Drug Target Review

OCTOBER 11, 2023

Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. Most of these conditions are genetic in origin and the majority have no effective treatment.

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Drug Target Review

OCTOBER 30, 2024

Imagine being able to create an in vitro replica of a diseased organ to study the molecular mechanism underlying the illness. Now take a step further: envision testing drugs in these organoids to identify the ones that can treat disease safely and effectively without needing to run expensive clinical trials first.

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Treatment Disease Clinical Trials Research 105

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. Monitoring biomarkers can help assess changes in a disease, its level of expression, or the extent of its progression.

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Drug Target Review

JUNE 9, 2023

This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.

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FDA Law Blog: Drug Discovery

JULY 17, 2024

Importantly, the Hub is intended to establish a new model within FDA, which leverages cross-Agency expertise in providing guidance and conducting reviews for products for rare disease populations. Early Direction for the Rare Disease Innovation Hub The Hub, which will be co-chaired by Drs. Valentine & Frank J.

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On Medicine

FEBRUARY 28, 2024

What is Perthes’ disease? Perthes’ Disease is a rare condition that affects the developing femoral head (the “ball” of the “ball and socket” hip joint) of a child due to poor blood supply. This in turn can cause significant pain for children with Perthes’ disease and limit their activity levels significantly.

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Conversations in Drug Development Trends

NOVEMBER 5, 2024

In preparation for World Orphan Drug Congress Europe, we interviewed Nathan Chadwick, Senior Director, Therapeutic Strategy Lead, Rare Disease, and Derek Ansel, MS, LCGC, Vice President, Therapeutic Strategy Lead, Rare Disease, to hear their insights into the current progress in rare disease research and their hopes for 2025.

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Drug Patent Watch

DECEMBER 9, 2024

High-Throughput Screening: Modern Technology Meets Natural Products Advanced technologies now allow researchers to rapidly test thousands of natural compounds against specific disease targets. The development of Taxol involved multiple patents, including those for the isolation method, synthetic production, and various formulations.

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DrugBank

MARCH 12, 2025

However, mRNA technology is not limited to infectious diseases. This unique mechanism has enabled scientists to rethink how they tackle diseases, paving the way for advancements in oncology, genetic disorders, and regenerative medicine.  Beyond vaccines, mRNA is being utilized to enhance the efficacy of existing cancer treatments.

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Cytel

MAY 28, 2024

Written by Susannah Sadler and Stacy Grieve Launching a treatment for inflammatory diseases can be a make-or-break endeavor. A robust evidence base and a compelling value story are crucial to success, but current challenges and unexpected bumps in the road will require creative solutions.

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Conversations in Drug Development Trends

NOVEMBER 21, 2023

Since the advent of modern medicine, the scientific community has developed more than 3,000 drugs for thousands of diseases. Despite significant progress, there are more than 6,500 rare diseases without a single approved treatment. There are so many patients suffering from diseases that have no treatments.

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PPD

MARCH 11, 2025

While individual rare diseases affect populations that are small in numbers, collectively they impact millions globally, posing significant health and research challenges. Real-world data (RWD) and real-world evidence (RWE) have emerged as transformational tools in addressing these hurdles, advancing rare disease drug development.

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Drug Target Review

AUGUST 31, 2023

In the pursuit of a remedy for Alzheimer’s disease, a frontier in medical science is illuminating a glimmer of hope. Chronic inflammation involving microglia has been linked to the disease, as the release of inflammatory molecules triggers an increase in β-amyloid production.

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Chemical Biology and Drug Design

FEBRUARY 25, 2025

A combination of network pharmacology and invitro validation was used to reveal the potential targets and molecular mechanisms of resveratrol for CRS treatment. ABSTRACT Resveratrol (RES) is a polyphenolic antioxidant derived from different plant products, which has anti-inflammatory and antioxidative stress effect.

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Drug Target Review

JULY 12, 2023

Molecular-level biochemical assays like transcriptomics, genomics and proteomics have emerged as valuable tools for identifying potential targets in cancer treatment through deep cyclic inhibition (DCI). Importantly, transcriptomics information shows that certain aspects of a disease change the gene expression profile.

