February, 2024

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'The Top Line': Gene editing's next act

Fierce Pharma

Gene editing’s therapeutic application has transitioned from hypothetical to reality, marked by the recent approval of a CRISPR-based therapy for sickle cell and beta thalassemia. | This week on "The Top Line," Max Bayer from Fierce Biotech explores the future of gene editing in an interview with the CEO of Verve Therapeutics.

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2024 Journal Citation Reports: Changes in Journal Impact Factor category rankings to enhance transparency and inclusivity

Clarivate

New – unified rankings for each of our 229 science and social science categories; no Journal Impact Factor (JIF) rankings for the arts and humanities categories The scholarly landscape is witnessing a significant increase in both the quantity and sophistication of fraudulent behaviors, posing a substantial threat to the integrity of the scholarly record.

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Overcoming Hearing Healthcare Barriers to Realize Gene Therapy Promise

MedCity News

The preliminary results from three clinical trials administering gene therapy to address deafness from otoferlin mutations mark inspiring and incredible progress in hearing healthcare.

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Enhancing Patient Care: 4 Strategies for Life Sciences Companies

Salesforce

For many people, a sudden tingling in the leg or a feeling of numbness in the arm isn’t too much cause for alarm. The tingling or the numbness goes away after a little while, and they go on with their lives. But for Brooke Eby, an abrupt limp on her way to a conference one day ultimately led to a diagnosis of amyotrophic lateral sclerosis (ALS) at the age of 33.

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AI Strategies for Sales Managers: How to Cut Down on Tedious Admin Work

What if you could help your sellers stop wasting 72% of their day on non-selling activities and focus on bringing in revenue? Incorporating AI in your enablement workflows can help you cut down on busy work, get projects done faster, and let your team (and you!) focus on making a bigger impact. We put together this guide to show you how to use AI to cut time and costs for projects, including collateral creation, development of training videos, and automating tedious processes.

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India’s Tata Institute develops tablet for cancer recurrence prevention

Pharmaceutical Technology

India’s Tata Institute has developed a tablet combining resveratrol and copper that has the potential to prevent the recurrence of cancer.

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Roche’s Xolair is first medicine for food allergy in US

pharmaphorum

Fast approaching the end of its patent life, Roche and Novartis’ Xolair is going out with a bang, picking up a new approval as the first and only drug therapy for people with food allergies in the US.

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The Path for Prescription Drug Sales

Pharmaceutical Commerce

A dive into this market reveals that the latest numbers yield promising results—a global value of a worldwide $1.6 trillion—but it will continue to be attributed to past, present, and future healthcare spending.

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Mayo Clinic Is Helping Develop a Tool Providers Can Use to Curb Clinical Bias

MedCity News

Mayo Clinic has entered into a collaboration with TruLite Health — Mayo is helping the Phoenix-based startup develop its software platform designed to address providers’ clinical bias. The health system said it chose to collaborate with TruLite because of the platform’s potential to mitigate health inequities and enhance patient outcomes at the point of care.

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Vertex secures European approval for CRISPR cell therapy

European Pharmaceutical Review

The first CRISPR/Cas9 gene-edited therapy has been granted a marketing authorisation by the European Commission (EC). Vertex Pharmaceuticals’ CASGEVY ( exagamglogene autotemcel [exa-cel]) is conditionally approved individuals 12 years and over with severe sickle cell disease characterised by recurrent vaso-occlusive crises or transfusion-dependent beta thalassemia.

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MHRA agrees licence change for Pfizer-BioNTech’s Comirnaty vaccine

Pharmaceutical Technology

The UK MHRA has granted approval for a variation in licence of Pfizer-BioNTech’s Comirnaty XBB.1.5 vaccine targeting Omicron variant.

