Drug Target Review

MARCH 17, 2025

Neuropsychiatric treatment is on the verge of a major transformation. Historically, treatment options have been limited, with patients relying on daily medications that have minimal efficacy and troublesome side effects. This has sparked the development of a new approach to treating neuropsychiatric disorders.

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Treatment Licensing Licensing Science 52

Drug Target Review

NOVEMBER 25, 2024

As the CEO of iOnctura, an innovative oncology biopharmaceutical company she co-founded in 2017, Catherine has played a key role in advancing the development of highly targeted small molecules aimed at revolutionising cancer treatment. This is exciting because often resistance builds up to these other treatments.

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Treatment Small Molecule Clinical Trials Trials 52

Drug Patent Watch

DECEMBER 9, 2024

In the vast realm of pharmaceutical research and development, there’s a fascinating intersection between ancient wisdom and modern science. “Pharmacognosy is the bridge between traditional medicine and modern pharmaceutical science, offering a treasure trove of potential new drugs waiting to be discovered.”

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Drugs Drug Development Pharmaceuticals Production 97

Drug Target Review

MARCH 12, 2024

Scientists from the Skaggs School of Pharmacy and Pharmaceutical Sciences at the University of California San Diego have discovered thousands of bile acids. The study’s findings offer new insights into the biochemical language microbes use to influence distant organ systems, which could revolutionise the way researchers approach disease.

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Disease Biochemical Pharmacology FDA Approval Pharmacy 111

Broad Institute

NOVEMBER 7, 2023

While RA therapies targeted to specific inflammatory pathways have emerged, only some patients’ symptoms improve with treatment, emphasizing the need for multiple treatment approaches tailored to different disease subtypes.

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Treatment RNA Hospitals Disease 96

Drug Patent Watch

DECEMBER 12, 2024

This approach not only helps maintain market exclusivity but also ensures a steady revenue stream for pharmaceutical companies. Understanding the Pharmaceutical Market Dynamics The pharmaceutical industry is a complex ecosystem where branded drugs and generics coexist, each playing a vital role in patient care and market dynamics.

Licensing 96

Licensing Licensing Drugs Biosimilars 96

New Drug Approvals

APRIL 11, 2025

1] [2] Adverse effects The US Food and Drug Administration prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal). [2] Fitusiran 1711.0g/mol, 1] It is an antithrombin-directed small interfering ribonucleic acid. [1]

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Clinical Trials FDA FDA Approval Trials 107

SCIENMAG: Medicine & Health

OCTOBER 31, 2023

— A new drug developed by professors from the School of Pharmacy and Pharmaceutical Sciences at Binghamton University has received Food and Drug Administration (FDA) approval for the treatment of patients with Duchenne muscular dystrophy (DMD), a common genetic disease that mostly affects young boys. BINGHAMTON, N.Y.

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Drug Target Review

MARCH 4, 2025

Obesity treatment is undergoing a major shift, much like the advances seen in cancer care. Phenomix Sciences , led by CEO Mark Bagnall, is using precision medicine to make treatments more targeted and effective. Phenomix Sciences, built on over a decade of clinical research at the Mayo Clinic, is disrupting this outdated approach.

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Therapies Treatment Clinical Trials Science 52

Conversations in Drug Development Trends

MAY 14, 2024

By: Amy Raymond, Derek Ansel, Nathan Chadwick, & Juliane Mills When choosing a CRO for a rare disease study, what truly sets them apart is their methodology: the CRO’s mindset, their approach to each unique study, and their agility in navigating the inherent complexities of rare disease research. Common goals empower change.

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Disease Clinical Trials Trials Pharmaceutical Companies 81

SCIENMAG: Medicine & Health

FEBRUARY 26, 2024

However, the underlying mechanisms of DCM are poorly understood, and treatment options are limited. DCM is the leading cause of heart failure in patients with chronic diabetes. Another mystery is the regulation of cytochrome P450 enzymes (CYPs) in the central nervous system.

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Disease Pharmaceuticals Regulations Treatment 75

Drug Target Review

AUGUST 6, 2024

A number of areas represent significant opportunities for Antibody Drug Conjugates (ADCs) beyond oncology, leveraging their ability to deliver therapeutic agents specifically to diseased cells or tissues while minimising off-target effects. Oncology also offers high returns and the opportunity to address severe, life-threatening conditions.

