Science Daily: Pharmacology News

FEBRUARY 17, 2025

The findings demonstrate the potential of this technology to pave the way for future treatments for a wide range of conditions such as Alzheimer's disease, amyotrophic lateral sclerosis, brain cancer, and drug addiction.

RNA 242

RNA Treatment Disease Drugs 242

BioPharma Drive: Drug Pricing

JUNE 17, 2025

The deal centers around a medicine that could “shift the treatment paradigm for cardiovascular disease,” Lilly said, but also follows a yearslong in decline in Verve’s share price amid skepticism over the need for such a therapy.

Disease 188

Disease Therapies Treatment 188

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.

Clinical Development 69

Clinical Development Production Clinical Trials Treatment 69

Drugs.com

JUNE 18, 2025

The deal gives Lilly a potential new treatment for heart disease, The Wall Street Journal reported. WEDNESDAY, June 18, 2025 — Drugmaker Eli Lilly plans to buy Verve Therapeutics, a gene-editing startup, for about $1 billion upfront.

Therapies 173

Therapies Treatment Disease Drugs 173

Broad Institute

MAY 21, 2025

Scientists create next generation of tools in battle against brain disease By Corie Lok May 21, 2025 Breadcrumb Home Scientists create next generation of tools in battle against brain disease The findings contained in eight studies could lead to targeted gene therapies for brain disorders. The tools are known as enhancer AAV vectors.

Disease 72

Disease Medical Schools DNA Therapies 72

Drug Target Review

DECEMBER 20, 2024

Their work focuses on creating ‘digital twin generators’ – AI-driven models that predict how a patients disease may progress over time. AI-powered digital twins A key component of Unlearns work is its focus on digital twin technology, which uses AI to create personalised models of disease progression for individual patients.

Clinical Trials 128

Clinical Trials Trials Pharmaceuticals Drug Development 128

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.

Treatment 105

Treatment Disease Clinical Trials Research 105

Drug Target Review

JULY 1, 2025

Placebo arms, in particular, create ethical and logistical hurdles, especially in areas like rare disease and oncology. They are patient-specific outcome predictions, generated using disease-specific machine-learning models trained on large, longitudinal clinical datasets. Digital twins offer a way forward.

Clinical Trials 99

Clinical Trials Trials FDA Disease 99

Drug Target Review

JUNE 16, 2025

Targeting GPCRs to fight inflammatory diseases GPCR-targeting drugs are well known therapies for a range of disease types, including cardio-metabolic, central nervous system and endocrinological disorders. It also holds potential for neuroinflammatory conditions such as certain types of migraine.

Disease 59

Disease Therapies Small Molecule Immune Response 59

Covalent Modifiers

MARCH 1, 2025

4c02665 Pharmacological inhibition of the cGAS-STING-controlled innate immune pathway is an emerging therapeutic strategy for a myriad of inflammatory diseases. Moreover, orally administered GHN105 exhibited on-target engagement in vivo and markedly reversed key pathological features in a delayed treatment of the acute colitis mouse model.

Disease 130

Disease Treatment 130

Chemical Biology and Drug Design

MARCH 6, 2025

ABSTRACT Parkinson's disease (PD) is the second most common neurodegenerative disease but has limited medications. Targeting leucine-rich repeat kinase 2 (LRRK2) has been identified as a potential strategy for the treatment of PD.

Treatment 100

Treatment Disease Drugs Research 100

Broad Institute

NOVEMBER 4, 2024

By Lucy Mutz November 4, 2024 Credit: Juliana Sohn Kasper Lage is managing director of the Novo Nordisk Foundation Center for Genomic Mechanisms of Disease. The overall goal is to translate genetic findings into biological mechanisms underlying disease that could be targeted with new medicines.

Disease 72

Disease Research Science International 72

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

Broad Institute

JUNE 11, 2025

First launched in 2015 and renewed in 2020, the program began as a recognition that while a majority of cardiovascular disease can be associated with lifestyle factors such as tobacco consumption, diet, and level of physical activity, genomics can influence an individual’s predisposition to cardiovascular disease, age of onset, and severity.