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ASPET

JUNE 4, 2024

The 8 -THC treatment (30 mg/kg) reduced paw swelling and qualitative signs of arthritis. Significance Statement Despite increasing use of cannabis products, the potential effects of minor cannabinoids are largely unknown. These data support the development of novel cannabinoid treatments for inflammatory arthritis.

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Conversations in Drug Development Trends

NOVEMBER 12, 2024

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. There are also nuances for operational planning in cell therapy development programs.

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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

DrugBank

JULY 20, 2023

of the global population) suffer from rare conditions without effective treatments or cures. Rare diseases, therefore, present compelling opportunities for Drug Development. Since each disease is present in small groups of patients, this research is also fraught with complex challenges. At any given time, millions of people ( 3.5–5.9%

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ASPET

SEPTEMBER 6, 2023

However, aberrant TLR7/8 activation by endogenous ligands has been implicated in the pathogenesis of autoimmune diseases including systemic lupus erythematosus. TLR activation and Type-I interferon (IFN) were shown recently to impart resistance to glucocorticoids (GC), which are part of the standard of care for multiple autoimmune diseases.

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FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

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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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Cytel

MAY 7, 2024

Here we explore the evolution and impact of market exclusivity policies in the EU and US, highlighting their role in fostering innovation and accessibility in rare disease treatment. This was a turning point, igniting a wave of research and development into treatments for rare diseases that had previously been neglected.

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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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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 Patent Watch

DECEMBER 12, 2024

Identifying branded drugs with a low likelihood of generic entry has become a crucial strategy for companies looking to expand their product portfolio through in-licensing. Drugs requiring specialized equipment, complex formulations, or intricate production methods are less likely to face immediate generic competition.

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FDA Law Blog: Biosimilars

APRIL 13, 2025

LYTGOBI was granted accelerated approval for the treatment of adult patients with previously treated, unresectable, locally advanced, or metastatic intrahepatic cholangiocarcinoma (iCCA) with FGFR2 gene fusions or rearrangements. DCR is defined as the sum of complete response (CR), partial response (PR) and stable disease (SD).

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Drugs FDA Trials Treatment 52

Chemical Biology and Drug Design

JANUARY 15, 2024

Abstract This study was designed to explore the protective effect and mechanism of naringin (NG) on radiation-induced heart disease (RIHD) in rats. The results showed that NG treatment significantly attenuated the 20 Gy XR-induced decline of LVEF and LVFS and the elevation of LVIDs.

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Drug Target Review

MAY 18, 2023

Foetal haemoglobin (HbF) is the predominant type of haemoglobin (Hb) in foetal life and remains so until just before birth when its production is turned down while the production of adult Hb is turned up. In sickle cell disease, however, this function is impaired, leading to a range of potential health consequences.

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Drug Target Review

APRIL 14, 2025

With many healthcare systems prioritising cost-effectiveness, there is limited demand for novel treatments, even though they are crucial to combatting antibiotic resistance. Traditional biologics manufacturing can be complex and expensive, making it difficult to offer affordable therapies for widespread diseases.

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Drug Patent Watch

FEBRUARY 26, 2025

The Complex World of Biologic Drugs: Navigating Patent Applications As a biotech professional, you're likely no stranger to the intricacies of developing life-changing treatments. They're used to treat a wide range of diseases, from cancer to autoimmune disorders. It's a complex world, to say the least.

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ProRelix Research

JULY 6, 2023

Biosimilar clinical trials and study designs’ Considerations The rising prevalence and large market share of biological products emphasize their importance as treatment options for several cancers and autoimmune diseases.

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Clinical Trials Biosimilars Trials Treatment 105

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. With cellular rejuvenation, our goal is to improve healthspan so as to maximise the number of healthy and productive years one has in their lifetime, not just their total number of years.

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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

ASPET

SEPTEMBER 12, 2023

In PAR2-null mice, disease severity of EAE is markedly diminished. We therefore tested whether inhibiting PAR2 activation in vivo might be a viable strategy for the treatment of MS. P2pal-18S treatment markedly diminishes disease severity and reduces demyelination, as well as the infiltration of T-cells and macrophages into the CNS.

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Disease Treatment Regulations Production 100

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

KIF1A

OCTOBER 7, 2023

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. Providing treatments for our community means understanding the different ways KAND can manifest, and learning from other disease groups with overlapping symptoms.

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