FDA Law Blog

DECEMBER 6, 2024

By Mark A. Tobolowsky & Charles G. Raver & James E. Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. As much of the content of this draft guidance for cellular and gene therapy (CGT) products is articulated elsewhere, this document serves as a one-stop shop or Cliffs Notes for the numerous guidance documents now covering CGT product development.

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Pharmaceutical Technology

DECEMBER 6, 2024

Eli has announced a $3bn investment to expand its newly acquired manufacturing facility in Kenosha County, Wisconsin, in the US.

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Manufacturing 173

Pharmaceutical Commerce

DECEMBER 6, 2024

Addressing the bottlenecks in cell and gene therapy manufacturing through a well-designed artificial intelligence approach is essential to avoiding data siloes.

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Gene Therapy Gene Manufacturing 98

Pharmaceutical Technology

DECEMBER 6, 2024

The new FDA guidance outlines how surrogate or intermediate clinical endpoints can support accelerated approval.

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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

Medicine

XTalks

DECEMBER 6, 2024

Kinevant Sciences, a unit in Roivant, announced that its Phase II RESOLVE-Lung study of namilumab, an investigational treatment for chronic active pulmonary sarcoidosis, did not meet its primary or secondary endpoints. Consequently, the company has decided to discontinue the development of the drug for this condition. This setback comes amid progress in Roivants broader clinical pipeline.

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Trials Drugs Antibody Clinical Trials 88

Pharmaceutical Technology

DECEMBER 6, 2024

A study published in Nature estimates the economic burden associated with obesity on major US industries to be in excess of $347bn in 2023.

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Pharmaceutical Technology

DECEMBER 6, 2024

Investment for companies developing cell and gene therapies (CGTs) was discussed during the GenScript Biotech Global Forum London 2024 conference held on 20 November 2024.

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Gene Therapy Gene Development 147

Medical Xpress

DECEMBER 6, 2024

Brainmachine interfaces (BMIs) have enabled a handful of test participants who are unable to move or speak to communicate simply by thinking. An implanted device picks up the neural signals associated with a particular thought and converts them into control signals that are fed into a computer or a robotic limb. For example, a quadriplegic person is asked to think about moving a cursor on a computer screen.

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Pharmaceutical Technology

DECEMBER 6, 2024

Centenarians present a unique opportunity for researchers to explore longevity, they demonstrate impressive resilience to illness and injury.

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Research 147

Medical Xpress

DECEMBER 6, 2024

The development of an effective vaccine against the hepatitis C virus (HCV) has posed a significant challenge for decades due to the high genetic diversity of the virus. A research team has now achieved promising results. Using epitope-focused immunogens, they were able to induce broadly neutralizing antibodies (bnAbs) in laboratory models for the first time.

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Vaccine Vaccination Genetics Antibody 80

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Clinical Supply

Pharmaceutical Technology

DECEMBER 6, 2024

The US FDA has granted RMAT designation to Affimed and Artiva Biotherapeutics combination therapy for lymphoma.

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XTalks

DECEMBER 6, 2024

The US Food and Drug Administration (FDA) has granted accelerated approval to Merus Bizengri (zenocutuzumab-zbco) for the treatment of metastatic non-small cell lung cancer (NSCLC) and advanced pancreatic adenocarcinoma with NRG1 gene fusions in patients who have had disease progression during or after prior systemic therapy. The approval makes Bizengri the first and only treatment for cancers that have a NRG1 gene fusion.

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Gene In-Vivo In-Vitro Genetics 83

Pharmaceutical Technology

DECEMBER 6, 2024

Muna has entered a research alliance with GSK to expedite Alzheimer's disease treatments by discovering and validating new drug targets.

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Drugs Research 147

Medical Xpress

DECEMBER 6, 2024

There is a U-shaped association between alcohol consumption and coronary heart disease (CHD) and type 2 diabetes (T2D) but no evidence for a causal association, according to a study published online Nov. 24 in Alcohol: Clinical and Experimental Research.

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Research Cardiology 79

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White paper that delves into the complex topic of Decentralized Clinical Trials and how to master them within the confines of FDA Regulations

Clinical Trials

Pharmaceutical Technology

DECEMBER 6, 2024

A study published in Nature estimates the economic burden associated with obesity on major US industries to be in excess of $347bn in 2023.

