Drug Discovery Today

OCTOBER 19, 2020

Former Lonza President of Biologics Manufacturing joins to continue rapid expansion of DNA contract manufacturing; Touchlight’s synthetic dbDNA in high demand as starting material in the manufacture of advanced therapies such as mRNA vaccines and cell and gene therapies

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Codon

FEBRUARY 13, 2024

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.

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Broad Institute

JUNE 27, 2024

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain.

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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. While iPSC therapies hold great promise, several challenges remain, including poor infiltration of cytotoxic lymphocytes into solid tumours and insufficient cytotoxicity of myeloid cells.

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Broad Institute

JUNE 28, 2023

Merkin Prize in Biomedical Technology for developing an efficient, automated technology for synthesizing DNA. The chemical reactions that he discovered in the early 1980s to accurately and quickly assemble nucleotides into strands of DNA provided an essential element in the development of modern molecular medicine.

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Drug Discovery Today

FEBRUARY 24, 2022

Global cell and gene therapy development and manufacturing business expands into Bruntwood SciTech’s Alderley Park to boost manufacturing capacity.The 17,000 sq ft high-specification lab space is currently under construction, expanding plasmid DNA and viral vector capabilities in Europe.The facility will support therapeutic developers with rapid access (..)

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Drug Target Review

JULY 3, 2023

Dr David Baram from gene therapy company EmendoBio provides a snapshot of the history of therapeutic genetic engineering, explaining the early pitfalls and reasons for recent renewed optimism.

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ASPET

JUNE 30, 2023

Significance Statement Drugs that target cellular DNA damage repair pathways are now being used as new ways to maximise the effect of multimodality treatments such as radiotherapy. Here, we develop a mathematical model to investigate the effects of ceralasertib and olaparib, two drugs that target DNA damage response pathways.

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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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PLOS: DNA Science

MARCH 21, 2024

The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). The history of gene therapy for MLD is compelling – DNA Science covered it for Rare Disease Day in 2021, here. She passed away in 2013.

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Fierce BioTech

NOVEMBER 1, 2023

Plasmid DNA Design Considerations for Cell and Gene Therapy Cell and Gene therapy organizations must balance the need for rapid clinical progression and stringent quality expectations throughout development.

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DrugBank

JUNE 13, 2024

  Gene Therapy: Reprogramming the Body's Cellular Code    Gene therapy is an exciting field that treats diseases at their genetic roots. This approach targets the fundamental instructions within a cell's DNA, either by correcting faulty genes or introducing entirely new ones to combat disease.

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Drug Target Review

OCTOBER 2, 2024

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.

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Drug Discovery Today

SEPTEMBER 16, 2021

Demand for Touchlight’s synthetic DNA vector has grown rapidly since last year.dbDNA™ is driving biomedical innovations such as DNA and mRNA vaccines, synthetic DNA-based neutralising antibody therapies, and more.Touchlight’s synthetic DNA vector is revolutionising genetic medicine manufacture by offering a superior alternative to traditional plasmid (..)

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DNA Vaccine Therapies 113

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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Drug Target Review

JULY 3, 2023

Gene editing, one of the most promising technologies in the field of medicine, involves modifying genes within living cells by inserting, replacing, or deleting specific DNA sequences to correct disease-causing genetic mutations.

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Disease DNA Therapies Treatment 98

Chemical Biology and Drug Design

SEPTEMBER 18, 2023

The presence of partially occupied water molecules in the layered organization in the crystal structure mimics the release of reactive oxygen species (ROS) for DNA damage resulting in cytotoxicity.

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ASPET

SEPTEMBER 16, 2024

Standard of care GBM therapies include radiation and cytotoxic chemotherapy that lead to DNA damage. Subsequent activation of DNA damage response (DDR) pathways can induce resistance. Elimusertib, a novel ATR kinase inhibitor, can prevent repair of damaged DNA, increasing efficacy of DNA damaging cytotoxic therapies.

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Drug Target Review

DECEMBER 13, 2024

2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates. These can include anti-tubulin compounds, potentially reducing side effects and improving quality of life.

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Chemical Biology and Drug Design

NOVEMBER 23, 2023

Nowadays, gene therapy is being implemented for its treatment as several of these genetic defects have been identified. There are challenges in delivering the gene/DNA to the site of action in the brain, and scientists are working to resolve the same. Scientists are trying to use viral vectors, miRNA, siRNA, and CRISPR technology.

