Drug Target Review

NOVEMBER 6, 2023

Advances in research and development and the utilisation of combination treatment strategies are revolutionising the haematology care landscape, but more must be done to address patients’ individual needs. 2 It is this complexity that necessitates powerful, targeted combination therapies.

Small Molecule 111

Small Molecule Therapies Treatment Disease 111

The Pharma Data

DECEMBER 23, 2020

” Dr. Stanley Plotkin , Professor Emeritus at The Wistar Institute, said, “INOVIO’s DNA vaccine appeared to be quite safe with few significant reactions but yet induced both antibody and T cell responses to SARS-CoV-2.” About INOVIO’s DNA Medicines Platform. mg and 2.0

DNA 40

DNA Vaccine Clinical Trials Immune Response 40

Drug Target Review

JUNE 6, 2023

In this IO arena, we continue to search for additional treatments that can further benefit patients. Generally, IO has been focused on harnessing the anti-tumour activity of certain cancer-fighting T-cells , a key cell type involved in the adaptive immune defense system.

Engineering 98

Engineering Immune Response Biochemical Pharmacology Clinical Trials 98

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.

Therapies 101

Therapies FDA Clinical Trials Disease 101

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.

Science 106

Science Clinical Development Treatment DNA 106

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).

DNA 52

DNA Drugs Therapies Regulations 52

Conversations in Drug Development Trends

JUNE 21, 2024

Worldwide Clinical Trials attends each year to hear from the research community, connect with our sponsors and sites, and explore potential partnerships to drive forward novel treatments. Currently, there are 12 approved ADC drugs, and several hundred more are in the development pipeline.

Clinical Trials 64

Clinical Trials Clinical Research Therapies Immune Response 64

PerkinElmer

MAY 6, 2022

Coined immunotherapy, this umbrella term describes several types of treatment, including immune checkpoint inhibitors, immune system modulators, and adoptive cell therapies. Each triggers the immune system in a different way to fight cancer.

Immune Response 52

Immune Response DNA Clinical Trials Clinical Development 52

The Pharma Data

JANUARY 18, 2021

Next Generation AAVs enhance gene therapies by increasing transduction efficiency and specificity while reducing immune responses and cost. Both parties aim to build a strategic partnership for long term success which will bring accessible AAV gene therapy treatment to patients around the world.”

Therapies 52

Therapies Immune Response DNA Treatment 52

The Pharma Data

NOVEMBER 18, 2020

.” Ming-Tain Lai, PhD, Chief Scientific Officer at OBI Pharma stated, “In the trial, OBI-833 demonstrated a favorable safety profile and generated detectable anti-Globo H IgM/IgG responses. Title: OBI-833 was safe and immunogenic, without treatment-related SAEs, in a Phase 1 dose-escalation trial. 9:00 – 20:00 Eastern Time.

Vaccine 52

Vaccine Small Molecule Immune Response Hospitals 52

The Pharma Data

MAY 15, 2023

The team at XinThera has developed research assets with the potential to target the DNA damage repair pathway in treating cancer and direct the body’s immune response in inflammatory diseases, both of which may improve outcomes for people living with these diseases,” said Flavius Martin, M.D.,

Small Molecule 40

Small Molecule DNA Immune Response Science 40

Drug Target Review

JANUARY 9, 2024

Dr Levin explained: “It is fascinating and completely unexpected that normal patient tracheal cells, without modifying their DNA, can move on their own and encourage neuron growth across a region of damage.” He continued: “We’re now looking at how the healing mechanism works, and asking what else these constructs can do.”

Laboratories 59

Laboratories Immune Response DNA Science 59

LifeSciVC

MARCH 1, 2023

Learning from Past Failures to Drive Toward a More Durable Immune Response Following the initial clinical successes of blocking inhibitory receptors, like CTLA4 and PD1, many in the immuno-oncology field explored the potential to further enhance anti-tumor immunity by simultaneously enhancing co-stimulatory pathways.

Small Molecule 52

Small Molecule Immune Response DNA Therapies 52

The Pharma Data

OCTOBER 14, 2020

(Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced that the first participant was enrolled in the observational PRECISION study (TNX-C002), to examine the immune responses to COVID-19 in healthy volunteers who have recovered from COVID-19 or were asymptomatic.