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Medical Xpress

DECEMBER 6, 2024

Experts have used magnetic resonance imaging (MRI) to better understand the impact a gluten free diet has on people with celiac disease, which could be the first step towards finding new ways of treating the condition.

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Radiology 75

Pharmaceutical Commerce

DECEMBER 6, 2024

A guide to overcoming the top obstacles facing patient services programs today.

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Medical Xpress

DECEMBER 6, 2024

A breakthrough by researchers at Peter Mac will allow scientists to detect, analyze and profile cancer tumors in patients via a simple blood test. The Dawson lab at Peter Mac has developed a method that can take circulating tumor DNA (ctDNA) from the patient's blood and use an algorithm to identify cancer-related changes and patterns in their DNA.

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DNA Scientist Development Research 73

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Pharmaceutical Commerce

DECEMBER 6, 2024

The companies will be financially committing $1 and $3 billion into their respective manufacturing facilities.

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Manufacturing 52

Medical Xpress

DECEMBER 6, 2024

Researchers have discovered that astrocytes play an important role in regulating stress responses in the lateral septum in the brain. It is expected to provide new clues for mental health treatments.

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Regulation Research 72

Pharmaceutical Commerce

DECEMBER 6, 2024

The importance of embracing universal values and business models that prioritize humanity.

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Medical Xpress

DECEMBER 6, 2024

Exactly why do people remember what they remember? A recently published review paper from researchers at Rice University sheds light on this fundamental question and the relationship between factors that influence human memory.

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Research 70

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This new white paper defines and details the impact of Decentralized Clinical Trials on the Pharmaceutical industry and how the impact can be measured along with steps companies can take to ensure adoption.

Clinical Trials

Pharmaceutical Commerce

DECEMBER 6, 2024

In the third part of this roundtable discussion, experts discuss the value of Michigan State Universitys Axia Instituteincluding its RFID testing effortsand dive into the Institutes end-to-end RFID pilot that it has been conducting.

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Medical Xpress

DECEMBER 6, 2024

Cleveland Clinic Genome Center researchers have unraveled how immune cells called microglia can transform and drive harmful processes like neuroinflammation in Alzheimer's disease. The study, published in the journal Alzheimer's & Dementia, also integrates drug databases with real-world patient data to identify FDA-approved drugs that may be repurposed to target disease-associated microglia in Alzheimer's disease without affecting the healthy type.

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FDA Approval Genomics Genome Drugs 68

Pharmaceutical Commerce

DECEMBER 6, 2024

The latest news for pharma industry insiders.

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Medical Xpress

DECEMBER 6, 2024

A significant advance in breast cancer research has uncovered a key mechanism driving cancer invasion and drug resistance. A University of Liverpool study reveals how two critical molecules present in "HER2-positive" breast cancerone of the most aggressive formsinfluence breast cancer survival and its spread. The findings are published in the journal Science Advances.

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Research Drugs 67

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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

Clinical Research

XTalks

DECEMBER 6, 2024

In a landmark legal decision, precision oncology company Guardant Health has been awarded nearly $293 million in a false advertising trial against liquid biopsy test competitor, Natera. A jury in the US District Court for the Northern District of California was unanimous in favor of Guardant Health on all of its claims from its May 2021 lawsuit against Natera for false advertising and unfair competition.

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Containment Marketing Trials DNA 83

Medical Xpress

DECEMBER 6, 2024

The gut microbiota might perhaps one day become a routine tool for the early diagnosis of many diseases and to guide treatment, but at present there is a lack of solid scientific evidence to support these claims. Yet, day by day, there are more and more offers of commercial kits for do-it-yourself testing, at the moment totally lacking in meaning and scientific solidity.

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Regulation Research 64

Medical Xpress

DECEMBER 6, 2024

As in most years that we've followed news reporting about the myth that suicides peak during the end-of-year holidays, an analysis of the past year showed again that more newspaper accounts supported the false idea that the suicide rate increases during the holiday season than debunked it.

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Medical Xpress

DECEMBER 6, 2024

According to a new study published in Nature Communications, researchers at the Department of Laboratory Medicine, Karolinska Institutet, have made a significant leap in drug discovery and development with a new method called CeTEAM. This approach connects how drugs bind to their targets inside cells with the effects they produce, offering a clearer understanding of how a drug works.

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Development Drugs Medicine Research 61

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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.

Clinical Trials