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

Broad Institute

OCTOBER 30, 2024

Researchers map communities of single cells in metastatic breast cancers By Corie Lok October 30, 2024 Breadcrumb Home Researchers map communities of single cells in metastatic breast cancers The team compared different single-cell and spatial profiling methods to explore the diversity of cells in metastatic breast cancer biopsies.

Research 127

Research RNA Medical Schools Treatment 127

Science Daily: Pharmacology News

JULY 18, 2025

Researchers found that disabling this gene in colorectal cancer cells not only crippled their survival but also disrupted thousands of other genes tied to cancer and metabolism. The findings hint at vast possibilities in treating diseases influenced by vitamin D, though long-term impacts remain uncertain.

Therapies 216

Therapies Disease Research 216

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

Disease 80

Disease Treatment Engineering Protein Production 80

Science Daily: Pharmacology News

MAY 7, 2025

Psychologists shows one type of curiosity can increase well into old age, contradicting prior research. Older adults who maintain curiosity and want to learn new things relevant to their interests may be able to offset or even prevent Alzheimer's disease. Psychology literature has shown that curiosity tends to decline with age.

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Disease Research 287

BioPharma Drive: Drug Pricing

JUNE 2, 2025

Phase 3 results revealed Monday led shares to surge and should yield an approval application in IgA nephropathy, a competitive corner of drug research, later this year.

Disease 177

Disease Drugs Drug Research Research 177

Broad Institute

MARCH 11, 2025

By Count Me In Communications March 11, 2025 Count Me In , a patient-partnered research initiative led by the Broad Institute of MIT and Harvard and Dana-Farber Cancer Institute, is launching a new project focused on translocation renal cell carcinoma (tRCC), a rare and aggressive form of kidney cancer.

Research 113

Research Medical Schools Treatment Disease 113

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

Chemical Biology and Drug Design

NOVEMBER 4, 2024

Alzheimer's disease (AD) is a progressive age-related neurodegenerative brain disorder that destroys memory and other important mental functions. ABSTRACT Alzheimer's disease (AD) is a progressive chronic age-related neurodegenerative brain disorder characterized by the loss of memory and other cognitive functions.

Disease 100

Disease Therapies 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 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

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.

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Clinical Trials Trials Pharmaceuticals Drug Development 128

Drug Target Review

JULY 1, 2025

Placebo arms, in particular, create ethical and logistical hurdles, especially in areas like rare disease and oncology. Chief Executive Officer Steve Herne has spent more than 25 years in clinical research, with senior roles at WCG, Bioclinica and Covance. Digital twins offer a way forward.

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Clinical Trials Trials FDA Disease 100

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 106

Treatment Disease Clinical Trials Research 106

Conversations in Drug Development Trends

OCTOBER 29, 2024

By: Juliane Mills, Senior Director, Therapeutic Strategy Lead, Rare Disease The rise of patient-led clinical research, particularly in rare disease, represents a significant shift in the clinical trial landscape. Why Is There an Increase in Patient-Led Rare Disease Research?

Clinical Research 72

Clinical Research Disease Research Clinical Trials 72

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 7, 2025

Persistent challenges with NHP use Despite their biological relevance, using live NHPs in research poses several major challenges: Ethical concerns NHPs, due to their advanced cognition and social behaviour, are at the centre of ongoing ethical debates. Supply chain disruptions Availability is a critical bottleneck in NHP-based research.

Drug Research 52

Drug Research Research Animal Testing Drugs 52

Science Daily: Pharmacology News

JULY 4, 2025

Scientists have discovered that the bacteria behind Lyme disease and anaplasmosis have a sneaky way of surviving inside ticks—they hijack the tick’s own cell functions to steal cholesterol they need to grow. The research opens the door to new methods of stopping these diseases before ticks ever get the chance to bite.

Disease 214

Disease Research 214

Drug Target Review

JUNE 17, 2025

Understanding health and disease requires more than reading the genomic code. Combining genomics with transcriptomics, proteomics, metabolomics, spatial profiling and cellular imaging, will enable researchers to move beyond single-variable analysis and begin to see the biological system as a whole.

RNA 72

RNA Engineering DNA Disease 72

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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Clinical Development Production Clinical Trials Treatment 69

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.

Molecular Biology 69

Molecular Biology Disease Therapies Bioinformatics 69

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. Schwamborns research focuses on creating patient-specific brain organoids.

Disease 52

Disease Treatment Cell Biology Therapies 52

Drug Target Review

DECEMBER 6, 2024

In drug discovery and biological research, the scientists workflow often follows a structured and iterative approach to ensure accuracy, reproducibility and scientific integrity. At the heart of any research is the scientific question. This acts as a safeguard against false positives and misinterpretation of the data.

RNA 52

RNA Drugs Research Science 52

Drug Target Review

JULY 15, 2025

Research by Markets and Markets projects the market to grow from $12.13 1 Emergen Research attributes this to the advantages of high-throughput sequencing technologies and declining sequencing costs​. 2 This trend reflects the increasing demand for genomic sequencing in research, clinical diagnostics and other applications.

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RNA DNA Laboratories Science 92

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.

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Disease Clinical Development Treatment Medical Schools 40

Conversations in Drug Development Trends

OCTOBER 14, 2024

Autoimmune diseases and bone health conditions are not just medical challenges – they’re personal battles that millions of people fight every day. Fortunately, advances in clinical research are providing hope for better treatments and outcomes.

Research 52

Research Clinical Trials Therapies Immune Response 52

Drug Target Review

JANUARY 14, 2025

Operating a workflow, such as the one described in Figure 1, involves several key considerations that affect both the accuracy of results and the efficiency of the research process. Defining the research question A well-defined research question is the cornerstone of an effective scientific workflow in drug discovery.

Drugs 69

Drugs Protein Expression Science Research 69

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.

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Drugs Treatment Disease Marketing 95

Drug Target Review

JUNE 30, 2025

. “I was increasingly drawn to understanding the underlying biology of disease at a molecular level – the ‘why’ – and how we might harness science to create better medicines for patients,” Lepore says. Today’s broad-spectrum drugs often miss the mark, especially for chronic diseases like COPD or heart failure.

Disease 59

Disease Medical Schools Science Therapies 59

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

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. This is especially relevant with today’s heavier focus on enhancing personalised medicine via broader emerging scientific findings.

Drug Development 65

Drug Development Drugs Animal Testing Engineering 65

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

MARCH 6, 2025

ABSTRACT Parkinson's disease (PD) is the second most common neurodegenerative disease but has limited medications. The results indicated a candidate 2 with IC 50 values of 2.396 and 5.996M against LRRK2 and LRRK2 G2019S, respectively, implying the reliability of this approach.

Treatment 100

Treatment Disease Drugs Research 100

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

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.

Science 144

Science Clinical Trials DNA Disease 144

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

Broad Institute

JANUARY 6, 2025

This and other molecular tools developed by Zhang have accelerated biomedical research worldwide. In 2023, the first CRISPR Cas9-based therapeutic, which is based on a design Zhang developed in 2015, was approved for clinical use to treat sickle cell disease. Lisa Yang and Hock E.

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Medical Schools Engineering Science Hospitals 138