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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Science Clinical Trials DNA Disease 139

Drugs.com

JULY 13, 2023

THURSDAY, July 13, 2023 -- More kids in the United States are getting a developmental disability diagnosis, with prevalence close to 9% in 2021, the U.S. Centers for Disease Control and Prevention reports. Among 3- to 17-year-olds, 8.56% have ever.

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Disease 189

Drug Discovery Today

FEBRUARY 17, 2021

LONDON, 17 February 2021 – Smiths Detection, a global leader in threat detection and security screening technologies, today reports that its BioFlash® Biological Identifier is capable of detecting SARS-CoV-2 in the air following tests conducted by the United States Army Medical Research Institute of Infectious Diseases (USAMRIID)

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Disease Research 113

Drug Discovery Today

JANUARY 25, 2021

OSLO/LONDON, JANUARY 20 2021: EXACT THERAPEUTICS AS (“EXACT-Tx” or the “Company”), a clinical stage precision medicine company today announced a collaboration with GE Healthcare to develop an innovative ultrasound probe to be used in forthcoming studies using Acoustic Cluster Therapy (ACT®) across multiple disease conditions.

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Therapies Disease 113

Drug Target Review

OCTOBER 25, 2024

How does precision neuroscience differ from traditional approaches in the treatment of neurodegenerative diseases, and what advantages does it offer? Traditionally, Alzheimer’s disease is diagnosed by clinical examination, medical history and cognitive testing. We believe we are at a breakthrough moment in Alzheimer’s disease research.

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Disease Clinical Trials Therapies Science 59

Broad Institute

JUNE 10, 2024

The advance, from the lab of Broad core institute member David Liu , 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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DNA Engineering Therapies Disease 144

Drug Target Review

FEBRUARY 8, 2024

3 Moreover, younger CRC patients have been shown to present with more advanced stages of the disease. Gene sequencing The team utilised gene sequencing technology to compare tissue samples taken from 136 young-onset colorectal cancer patients with 140 average-age patients with the disease. 2021 April 22 [2024 February 6]; 13(9):2025.

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Disease Research Drugs 106

Drug Target Review

JUNE 3, 2024

How does COUR Pharmaceuticals’ immune-modifying nanoparticle platform differ from traditional approaches to treating immune-mediated diseases? Essentially, our CNPs reprogramme the immune system by restoring balance and returning the body to a homeostatic state, offering a precise approach to treating immune-mediated diseases.

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Drug Target Review

JULY 4, 2023

In practice, some patients may be left managing a syndrome associated with the therapy along with the disease. For instance, we can compare the data from the 2021 DESTINY gastric cancer trial using the ADC Trastuzumab Deruxtecan with data from a gastric cancer trial in 2004 using the small molecule Irinotecan. Colombo R, Rich JR.

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Small Molecule Drugs Clinical Pharmacology Trials 145

KIF1A

MARCH 16, 2024

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. Like hereditary spastic paraplegia, CMT is an umbrella disorder, associated with mutations in over 140 genes, but many diagnosed patients do not know their disease-causing mutation.

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Disease Clinical Development Research Treatment 105

biobide

SEPTEMBER 15, 2023

The need to protect crops from pests, disease, and weeds has existed for centuries, with the Roman’s having used ash, crushed cypress leaves and diluted urine to protect their crops. In 2021, the UK government introduced new “Maximum Residue Levels”. Pesticides are a vital tool in safeguarding the world’s food supply.

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Regulations Government Disease Production 112

Drug Target Review

SEPTEMBER 21, 2023

Some report that they believe that if they don’t take the medication, they may develop onchocerciasis-related severe skin diseases and may go blind (river blindness) and would prefer a treatment that does not repeat every year. Need for adulticidal drugs More than 99 percent of countries worldwide has been affected by this disease.

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Drugs Treatment Disease Research 106

Chemical Biology and Drug Design

OCTOBER 10, 2023

Abstract Human immunodeficiency virus (HIV) causes acquired immunodeficiency syndrome (AIDS), a lethal disease that is prevalent worldwide. million people worldwide were living with HIV in 2021. According to the Joint United Nations Programme on HIV/AIDS (UNAIDS) data, 38.4 million

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FDA Approval Clinical Trials FDA Virus 100

KIF1A

OCTOBER 14, 2023

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. But the opposite relationship is just as important: knowing which diseases are associated with a symptom means earlier diagnosis, and an earlier search for other potential symptoms.

