thought leadership

DECEMBER 9, 2024

Today's manufacturing environment is shaped by the rise of advanced therapies, including biologics, cell and gene therapies , and personalized medicine, each demanding highly specialized processes and controls.

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Science Compliance Regulations FDA 75

Advarra

NOVEMBER 14, 2024

In an era where clinical trials are increasingly global, it’s more imperative than ever to leverage international expertise. Enhanced objectivity: A DMC composed of diverse international experts can mitigate potential biases arising from local or regional perspectives. local standards of care).

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Clinical Trials Trials International Clinical Research 97

ASPET

SEPTEMBER 28, 2023

Benzodiazepine pharmacoresistance is thought in part to result from internalization of synaptic GABA A receptors, which are the main target of the drug. The naturally occurring neurosteroid allopregnanolone is a therapy of interest against SE for its ability to modulate all isoforms of GABA A receptors.

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Therapies Treatment International Drugs 100

DrugBank

JUNE 13, 2024

Unlike traditional oral medications with limited bioavailability and potential for erratic absorption, IDDS function as internal drug repositories, delivering precise doses of medication directly at the site of action or into the systemic circulation via precisely adjusted release mechanisms. 

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Fierce BioTech

JUNE 11, 2025

The biologics revolution is in full swing, and as innovation accelerates, so does the complexity of bringing these life-changing therapies to market. | The biologics revolution is in full swing, and as innovation accelerates, so does the complexity of bringing these life-changing therapies to market.

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Therapies International Science Marketing 52

Drug Discovery Today

NOVEMBER 10, 2022

A team of surgeons and scientists from the UK, Sweden and Canada, funded by Rinri Therapeutics, has confirmed secure surgical access to the central core of the human cochlea The research, published in Scientific Reports, is critical to the first in-human trials of new cell, gene and drug therapies for the inner ear, and will assist with treatment for (..)

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International Research Therapies Trials 100

Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Safety biomarkers account for adverse effects of a therapy under study. There are several types of biomarkers to consider.

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Clinical Development Clinical Trials Clinical Pharmacology Trials 69

Drug Target Review

JUNE 5, 2025

Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.

Disease 52

Disease Treatment Therapies Nurses 52

The Pharma Data

JUNE 16, 2025

Teva and Fosun Pharma Forge Strategic Partnership to Develop and Commercialize Innovative Anti-PD1-IL2 Therapy (TEV-56278) in Immuno-Oncology Teva Pharmaceutical Industries Ltd. TEV-56278 is a first-in-class anti-PD1-IL2 ATTENUKINE therapy designed by Teva’s internal team of innovative scientists.

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PPD

JANUARY 2, 2025

The need for FSP support across different functions may arise because the sponsor has chosen to focus its core workforce on critical internal competencies, leaving other in-house functions undeveloped. Using this approach, CROs can integrate single or multiple FSP services into an existing FSO arrangement or from the outset.

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Clinical Development Clinical Trials Trials Regulations 98

PPD

DECEMBER 16, 2024

Rising costs have become a persistent challenge for drug developers, driven by a combination of internal and external pressures that have intensified in recent years. Lacking internal expertise or capacity often slows progress, making outsourcing a strategic necessity.

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Drug Development Clinical Trials Compliance Trials 52

The Premier Consulting Blog

JANUARY 14, 2025

As the oncology landscape continues to evolve, the OCE aims to improve access to safe, effective, and cutting-edge therapies through streamlined regulatory processes and patient-centered approaches. These include Fast Track Designation, Breakthrough Therapy Designation, Priority Review Designation, and the Accelerated Approval Pathway (AAP).

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Clinical Trials Therapies FDA Trials 52

Drug Target Review

JUNE 11, 2025

Drug development faces significant challenges: long timelines, high costs , complex processes and low probabilities of success (PoS), exacerbated by the shift towards more complex molecules, biologics and cell and gene therapies, hindering patient access to vital treatments. flash (powering the free Google App tier) and Claude Sonnet 3.5.

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Drugs Clinical Trials Drug Development Pharmaceuticals 64

New Drug Approvals

APRIL 11, 2025

2] [3] Names Fitusiran is the international nonproprietary name. [4] This article incorporates text from this source, which is in the public domain. ^ “Qfitlia approved as the first therapy in the US to treat hemophilia A or B with or without inhibitors” Sanofi (Press release). 26 March 2025. 28 March 2025.

