September, 2021

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FDA halts tests of BioMarin drug amid heightened focus on gene therapy safety

Bio Pharma Dive

On the heels of a two-day FDA meeting in which experts debated the risks of gene therapy, the biotech said its early-stage PKU treatment was associated with cancer in mice.

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New report sheds light on medicine shortages in Northern Ireland following Brexit

Pharma Times

Informa Pharma Intelligence (IPI) has found that some drug firms may withdraw up to 90% of their products from Northern Ireland, once the Brexit grace period has passed.

Medicine 151
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FDA Approves First Human Trial for Potential CRISPR-Led HIV Cure

BioSpace

?The FDA's IND approval sets the stage for the very first Phase I/II trial to evaluate EBT-101 as a functional cure for chronic HIV based on the endpoints of safety, tolerability, and efficacy.

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AI could be used to guide drug therapy for heart failure

pharmaphorum

UK researchers have used artificial intelligence (AI) to develop a way to identify patients with heart failure who would benefit from treatment with beta blockers, by interrogating data from nine landmark clinical trials. The study applied machine learning to trial data involving 15,659 patients who were being treated for heart failure with reduced ejection fraction (HFrEF), a form of heart failure where the left side of the heart isn’t able to pump blood effectively around the body.

Drugs 145
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European Clinical Supply Planning: Balancing Cost, Flexibility and Time

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Telling it how it is: The need for plain language summaries in medical journals

BioPharma Reporter

Open Pharma wants to see articles in peer-reviewed medical journals provide plain language summaries: those written in everyday language that is easy to understand by anyone from patients to policymakers. It's now set out its recommendations on how to write a good plain language summary.

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How Has COVID-19 Changed the Way We Work in the Life Sciences?

XTalks

The world as we know it has changed because of the COVID-19 pandemic. We’ve adopted and embraced new technologies at a much faster pace in order to conform to these changes. Simple examples include the increased popularity of food delivery apps and at-home meal kits, but in the larger picture, the nature of working has changed entirely for many companies and people.

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JCVI does not recommend COVID-19 vaccination for all 12 to 15-year-olds

Pharma Times

UK’s chief medical officers will now provide ‘further advice’ on COVID-19 vaccination in this age group

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Growing Glioblastomas in a Laboratory: 3D Bioprinting a Better Brain Model

BioSpace

This newfound ability to take tumor cells directly from a patient and incorporate them into a more clinically accurate glioblastoma model could lead to greater patient personalization in cancer therapeutics.

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Italian ‘chatbot’ predicted COVID-19 spikes, says study

pharmaphorum

An online tool used by people in Italy to manage their potential exposure to COVID-19 using a simple chat interface has shown that it can spot emerging trends days ahead of the national surveillance system. The decision support system (DSS) – developed by Italian digital health startup Paginemediche – provided a way for people to assess and interpret their symptoms and provide guidance on what to do next, such as self-isolate or seek out a test.

Scientist 145
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Moderna to develop mRNA therapeutic for very rare disease

BioPharma Reporter

Moderna and the nonprofit Institute for Life Changing Medicines (ILCM) are collaborating to develop a new messenger RNA (mRNA) therapeutic (mRNA-3351) for Crigler-Najjar Syndrome Type 1 (CN-1), an ultra-rare disease.

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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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The right tech can add real-world context to clinical trials: Sharecare

Outsourcing Pharma

According to a leader from the health engagement solutions firm, intelligent use of technology tools can add patient-centricity and realism to a study.

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FDA advisers support booster dose of Pfizer vaccine in older or high-risk adults

Bio Pharma Dive

In a back-and-forth meeting Friday, the committee opposed clearing a third shot widely for use in people older than 16, but agreed the benefits outweighed the risks for adults over 65 or those at risk of severe COVID-19.

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Dr. Daniel Pauly’s extraordinary life and work revealed in new book

Scienmag

Dr. Daniel Pauly is the world’s most-cited fisheries scientist, but life for the UBC professor has been far from easy. The biracial son of a French woman and an American GI, he was born in Paris and kidnapped as a child to be a live-in servant for a Swiss family. He escaped to Germany at […].

Scientist 110
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Pharma in the Courts: Takeda, AbbVie, RedHill, Pfizer and Gilead

BioSpace

Aside from the fraud trial of Theranos founder Elizabeth Holmes, other court-related cases are taking place, including between Takeda and AbbVie over cancer drug, Lupron.

Trials 135
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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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Scientific study finds music can ‘tune out pain’

pharmaphorum

A study has found that listening to a specially composed music track can achieve “clinically significant” reductions in pain intensity and unpleasantness, according to the researchers behind the work. The All of Us track – composed by musician Anatole with the help of psychologist Dr Claire Howlin of University College Dublin and available via Spotify – reportedly showed a benefit in 286 people suffering from various types of acute pain, including headache, backache or period pain in

Hormones 136
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CyanVac starts Phase 1 clinical trial for intranasal COVID-19 vaccine

BioPharma Reporter

CyanVac LLC and its subsidiary, Blue Lake Biotechnology, Inc., have enrolled the first participant in a Phase 1 clinical trial of their intranasal COVID-19 vaccine in Rochester, NY.

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Purdue Pharma Is Dissolved and Sacklers Pay $4.5 Billion to Settle Opioid Claims

NY Times

The ruling in bankruptcy court caps a long legal battle over the fate of a company accused of fueling the opioid epidemic and the family that owns it.