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Disease Treatment Research Production 105

The Pharma Data

MAY 31, 2022

the completion of enrollment of its Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease (PD). of BRT-DA01 in patients with Parkinson’s disease.?. In fiscal 2021, the Group employed around 100,000 people and had sales of 44.1 The purpose of the?Ph1

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Trials Disease Clinical Trials Engineering 52

The Pharma Data

MARCH 16, 2022

(Nasdaq: BIIB) announced that The Journal of Prevention of Alzheimer’s Disease (JPAD) today published a peer-reviewed manuscript detailing data from the pivotal Phase 3 EMERGE and ENGAGE trials for ADUHELM ® (aducanumab-avwa) 100 mg/mL injection for intravenous use in early Alzheimer’s disease. In June 2021, the U.S.

Disease 52

Disease Clinical Trials Treatment Trials 52

Broad Institute

SEPTEMBER 15, 2023

We apply STACI to analyze the spatio-temporal progression of Alzheimer’s disease and identify the associated nuclear morphometric and coupled gene expression features.

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Disease 40

DrugBank

JUNE 27, 2023

Out of the millions of genetic variants present in human populations, GWAS are designed to uncover those associated with specific traits or diseases. 9 Polygenic diseases are much more common, and thousands of genetic variants with very small effects could impact their phenotype.

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Disease Treatment Drugs Clinical Trials 98

LifeSciVC

NOVEMBER 17, 2023

This year we start with a deep dive into the biomedical innovation across our industry – “putting the Big back into Big Pharma” with obesity and Alzheimer’s, and the state of play across other disease areas, industry R&D productivity, and the risks facing the sector. As usual, we close with a brief update on Atlas itself.

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Science Marketing Production Disease 97

SugarCone Biotech

MARCH 24, 2025

Consequently, these drugs have limited ability to keep patients disease-free and limited impact on patient survival. doi: 10.1136/jitc-2021-004348 6. Our goal therefore is to safely improve these types of therapeutics for longer-lasting clinical efficacy. Oncoimmunology 11: 2111904. doi: 10.1080/2162402X.2022.2111904 2022.2111904 3.

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Immune Response Therapies Treatment Disease 67

Drug Target Review

NOVEMBER 29, 2023

We focus on patients who are resilient to disease and conduct an unbiased search for protective autoantibodies in these patients that may underlie their resiliency. There is a huge push for multiplexed biological readouts, coupled with more advanced translatable model systems that capture more of the complexity of human neurological diseases.

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Engineering Drug Development Disease Treatment 112

Broad Institute

JULY 10, 2023

Credit: Allison Colorado, Broad Communications Arriving in the Bay Area after a childhood in increasingly diverse surroundings, Martin became acutely aware of health disparities and the profound differences in disease prevalence among different populations. I wanted to know how I could have any impact on studying genetic diversity.”

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Disease Bioinformatics Medical Schools Research 98

LifeSciVC

APRIL 26, 2024

With just over 60 new biotechs raising their first round of financing, the sector’s company formation activity has slowed 50-60% from its historic peak in 2021. Further, this lemming challenge has also moved well beyond oncology – think of all the “not-so-fast follower” autoimmune programs or metabolic disease stories.

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DNA Science Marketing Clinical Trials 112

Eye on FDA

JANUARY 26, 2022

That said, the last two letters – the final one of 2021 and the first of 2022 both went to the same company – Lilly. Note: In a prior posting reviewing 2021 regulatory actions, it was stated that FDA issued 5 regulatory action letters for the year.

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FDA Production Drugs Disease 108

Drug Target Review

SEPTEMBER 19, 2023

Prior to this European Union Parliament, in 2021, voted for animal testing phase out. Viable in vitro alternatives to animal Models: The European Medicines Agency has launched the new approach methodologies in 2021 promoting the use of animal experiment alternatives in drug discovery and development. The US FDA Modernisation Act 2.0.,

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Animal Testing Clinical Trials Pharmaceutical Companies FDA 98

LifeSciVC

JANUARY 16, 2024

recent announcements from Merck and Sanofi for obesity and broader metabolic disease). ongoing tirzepatide CVOT to assess head-to-head CV outcomes vs. Trulicity in T2D) will continue to build the case for weight loss (and incretins) in broader metabolic disease and suggest overall healthcare system cost benefits to anti-obesity therapies.