Vaccine 40

Vaccine Pharmaceuticals Small Molecule Virus 40

The Pharma Data

DECEMBER 27, 2020

Notably, we never modify the DNA of these cells, which avoids some of the safety concerns that have been reported with certain gene-editing technologies. From these cells we apply proprietary methods to manufacture pure populations of only the cell types which we wish to use in patients.

Therapies 40

Therapies Clinical Trials Production Treatment 40

Broad Institute

JUNE 27, 2024

Neurons were classified by software as either positive for prion protein expression (yellow) before treatment with CHARM (left panel), or negative (purple) after treatment (right panel). Like Cas9, ZFPs can serve as guide proteins to direct the tool to a target site in DNA. Credit: Neumann EN, Bertozzi TM, et al.

Disease 142

Disease Therapies Clinical Trials Engineering 142

Broad Institute

OCTOBER 11, 2023

Manguso, who’d recently graduated from college and was conducting research at the University of Copenhagen as a Fulbright scholar, moved back to the Boston area to be with his mother as she underwent treatment. His mother had a presentation of the disease that suggested her immune system was already on the job.

Research 137

Research Immune Response Clinical Trials Therapies 137

Drug Target Review

JANUARY 15, 2024

Monoclonal antibodies (mAbs) targeting tumour-specific antigens play an essential role in the treatment of many cancers. Unlike almost every other cell type (except B cells), T cells do not have the exact same chromosomal DNA sequences as other cells in the body. Many potential candidate antigens are under consideration.

Engineering 110

Engineering DNA Virus Medical Schools 110

PLOS: DNA Science

OCTOBER 3, 2024

“BCG” is the “treatment” vaccine’s technical name, for Mycobacterium bovis Bacillus Calmette-Guérin. ’” Coaxing BCG to Tame TB and then Cancer The novel vaccine strategy builds on the accumulation of natural mutations when DNA copies itself as cells divide.

Vaccine 52

Vaccine Immune Response DNA Treatment 52

PLOS: DNA Science

SEPTEMBER 5, 2024

Diagnosis began to shift from a body-part basis to a molecular one, which I wrote about in “Mutation and location important in cancer treatment” for The Lancet in 2015. This removes the cancer’s blocking of the immune response, so that T cells can fight the cancer.

Treatment 65

Treatment FDA Approval DNA Small Molecule 65

Codon

FEBRUARY 13, 2024

doi: 10.2210/rcsb_pdb/goodsell-gallery-048 The Virus that Cures It’s been over 25 years since the science magazine Discover first ran an extraordinary article about how a long-forgotten medical treatment, used in the former Soviet country of Georgia, could save us from the growing threat of untreatable, drug-resistant infections.

Therapies 138

Therapies Virus DNA Clinical Trials 138

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. By correcting these mutations, gene-modifying and editing therapies hold the promise of long-term, potentially curative treatments.

Therapies 98

Therapies Disease RNA Engineering 98

Drug Target Review

MAY 14, 2024

Since cancer cells are under chronic stimulation of ER stress, the polypeptides cannot activate innate immune sensors in cancer cells even upon the polypeptide treatment. Which natural agonists impede the activation of intracellular DNA sensors in APCs, and what challenges do they pose?

Engineering 59

Engineering Immune Response FDA Approval Clinical Trials 59

The Pharma Data

APRIL 8, 2021

Leadership in DNA damage response demonstrated in multiple presentations for AZD5305, a next-generation PARP1 selective inhibitor. Data for AZD5305 will demonstrate how the next wave of DNA damage response medicines can build on the success of PARP inhibitors, potentially allowing patients to stay on treatment longer.

DNA 52

DNA Immune Response Therapies Treatment 52

PLOS: DNA Science

OCTOBER 12, 2023

Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases. Can they deliver healing genes without triggering an overactive immune response? million DNA bases. The implicated gene is the largest in a human genome, with 2.2

Therapies 69

Therapies Immune Response DNA Virus 69

Broad Institute

AUGUST 7, 2023

Related people Paul Blainey Nir Hacohen A signaling protein known as STING is a critical player in the human immune system, detecting signs of danger within cells and then activating a variety of defense mechanisms.