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CDC panel clears way for Pfizer booster shots, but divided on broader use

Bio Pharma Dive

Agency advisers supported giving a third Pfizer vaccine dose to older adults and those with medical conditions. But in a break with the FDA, they voted against recommending it for younger people in high-risk jobs.

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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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TDP-43 DNA methylation may contribute to the mystery of age-dependent motor cortex involvement in ALS

Scienmag

Niigata, Japan – Amyotrophic lateral sclerosis (ALS) is an intractable neurological disease. Aging is a risk factor for the accumulation of TDP-43 (transduction responsive region DNA-binding protein of 43kDa) in the motor cortex. TDP-43 is a protein that tends to accumulate in increasing amounts, and the amount is strictly controlled by the splicing of TDP-43. […].

DNA 108
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3 Ways to Encourage Positive Mental Health at Work

BioSpace

The pandemic and its subsequent effect on the workplace has been the source of stress for many professionals and executives in various industries.

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Digital health approach improves neurosurgery care, says study

pharmaphorum

Deployment of digital health technologies during the pandemic improved patient satisfaction, expanded the delivery of care and augmented training of staff at a leading neurosurgery provider in the US, according to a new study. Faced with the disruption caused by the COVID-19 crisis, Cleveland Clinic centres implemented a number telehealth and audio-visual technologies to compensate for the difficulties in having face-to-face interactions with patients.

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Plant-based biopharma firm iBio licenses novel antibody targeting regulatory T cells

BioPharma Reporter

Anti-CD25 antibodies have emerged as one of the most promising next-generation cancer therapeutics, says US plant-based manufacturer of therapeutics and vaccines, iBio, as it enters the field.

Antibody 111
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2022 Research: The Rapid Rise of Ocean Freight Visibility

A research study conducted by The Journal of Commerce and FourKites surveyed hundreds of international shippers, exploring how their usage of global supply chain visibility technology has evolved since the onset of global disruptions caused by COVID-19. For international shippers, ocean freight visibility has evolved from optional to essential and satisfaction with visibility varies greatly depending on how it is obtained and delivered.

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NHS patients gain access to COVID-19 treatment Ronapreve

Pharma Times

Ronapreve will be targeted at hospitalised patients who have not mounted an antibody response against COVID-19

Antibody 131
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Theravance to lay off 75% of employees alongside larger restructuring

Bio Pharma Dive

The move, which will affect an estimated 270 positions, follows a review by the company's board, and comes just weeks after one of its most advanced drugs failed a key test.

Drugs 343
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Workshop and lecture on generic portfolio management

Drug Patent Watch

I’ll be leading a workshop on generic portfolio management, and also giving a talk, at the 14th annual Marcus Evans event on Portfolio Planning and Partnerships for Generics. The event…. The post Workshop and lecture on generic portfolio management appeared first on DrugPatentWatch - Make Better Decisions.

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Cost, Confusion Proving Potentially Prohibitive in Aduhelm Rollout

BioSpace

Biogen CEO Michel Vounatsos admitted Thursday that the company’s rollout of Aduhelm (aducanumab) for Alzheimer’s disease has been “slower than we anticipated.

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An Innovative & Creative Problem Solver Approach to Selling in the Medical Device Space

Speaker: Steve Goldstein, Sales Leader

Are you currently in sales, or involved in a business that depends on strong sales results? What about the extremely competitive world of medical device sales? What are some of the top challenges your customers face and how do you approach understanding what’s most important to them? Join Steve Goldstein, Sales Success Coach, Motivational Speaker and Medical Device Sales Leader from Gold Selling LLC., to discover critical strategies and approaches you can take to engage your customers, achieve g

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Bayer teams up with Huma to apply AI to lung cancer diagnosis

pharmaphorum

A research project between Bayer and digital health company Huma will use artificial intelligence to detect lung cancer in CT scans – and determine which type a patient has, in order to direct treatment. AI is already being applied by a number of groups, with some studies indicating it can even be more effective than trained radiologists in detecting subtle patterns that indicate the presence of tumours.

Genome 132
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Touchlight extends funding round, sees hike in demand for synthetic DNA vectors

BioPharma Reporter

Synthetic DNA vector producer, Touchlight, has announced the extension of its fundraising round to a current aggregate total of US$125m (Â92m). The financing was led by Bridford Investments Limited and Novator Partners.

DNA 105
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Automatic Student Loan Forgiveness for People with Disabilities

Triage Cancer

Breaking News: Thanks to a new decision by the U.S. Department of Education , many people with disabilities will receive automatic student loan forgiveness through the Total and Permanent Disability (TPD) Discharge program! What is a Total and Permanent Disability (TPD) Discharge? If you have a disability and are unable to work because of your medical condition, you might not have to pay off your remaining student loan balance.

Doctors 105
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Worrisome side effects lead Pfizer to narrow Duchenne gene therapy trial

Bio Pharma Dive

Outside trial data reviewers have attributed three cases of severe muscle weakness to Pfizer’s treatment, causing the company to change the design of its closely watched Phase 3 study.

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Unlocking Excellence: How Catalent Is Transforming Japan’s Clinical Research

Planning on running clinical trials in Japan? How can you reliably supply these studies? Discover Catalent’s clinical supply packaging facility in Shiga, Japan. Strategically located between Tokyo and Osaka, and one of largest in Japan, this 6,000 square meter facility offers comprehensive services including primary and secondary clinical packaging and labelling, comparator sourcing, cold chain storage, local and global distribution, local language support and white glove service to support stud