Fri.Dec 08, 2023

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FDA approves bluebird's sickle cell disease gene therapy. Can Lyfgenia overcome CRISPR’s halo?

Fierce Pharma

Alongside a historic approval for the first therapy utilizing the Nobel Prize-winning CRISPR/Cas9 gene-editing technology, the FDA has cleared a rival gene replacement therapy, also for sickle cell | Alongside a historic approval for the first therapy utilizing the Nobel Prize-winning CRISPR/Cas9 gene-editing technology, the FDA has cleared a rival gene replacement therapy, also for sickle cell disease (SCD).

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‘Increasingly Concerned’ Senators Launch Investigation Into Private Equity’s Effect on Healthcare

MedCity News

This week, two bipartisan members of the Senate Budget Committee launched an investigation into private equity and its impact on healthcare. They initiated the probe to get answers about “questionable financial transactions” that could be hurting care quality for patients at hospitals owned by private equity firms.

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Vertex, CRISPR score landmark FDA approval for sickle cell disease gene therapy Casgevy

Fierce Pharma

Groundbreaking? Game-changing? Transformational? Historic? | Vertex Pharmaceuticals and CRISPR Therapeutics have won an historic FDA approval for their sickle cell disease gene therapy Casgevy. The transformational treatment is a potential cure for the debilitating and life-threatening disease which affects more than 100,000 in the United States, most of them Black.

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How IT Became So Integral in Healthcare in So Little Time

MedCity News

Looking back 20 years, when fewer than 18% of physician practices used electronic health records, few experts would have anticipated how information technology has changed healthcare. Thanks to IT, the volume and types of data generated and the speed at which they can be analyzed are vastly different than decades ago.

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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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Bristol Myers Squibb bids farewell to dealmaking czar Elizabeth Mily

Fierce Pharma

Three years on, Bristol Myers Squibb is bidding adieu to its top dealmaker. | Three years on, Bristol Myers Squibb is bidding adieu to its top dealmaker. Elizabeth Mily, who replaced BMS’ former M&A czar Paul Biondi back in 2020, is headed for the exit, the company confirmed Friday.

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Are Registered Dietitians the Key To Optimizing Weight Loss With GLP-1s?

MedCity News

Prescriptions for GLP-1s are soaring, but fewer than a third of people prescribed the costly drugs will continue taking them one year later without personalized support. Without the expert guidance of a registered dietitian, you risk not achieving the significant clinical benefits these drugs offer and forfeiting the potential for sustainable, transformative health outcomes.

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FDA Approves Two Landmark Cell-Based Gene Therapies for Sickle Cell Disease

PharmExec

FDA approval of bluebird bio’s Lyfgenia and Vertex Pharmaceuticals' and CRISPR Therapeutics’ Casgevy marks significant milestone in the treatment of sickle cell disease.

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Applying the Economy-Wide Lessons of Digital Payments to Clinical Trial Retention

MedCity News

To help keep participants engaged, and on trial, payment and reimbursement systems should offer the same level of flexibility that people have come to expect in every other walk of life.

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AbbVie Collaborates with BigHat Biosciences on Next-Gen Therapeutic Antibodies

PharmaTech

This collaboration will combine BigHat Biosciences’ AI/ML-guided Milliner platform with AbbVie's expertise in oncology and neuroscience to develop next-generation antibodies.

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FDA Approves Not One, But Two Sickle Cell Disease Gene Therapies

MedCity News

The first gene therapies for sickle cell disease are now approved: Casgevy from Vertex Pharmaceuticals Casgevy and Lyfgenia from Bluebird Bio. Despite the same-day approval, key differences give one of these therapies commercialization advantages over the other.

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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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AbbVie to buy neuroscience specialist Cerevel Therapeutics for $8.7bn

European Pharmaceutical Review

AbbVie has signed a definitive agreement to acquire Cerevel Therapeutics and its neuroscience pipeline – including antipsychotic emraclidine – for approximately $8.7 billion. Cerevel’s pipeline includes multiple clinical-stage and preclinical candidates with potential across several diseases including schizophrenia, Parkinson’s disease, and mood disorders.

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Cambridge researchers redesign future mRNA therapeutics

PharmaTimes

One-third of patients who received Pfizer’s COVID-19 vaccine had unintended immune responses - News - PharmaTimes

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MHRA authorises Mounjaro for weight management

European Pharmaceutical Review

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has authorised diabetes medicine, Mounjaro (tirzepatide), for weight loss and weight management in adults aged 18 and over. Mounjaro is now authorised for adults with a BMI of 30kg/m² or more (obesity), as well as those with a BMI between 27-30kg/m² (overweight) who also have weight-related health problems eg, prediabetes, high blood pressure, high cholesterol, or heart problems.

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AAP Responds To “Flawed” GenAI Arguments

Copyright Clearance Center

AAP responded to tech industry assertions that respect for copyright is an obstacle to their innovation by labeling that as, “nonsense.

