Science Daily: Pharmacology News

MARCH 19, 2025

An experimental drug appears to reduce the risk of Alzheimer's-related dementia in people destined to develop the disease in their 30s, 40s or 50s, according to the results of a new study.

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FDA Law Blog: Drug Discovery

JANUARY 30, 2025

However, as we note in that post, the design, timing of initiation, and timely conduct of confirmatory trials are also important considerations in FDAs determination of whether accelerated approval is appropriate. This blog post focuses on interpreting these new authorities with respect to timely conduct of confirmatory trials.

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FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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BioPharma Drive: Drug Pricing

OCTOBER 23, 2023

The trial, which is ongoing in the U.K. since late last year as the FDA sought more details on Verve’s in vivo treatment for heart disease. and New Zealand, has been on hold in the U.S.

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BioPharma Drive: Drug Pricing

JANUARY 19, 2024

Phase 3 trial results showed Lutathera cut the risk of disease progression or death by 72% as initial treatment for gastroenteropancreatic neuroendocrine tumors, or GEP-NETs.

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Science Daily: Pharmacology News

JULY 15, 2024

Importantly the research team isolated a molecule produced by gut bacteria that in the future could potentially be trialed as a simple treatment, in the form of a dietary supplement, for children at risk of asthma to prevent them developing the disease.

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Drugs.com

JUNE 18, 2024

TUESDAY, June 18, 2024 -- There's more good news in the battle against sickle cell disease, with another trial finding CRISPR gene-editing therapy delivering impressive results for patients.“It’s It’s encouraging that this gene-editing treatment con.

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BioPharma Drive: Drug Pricing

JUNE 3, 2024

Liquid biopsy has emerged as a revolutionary tool for clinical trials, offering cellular and cell-free methods that provide valuable insights into disease progression and treatment response.

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Clinical Trials Trials Treatment Disease 255

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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BioPharma Drive: Drug Pricing

OCTOBER 7, 2024

The study, of Intellia’s treatment for hereditary angioedema, is the second Phase 3 trial the biotech has begun testing its “in vivo” gene editing medicines.

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BioPharma Drive: Drug Pricing

AUGUST 28, 2023

The treatment, developed by a biotech Bayer acquired in 2019, appeared safe in a small trial and showed signs of an effect on disease progression.

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Therapies Trials Treatment Disease 246

Drug Discovery Today

FEBRUARY 8, 2021

A new approach to treating one of the leading causes of blindness among patients with diabetes is being tested in clinical trials which begin this month.

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Broad Institute

JUNE 27, 2024

Neurons were classified by software as either positive for prion protein expression (yellow) before treatment with CHARM (left panel), or negative (purple) after treatment (right panel). Therapy search Vallabh, along with her husband Eric Minikel, leads a lab at Broad focused on developing drugs to prevent and treat prion diseases.

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Science Daily: Pharmacology News

FEBRUARY 22, 2024

A large-scale clinical trial of treatment strategies for Crohn's disease has shown that offering early advanced therapy to all patients straight after diagnosis can drastically improve outcomes, including by reducing the number of people requiring urgent abdominal surgery for treatment of their disease by ten-fold.

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ProRelix Research

SEPTEMBER 7, 2023

Autoimmune disorders comprise several diseases in which the immune system mistakenly attacks and destroys the body’s own healthy cells instead of destroying bacteria and viruses to keep the body healthy. […] The post Autoimmune disorders and current developments of novel treatments under clinical trials appeared first on ProRelix Research.

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BioPharma Drive: Drug Pricing

OCTOBER 9, 2024

The Series B round will fund a Phase 1/2 trial of the company’s experimental treatment for IgA nephropathy.

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ProRelix Research

SEPTEMBER 17, 2023

The rising prevalence of ophthalmology disorders such as dry eye disease (DED) and vision-related issues is largely related to the increasing use of mobile phones, tablet screens, and laptops.

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Clinical Trials Trials Treatment Disease 98

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. “Zelquistinel is a positive modulator of NMDA receptors,” Donello explains.

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BioPharma Drive: Drug Pricing

OCTOBER 7, 2024

The development of cancer vaccines has provided some hope in the battle against cancer worldwide; however, there are still many challenges to overcome when developing these life-saving treatments.

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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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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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Quanticate

DECEMBER 16, 2024

A randomised controlled clinical trial is the most prevalent design used in Phase II and Phase III clinical trials, whereby participants are randomly assigned to a control or experimental group with a fixed probability, normally 50% for each treatment.

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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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SCIENMAG: Medicine & Health

SEPTEMBER 7, 2023

7, 2023 – Alzheimer’s disease is the most common cause of dementia that affects more than 6.5 To find effective treatments and slow the progression of this debilitating disease, researchers have made much progress in developing new drugs that target beta-amyloid plaques, one of […] WINSTON-SALEM, N.C.

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Drug Target Review

MARCH 3, 2025

The search for effective treatments for neurodegenerative diseases like Parkinson’s disease has long been hindered by the brain’s complexity and the absence of adequate models for drug discovery. “For diseases like Parkinson’s, it’s more than sufficient,” he explains.

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Science Daily: Pharmacology News

SEPTEMBER 15, 2023

Researchers have achieved a significant breakthrough in identifying the primary cause and potential treatment for preeclampsia, a severe pregnancy complication. Experts pinpointed a toxic protein named cis P-tau in the blood and placenta of individuals with preeclampsia.

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Drug Hunter

AUGUST 15, 2023

LRRK2 has been a hotly pursued drug target for Parkinson’s Disease based on human genetics. On Sunday, it was disclosed that additional, likely on-target adverse findings were observed in a primate GLP study on long-term treatment with Merck’s second-generation LRRK2 inhibitors. Get ahead now by requesting a trial.

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BioPharma Drive: Drug Pricing

APRIL 29, 2024

The company will soon start a Phase 1/2 study of its treatment for chronic granulomatous disease, a milestone for the search-and-replace gene editing technology.

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SCIENMAG: Medicine & Health

JULY 17, 2023

Osaka, Japan – Systemic autoimmune diseases are characterized by inflammation of multiple organs and can have devastating consequences for patients. There is a dire need for treatments against these diseases. RNase treatments seem promising in some clinical trials but not all. Credit: Ryota Naito et […]

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Conversations in Drug Development Trends

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.

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Broad Institute

APRIL 5, 2025

The organization has honored Liu for the development of the gene editing platforms base editing and prime editing, which can correct the vast majority of known disease-causing genetic variations and have already been used in at least 15 clinical trials, with life-saving results.

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BioPharma Drive: Drug Pricing

MAY 7, 2024

Weeks after a safety setback derailed its lead candidate, the company has treated the first participant in a trial of its heart disease treatment Verve-102.

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Quanticate

SEPTEMBER 25, 2024

In rheumatoid arthritis (RA) clinical trials, accurately measuring the effectiveness of treatments is critical for determining their value in managing this chronic and debilitating condition.

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Clinical Trials Trials Therapies Treatment 97

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

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. Additionally, we are preparing to launch several other trials over the coming months.

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BioPharma Drive: Drug Pricing

SEPTEMBER 22, 2023

The drug, which is already cleared as a neoadjuvant lung cancer treatment, could further compete with Merck’s Keytruda if it gains approval in the adjuvant setting for patients with the non-small cell form of the disease.

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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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