Disease 93

Disease Therapies Treatment Research 93

Drug Target Review

JULY 21, 2025

Disorders such as Alzheimer’s disease , primary glioblastoma and amyotrophic lateral sclerosis (ALS) all affect the CNS and are considered fatal, while more common conditions, including depression , strokes and epilepsy , require long-term treatments. Hundreds of life-altering conditions attack the central nervous system (CNS).

Drug Development 64

Drug Development Small Molecule Drugs Therapies 64

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.

Clinical Development 69

Clinical Development Clinical Trials Clinical Pharmacology Trials 69

Chemical Biology and Drug Design

APRIL 4, 2025

Currently, the FDA has granted approval for the use of the small molecule inhibitor Ivosidenib (AG-120) in the treatment of IDH1-mutated AML and cholangiocarcinoma. Although AG-120 has benefited patients clinically, drug resistance has gradually emerged and has become a major problem in the treatment of mutant IDH1 (mIDH1) diseases.

Small Molecule 100

Small Molecule Drugs DNA Treatment 100

The Pharma Data

JUNE 12, 2025

The results come from a open-label, proof-of-concept, Phase 2 study (NCT04520451) and highlight rilzabrutinib’s potential as a disease-changing treatment option for a condition that currently has limited and non-specific treatment options and involves substantial patient suffering due to its chronic and progressive course.

Disease 40

Disease Clinical Development Treatment Medical Schools 40

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.

Disease 52

Disease Treatment Cell Biology Therapies 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.

Treatment 52

Treatment Small Molecule Clinical Trials Trials 52

Nvidia Developer: Drug Discovery

OCTOBER 30, 2024

The integration of AI in drug discovery is revolutionizing the way researchers approach the development of new treatments for various diseases. The integration of AI in drug discovery is revolutionizing the way researchers approach the development of new treatments for various diseases. Traditional.

Drugs 95

Drugs Treatment Disease Marketing 95

Broad Institute

MARCH 11, 2025

The tRCC Project aims to address these challenges, by partnering directly with patients, caregivers, and advocates to deepen understanding of this rare disease and generate new insights that could lead to new treatments. Tags: Count Me In Cancer Rare Disease

Research 113

Research Medical Schools Treatment Disease 113

New Drug Approvals

JUNE 24, 2025

Safety: Diarrhea was the most frequent adverse event, but serious adverse events were balanced across treatment groups. Progressive Fibrosing ILDs: Nerandomilast is also being investigated in other progressive fibrosing interstitial lung diseases (ILDs) beyond IPF. SCHEME 1H NMR (400 MHz, DMSO-D6)  1.57–1.84 Hz, 2H), 2.10–2.21

Clinical Trials 62

Clinical Trials Treatment Disease Trials 62

Drug Target Review

JULY 3, 2025

Given macro healthcare influences (eg, economic uncertainty, environmental changes) and the numerous available treatments for major diseases, drug developers may need to reassess their therapeutic strategies. Preclinical advantage. Lifecycle planning and repurposing.

Drug Development 64

Drug Development Drugs Animal Testing Engineering 64

The Pharma Data

JUNE 25, 2025

Designed using the same fundamental mechanism as Biogen’s approved SMA treatment SPINRAZA (nusinersen), salanersen represents a next-generation approach aimed at greater potency and less frequent dosing—potentially requiring only once-yearly administration.

Therapies 52

Therapies Trials RNA Treatment 52

Broad Institute

OCTOBER 30, 2024

Researchers have typically analyzed these cells as a mixed-together group, but this approach can miss rare cell types, and makes it difficult to draw conclusions about how cells interact to drive the disease. Eventually, we hope that these methods can help inform the development of drugs or even guide patients’ individual treatments.”