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Small Molecule Trials Therapies Disease 140

Drug Target Review

SEPTEMBER 26, 2023

Human derived induced pluripotent stem cells (hiPSCs) have revolutionised research and are increasingly used for toxicology screening and disease modelling. 2021 Jan 1;179(1):3–13. Using stem cell-derived neurons in drug screening for neurological diseases. 2021 Jun;17(6):381–92. J Pharmacol Toxicol Methods. Toxicol Sci.

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Drugs FDA Disease Animal Testing 112

PPD

AUGUST 16, 2023

The study’s objective was to supplement the findings of PPD surveys conducted in 2020 and 2021 that examined similar issues from the perspective of sponsor pharmaceutical companies. However, the accessibility of advanced technologies enables the expansion of DCT models to other disease states.

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Clinical Trials Trials Clinical Research FDA 98

Drug Target Review

JULY 5, 2024

Since brain organoids can be derived from either diseased or healthy patients, they can recapitulate human (patho)physiology making them a better choice compared to mammalian cell systems that often fail to recapitulate the human phenotype. 13(2):280 (2021). Pharmaceutics. link] Daviaud N, et al. 12(3) (2023). link] Tan SJ, et al.

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Targeted Protein Degradation Drugs Disease FDA Approval 65

Drug Target Review

AUGUST 24, 2023

These findings were mirrored in the clinical results from an analysis of two metastatic melanoma patient cohorts who received anti-PD-1 treatment: patients with responses and disease control had significantly lower GDF-15 serum levels (measured before treatment start), while none of the patients with elevated GDF-15 levels showed a lasting response.

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Treatment Therapies Clinical Development Cell Biology 98

Drug Target Review

DECEMBER 16, 2024

Over the past three years in Oncology Early Development at AbbVie, I have worked on several antibody-drug conjugates from discovery to the clinic in disease areas of high unmet need. Opportunities in clinical research and drug development were not always easy to come by.

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Medical Schools Clinical Research Drug Development Trials 52

Tecan

DECEMBER 7, 2022

By Joe Guterl More than $72 billion – that is what some researchers estimate will be the global point-of-care (POC) biochemical diagnostic testing market size in 2027, up from $36 billion in 2021.

Marketing 52

Marketing Disease Research 52

DrugBaron

JANUARY 8, 2021

Here is a disease that is caused by the lack of functional dystrophin in the muscles of these boys. The underlying assumption of most gene therapy procedures, irrespective of the disease being targeted, is that restoration of a wild-type DNA sequence will alone be sufficient to normalise the phenotype of the individual.

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DNA Therapies Disease Production 100

KIF1A

MAY 27, 2023

Each week, Dr. Dominique Lessard and Dr. Dylan Verden of KIF1A.ORG summarize newly published KIF1A-related research and highlight progress in rare disease research and therapeutic development. And while an individual disease might be rare, collectively over 30 million people suffer from rare diseases in the United States alone.

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Science Small Molecule Disease Research 94

The Pharma Data

MARCH 8, 2021

These preliminary findings were presented today during Science Spotlights TM at the 2021 Conference on Retroviruses and Opportunistic Infections (CROI 2021). Headquartered in Miami, Florida, Ridgeback Biotherapeutics LP is a biotechnology company focused on emerging infectious diseases. About Ridgeback Biotherapeutics.

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Trials Virus RNA Research Laboratories 69

Drug Target Review

MAY 23, 2023

Inflammatory bowel disease (IBD) is an umbrella term that describes complex disorders that cause chronic inflammation in the digestive tract with alternating periods of relapse and remission. A changed composition of the gut microbiota is a characteristic trait of inflammatory bowel disease. More than 6.8

Drugs 75

Drugs Small Molecule Production Disease 75

LifeSciVC

FEBRUARY 27, 2024

Our lead asset is in development for adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), a rare disease with no approved treatments, so we have prioritized incorporating patient and care partner voices across every stage of development. Building Community Understanding the biology of the disease is the beginning.

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