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Broad Institute

OCTOBER 23, 2024

In recent years, genetic editing technologies and other gene therapy approaches have given scientists the ability to alter the genes inside living cells. That is in part because of the ongoing challenge of understanding the DNA switches, called cis-regulatory elements (CREs), that control the expression and repression of genes.

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Chemical Biology and Drug Design

SEPTEMBER 16, 2024

This study identified MAPK9 as the directly binding target of baicalein by the DNA-programmed affinity labeling method and tested the binding affinity as 89.7 nM Baicalein stabilized MAPK9 to induce apoptosis in hepatocarcinoma cells, which would be helpful to understand and use baicalein in hepatocarcinoma therapy.

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Drug Discovery Today

JULY 7, 2022

Pfizer and Touchlight agree to patent license for Pfizer to utilise rapid, scalable, enzymatic doggybone DNA (dbDNA) in Pfizer’s clinical and commercial manufacture of its mRNA vaccines, therapeutics, and gene therapiesAgreement includes upfront payment, potential development and commercial milestone payments, and royalties upon commercializationAccess (..)

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Drug Target Review

JULY 7, 2023

The reasons for this are multifaceted, including concerns over the safety of directly altering DNA sequences and subsequent regulatory restrictions that have arisen as a result. The epigenome (meaning ‘above the genome’) is a system of reversible marks regulating how the DNA is read, translated and used. What is epigenetic editing?

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DNA Engineering Therapies Regulations 105

Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. Hearing aids also have all sorts of deficits.

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PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. But even if gene therapy could reach sufficient muscle cells to improve quality of life, another hurdle looms: the dystrophin gene that’s deleted in DMD is huge, the largest in a human genome at 2.2

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PLOS: DNA Science

OCTOBER 12, 2023

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

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ASPET

MARCH 20, 2024

DNA topoisomerase IIβ (TOP2β/180; 180 kDa) is a nuclear enzyme that regulates DNA topology by generation of short-lived DNA double-strand breaks primarily during transcription. TOP2β/180 can be a target for DNA damage-stabilizing anticancer drugs, whose efficacy is often limited by chemoresistance.

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DNA Laboratories Therapies Regulations 100

KIF1A

APRIL 6, 2024

When a mutation causes a deletion or duplication of larger areas of DNA, we call it Copy Number Variation. DNA deletion and duplication may impact large sections of genes, or multiple neighboring genes. The altered expression of so many genes has profound consequences for the nervous system. She graduated from UC San Diego with a B.S.

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Drug Target Review

MAY 22, 2024

What are the key findings of Circio’s in vivo proof-of-concept for its circVec circular RNA platform technology compared to conventional mRNA-based expression with DNA vectors? How does Circio’s circVec technology aim to enhance the potency and reduce the cost of current gold-standard gene therapy? DNA vectors in mouse models?

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DrugBaron

JANUARY 8, 2021

Yesterday, Sarpeta (NASDAQ: $SRPT) announced that its gene therapy for Duchenne Muscular Dystrophy failed to improve muscle function in a study of 40 boys, despite achieving impressive expression of micro-dystrophin (at least in the short term). Surely, then, a correct set of DNA plans will ensure a correctly functioning cell?

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SCIENMAG: Medicine & Health

SEPTEMBER 14, 2023

million from the National Institutes of Health to study how unique DNA circles found in cancer cells drive deadly pediatric brain tumors. The research aims to expose how the circular pieces of DNA contribute to therapy resistance and open new therapeutic avenues to improve the lives of people affected […] has received $3.2

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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. We’ve miniaturised the chemical process to making DNA and synthesize or ‘write’ DNA on a silicon chip.

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Chemical Biology and Drug Design

APRIL 3, 2023

As a life-threatening disease, it necessitates the emergence of new therapies. One of the five-membered aromatic nitrogen heterocycles identified in American therapies that have been approved by the Food and Drug Administration (FDA). Abstract Cancer is the second leading cause of death globally, with 9.6 million million deaths yearly.

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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. Gene therapy trials explore using lentiviral vectors to deliver a functional copy of the HBB gene to hematopoietic stem cells, the progenitors of all blood cells.

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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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Broad Institute

NOVEMBER 13, 2023

Liquid biopsies enable clinicians to find and analyze tumor DNA in a patient’s blood sample to detect cancer early, monitor cancer recurrence, assess the patient’s response to treatment, and measure other clinically important features in real time, without invasive procedures.

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