Thu.Nov 16, 2023

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STAT+: U.K. approves world’s first CRISPR-based medicine, giving greenlight to therapy for sickle cell, thalassemia

STAT

LONDON — Regulators in the U.K. on Thursday approved a CRISPR-based medicine to treat both sickle cell disease and beta thalassemia, making it the world’s first therapy built on the revolutionary gene-editing technology and ushering in a new phase of genetic medicine.   The authorization of the therapy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is itself not a surprise.

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AstraZeneca showed the pandemic isn’t over for immunocompromised patients. What’s next?

PharmaVoice

Dr. Paul Moss, a hematology expert in the U.K., conducted a study with AstraZeneca showing the alarming extent to which immunocompromised patients are burdened with COVID-19.

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How the Bad River Tribe flipped the script on the Native American opioid crisis

STAT

BAD RIVER RESERVATION, Wis. — Sunlight is streaming through holes in the walls of a disused, corrugated metal shack, revealing its modest insides: Dirt floors, stacks of two-by-fours, and a pile of Little Caesars pizza boxes under attack by a work crew on lunch break. This building is clearly not ready to be a home. But in the next 24 hours, it must become one.

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Top FDA official Woodcock to retire early next year

PharmaVoice

The 37-year veteran helmed the agency’s pandemic response, and was a key decision-maker in controversial calls on opioids, an Alzheimer's therapy and muscular dystrophy drugs.

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From Diagnosis to Delivery: How AI is Revolutionizing the Patient Experience

Speaker: Simran Kaur, Founder & CEO at Tattva Health Inc.

The healthcare landscape is being revolutionized by AI and cutting-edge digital technologies, reshaping how patients receive care and interact with providers. In this webinar led by Simran Kaur, we will explore how AI-driven solutions are enhancing patient communication, improving care quality, and empowering preventive and predictive medicine. You'll also learn how AI is streamlining healthcare processes, helping providers offer more efficient, personalized care and enabling faster, data-driven

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Report: Measles cases and deaths increase worldwide, as childhood vaccinations rates decline

STAT

A dangerous decline in the rate of children vaccinated against measles is spurring a global increase in cases and deaths from the highly contagious virus, according to a report released Thursday. Estimated measles cases rose 18% to 9 million in 2022 when compared to the previous year, and deaths rose by 43% to 136,200, according to the report , jointly authored by the World Health Organization and the Centers for Disease Control and Prevention.

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UK Authorizes World-First Gene Therapy from Vertex and CRISPR Therapeutics for Blood Disorders

Big Molecule Watch

Earlier today, UK’s Medicines and Healthcare products Regulatory Agency (MHRA) granted conditional marketing authorization to Vertex Pharmaceuticals and CRISPR Therapeutics for their CRISPR/Cas9 gene therapy, CASGEVY (exagamglogene autotemcel), marking the world’s first regulatory approval for a CRISPR-based gene-edited therapy. CASGEVY is the first medicine to be licensed that uses the innovative gene-editing tool CRISPR.

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World’s first CRISPR medicine approved in UK for sickle cell, beta thalassemia

BioPharma Dive

Clearance of Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy in the U.K. comes ahead of expected regulatory decisions in the U.S. and Europe.

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Opinion: What antidepressants are saying 30 years after the publication of ‘Listening to Prozac’

STAT

In 1993, I published “Listening to Prozac,” a book that grew out of my clinical experience prescribing what was then a new class of medications, ones thought to moderate depression through their effect on the way that the brain handles the neurotransmitter serotonin. Some of my patients had reported marked favorable reactions to the drugs — first Prozac and, soon after, Zoloft.

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First CRISPR-based gene-editing therapy authorised

European Pharmaceutical Review

Casgevy (exagamglogene autotemcel), also known as exa-cel, is the first treatment to be licensed that uses CRISPR. It has been conditionally authorised by the UK Medicines and Healthcare products Regulatory Agency (MHRA) as a gene-editing therapy for certain patients 12 years old and over with sickle-cell disease and transfusion-dependent β-thalassemia.

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Opinion: I worked for CMS. Even I struggle to help family navigate dual eligibility

STAT

Three months ago, my aunt Margaret had a stroke. Days before, she was repainting her bathroom and driving herself. Now, she needs help with dressing and getting in and out of bed, and our family is grappling with the fact that this fiercely self-sufficient woman will never live independently again. This health crisis has been compounded at every turn by failures of the health care system — failures that are particularly infuriating to me because I have devoted my life to solving these ver

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Why Every Small Business Needs an HCM Solution: A Comprehensive Guide

Managing HR tasks like payroll, compliance, and employee data can overwhelm small businesses. That’s where a Human Capital Management (HCM) solution comes in. Our eBook, Why Every Small Business Needs an HCM Solution: A Comprehensive Guide , shows how an HCM system automates tedious processes, ensuring your business stays compliant and efficient. You’ll learn how to simplify payroll, eliminate costly errors, and empower your employees with self-service tools.

