Conversations in Drug Development Trends

NOVEMBER 12, 2024

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations.

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Drug Target Review

JULY 5, 2023

Induced pluripotent stem cell (iPSC) therapies have the potential to treat a wide variety of devastating diseases. Please summarise Eterna Therapeutics’ ($ERNA) new research at ASGCT on a cell therapy approach that integrates multiple cell types to emulate the natural immune system’s fight against diseases?

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Broad Institute

OCTOBER 3, 2024

Epilepsy also has several subtypes, and while one group called developmental encephalopathies have been connected to several genes, other forms of the disease are less well understood. The exomes revealed connections between disease risk and several genes involved in the transmission of signals across the synapses between neurons.

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Drug Target Review

SEPTEMBER 28, 2023

After some time in that role and launching several products, I received a call from Bill Banyai and Bill Peck, or ‘The Bills’ as we call them, who were building a company around technology that creates DNA by ‘writing’ it on a silicon chip. They needed reference materials for the disease in order to develop and validate diagnostic tests.

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PLOS: DNA Science

AUGUST 24, 2023

“Pangenomics” is a newish term, referring to ways that a species’ genome can vary, DNA base by DNA base. I wrote The Age of the Pangenome Dawns here at DNA Science last year, about the Human Pangenome Reference Consortium. A genome of 3,054,832 billion DNA base pairs can vary in many ways.

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PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. A Scant Protein with Outsize Importance Slowing the muscle decline of DMD presents two huge challenges: the ubiquity of muscle and a giant gene. million DNA bases.

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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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Chemical Biology and Drug Design

NOVEMBER 23, 2023

The symptoms of the disease include delusions and hallucinations, associated with extremely disordered behavior and thinking, which may affect the daily lives of the patients. Advancements in technology have led to understanding the dynamics of the disease and the identification of the underlying causes.

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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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Broad Institute

MARCH 26, 2024

The team analyzed ancient DNA and traced the variant all the way back to the first people to enter the Americas about 13,000 years ago. "We The researchers then analyzed ancient DNA samples to understand when the risk variant arose in human history. Further investigation revealed that the variant was promoted by natural selection.

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Drug Target Review

DECEMBER 21, 2023

To provide comprehensive insights into ongoing developments, we are pleased to present the latest edition of Drug Target Review. Dr Piotr Trzonkowski, from the University of Gdansk, highlights how TREGs offer hope for personalised medicine in autoimmune diseases.

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Drug Target Review

NOVEMBER 13, 2023

Regulatory T cells (Treg) cells are specialised immune cells that are essential for maintaining peripheral tolerance, preventing autoimmune diseases, and limiting chronic inflammatory diseases. However, they also limit beneficial responses by suppressing sterilising immunity and limiting antitumour immunity.

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KIF1A

JANUARY 13, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. As one cell turns into two, its DNA must be replicated and then pulled apart, so that when the cell divides each has all the DNA it needs to function.

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NIH Director's Blog: Drug Discovery

MARCH 8, 2016

Instead of the black, printed stripes of the Universal Product Codes (UPCs) that we see on everything from package deliveries to clothing tags, they used short, unique snippets of DNA to label cells. DNA barcoding has already empowered single-cell analysis, including for nerve cells in the brain.

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Broad Institute

FEBRUARY 16, 2024

We didn’t expect that frequency of genetic variants would correlate with clinical presentation, but that amazingly seems to be what we’re seeing.” Their analyses demonstrated that the combination of common and rare genetic variants in youth-onset T2D have a greater influence on disease risk than in the adult-onset form.

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PLOS: DNA Science

OCTOBER 19, 2023

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.

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Drug Target Review

AUGUST 16, 2023

However, a study published in eLight , led by Professor Xiangheng Xiao from Wuhan University, presents a game-changing approach that merges SERS with artificial intelligence (AI) for cancer screening (SERS-AICS). Direct detection of early-stage cancers using circulating tumor DNA. percent, sensitivity of 95.40 References 1.

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The Pharma Data

NOVEMBER 15, 2020

Triplet’s chief executive officer, president, and founder, will present at the Stifel Virtual Healthcare Conference taking place November 16-18, 2020. Dr. Bermingham will discuss Triplet’s recent activities, including: Dr. Bermingham’s presentation details are as follows: Date: Wednesday, November 18 Time: 9:20 a.m.

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The Pharma Data

SEPTEMBER 6, 2021

First data to be presented from the phase II coopERA Breast Cancer study evaluating neoadjuvant giredestrant treatment for oestrogen receptor (ER)-positive, HER2-negative breast cancer. Breast Cancer Highlights. Lung Cancer Highlights.

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DrugBank

MAY 22, 2024

In the dynamic world of drug discovery, the notion of "undruggable" targets presents both a significant challenge and an intriguing frontier for researchers and pharmaceutical companies. Beyond Proteins: DNA and RNA Frontier The story doesn’t end with proteins.