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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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AbbVie’s 24-hour Parkinson’s therapy to roll out on NHS

pharmaphorum

Around 1,000 people with Parkinson’s in England will be able to access treatment with AbbVie’s Produodopa, the first therapy delivered by a 24-hour subcutaneous infusion pump

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FDA approves Roche, Novartis' Xolair to prevent severe outcomes from common food allergies

Fierce Pharma

People with food allergies finally have a drug that can help prevent severe outcomes—and it’s a drug that’s been on the market for two decades. | People with food allergies finally have a drug that can help prevent severe outcomes—and it’s a drug that’s been on the market for two decades. The FDA has blessed Roche and Novartis’ Xolair as the first medicine to reduce allergic reactions that can occur with accidental exposure to certain foods.

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Long-Acting Injectable Cabenuva Found More Effective Than Daily ART at Suppressing HIV in Patients With Adherence Challenges

Pharmaceutical Commerce

Cabenuva (cabotegravir + rilpivirine) showed superior efficacy in maintaining viral load suppression in patients with HIV compared with standard of care daily oral antiretroviral treatment.

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Increasing the Impact of Opioid Settlement Funds by Investing in Health IT Infrastructure

MedCity News

Strengthening health IT infrastructure is a critical and necessary step toward abating the opioid crisis and evaluating the impact of how opioid settlement dollars are allocated across communities.

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Optimizing Clinical Supply Strategy: Navigating Challenges & Finding Your Ideal Model

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MHRA Chief Executive to step down

European Pharmaceutical Review

The Chief Executive of the Medicines and Healthcare products Regulatory Agency (MHRA), Dame June Raine, will step down in the Autumn following five years in the role. Dame June will remain in the position until then to support the new Chief Executive’s transition into the role. Leading the MHRA – a five year journey Professor Graham Cooke, interim chair of the MHRA board, remarked that Dame June’s “leadership of the MHRA over the last five years, particularly during the COVID-19 pand

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Alternative therapies are needed to combat the impending threat of AMR

Pharmaceutical Technology

The potentially disastrous consequences of antimicrobial resistance (AMR) were highlighted at the recent World Economic Forum Meeting 2024.

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FDA starts review of Argenx drug in rare disease CIDP

pharmaphorum

The FDA has said it plans to deliver a decision in June on the use of Argenx’ Vyvgart Hytrulo in rare autoimmune disease chronic inflammatory demyelinating polyneuropathy (CIDP).

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FDA sets date for high-profile CAR-T adcomm for Bristol’s Abecma, J&J’s Carvykti

Fierce Pharma

Mark your calendars, oncology drug and CAR-T therapy developers. | Mark your calendars, oncology drug and CAR-T therapy developers. The FDA has decided on a date for a highly anticipated advisory committee meeting for Bristol Myers' Abecma and J&J's Carvykti.

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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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CCC to Host Town Hall on Copyright, Artificial Intelligence, and the Training of Large Language Models

Copyright Clearance Center

February 22, 2024 – Danvers, Mass. – CCC , a leader in advancing copyright, accelerating knowledge, and powering innovation, will host the Town Hall event “The Heart of the Matter: Copyright, AI Training and LLMs” via LinkedIn Live on Thursday, 29 February, 11:00 EST/16:00 GMT. A panel of legal experts including Prof. Daniel Gervais , Vanderbilt University Law School, and Prof.

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Tampa General Hospital CEO on Change Healthcare Breach: They Are Going To Have To Give an Update Soon

MedCity News

In an interview at the Vive conference in Los Angeles, John Couris said that while he is sympathetic toward Change Healthcare’s current woes, he wants definitive answers soon.

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BioNTech agrees collaboration to accelerate autologous CAR-T therapies

European Pharmaceutical Review

BioNTech SE and Autolus Therapeutics have agreed to collaborate to advance autologous CAR-T cell therapies. Under the terms of the agreement, BioNTech will pay $50 million to Autolus, granting the immunotherapy company rights including the option to access Autolus’ commercial and clinical site network, manufacturing capacities in the UK and commercial supply infrastructure.

