Drug Target Review

JUNE 21, 2023

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.

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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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Treatment Disease Clinical Trials Molecular Biology 105

Eye on FDA

MARCH 7, 2024

Back in December 2023, FDA announced intention in the Federal Register and in a press release to form a new FDA Advisory Committee to be called the Genetic Metabolic Diseases Advisory Committee (GeMDAC).

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FDA Disease Government Drugs 78

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.

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Clinical Development Clinical Trials Clinical Pharmacology Trials 59

Broad Institute

JUNE 18, 2024

By Allessandra DiCorato June 18, 2024 Credit: Jon Arizti-Sanz SHINE, a rapid diagnostic test developed by Pardis Sabeti's lab in 2020, uses paper strips and CRISPR enzymes to identify specific sequences of viral RNA in samples. Tags: Infectious Disease Diagnostics Pardis Sabeti Paper cited Zhang YB, Arizti-Sanz J, et al. 2024.04.004.

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Drug Target Review

MAY 7, 2024

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

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Disease Clinical Trials Clinical Pharmacology Trials 59

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

Drug Discovery Today

MAY 21, 2020

Glasgow, Scotland, 21 May 2020 - Amphista Therapeutics, a biopharmaceutical company creating first-in-class cancer therapeutics that harness the body’s natural processes to remove disease causing proteins selectively and efficiently, today announced the appointment of Dr Ian Churcher as Chief Scientific Officer (CSO).

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Disease 100

Drug Target Review

FEBRUARY 14, 2024

Researchers from the Michigan State University have created an advanced human heart organoid system that models the characteristics of pregestational diabetes-induced congenital heart disease found in mice and humans. The most common type of congenital defect in humans is congenital heart disease.

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Disease Research Drugs 59

FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

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Drug Discovery Today

JULY 15, 2020

Amsterdam, The Netherlands, 23 June 2020 - Scenic Biotech BV (“Scenic”), a pioneer in the discovery of genetic modifiers to enable the development of disease modifying therapeutics for rare genetic disorders and other devastating illnesses, today announced that it has been awarded a €3.1

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Government Disease 100

Drug Target Review

NOVEMBER 1, 2023

2 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. 1-5 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. 2020 Jan 1;57(1):372–92. References: Ninou I, Magkrioti C, Aidinis V.

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addgene Blog

FEBRUARY 13, 2024

Monoclonal antibody drugs are popular therapeutics for a plethora of disease conditions, from cancer to autoimmune disorders. Antibodies administered as drugs are still immunogenic, meaning that they elicit an immune response from the body.

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Immune Response Drugs Production Disease 97

Broad Institute

APRIL 25, 2024

He started to imagine how the deadly and contagious disease, if confirmed, might spread to half the city’s population. Eight of the 20 patients died, but the spread of the disease in Nigeria stopped there. Blood and urine samples from the man were waiting for Happi in his lab. Happi felt chilled. They called the idea Sentinel.

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Drug Discovery Today

APRIL 25, 2020

AUSTIN, Texas, April 23, 2020 /PRNewswire/ -- VolitionRx Limited (NYSE AMERICAN: VNRX) ("Volition") today announced that it is actively developing a COVID-19 triage test aiming to predict the likelihood that an individual who is COVID-19 positive will develop complications and severe disease, using its propriety Nu.QTM platform.

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Hospitals Disease 100

The Pharma Data

NOVEMBER 9, 2020

DAEGU, KOREA, November 09, 2020 /24-7PressRelease/ — South Korean religious group Shincheonji Church of Jesus has announced their third convalescent plasma donation drive to take place for 3 weeks starting November 16th at an Indoor Athletics Center in Daegu. Approximately 4,000 members of the group are expected to participate.

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Advarra

AUGUST 16, 2022

A rare, or orphan, disease by definition affects a small percentage of the population — fewer than 200,000 people in the U.S. But the numbers add up, and taken together, rare diseases impact an estimated 30 million Americans. Since then, the FDA has significantly changed its approach to rare and orphan diseases.

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Drug Target Review

FEBRUARY 2, 2024

What specific limitations in mirroring diseases in humans are commonly observed when using animal models for preclinical research? Our cells are fully differentiated, non-tumour, primary cells, which have a more natural growth pattern and gene expression profile, and the disease signature is often carried over epigenetically.

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Drugs Clinical Trials Disease Science 116

The Premier Consulting Blog

JUNE 18, 2023

One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases. However, with rare diseases there may be no available treatments that could serve as SoC or active control in a clinical trial and assigning patients to placebo may be unethical.

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Drug Development Disease Clinical Trials Drugs 52

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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SCIENMAG: Medicine & Health

JUNE 23, 2023

are 40% more likely to develop heart disease and have a 30% higher risk of stroke than people who live in urban areas, according to data reported in the 2020 Call to Action: Rural Health: A Presidential Advisory From the American Heart […] DALLAS, June 23, 2023 — People who live in rural areas of the U.S.

