LifeSciVC

JUNE 12, 2025

The ability of modifying RNA enabled us to sidestep many of the potential risks associated with permanent DNA editing. We aimed to achieve this by modifying and modulating proteins by precisely changing a single alphabet on RNA with the use of an oligonucleotide (think short stretches of chemically modified RNA or DNA).

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RNA Protein Expression DNA Science 67

Codon

NOVEMBER 3, 2024

As an undergraduate biology student, I spent some time in a TB lab working on antibiotic resistance — a growing concern for drug developers. Some fluoroquinolones — a class of antibiotics that inhibits DNA replication — may even cause psychosis. However, hope prevails.

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Vaccine Trials Therapies Treatment 114

Broad Institute

NOVEMBER 21, 2024

In IDH-driven gliomas, the mutated IDH enzyme fosters the addition of methyl groups to the cells’ DNA. These chemical changes alter the expression of genes without modifying the underlying DNA sequence. Later in the disease, other genetic drivers that are not targeted by the drug take over.

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Research Medical Schools DNA Hospitals 124

SugarCone Biotech

JULY 6, 2025

It follows that HER3-targeting therapies have emerged as a class of anti-cancer treatments designed to treat various tumors that have developed resistance to EGFR or HER2-directed therapies or have emerged due to a NRG1 gene fusion. This is an active drug development landscape with a lot of recent news.

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Drug Development Drugs Clinical Trials Therapies 56

BioPharma Drive: Drug Pricing

JUNE 11, 2025

billion buyout of Blueprint Medicines is the sector’s second-largest M&A deal this year and represents the highest value paid for an immune drug developer since April 2023. Sanofi’s $9.1 Sanofi’s $9.1

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Conversations in Drug Development Trends

NOVEMBER 12, 2024

For example, once considered incurable and terminal, patients with sickle cell disease may reach new summits in their lives with gene editing technologies such as CRISPR to repair affected DNA and, in some cases, functionally cure the condition.

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FDA Law Blog: Drug Discovery

DECEMBER 15, 2024

For adeno-associated viral vectors, examples may include characterization of non vector DNA impurities in capsids by next-generation sequencing, vector genome size analysis, and detection of capsid amino acid modifications by mass spectrometry.

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Conversations in Drug Development Trends

APRIL 7, 2025

Liquid biopsies, particularly those that detect circulating tumor DNA amongst other known blood-based biomarkers, make the screening process less invasive and more approachable to patients who already undergo multiple blood draws daily, reducing participation burden.

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Conversations in Drug Development Trends

APRIL 29, 2025

We welcome opportunities to engage with the rare disease community and help contribute to the conversation between advocates, researchers, drug developers, and more. For any collaboration inquiries or questions about the 2025 Rally for Rare event, reach out to rare@worldwide.com.

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Perficient: Drug Development

APRIL 28, 2025

They’re asking: Who owns this dataset? How do we know it’s complete? What biases are hiding here? How do we certify and monitor trustworthiness over time?

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Government Compliance Engineering DNA 59

Collaborative Drug

JULY 22, 2024

Is Revolutionizing Drug Development. Holy Grail for Weight-Loss Drugs Is Sleep Apnea? DNA Discovery that Could Help Find Pancreatic Cancer Cure.

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DrugBank

OCTOBER 17, 2024

Data science has emerged as an innovative tool in the biopharmaceutical industry, leveraging the power of machine learning and artificial intelligence to drive innovation and efficiency across the entire drug development lifecycle. This was seen in the case of the BRAF V600E mutation test for melanoma patients receiving vemurafenib.

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Drug Development Metabolic Stability Clinical Trials Drugs 98

Drug Target Review

JULY 18, 2023

Artificial Intelligence (AI) is poised to transform the field of target discovery in drug development, offering immense potential to enhance efficacy, personalised medicine, and accelerate the development of innovative compounds. This means that we are not doing something right.

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NIH Director's Blog: Drug Development

MARCH 11, 2014

Credit: Jane Ades, National Human Genome Research Institute, NIH Type 2 diabetes (T2D) tends to run in families, and over the last five years the application of genomic technologies has led to discovery of more than 60 specific DNA variants that contribute to risk.

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DNA Drugs Disease Research Laboratories 52

Chemical Biology and Drug Design

MAY 21, 2025

Molecular docking studies further supported the experimental findings, showing favorable interactions with bacterial DNA gyrase (PDB ID: 1KZN). Additionally, SwissADME profiling revealed that the compounds possess promising drug-like properties and oral bioavailability.

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KIF1A

JANUARY 13, 2024

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. The strands that pull the DNA apart are composed of microtubules, and kinesins like KIF1A and KIF13B can impact the direction the strands pull in.

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DNA Regulations Drug Development Disease 105

PLOS: DNA Science

MARCH 28, 2024

It “could have been easily repurposed for DMD,” said Pier Lorenzo Puri at Sanford Burnham Prebys Medical Discovery Institute in San Diego, who has been working on the drug development since 2004. MDA supported development of a mouse model that enabled the preclinical studies of what would become Duvyzat.

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Chemical Biology and Drug Design

SEPTEMBER 12, 2023

In the drug development process, linker hybrids acquire the top position due to their excellent π-stacking and Van der Waals interaction with the DNA active sites of pathogens. This review highlights the recent advances (2013–2023) in the pharmacology of rhodanine-linked quinoline hybrids as more effective antimicrobial agents.

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Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

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DrugBank

MAY 22, 2024

Beyond Proteins: DNA and RNA Frontier The story doesn’t end with proteins. DNA and RNA are also key players, each bringing unique challenges. Many cancer treatments target DNA, but often these can cause collateral damage, leading to severe side effects.

