have huge returns on the back end, according to speakers at Informa Connect’s Decentralized Clinical Trials meeting.

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Feelings of detachment predict worse mental health outcomes after trauma

Scienmag

Results from the largest prospective study of its kind indicate that for individuals who experience trauma, the presence of dissociation—a profound feeling of detachment from one’s sense of self or surroundings—may indicate a high risk of later developing severe post-traumatic stress, depression, anxiety, physical pain, and social impairment. The research, which was led by investigators […].

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Biden Nominates First Woman and Person of Color to Serve as Science Advisor

BioSpace

U.S. President Joe Biden nominated Arati Prabhakar, Ph.D., to be his science advisor. If confirmed by the Senate, Prabhakar will become the first woman, first person of color and the first immigrant to hold the position.

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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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New WVU program trains next generation of toxicologists to collect, analyze air samples from mining, fracking sites

Scienmag

West Virginia University is launching a toxicology training program that offers students something no other academic program does: experience collecting air samples, identifying the toxicants they contain and determining how those toxicants might harm a community’s health. Credit: WVU Photo West Virginia University is launching a toxicology training program that offers students something no other academic program […].

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Krystal Biotech Submits BLA for Rare Skin Disease Treatment

BioSpace

Krystal Biotech announced that it submitted a Biologics License Application to the FDA for its candidate B-VEC, intended for the treatment of dystrophic epidermolysis bullosa.

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MIT engineers devise a recipe for improving any autonomous robotic system

Scienmag

Autonomous robots have come a long way since the fastidious Roomba. In recent years, artificially intelligent systems have been deployed in self-driving cars, last-mile food delivery, restaurant service, patient screening, hospital cleaning, meal prep, building security, and warehouse packing. Credit: Image courtesy of Charles Dawson, ChuChu Fan, et al Autonomous robots have come a long […].

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As Plenity Gains Popularity for Weight Loss, Some Question its Efficacy

BioSpace

Gelesis' weight loss device, Plenity, has been gaining popularity since the FDA approved its commercialization, but some in the medical community question whether its popularity is deserved.

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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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Where do COVID-19 variants come from?

Scienmag

Patients with weakened immune system are at risk for chronic infection and develop highly mutated virus variants Credit: Tel Aviv University Patients with weakened immune system are at risk for chronic infection and develop highly mutated virus variants New study finds that immunocompromised patients tend to develop chronic coronavirus infections, leading to highly mutated […].

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National Association of Specialty Pharmacy (NASP) 2022 Annual Meeting & Expo

Drug Channels

National Association of Specialty Pharmacy (NASP) 2022 Annual Meeting & Expo. September 19-22, 2022 | Gaylord Palms Resort & Convention Center | Orlando, FL. www.naspnet.org/annualmeeting. The National Association of Specialty Pharmacy (NASP) will host the NASP 2022 Annual Meeting & Expo September 19-22. NASP will celebrate their 10th Anniversary and welcome attendees to a new conference venue – the gorgeous Gaylord Palms Resort & Convention Center, located in Orlando, Florida.

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NCCR MARVEL team wins award for ground-breaking work on 2D materials

Scienmag

Investigations into a fascinating class of novel materials that could underpin next-generation electronic and optoelectronic applications and quantum computing has won a team led by Nicola Marzari the inaugural PRACE (Partnership for Advanced Computing in Europe) HPC Excellence Award. It was established to honor “the most distinguished projects and researchers in the field of high-performance […].

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TC BioPharm’s Co-Stimulatory CAR Therapy May Be Safer and More Economical

BioSpace

TC BioPharm is developing a new approach to chimeric antibody receptor (CAR) T-cell therapies with the potential to eliminate off-target effects, and possibly help more patients.

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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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Study highlights undiscovered potential of bacterial compounds and genes linked to colon cancer-related toxin

Scienmag

The last two decades have seen the development of sophisticated computational tools that explore the DNA of bacteria. These tools are on the lookout for interesting metabolites (metabolism-related molecules) that illicit a strong biological reaction. Their impact might be toxic, or it might be life enhancing; for example, informing the development of new antibiotics, anti-cancer […].

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Carbon Biosciences launches with $38m Series A financing round to advance cystic fibrosis gene therapy candidate to clinic

BioPharma Reporter

The developer says it is harnessing parvovirus vectors that can deliver larger gene therapy payloads with enhanced tissue specificity and with minimal neutralizing immunity.

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University of Illinois researchers derive new theory on behavior of new class of materials

Scienmag

Researchers led by CEE Professor Oscar Lopez-Pamies have derived the governing equations that describe and explain the macroscopic mechanical behavior of elastomers filled with liquid inclusions directly in terms of their microscopic behavior. The work is described in an article by Lopez-Pamies and Ph.D. student Kamalendu Ghosh recently published in the Journal of the Mechanics and Physics […].

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Single MRI-based brain scan could diagnose Alzheimer’s disease, says study

pharmaphorum

Alzheimer’s disease could be diagnosed from a single MRI brain scan, according to a new study by Imperial College London. Using machine learning technology, researchers were able to examine structural features of the brain, including regions that are not traditionally associated with Alzheimer’s. The simplicity of this process, scientists suggest, could aid clinicians in identifying the disease at an early stage when it can be challenging to diagnose.

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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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Nature journal publishes UTEP-led pollution study

Scienmag

EL PASO, Texas (June 16, 2022) — Residents of public housing throughout the United States experience higher levels of air pollution, according to an inter-institutional study led by a researcher from The University of Texas at El Paso that appeared in Scientific Reports, one of Nature’s portfolio of journals. According to its website, Scientific Reports […].

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AbbVie Seeks New Indication for Migraine Drug Qulipta, Drops Deal with Morphic

BioSpace

AbbVie filed a supplemental New Drug Application with the U.S. Food and Drug Administration seeking approval for Qulipta for the preventative treatment of migraine.

Drugs 97
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New insights of how the HIV-1 assembles and incorporates the Env protein

Scienmag

BIRMINGHAM, Ala. – Assembly of HIV-1, which causes AIDS, takes place on the inner plasma membrane leaflet of infected cells, a geometric building process that creates hexamers out of trimers of the viral Gag protein, as guided by Gag’s N-terminal matrix domain. Credit: UAB BIRMINGHAM, Ala. – Assembly of HIV-1, which causes AIDS, takes place […].

Protein 83
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New Research Adds to Momentous Month for Colorectal Cancer Treatment

BioSpace

Researchers at the University of California, San Diego published promising preclinical work on a new combination therapy to treat colon cancer.

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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.