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Clinical Trials FDA FDA Approval Trials 107

Drug Target Review

DECEMBER 11, 2024

By employing advanced analytical techniques like high-resolution mass spectrometry (HRMS) and carefully calibrating systems with optimal internal standards (IS), we effectively mitigate matrix effects and provide reliable preclinical toxicology data. These solutions must contain analytes and internal standards at known concentrations.

International 52

International Laboratories Drugs Pharmacokinetics 52

The Pharma Data

JUNE 6, 2025

Sanofi Highlights Robust Pipeline of Innovations in Rare Blood Disorders at ISTH 2025 Sanofi is poised to take center stage at the 33rd Congress of the International Society on Thrombosis and Haemostasis (ISTH), set to be held in Washington, D.C., and EU, in late 2024 as the first therapy of its kind. from June 21 to 25, 2025.

Disease 40

Disease Therapies Treatment International 40

SCIENMAG: Medicine & Health

JUNE 21, 2023

New research published in the Journal of Internal Medicine demonstrates that optogenetics—which uses light-sensitive proteins to control the activity of targeted cells—is a promising shock-free approach to treating atrial fibrillation (AF), or an irregular, often rapid heart rate, for immediate restoration of regular rhythm.

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Therapies International Treatment Research 64

Translation

JUNE 11, 2025

Kazuyuki Dan, Scientific Finding Manager, Collaborate to Cure Hub Japan A Look at the Startup: Rege Nephro (Japan) Led by CEO Akifumi Morinaka , Rege Nephro is advancing therapies for autosomal dominant polycystic kidney disease (ADPKD), a life-threatening condition marked by progressive cyst growth in the kidneys. market preparation.

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Science International Disease Laboratories 52

ASPET

AUGUST 2, 2024

Based on previous studies, we hypothesized that vascular senescence increases the risk of bleeding during warfarin therapy. After validating the models, we examined changes in the International Normalized Ratio (INR) at fixed warfarin doses (0.20 This study aimed to explore these effects using animal models and clinical cohorts.

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The Pharma Data

JUNE 16, 2025

a principal investigator for the TRANSCEND FL study and a lymphoma and cell therapy specialist at Memorial Sloan Kettering Cancer Center in New York. This high durability highlights the sustained Benefit provided by liso-cel in a population typically faced with poor prognosis after multiple lines of standard therapy. months, and 24.5

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ASPET

NOVEMBER 17, 2023

This response may result from gamma-aminobutyric acid A receptors (GABA A R) internalization that follows prolonged SE; this receptor trafficking results in fewer GABA A R in the synapse to restore inhibition. Benzodiazepine pharmacoresistance develops when treatment of status epilepticus (SE) is delayed.

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Treatment Therapies International Drugs 100

SCIENMAG: Medicine & Health

JUNE 15, 2023

A new therapy is on the horizon for patients with metastatic colorectal cancer who have run out of treatment options. Credit: Vanderbilt University Medical Center A new therapy is on the horizon for patients with metastatic colorectal cancer who have run out of treatment options.

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Treatment Clinical Trials Therapies International 83

The Pharma Data

JANUARY 19, 2021

Estrogen Therapy Need Not Stop for Gender-Affirming Surgery. 19, 2021 — For transgender and gender nonbinary (TGNB) patients undergoing primary vaginoplasty, perioperative venous thromboembolism (VTE) occurs rarely, regardless of whether hormone therapy is suspended prior to surgery, according to a study published online Jan.

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Therapies International Research 52

Elrig

MARCH 25, 2025

GOLD Lab collaborates with leading international consortia, including Genomics England and FANTOM (Functional Annotation of the Mammalian Genome). His team has developed CRISPR-Cas and cancer-driver methods for the discovery of oncogenic lncRNAs that are promising targets for therapeutic oligonucleotides.

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RNA Bioinformatics Science Pharmacokinetics 59

Drug Target Review

FEBRUARY 13, 2024

In light of the International Day of Women and Girls in Science, which celebrates the achievements of women in STEM, could you share an example of a project or research that has been particularly impactful within your field? I started my career in the early stages of the targeted approach to drug therapies.