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Small Molecule RNA DNA Pharmaceutical Companies 96

LifeSciVC

AUGUST 5, 2024

We are constantly reminded how we are in the midst of an artificial intelligence revolution of the drug development process which promises to completely transform how we develop drugs with increases in productivity of an order of magnitude or more.

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Conversations in Drug Development Trends

JUNE 13, 2024

At Worldwide, our recent study delved into the intricacies of PGx, comparing two common methods of DNA sample collection: buccal swabs and blood samples. These results are largely due to the higher cell density in blood, which includes DNA-rich white blood cells. for buccal swabs and 93.2% for blood samples.

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DNA Compliance Clinical Trials Trials 72

Drug Target Review

JANUARY 8, 2024

We are at the forefront of drug development in an area of research called cellular rejuvenation, which is an approach that has the potential to address many diseases of ageing by restoring aged and injured cells to a more youthful and resilient state. The epigenome drifts with age, leading to dysregulated gene expression.

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Science Disease FDA Approval Pharmaceutical Companies 145

Drug Target Review

DECEMBER 14, 2023

Throughout the course of our lives, natural and environmental factors can break DNA. Breaks in both DNA strands are a particularly dangerous threat to genome stability. At a DNA double-strand break (DSB), potentially lost sequence information cannot be recovered from the same DNA molecule.

DNA 59

DNA Clinical Trials Treatment Research 59

DrugBank

APRIL 24, 2025

Epigenetics , the study of changes in gene activity that occur without altering the DNA sequence, has revolutionized our understanding of gene expression regulation. DNA methylation involves adding methyl groups to cytosine residues in CpG dinucleotides , a process catalyzed by DNA methyltransferases (DNMT).

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DNA Drugs Therapies Regulations 52

DrugBank

MAY 15, 2024

Its structure makes it incredibly difficult for drugs to bind effectively, which has stymied drug development for decades. The binding pockets on KRAS are shallow and polar , not ideal for the kinds of interactions needed for strong, effective drug binding.

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Small Molecule Molecular Biology Drugs Disease 98

Alta Sciences

APRIL 16, 2024

Inside The Altascientist: The Benefits of PCR for Your Gene Therapy Programs pmjackson Tue, 04/16/2024 - 19:04 The gene therapy landscape continues to accelerate in preclinical and clinical research, with programs constantly in development for targeted, personalized medicines. But how are PCR analysis techniques applied?

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Therapies DNA RNA Laboratories 52

NIH Director's Blog: Drug Development

APRIL 18, 2017

Every person’s genetic blueprint, or genome, is unique because of variations that occasionally occur in our DNA sequences. As promising as it may be, this collection of 1,300 or so human gene knockouts in Pakistan represents what is likely just the start of a rapidly growing area of scientific research and drug development.

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The Pharma Data

DECEMBER 20, 2020

UK drug developer Scancell said it has chosen a COVID-19 vaccine candidate, SN14, from more than a dozen potential products to advance to a clinical trial. . SN14 works by targeting the coronavirus’ nucleocapsid and spike proteins to prevent viral replication using the company’s ImmunoBody DNA vaccine technology.

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The Pharma Data

SEPTEMBER 11, 2021

We have the ability to generate precise antibodies to a diverse range of targets, which together with Boehringer Ingelheim’s strength in drug development capabilities, could mean multiple new, more personalized treatments in the future for patients,” said Emily M. Leproust, Ph.D., CEO and co-founder of Twist. About Twist Biopharma.

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DNA Bioinformatics Production Engineering 52

Drug Target Review

SEPTEMBER 10, 2024

The current landscape of protein drug development is characterised by accelerated timelines where new drugs are approved in months rather than years. Hence, in many cases an earlier IND may be prevented by the timely provision of representative Drug Substance (DS) to execute such toxicology studies.

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Drug Target Review

NOVEMBER 6, 2023

To be successful, surrogate or intermediate endpoints will need to be established to accelerate the development of the new regimens. At AstraZeneca and beyond we are exploring the use of cell-free DNA (cfDNA) methods to assess minimal residual disease as a possible intermediate endpoint across several disease types.

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Small Molecule Therapies Treatment Disease 111

DrugBaron

OCTOBER 17, 2022

Buoyed by the string of successes in early discovery delivered by this approach, attention inevitably turned to how we might leverage the same insights to improve return on capital in the rest of the long path through drug development. But how to proceed? Will it work?

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DrugBaron

JULY 19, 2022

The first is the growing popularity of DNA-encoded libraries (DELs). Here, libraries are constructed on DNA tags in a variety of different ways, but in essence in a broadly similar way to the creation of a virtual chemical space. Two solutions, one physical and one computational have emerged.

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Small Molecule Drugs DNA Pharma Companies 100

DrugBank

JULY 24, 2024

This was the first successful drug development against a transcription factor, opening the door to targeting a whole new class of previously unreachable targets. The Promise of Multispecific Drugs The future of drug discovery looks even brighter with the development of multispecific drugs.

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Small Molecule Drugs Clinical Trials Therapies 98

Conversations in Drug Development Trends

AUGUST 14, 2024

A few notable developments include: Liquid Biopsy Technologies Liquid biopsy technologies involve analyzing biomarkers from bodily fluids to detect and monitor cancers, offering a non-invasive alternative to traditional biopsies. However, at Worldwide, we have a proven track record of successfully utilizing biomarkers in oncology development.

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The Premier Consulting Blog

JULY 9, 2023

Assessment and control of DNA reactive impurities Another important guidance document related to impurities and degradants in new small molecule drugs is ICH M7(R1). What sets this guidance apart from ICH Q3A and ICH Q3B is that it applies to all stages of clinical development, not just to approval. link] ICH M7 (R2)- Q&A.

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