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Therapies Pharmaceutical Companies Disease Treatment 119

The Premier Consulting Blog

NOVEMBER 6, 2024

Inhaled combination products (ICP) have emerged as a significant advancement in the treatment of respiratory diseases such as asthma, chronic obstructive pulmonary disease (COPD), and other pulmonary conditions. The goal of ICPs is to improve the quality of life for patients suffering from chronic respiratory diseases.

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Production FDA Compliance Disease 52

LifeSciVC

JULY 17, 2024

By Mike Cloonan, Chief Executive Officer of Sionna Therapeutics, as part of the From The Trenches feature of LifeSciVC The drug development process in rare diseases is rife with challenges especially when companies target significant differentiation or first-in-class targets.

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Clinical Research Disease Research Clinical Trials 74

Broad Institute

DECEMBER 18, 2023

Brain disorders are difficult to study and many drug candidates have failed in clinical trials, causing pharmaceutical companies to reduce their investments or even exit the field entirely. But a new path for bringing treatments to patients is starting to emerge. The following conversation was edited for length and clarity.

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Drug Development Clinical Trials Drugs Disease 130

Drug Target Review

SEPTEMBER 9, 2024

functions to reset the immune system and potentially provide remission for allergic diseases? In allergic diseases, APCs identify and present unknown antigens to the immune system. This can lead to inadequate long-term effectiveness with low rates of disease remission. They also require frequent administration.

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Disease Immune Response Clinical Trials Treatment 64

Broad Institute

NOVEMBER 25, 2024

Ladders to Cures (L2C) Accelerator By Maria Nemchuk November 25, 2024 Breadcrumb Home Ladders to Cures (L2C) Accelerator The Ladders to Cures (L2C) Accelerator aims to catalyze progress across the research ecosystem and accelerates advances leading to treatments and cures for patients with rare genetic diseases.

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Clinical Trials Disease Trials Therapies 52

Cytel

APRIL 10, 2024

Orphan drug designation is a regulatory status granted to pharmaceuticals developed for the treatment of rare diseases. It provides incentives to encourage the research, development, and approval of therapies targeting small patient populations.

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Disease Drugs Therapies Pharmaceuticals 59

Conversations in Drug Development Trends

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.

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Treatment FDA Clinical Trials Therapies 75

Drug Target Review

JANUARY 8, 2024

It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.

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Science Disease FDA Approval Clinical Trials 145

National Institute on Drug Abuse: Nora's Blog

MARCH 6, 2025

Advancing reduction of drug use as an endpoint in addiction treatment trials astewart Thu, 03/06/2025 - 09:59 Nora's Blog March 18, 2025 Image Getty Images/ SolStock This blog was also published in the American Society of Addiction Medicine (ASAM) Weekly on March 18, 2025.&

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Treatment Trials Clinical Trials Drugs 69

Drug Channels

JUNE 14, 2022

The authors obscure the real story with mathematical sleight-of-hand that misrepresents the underlying data and overlooks the true nature of today’s pharmaceutical innovations. Their policy recommendations would therefore have a devastating impact on these patients and their hope for treatments and cures.

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Drugs Disease Pharmaceuticals Treatment 98

Drug Target Review

JUNE 5, 2024

How do you prioritise which therapeutic areas or diseases to focus on in your drug discovery efforts? percent) reduction in the treatment group. About the author James Graham, CEO of Recce Pharmaceuticals Recce Pharmaceuticals. James Graham is the CEO of Recce Pharmaceuticals. gonorrhoeae , with a 4-log (99.99

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Pharmaceuticals Hospitals International Trials 72

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options.

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Small Molecule Drugs Clinical Pharmacology Trials 145

DrugBank

DECEMBER 18, 2024

A new shift is occurring in the pharmaceutical industry, leading to a rapidly expanding field known as digital therapeutics (DTx). These interventions target a broad spectrum of medical conditions, encompassing chronic diseases and even substance abuse. Consider BlueStar's success in diabetes management.

