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

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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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Behind the breakthrough cancer therapy that just won a historic FDA nod

PharmaVoice

The first-of-its-kind TIL therapy for solid tumors developed by Iovance Biotherapeutics won FDA approval last week.

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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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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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European Clinical Supply Planning: Balancing Cost, Flexibility and Time

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Aurlumyn Gets First FDA Approval for a Severe Frostbite Medication

Pharmaceutical Commerce

Patients with severe frostbite who were administered Aurlumyn monotherapy experienced a significantly reduced risk of the need for amputation.

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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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Viking Therapeutics Obesity Drug’s Data Raise Best-in-Class Expectations

MedCity News

The weight loss achieved by Viking Therapeutics’ obesity drug tops results posted by Eli Lilly’s Zepbound in its pivotal test. But Viking still needs to show its results can hold up in a larger Phase 3 clinical trial.

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Positive results for MAIA’s SCLC treatment published in Nature Communications

PharmaTimes

The aggressive type of cancer accounts for 13% of all lung cancer cases worldwide

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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

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Lutris wins FDA orphan drug tag for EGFR inhibitor-induced rash candidate

Pharmaceutical Technology

LUT014 is being investigated in an ongoing Phase II trial as a first-line treatment for EGFR inhibitor-induced acneiform rash, for which there is no approved drug.

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Gilead drops CD47 drug magrolimab for blood cancers

pharmaphorum

Gilead Sciences has said it will no longer develop its CD47-targeting antibody magrolimab in blood cancers after data suggested that patients taking the drug in clinical trials were more likely to die than those in control groups.

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What Goes into Planning LogiPharma Europe?

Pharmaceutical Commerce

A dive into the intricacies of how to ensure a long-running event stays fresh and relevant to the needs of the pharmaceutical industry.

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AstraZeneca brings its cell therapy ambitions to Maryland with $300M plant investment, 150 planned hires

Fierce Pharma

Following a slew of cell therapy biotech team-ups, AstraZeneca is taking manufacturing into its own hands. | AstraZeneca is plugging $300 million into a new facility in Rockville, Maryland, to launch its cell therapy platforms in the United States for cancer trials and future commercial supply. In turn, AZ will create more than 150 new jobs.

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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.