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The Correlation Between a New York State Law Repeal and Vaccine Coverage in Schools

Pharmaceutical Commerce

A cohort study investigates the impact of Senate Bill 2994A and answers the question: does the repeal of school-entry nonmedical vaccination exemptions result in a rise in school vaccinations?

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How to Stay Competitive in the Evolving State of Martech

Marketing technology is essential for B2B marketers to stay competitive in a rapidly changing digital landscape — and with 53% of marketers experiencing legacy technology issues and limitations, they’re researching innovations to expand and refine their technology stacks. To help practitioners keep up with the rapidly evolving martech landscape, this special report will discuss: How practitioners are integrating technologies and systems to encourage information-sharing between departments and pr

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Dermatology pharma Alys launches with $100m from Medicxi

pharmaphorum

Medicxi has combined six biotechs with dermatology medicines in development in a single drugmaker, called Alys Pharma, which starts life with $100 million in financing.

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Perrigo plots hundreds of job cuts as it embarks on restructuring initiative

Fierce Pharma

Over-the-counter self-care specialist Perrigo is poised to leave hundreds of workers by the wayside as it embarks on the next leg of its corporate journey. | As part of a cost-cutting and restructuring initiative dubbed "Project Energize," Perrigo expects to trim roughly 6% of its total staff, the company said in a fourth-quarter earnings release Tuesday.

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For Moderna, COVID vaccines were a ‘proof of concept’ — now comes the big test

PharmaVoice

Following Moderna’s COVID-19 vaccine success, therapeutics head Dr. Kyle Holen discusses scaling mRNA development to bring candidates to the finish line in oncology and more.

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‘Nation-State’ Cyberattack Hits Change Healthcare, Disrupting Pharmacy Services Across the Country

MedCity News

Pharmacies across the country are facing disruptions following a cyberattack on Change Healthcare — which is owned by Optum, a subsidiary of UnitedHealth Group. In a filing with the SEC, UnitedHealth stated that the unauthorized party that gained access to Change Healthcare’s systems was a “suspected nation-state associated cyber security threat actor.

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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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Beacon Therapeutics unveils promising interim results for gene therapy AGTC-501

Pharmaceutical Technology

AGTC-501 is in Phase II development for retinitis in the SKYLINE trial (NCT03316560) and addresses unmet needs within the retinitis space

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NICE recommends first licenced treatment for AL amyloidosis

European Pharmaceutical Review

Eligible individuals in England and Wales can access the first licensed treatment for amyloid light-chain (AL) amyloidosis on the NHS. The National Institute for Health and Care Excellence (NICE) recommended DARZALEX ® (daratumumab) plus VELCADE ® (bortezomib), cyclophosphamide and dexamethasone (DVCd) as an option for people newly diagnosed with the systemic form of the disease.

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Study backs safety of Gilead CAR-T as outpatient therapy

pharmaphorum

CAR-T therapies can achieve remarkable efficacy in the treatment of haematological cancers, but the risk of side effects means that the cell infusions are almost always administered to inpatients under close supervision in clinics.

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PBMs take bulk of blame as senators question BMS, J&J and Merck CEOs on high drug prices in US

Fierce Pharma

Thursday, when senators questioned three Big Pharma CEOs about the high price of drugs in the U.S., Health, Education, Labor and Pensions (HELP) committee chairman Bernie Sanders, I-Vermont, put th | CEOs from Bristol Myers Squibb, Johnson & Johnson and Merck faced tough questions from senators on why prices for drugs in the U.S. exceed those in other countries.

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How to Prove Marketing Value: Demonstrate Content ROI and Contribution to Sales

Every marketer knows how important it is to prove their efforts drive sales opportunities, but that’s easier said than done. When problems like sales and marketing misalignment, lack of data, and wasted efforts persist, marketers can’t measure, prove, or increase their impact on revenue at a time when demonstrating marketing value is critical. Using analyst and expert data, this guide to marketing impact and content attribution explains: How B2B buyers use content The most common types of conten