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Pharmaceuticals Pharmaceutical Companies Treatment Therapies 52

Drug Target Review

JUNE 3, 2024

How does COUR Pharmaceuticals’ immune-modifying nanoparticle platform differ from traditional approaches to treating immune-mediated diseases? Essentially, our CNPs reprogramme the immune system by restoring balance and returning the body to a homeostatic state, offering a precise approach to treating immune-mediated diseases.

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Disease Therapies Pharmaceuticals Clinical Trials 49

Drug Target Review

SEPTEMBER 19, 2024

Based on Nobel Prize-winning research, IRLAB has grown rapidly to become recognised and respected as a world-leader in understanding the complex neuropharmacology of CNS disorders, especially Parkinson’s disease (PD). The pipeline currently includes five drug candidates, all of which have the potential to revolutionise the treatment of PD.

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Disease Drugs Treatment Drug Development 52

DrugBank

OCTOBER 17, 2024

By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.

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Drug Development Metabolic Stability Clinical Trials Drugs 98

PPD

JUNE 27, 2024

AA is considered a dermatology autoimmune disease, wherein the body fails to recognize its own cells and leads to subsequent immune-mediated destruction of the hair follicle. Accelerate your alopecia areata treatments. Learn more The post Advancing New Treatments for Alopecia Areata appeared first on PPD.

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Treatment Clinical Trials Clinical Research Small Molecule 52

Drug Target Review

JANUARY 12, 2024

How does the integration of Real-World Data (RWD) with genomic biomarker data contribute to a more comprehensive understanding of disease progression and treatment response? Biomarker information is also useful for tracking progression of the disease and treatment response. Most drugs do not work in all people.

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Doctors Clinical Trials Treatment Therapies 111

Elrig

MARCH 25, 2025

It will also cover recent advancements in broadening their application, including innovative approaches to ensure their safety and efficacy, the use of targeted delivery to reach disease-relevant tissues as well as success stories in bringing therapeutic oligonucleotides to the clinic.

RNA 59

RNA Bioinformatics Science Pharmacokinetics 59

Conversations in Drug Development Trends

JULY 9, 2024

The recent surge in GLP-1 treatments has ignited a profound shift in the pharmaceutical landscape, with more studies being announced than ever before. However, the treatment journey does not end with weight reduction, as maintaining weight loss overall is equally crucial.

Clinical Research 52

Clinical Research Treatment Research Clinical Trials 52

Chemical Biology and Drug Design

SEPTEMBER 26, 2024

Compound 22 might become a novel antioxidant for the treatment of oxidative stress-related diseases. ABSTRACT Paeoniflorin (PF) is one of the active constituents of the traditional Chinese medicine Paeoniae Radix Rubra and has been actively explored in the pharmaceutical area due to its numerous pharmacological effects.

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Protein Expression Pharmaceuticals Treatment Disease 100

Drug Target Review

NOVEMBER 27, 2023

In cancer, the eIF4F complex becomes dysregulated, leading to the excessive production of these proteins, which contribute to tumour formation and resistance to treatment. STRIs represent a fundamentally different approach to cancer therapy compared to traditional treatments like chemotherapy, radiation therapy, and targeted therapies.

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Molecular Biology Therapies Pharmaceuticals Treatment 86

Drug Target Review

OCTOBER 24, 2023

Live Bacterial Products (LBPs) will provide patients with a desirable, credible, safe and effective treatment option. The human microbiome, a complex ecosystem of microorganisms residing within our bodies, has emerged as a promising frontier in the quest for innovative medical treatments.

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Treatment Pharmaceuticals Pharmacokinetics Trials 122

DrugBank

AUGUST 20, 2024

The world of drug discovery is in a state of transformation, with advancements in biotechnology opening doors to new possibilities for targeting previously "undruggable" disease mechanisms. We also explore why some disease-associated targets remain elusive and how recent innovations are changing that narrative.

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Therapies Pharmaceuticals Disease Research 98

Conversations in Drug Development Trends

MAY 14, 2024

By: Amy Raymond, Derek Ansel, Nathan Chadwick, & Juliane Mills When choosing a CRO for a rare disease study, what truly sets them apart is their methodology: the CRO’s mindset, their approach to each unique study, and their agility in navigating the inherent complexities of rare disease research. Common goals empower change.

Disease 52

Disease Clinical Trials Trials Pharmaceutical Companies 52