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Wales launches first electronic prescription service

The Pharmacist

The first electronic prescription service (EPS) in Wales has launched today, allowing patients and healthcare professionals to benefit from ‘easier, safer and more efficient’ prescribing and dispensing, the Welsh Government has said. Instead of printing, signing and giving a patient a paper form to take to a pharmacy, GPs will be able to send the […] The post Wales launches first electronic prescription service appeared first on The Pharmacist.

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STAT+: FDA cites Dr. Reddy’s for quality control problems at a biologics plant

STAT

Dr. Reddy’s Laboratories, one of the world’s largest generic drugmakers, was cited by the U.S. Food and Drug Administration for a host of manufacturing violations at one of its plants in India, the latest instance in which the company was tagged by the regulator for quality-control problems. In a recent inspection report , the agency found several notable failures, such as major production equipment that was not appropriately cleaned; a quality-control unit that did not investigate

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More pharmacy staff able to deliver blood pressure checks from December

The Pharmacist

All suitably trained members of the pharmacy team will be able to deliver blood pressure checks from 1 December, it has been announced today. And there will be a 'renewed focus' on ambulatory blood pressure monitoring, helping to identify even more people at risk of heart attack and stroke, the pharmacy negotiator said. Currently, under […] The post More pharmacy staff able to deliver blood pressure checks from December appeared first on The Pharmacist.

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Colon cancer screening kits have a high rate of untestable samples

STAT

More than a tenth of fecal immunochemical tests, used for routine colorectal cancer screening, contained samples that could not be processed by labs, according to a study published this week in Cancer Epidemiology, Biomarkers and Prevention, a journal of the American Association for Cancer Research. This is over twice the amount recommended by the U.S.

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Best Practices to Streamline Compensation Management: A Foundation for Growth

Speaker: Joe Sharpe and James Carlson

Payroll optimization can be one of the most time-consuming and complex factors of small business management. Yet, organizations that crack the code on streamlining employee compensation often discover innovative avenues for growth. With the right strategies in place, outsourcing and streamlining payroll processes can result in substantial time and resource savings.

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Regional inequalities threatening Europe’s biosimilars market

European Pharmaceutical Review

While Europe has historically been a pioneer in the adoption of biosimilars, a new report by the global initiative Act4Biosimilars has revealed differences country by country, including disparities between Eastern and Western Europe. This is based on research from a report published in April this year by IQVIA. [Act4Biosimilars’ Regional Deep Dive Report Europe] noted that biosimilar adoption levels vary significantly across Europe” Act4Biosimilars shared that their Deep Dive Report

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Progress in childhood cancer has stalled for Blacks and Hispanics, report says

STAT

Advances in  childhood cancer  are a success story in modern medicine. But in the past decade, those strides have stalled for Black and Hispanic youth, opening a gap in death rates, according to a new report published Thursday. Childhood cancers are rare and treatments have improved drastically in recent decades, saving lives.

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Glox Therapeutics secures £4.3m seed funding to combat AMR

Pharma Times

The bacteriocin development programme will first target pseudomonas aeruginosa - News - PharmaTimes

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STAT+: Bristol wins U.S. approval for lung cancer drug targeting rare genetic mutation

STAT

The Food and Drug Administration on Wednesday approved a new treatment for people with non-small cell lung cancer that contains a rare genetic alteration called Ros1. The genetically targeted medicine, called Augtyro, will be sold by Bristol Myers Squibb. It was developed by a small biotech company, Turning Point Therapeutics, that was acquired by Bristol in June 2022.

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Enhance Healthcare Efficiency With Top Payroll & HCM Services

Running a healthcare facility requires precision and care, not just for patients but also for your staff. Our guide, "A Buyer’s Guide to Payroll & HCM Services," helps healthcare providers choose the best provider. Efficient payroll management ensures timely, accurate payments, critical for maintaining staff morale and trust. Compliance support helps navigate complex healthcare regulations and avoid costly fines.

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Community pharmacists to gain greater access to GP record from January

The Pharmacist

Community pharmacists will be able access and add to patient records currently maintained by GPs from January next year, in a bid to free up millions of GP appointments. The Government, NHS England and Community Pharmacy England said they agreed that the Pharmacy First scheme will launch on 31 January 2024, subject to the ‘appropriate digital […] The post Community pharmacists to gain greater access to GP record from January appeared first on The Pharmacist.