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International Science Drugs Engineering 59

Drug Target Review

OCTOBER 30, 2024

Organoids will play increasingly crucial roles not only in research and diagnostics, but also in therapies to restore and enhance the human body. About the author Anirban Datta, Head of Discovery Biology at Verseon International Corporation Anirban Datta PhD is the head of discovery biology at Verseon International Corporation.

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Treatment Disease Clinical Trials Research 105

The Pharma Data

JUNE 15, 2025

Extramedullary Disease — An Unmet Need in Multiple Myeloma Extramedullary disease occurs when multiple myeloma evolves and forms tumors (plasmacytomas) outside of bone structures — for instance, in soft tissues and various internal organs. The combination therapy resulted in 61% of patients progression-free and alive at 1 year.

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Therapies Treatment Disease FDA Approval 40

The Pharma Data

JUNE 9, 2025

The acceptance, which follows the CDE’s grant of Priority Review status in May, represents an accelerated regulatory pathway for what may become the first approved systemic therapy for TGCT in China. As Hong Chow emphasized, “We are proud to bring forward a therapy that has the potential to be truly life-changing for people with TGCT.

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Treatment Therapies Small Molecule Marketing 40

FDA Law Blog: Drug Discovery

NOVEMBER 2, 2023

Lewis, Senior Regulatory Device & Biologics Expert — On October 20, 2023, FDA announced the availability of the final guidance authored by CBER titled “Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies.” It finalized a draft guidance published in 2022.

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Therapies FDA Production Regulations 59

The Pharma Data

JUNE 16, 2025

FDA Approves ANDEMBRY (garadacimab-gxii) The First Once-Monthly Prophylactic HAE Therapy Targeting Factor XIIa CSL a leading biotechnology company with a strong track record of developing innovative medicines for patients with rare and serious disorders, today announced that the U.S. when directly comparing ANDEMBRY to placebo.

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The Pharma Data

JULY 15, 2021

Amgen Inflammation Amgen brings therapies to millions of people with inflammatory diseases, with a focus on serving unmet patient needs. In doing so, we have introduced and evolved novel therapies that have changed the lives of patients. It’s a commitment that extends beyond introducing novel therapies. Source link:

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International Disease Treatment Science 52

The Pharma Data

JULY 23, 2021

(Nasdaq: BIIB) today announced it will share multiple oral and poster presentations from its Alzheimer’s disease clinical development portfolio at the Alzheimer’s Association International Conference (AAIC), which will be held in Denver, Colorado and online July 26-30, 2021.

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International Disease Clinical Trials Trials 52

The Pharma Data

JUNE 16, 2025

BioMarin Boosts Enzyme Therapy Business with Inozyme Acquisition BioMarin Pharmaceutical and Inozyme Pharma today announced that BioMarin has entered into a definitive agreement to acquire Inozyme for $4.00 per share in an all-cash transaction valued at approximately $270 million in total consideration. This is where INZ-701 comes in.

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The Pharma Data

JUNE 11, 2025

Notably, BRUKINSA has emerged as the market leader in new CLL patient starts across all lines of therapy in the U.S. According to internal data, BRUKINSA has surpassed its competitors to become the BTK inhibitor with the largest overall market share in the country. for the first time. With its growing presence in the U.S.

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The Pharma Data

AUGUST 30, 2021

.–(BUSINESS WIRE)– Showcasing Teva’s Commitment to Helping Patients Have More Migraine-Free Days, 18 Abstracts will be presented, Including One Late-Breaker, on AJOVY ® (fremanezumab-vfrm) Injection at the International Headache Society and European Headache Federation Joint Congress 2021. Teva Pharmaceuticals USA, Inc.,

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International Clinical Trials Treatment Trials 52

The Pharma Data

JUNE 6, 2025

Designed to serve a diverse international audience—including patients, healthcare providers, business partners, and investors—the site embodies the company’s twin commitments to scientific rigor and human empathy. “In In September, we celebrate the 50th anniversary of the company I founded in 1975. JCR Pharmaceuticals Co.,

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Pharmaceuticals Science Disease International 40

The Pharma Data

MAY 6, 2021

INDIANAPOLIS , May 6, 2021 /PRNewswire/ — With a goal of integrating data and technology to help streamline diabetes management, Eli Lilly and Company (NYSE: LLY) signed strategic international agreements with four companies – DexCom, Inc., 1 International Diabetes Federation. Glooko Inc. IDF Diabetes Atlas, 9 th edn.

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