Research 127

Research RNA Medical Schools Treatment 127

The Pharma Data

JUNE 23, 2025

These findings represent a pivotal advancement in the treatment of this rare, progressive, and life-threatening disease. Participants were classified as WHO Group 1 PAH, functional class (FC) II or III, indicating moderate to severe disease at either intermediate or high risk of clinical deterioration.

Trials 40

Trials Therapies Treatment Disease 40

The Pharma Data

JUNE 25, 2025

FDA Approves Label Update for Lilly’s Amyvid, Expanding Its Role in Alzheimer’s Disease Diagnosis and Therapy Guidance In a major development for Alzheimer’s diagnostics, Eli Lilly and Company (NYSE: LLY) announced that the U.S.

FDA 52

FDA FDA Approval Therapies Disease 52

Drug Target Review

DECEMBER 6, 2024

In cases where control or treatment groups show large variability, this could reflect true biological responses to a treatment, and removing such data could reduce the accuracy of the analysis. It is crucial during this phase to strike a balance between removing erroneous data and preserving meaningful biological variability.

RNA 52

RNA Drugs Research Science 52

The Pharma Data

JUNE 6, 2025

These new findings reinforce the company’s growing leadership in rare hematologic diseases, with a focus on transformative treatments for immune thrombocytopenia (ITP) and hemophilia. Rilzabrutinib is currently under regulatory review in major markets including the United States, European Union, and China for the treatment of ITP.

Disease 40

Disease Therapies Treatment International 40

The Pharma Data

JUNE 25, 2025

Novartis Finalizes Acquisition of Regulus Therapeutics, Strengthening Its Renal Disease Portfolio with Promising ADPKD Therapy Novartis AG, a leading global pharmaceutical company, has officially completed its acquisition of Regulus Therapeutics Inc. , a biotechnology firm known for its expertise in microRNA-targeting therapies.

RNA 52

RNA Clinical Trials Disease Small Molecule 52

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.

Drug Development 52

Drug Development Clinical Trials Disease Trials 52

Broad Institute

MAY 19, 2025

With AI, researchers predict the location of virtually any protein within a human cell By Corie Lok May 19, 2025 Breadcrumb Home With AI, researchers predict the location of virtually any protein within a human cell Trained with a joint understanding of protein and cell behavior, the model could help with diagnosing disease and developing new drugs.

Research 105

Research Engineering Science Disease 105

Drug Target Review

JUNE 17, 2025

Understanding health and disease requires more than reading the genomic code. The result is a more holistic, actionable view of cellular and tissue dynamics that is essential for uncovering causal mechanisms in disease. A genome is only one layer in a highly interconnected system of molecular and cellular processes.

RNA 71

RNA Engineering DNA Disease 71

Chemical Biology and Drug Design

JANUARY 20, 2025

ABSTRACT Depression is a mental health disorder and is the fourth most prevalent disease. Following treatment, changes in behavior and the activation of the NLRP3/ASC/caspase-1 inflammasome pathway in the depression model rats were observed. Interestingly, the P2X7/NLRP3 pathway was predicted as a potential target for simvastatin.

Treatment 100

Treatment Disease Research 100

The Pharma Data

JUNE 11, 2025

Dapirolizumab Pegol Demonstrates Promise in Phase 3 SLE Trial, Showing Improvements in Fatigue and Disease Activity at EULAR 2025 UCB (Euronext Brussels: UCB) and Biogen Inc. The latest data from the PHOENYCS GO trial reinforce the potential of dapirolizumab pegol to improve disease outcomes in people with moderate-to-severe SLE.

Disease 40

Disease Treatment Trials Therapies 40

Drug Target Review

JULY 15, 2025

The latest technologies are empowering researchers with faster, more efficient and more accessible genomic tools, leading to richer data generation and accelerating progress in areas like cancer genomics, rare disease research and infectious disease surveillance.​ hours to 24 hours.

RNA 91

RNA DNA Laboratories Science 91