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Research Disease 81

Broad Institute

OCTOBER 23, 2024

They knew that discovering the genetic cause of her disorder would help them find other people like her, help get the condition formally recognized as a new disease, and help them better advocate for research into new treatments. This is the first human disease caused by loss of one copy of a lncRNA gene.

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DNA RNA Disease Hospitals 128

Drug Target Review

AUGUST 9, 2023

Published on bit.bio’s website, this article authored by Dr Tony Oosterveen dives into the close relationship between FTD and ALS with a focus on mechanisms underlying the neuropathology of both diseases. ALS is the most common motor neuron disease and is estimated to affect more than 80,000 people worldwide.

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RNA Regulations Disease DNA 98

The Pharma Data

MAY 31, 2022

BIIB122 is an inhibitor of LRRK2, a potential novel target intended to impact the underlying biology and slow the progression of Parkinson’s disease Phase 2b LUMA to enroll approximately 640 participants with early-stage Parkinson’s disease; most advanced clinical study of a LRRK2 inhibitor. Denali Therapeutics Inc.

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Disease Small Molecule Engineering Treatment 52

Drug Target Review

JULY 6, 2023

million scientific procedures involving live animals were carried out in 2020. Three-dimensional organoids have been a popular choice in testing drugs to treat cancer and disease. Animal testing plays a significant role in pre-clinical research and therefore requires the use of millions of animals. In Britain, 2.88

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Drug Target Review

OCTOBER 5, 2023

The research team identified 231 individuals diagnosed with relapsing-remitting MS, with 174 of them having undergone aHSCT treatment before the year 2020. Around three years following their aHSCT, 20 patients (11 percent) were administered disease-modifying drugs.

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Drug Target Review

APRIL 7, 2025

The challenge of GPCR drug discovery G protein-coupled receptors (GPCRs) are one of the most desirable and challenging target classes in drug discovery, as their mutation can lead to a wide range of diseases such as cancer, cardiovascular disorders and neurological conditions. References Sun D, et al. Acta Pharmaceutica Sinica B , 12(7), pp.

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Drug Target Review

JANUARY 15, 2024

Chronic obstructive pulmonary disease Chronic obstructive pulmonary disease (COPD) is the third leading cause of global deaths. The Pathology of Chronic Obstructive Pulmonary Disease. Annual Review of Pathology: Mechanisms of Disease. 2020 October 1 [2024 January 5]; 27(4):523-31. References 1 Hogg JC, Timens W.

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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.

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Disease Clinical Trials Trials Treatment 52

New Drug Approvals

SEPTEMBER 29, 2024

gingivalis has been called a risk factor for Alzheimer’s disease ( Kanagasingam et al., gingivalis is associated with cardiovascular disease. Both were reversed by treatment with COR388 ( 2020 AAIC abstract ). A dental substudy of 228 participants is assessing effects of COR388 on periodontal disease. and Europe.

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Small Molecule DNA Disease Trials 62

New Drug Approvals

SEPTEMBER 23, 2024

Target: DNA/RNA Synthesis Pathway: Cell Cycle/DNA Damage Huntington’s disease (HD) is a progressive, autosomal dominant neurodegenerative disorder of the brain, having symptoms characterized by involuntary movements, cognitive impairment, and mental deterioration. 2020-01-02. SCHEME PATENT PTC Therapeutics Inc., Audiol Res. J Pers Med.

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Small Molecule RNA DNA Disease 57

Drug Target Review

AUGUST 11, 2023

By selecting specific segments of proteins that elicit robust immune reactions, these vaccines could offer enhanced protection against diseases. Elsevier; 2020 [cited 2023 Jul 25]. Since December 2019, SARS-CoV-2 (COVID-19) infection has become a worldwide urgent public health concern. References Mehdi Hassanpour, et al.

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SCIENMAG: Medicine & Health

JULY 5, 2023

New drugs are often used not only for one disease (first approved indication) but also for other diseases (supplemental indications).

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Treatment Drugs Disease 67

PLOS: DNA Science

MAY 16, 2024

These factors are converging to enable both identification of novel infectious diseases as well as microbial resistance, before these threats can impact public health, write a team from the European Society for Clinical Microbiology and Infectious Diseases in Frontiers in Science. COVID clearly caught us off guard.

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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

The Pharma Data

APRIL 7, 2022

These coverage restrictions, including the distinction between accelerated approval and traditional approval, have never been applied to FDA-approved medicines for other disease areas. ADUHELM is indicated for the treatment of Alzheimer’s disease. It may also limit coverage for any future approved treatment in the class. About Biogen.

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Treatment Therapies Disease FDA Approval 52

Drug Target Review

JUNE 8, 2023

An important research area at AbbVie involves blood cancers, where our ultimate goal is to transform standards of care across these diseases and advance a dynamic cancer research and treatment pipeline. Chronic lymphocytic leukemia in 2020: a surfeit of riches? 2020 Aug;34(8):1979-1983. Epub 2020 May 11.

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