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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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Pfizer’s multiple myeloma treatment secures conditional approval in EU

Pharmaceutical Technology

Pfizer has added the EU to its growing number of markets for its targeted immunotherapy intended for relapsed/refractory multiple myeloma.

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The Human Behind the Screen: Why Empathy Matters in your Healthcare IT Strategy

MedCity News

As healthcare IT undergoes continual evolution, the integration of automation and human connection is poised to define the trajectory of IT support. This synergy promises a smooth experience for users and ultimately contributes to enhanced patient care.

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A Guide on Determining Stability of Molecular Crystals in Pharmaceutical Drug Development

Pharmaceutical Commerce

Study analyzes and predicts how conditions in research and development can be predicted via computer simulation.

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Signal: US lays out plan to seize govt-funded drug patents to lower cost

Pharmaceutical Technology

The newly announced plans would allow third parties to manufacture such drugs if manufacturers make them unaffordable.

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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.

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FDA clears two sickle-cell gene therapies, including CRISPR first

pharmaphorum

The US FDA has cleared two novel gene therapies for sickle cell disease: Vertex Pharmaceuticals' Casgevy and bluebird bio's Lyfgenia.

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BioMed X launches call for applications in autoimmune disease research

PharmaTimes

The project will explore the biology of human autoreactive plasma cells - News - PharmaTimes

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PharmStars digital health accelerator names fall 2023 grads

pharmaphorum

PharmStars digital health accelerator names fall 2023 grads Phil.

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StartUPDATES: New developments from healthcare startups

MedCity News

Check out news from FreezeNit, Vascular Perfusion Solutions, Kandu, and BetterYou.

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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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Africa-led HIV vaccine trial ends in failure

pharmaphorum

Africa-led HIV vaccine trial ends in failure Phil.

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Patient Referrals: A Game-Changer for Dermatology Clinics

Referral MD

Discover the best practices to enhance, improve, and boost patient referrals for your dermatology clinic. Understanding the Importance of Patient Referrals Patient referrals play a crucial role in the success of dermatology clinics. When patients refer someone to your clinic, they are satisfied with your services. Referrals also help to build trust and credibility for your clinic, as people are more likely to trust the recommendation of a friend or family member.

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Spyre raises $180m to advance IBD antibody pipeline

pharmaphorum

Spyre raises $180m to advance IBD antibody pipeline Phil.

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Lyfgenia, Casgevy Become First Cell-Based Gene Therapies to Gain FDA Approval for Sickle Cell Disease

Pharmaceutical Commerce

FDA approves bluebird bio’s Lyfgenia and Vertex Pharmaceuticals' and CRISPR Therapeutics’ Casgevy for the treatment of sickle cell disease, which affects approximately 100,000 people in the United States.

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Clinical Data Like You´ve Never Seen It Before: Why Spotfire Is the Leading Tool for Clinical Analytics

Clinical development organizations face a wide array of challenges when it comes to data, many of which can impact the operational effectiveness of their clinical trials. In this whitepaper, experts from Revvity Signals explore how solutions like TIBCO® Spotfire® enable better, more streamlined studies. The whitepaper also features a success story from Ambrx, a leading biopharmaceutical company, detailing how it has leveraged Spotfire to tackle data quality and collaboration challenges in clinic

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AbbVie to Acquire ImmunoGen in Deal Valued at $10.1 Billion

PharmaTech

AbbVie’s proposed acquisition of ImmunoGen is expected to accelerate AbbVie's entry into the commercial market for ovarian cancer and includes ImmunoGen’s flagship cancer therapy, ELAHERE (mirvetuximab soravtansine-gynx).

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MSD’s Keytruda and Eisai’s Lenvima fail in Phase III uterine cancer trial

Pharmaceutical Technology

The combination therapy of Keytruda and Lenvima failed to meet the dual primary endpoints in a Phase III advanced endometrial cancer trial.

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MilliporeSigma Launches AI Solution for Integrating Drug Discovery and Molecule Synthesis

PharmaTech

MilliporeSigma’s new platform combines generative AI, machine learning, and computer-aided drug-design aimed at increasing the success rate of new drugs and therapies.

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FDA accepts Alpha Cognition’s NDA for Alzheimer’s disease

Pharmaceutical Technology

The US FDA has accepted Alpha Cognition’s new drug application (NDA) for ALPHA-1062 to treat mild-to-moderate Alzheimer’s disease.

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Clinical Supply Strategies for CROs

When a CRO is bidding on a project where clinical supplies will be one of the aspects to manage on behalf of the client via a partner, leveraging the expertise of a chosen clinical supply partner can be a valuable resource in demonstrating the CRO’s understanding of and ability to deliver upon critical drug-supply related aspects of the project, and ability to hit key milestones such as FPI for their client.