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DrugBank

MARCH 12, 2025

However, mRNA technology is not limited to infectious diseases. This unique mechanism has enabled scientists to rethink how they tackle diseases, paving the way for advancements in oncology, genetic disorders, and regenerative medicine. 

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The Pharma Data

NOVEMBER 18, 2020

Presentations to highlight the OBI-833 Phase 1 clinical study results in non-small cell lung cancer (NSCLC) and the dose escalation cohort. These results will be presented by the lead investigators of OBI Pharma’s novel anti-Globo H therapeutic cancer vaccine, OBI-833. ” Presentation number: 397P / Poster: ID 680.

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DrugBank

JULY 24, 2024

This is a game-changer, especially in the fight against cancer and other complex diseases. The versatility and potential of PROTACs are vast, paving the way for new therapies that could transform how we treat diseases. This can potentially correct the root causes of some diseases at the genetic level.

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Drug Target Review

JUNE 9, 2023

One aspect of the collaboration focuses on screening and identification of therapeutic T-cell receptors (TCRs) to target a common shared neoantigen present in many cancer patients. What types of diseases or conditions were targeted in the pre-clinical research phase? a healthcare informatics company.

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The Pharma Data

APRIL 11, 2023

The presentation highlights the synergistic anti-cancer effects of a PSMA (prostate-specific membrane antigen)-actinium-225 conjugate in combination with darolutamide in preclinical prostate cancer models. Data from all three areas of scientific focus will be showcased during this year’s meeting.

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Drug Target Review

SEPTEMBER 8, 2023

Remarkably, these findings propose that the presence of a cytoplasmic version of RARα is indispensable for T cells to initiate their defence against diseases. The cell’s nucleus, containing compacted DNA, is centrally positioned within the cell.

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PPD

AUGUST 5, 2024

Pulmonary arterial hypertension (PAH) is a progressive and fatal lung disease that is caused or influenced by multiple factors. As your CRO partner, we deliver equally dedicated and experienced teams that understand this disease and the range of assessments used for collecting endpoint data in PAH clinical trials.

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The Pharma Data

JULY 11, 2021

years with TAKHYZRO is Consistent with Initial Period of Treatment, Building on Growing Body of Evidence on the Long-term Safety and Efficacy Final Patient Subgroup Analysis Suggests Reductions of HAE Attacks Across Range of Patient Demographics and Disease Characteristics with TAKHZYRO. Analysis of Safety and Efficacy for up to 2.5

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DrugBank

JUNE 27, 2023

” 4 The term originally rose to prominence after the completion of the Human Genome Project 5 as a call to action in the face of wide disparities between treatment outcomes within the same clinical presentation; however, the exact meaning has changed as technology continues to advance.

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Drug Target Review

APRIL 9, 2024

How has nucleolar stress (NS) been linked to age-related diseases, and what approach did researchers use to investigate its toxicity? The nucleolus (and nucleolar stress) had been previously linked to aging and age-related diseases such as cancer or degeneration.

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DrugBank

JANUARY 29, 2025

Gene Therapy Gene therapy operates on the principle of modulating the DNA blueprint of cells to induce a therapeutic response. Each of these mechanisms represents a distinct strategy to address the underlying genetic perturbations contributing to disease pathogenesis.

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Drug Target Review

APRIL 3, 2024

Everything started in school with an experiment on isolating DNA from bananas. Making a little tangle of DNA visible to the eye and understanding that this is the basis of complex organisms, which might be altered in disease, was the defining moment for my future path in life sciences.

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New Drug Approvals

SEPTEMBER 6, 2024

US10071079, Example 15 Ref WO2018005586 [link] The invention further pertains to pharmaceutical compositions containing at least one compound according to the invention that are useful for the treatment of conditions related to TLR modulation, such as inflammatory and autoimmune diseases, and methods of inhibiting the activity of TLRs in a mammal.

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Broad Institute

MAY 25, 2023

By coupling the experiments with LipocyteProfiler , an imaging assay tailored for cardiometabolic disease research, they found that changes in COBLL1 expression alter how a developing fat cell's cytoskeleton matures. In cells where COBLL1 activity has been dialed down, few fat deposits (green) are present.

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Broad Institute

JULY 25, 2024

But she soon realized that to truly capture the complexity of human disease, she would need to study a large number of cells from many people with or without the disease and with different genetic backgrounds. But human cells include that genetic background, which can really influence disease.

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PerkinElmer

JANUARY 19, 2020

Here are ten key tutorials, presentations and posters designed to help you achieve fast, accurate and reproducible results in precision medicine, next generation sequencing, homogenous assays and more. Time away from the lab is limited and valuable. Here we describe a cloud-enabled AI platform for analyzing and visualizing HCS data.

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The Pharma Data

OCTOBER 18, 2020

(NASDAQ:CDXC) today highlighted a new study published in The European Molecular Biology Organization Journal looking at the effect of nicotinamide riboside (NR) on maintaining telomeres, the protective regions at the end of DNA strands. Telomeres are “caps” at the end of chromosomes that protect DNA from getting worn away as cells replicate.

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