Regulations 91

Regulations Clinical Trials Engineering Science 91

Drug Target Review

OCTOBER 26, 2023

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. 1 Adeno-associated virus (AAV) vectors are the leading platform for in vivo gene delivery for the treatment of various human diseases.

Engineering 105

Engineering Virus Therapies Immune Response 105

The Pharma Data

JUNE 30, 2021

The EC’s decision is based on results from the Phase 2 CheckMate -142 trial in which Opdivo plus Yervoy demonstrated a clinically meaningful improvement in objective response rate (ORR) in patients with MSI-H/dMMR mCRC who received prior treatment with fluoropyridine, oxaliplatin and irinotecan. achieving a complete response.

Treatment 52

Treatment Trials FDA Approval Disease 52

PLOS: DNA Science

JANUARY 4, 2024

.” CRISPRs are short DNA sequences, peppered with repeats, that latch onto DNA-cutting enzymes, commandeering and directing them to snip certain parts of a chromosome. The microbes deploy them to dismantle the genetic material of infecting viruses, a little like an immune response. Perhaps one of these.

FDA Approval 52

FDA Approval DNA Therapies Clinical Trials 52

DrugBank

FEBRUARY 26, 2025

However, bacteria are evolving resistance to even the most potent antibiotics, rendering many treatments ineffective. Multidrug-resistant (MDR) bacteria exacerbate the problem by evading multiple classes of antibiotics, leaving healthcare providers with few or no treatment options.

Therapies 52

Therapies Pharmaceutical Companies Pharmacokinetics Treatment 52

Drug Target Review

APRIL 26, 2023

Gene-editing methods allow researchers to modify DNA, resulting in alterations in physical characteristics such as eye colour and susceptibility to diseases. To achieve this, scientists utilise various technologies that function similarly to scissors, precisely cutting the DNA at a particular location.

RNA 98

RNA Medical Schools Engineering Disease 98

Codon

APRIL 23, 2023

Unfortunately, they can also trigger immune responses, and they are not super efficient at gene-editing some parts of the brain. These ribonucleoproteins also caused a lower immune response compared to the viruses, and are much easier to make in the lab. Image from Everette K.A. Nature Communications.

Vaccine 52

Vaccine DNA Virus Engineering 52

Codon

APRIL 23, 2023

Unfortunately, they can also trigger immune responses, and they are not super efficient at gene-editing some parts of the brain. These ribonucleoproteins also caused a lower immune response compared to the viruses, and are much easier to make in the lab. Image from Everette K.A. Nature Communications.

Vaccine 52

Vaccine DNA Virus Engineering 52

The Pharma Data

AUGUST 3, 2021

The FDA has broadened the Emergency Use Authorization (EUA) for baricitinib to allow for treatment with or without remdesivir. The European Commission granted marketing authorization for Jardiance ® as a treatment for adults with symptomatic chronic heart failure with reduced ejection fraction (systolic heart failure).

Production 52

Production Marketing Research Government 52

Codon

APRIL 30, 2023

Yeast die for two reasons: Either their nucleolus (where the DNA is kept) degrades and dies, or their mitochondria whimpers out and they stop making energy. The vaccine printer can make lots of different types of vaccines, including protein, DNA, and mRNA ones, but I’m sure this is all quite expensive right now. From Zhang et al.

Vaccine 52

Vaccine DNA Engineering RNA 52

Codon

APRIL 30, 2023

Yeast die for two reasons: Either their nucleolus (where the DNA is kept) degrades and dies, or their mitochondria whimpers out and they stop making energy. The vaccine printer can make lots of different types of vaccines, including protein, DNA, and mRNA ones, but I’m sure this is all quite expensive right now. From Zhang et al.

Vaccine 52

Vaccine DNA Engineering RNA 52

The Pharma Data

APRIL 7, 2023

1 Over two thirds of patients are diagnosed with advanced disease which can progress quickly, often within two years, diminishing their quality of life despite treatment. DUO-O enrolled over 1200 patients across all treatment arms at 179 study locations. Ovarian cancer is one of the most common gynaecologic cancers.

Trials 40

Trials DNA Treatment Therapies 40