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How do we know Alzheimer’s drugs ‘work’?

STAT

Want to stay on top of the science and politics driving biotech today?  Sign up  to get our biotech newsletter in your inbox. Good morning, everyone. Damian here with an update on a thwarted gene therapy study, a head-to-head fight in GLP-1, and a critical look at Alzheimer’s treatment.

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Retaining the human element of AI within pharma and healthcare

pharmaphorum

In this episode of the pharmaphorum podcast, web editor Nicole Raleigh speaks with Venu Mallarapu, vice president of global strategy and operations at eClinical Solutions, about the ins and outs of successful AI implementation within pharma and healthcare.

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Listen: A CRISPR milestone, algorithms amok, & biotech mixology

STAT

Who polices algorithms? How would you name the first approved CRISPR medicine? And what pairs well with a biotech downturn? We cover all that and more this week on “The Readout LOUD,” STAT’s biotech podcast. Our colleague Casey Ross joins us to tell the bombshell story of how the nation’s largest health insurer used a computer algorithm to deny patient care and boost its profitability.

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Position Your Pharmacy for Expansion

Speaker: Chris Antypas and Josh Halladay

Access to limited distribution drugs and payer contracts are key to pharmacy expansion. But how do you prepare your operations to take the next step? Meaningful data: Collect and share clinical data regarding outcomes, utilization, and more Reporting: Limited distribution models require efficient tracking and reporting systems Workflows: Align workflows with specific pharma and payer contractual requirements For in-depth, expert insights on pharmacy expansion, watch this webinar from Inovalon.

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LifeArc and UKDRI invest £14.5m into neurodegeneration research

Pharma Times

The funding will be used to develop new diagnostic tests, treatments and devices - News - PharmaTimes

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STAT+: Pharmalittle: U.K. approves world’s first CRISPR-based medicine; FDA to review pricey cancer drug lacking confirmatory data

STAT

Rise and shine, everyone, another busy day is on the way. We can tell because the official mascots are racing about the Pharmalot grounds and the parade of vehicles outside our window is picking up. As for us, we are dutifully firing up the coffee kettle to brew another cup of needed stimulation. Our choice today is butter pecan. Please feel free to join us.

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How Digital Twins Drive Sustainability in Clinical Supply Planning

PharmExec

Emerging technologies like digital twins offer supply chain efficiency solutions, allowing the industry to integrate sustainable practices while ensuring patient safety.

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FDA approves new Bristol Myers drug for lung cancer

BioPharma Dive

Acquired via a $4 billion biotech buyout, Augtyro is one of an array of new products the pharma hopes will offset patent expirations for current top sellers.

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5 Reasons to Upgrade Your Pharmacy Management Software

Are you still using workarounds to manage your daily operations? To achieve peak performance, it's time to explore other options for specialty and infusion pharmacy software. Streamline pharmacy operations and improve clinical performance with automated processing, real-time data exchange, and electronic decision support. Download this helpful infographic to: Drive efficiency and patient adherence from referral receipt to delivery and ongoing care – all with our Pharmacy Cloud.

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Single-use bioprocessing market to value $84.14bn by 2032

European Pharmaceutical Review

The global single-use bioprocessing market is expected to value estimated $84.14 billion by 2032, according to Towards Healthcare. This is due to rising demand for personalised medicines, rising investment in R&D, and adoption of decentralised clinical trials. The organisation’s report asserted that growth of the market is being driven by numerous factors including the greater demand for biologics, the expanding biopharmaceutical industry, and the numerous advantages that single-use bioproce

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West Coast investor Red Tree on its San Diego expansion and staying bullish in biotech

BioPharma Dive

Red Tree Venture Capital plans to tap into San Diego’s biotech pipeline as it works to build a West Coast competitor to Boston-area investors.

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World’s first CRISPR-based gene therapy approved for sickle cell anaemia

Pharmaceutical Technology

Vertex and CRISPR's Casgevy has received conditional approval in the UK for treating sickle cell and transfusion-dependent beta-thalassemia.

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Homology enters reverse merger with Q32 Bio

BioPharma Dive

The deal, which will result in a combined company operating under Q32's name, comes months after Homology laid off much of its staff and halted research.

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Pharmacy First: Services ‘bundle’ required by March 2025 for monthly payments

The Pharmacist

Payments for Pharmacy First, the expanded Pharmacy Contraception Service and the re-launched blood pressure check service will be ‘bundled’ together by March 2025, meaning pharmacies will have to provide all three to be eligible for funding. It was confirmed today that the highly anticipated Pharmacy First service is set to launch at the end of […] The post Pharmacy First: Services ‘bundle’ required by March 2025 for monthly payments appeared first on The Pharmacist.

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