Science Daily: Pharmacology News

AUGUST 30, 2023

Scientists have made a `paradigm shifting' discovery on the mechanisms required for learning and memory that could lead to new therapies for Alzheimer's disease and potentially Down syndrome.

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Science Daily: Pharmacology News

FEBRUARY 14, 2025

One day, researchers want to use switches of this kind to trigger cell therapies for various metabolic diseases. Researchers have developed a new gene switch that can be activated using a commercially available nitroglycerine patch applied to the skin.

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Therapies Disease Research 253

Drug Target Review

JUNE 9, 2025

The landscape of genetic medicine is undergoing a profound transformation, driven by innovative approaches that challenge the traditional, disease-specific paradigms. My transition to biotech and rare diseases was deeply personal – my son was diagnosed with Duchenne muscular dystrophy in 2020. “My

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Science Daily: Pharmacology News

APRIL 9, 2025

By inserting an entire gene, the tool offers a one-and-done approach that overcomes hurdles from CRISPR gene editing technology -- which is programmed to correct individual mutations -- offering a promising step forward for gene therapy.

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RNA DNA Disease Therapies 345

Science Daily: Pharmacology News

NOVEMBER 13, 2023

This discovery opens up new opportunities for therapeutic intervention to control cholesterol uptake that could complement other therapies and potentially save lives. Researchers have discovered the mechanism by which cholesterol in our diet is absorbed into our cells.

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Science Daily: Pharmacology News

AUGUST 30, 2023

A clinical trial has tested a potentially curative stem cell gene therapy for sickle cell disease. The results were promising.

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Therapies Clinical Trials Trials Disease 213

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.

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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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Science Daily: Pharmacology News

MARCH 13, 2025

Such neurons could be used to treat spinal cord injuries or diseases such as ALS. Researchers devised a process to convert a skin cell directly into a neuron, eliminating the need to generate induced pluripotent stem cells.

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Engineering Therapies Disease Research 257

Science Daily: Pharmacology News

JANUARY 3, 2025

The research could revolutionize therapies for complex conditions like autoimmune disease and cancer. Bioengineers have developed a new construction kit for building custom sense-and-respond circuits in human cells.

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Science Daily: Pharmacology News

NOVEMBER 28, 2023

Heart disease kills 18 million people each year, but the development of new therapies faces a bottleneck: no physiological model of the entire human heart exists -- so far.

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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. As Smith puts it, “now the viewpoint is that computational science is very powerful, and these AI/ML methods are going to be transformative in a lot of ways.”

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BioPharma Drive: Drug Pricing

JUNE 3, 2024

With new modalities such as gene and cell therapies, RNA therapeutics, complex biologics and more, today’s science brings unprecedented opportunities to address diseases that have long remained out of reach.

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Science RNA Therapies Disease 141

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. Stem cell therapies have already demonstrated their prowess in treating diverse cancers and ailments linked to the blood and immune system.

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Therapies Disease Research Treatment 105

The Pharma Data

JUNE 25, 2025

Biogen Reports Promising Interim Phase 1 Results for Salanersen in Spinal Muscular Atrophy, Prepares for Registrational Trials Biogen has announced encouraging topline results from its Phase 1 clinical trial evaluating salanersen (BIIB115/ION306) , an investigational antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA).

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Science Daily: Pharmacology News

JULY 4, 2024

The new T cell atlas is publicly available and should help in the development of new drug therapies for immune-mediated diseases. The discoveries were made possible by a newly developed technology they call ReapTEC.

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Broad Institute

MAY 16, 2024

Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. Because the vehicle binds to a well-studied protein in the blood-brain barrier, the scientists say it has a good chance at working in patients.

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Science Daily: Pharmacology News

FEBRUARY 14, 2024

A groundbreaking scientific study has unveiled a remarkable discovery that may have far-reaching implications for the treatment of heart disease. The implications are immense offering glimpses of a future where heart disease may no longer be an irreversible condition but a challenge that can be overcome through medical intervention.

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Drug Target Review

MARCH 4, 2025

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. Obesity treatment is undergoing a major shift, much like the advances seen in cancer care.

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Therapies Treatment Clinical Trials Science 52

Drug Target Review

JULY 11, 2025

Neurological diseases are among the most complex and least understood conditions in medicine. Despite the knowledge that certain genes increase the risk of contracting diseases like Alzheimer’s and Parkinson’s, it is still not fully understood how those diseases develop in the brain. But that is beginning to change.

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Molecular Biology Disease Therapies Bioinformatics 69

Science Daily: Pharmacology News

APRIL 9, 2025

Researchers have discovered a way to get anti-inflammatory medicine across the blood-brain barrier, opening the door to potential new therapies for a range of conditions, including Alzheimer's disease, multiple sclerosis, Parkinson's disease and cancer cachexia.

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Treatment Therapies Disease Drugs 253

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.

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Science Daily: Pharmacology News

JUNE 10, 2024

The advance could one day help researchers develop a single gene therapy for diseases such as cystic fibrosis that are caused by one of hundreds or thousands of different mutations in a gene.

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Therapies Disease Research 291

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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The Pharma Data

JUNE 24, 2025

This alliance aims to address mounting challenges in the development and commercialization of mAb therapies and gene therapies, particularly those involving AAV vectors. Biologic drugs, including monoclonal antibodies, have become essential in the treatment of a wide array of conditions, from autoimmune diseases to various cancers.

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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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Drug Target Review

OCTOBER 16, 2023

How can PDOs revolutionise drug discovery and deepen our understanding of disease? Patient-derived organoids (PDOs) are proliferative 3D cell structures derived from tissue samples of both healthy and diseased tissue. Improved translatability to the patient and the disease state.

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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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Disease Treatment Cell Biology Research 52

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. By Sarah Wicks & James E.

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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.

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RNA Engineering DNA Disease 72

Broad Institute

SEPTEMBER 6, 2023

Diabetes and cardiac fibrosis are two conditions in dire need of new therapies,” said Todd Golub, director of the Broad Institute. For both patient populations, there are no safe and effective therapies for reversing disease. The collaboration will focus on advancing three programs over the next three years.

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Disease Research Therapies Treatment 136

Drug Target Review

JULY 8, 2025

Her team sits right at the edge of innovation, where discovery science meets clinical execution. Not only are they chasing new targets, they are also working to understand the biology behind resistance, discover biomarkers that matter and build smarter therapies from the ground up.

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Covalent Modifiers

SEPTEMBER 7, 2023

Science 2023 , 380, 1349-1356 DOI: 10.1126/science.adh0614 Millions who live in Latin America and sub-Saharan Africa are at risk of trypanosomatid infections, which cause Chagas disease and human African trypanosomiasis (HAT). Srinivasa P.

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Drug Target Review

NOVEMBER 8, 2023

Problem w/ CTs and foundational understanding of Vittoria: can you explore the current limitations of cell therapies and the challenges faced by patients and providers? Currently, only a small percentage of cancers can be effectively treated with cell therapies, and there is little diversity in the currently approved products.

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Science Daily: Pharmacology News

MAY 15, 2025

A new gene editor may soon open the door to gene therapies for a wider array of diseases.

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Therapies Disease 214

Drug Target Review

NOVEMBER 17, 2023

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. NK cells can be readily extracted from umbilical cord blood or peripheral blood of adult donors.

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Therapies Clinical Trials Cell Biology Trials 118

Fierce BioTech

SEPTEMBER 5, 2024

How can life sciences organizations effectively identify and recruit participants with specific genetic variants? How can life sciences organizations effectively identify and recruit participants with specific genetic variants? How can they engage providers and raise awareness of gene therapies for the patients who need them most?

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