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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Codon

NOVEMBER 3, 2024

Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. But even now, more than a century later, TB remains the deadliest infectious disease on Earth, killing about 1.2 million people every year. tuberculosis.

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Vaccine Trials Therapies Clinical Trials 92

Codon

AUGUST 27, 2024

Allison Berke makes the case for real-time DNA sequencing and AI tools to detect pathogens before they spread widely. Doctors in training are told that when they hear hoofbeats, they should think horses, not zebras; rare diseases are the exception, not the rule. After copying the DNA to form a big pool, each piece is sequenced.

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DNA Laboratories Hospitals Virus 93

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.

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RNA Medical Schools Engineering DNA 98

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.” . PLYMOUTH MEETING, Pa. , mg and 2.0

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DNA Vaccine Clinical Trials Immune Response 40

Drug Target Review

OCTOBER 4, 2023

Antibody-drug conjugates (ADCs) have been a groundbreaking approach to cancer treatment with their ability to deliver cytotoxic drugs directly to diseased cells while sparing healthy tissues. 2 In response, DNA-damaging agents that could target the entire cell cycle received renewed attention as ADC payloads. Chang, P. &

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Small Molecule Drugs Immune Response Targeted Protein Degradation 126

Drug Target Review

JANUARY 15, 2024

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. This produces a great advantage in infectious disease, as it becomes difficult for a virus to adapt and spread from person to person, because each person’s HLA array is genetically distinct.

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Engineering DNA Virus Immune Response 110

Drug Target Review

NOVEMBER 6, 2023

Advances in disease understanding and combination strategies in haematology We now know that blood cancers are characterised by molecular and phenotypic heterogeneity. 2 It is this complexity that necessitates powerful, targeted combination therapies.

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Small Molecule Therapies Treatment Disease 111

Drug Target Review

JUNE 9, 2023

C ould you explain the specific mechanisms or techniques used in the pre-clinical research to translate the genetics of the adaptive immune system into clinical products for disease detection and treatment? What types of diseases or conditions were targeted in the pre-clinical research phase? a healthcare informatics company.

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Treatment Packaging Clinical Research Therapies 105

Codon

SEPTEMBER 29, 2024

The earliest ones relied on simple linear regression and attempted to correlate genetic variations with observable traits or disease risks — such as drug metabolization rates or cancer susceptibility. Over the decade, entire categories of diseases disappeared. 2024 Statistical models of organisms have existed for decades.

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Engineering Virus DNA Therapies 137

PLOS: DNA Science

OCTOBER 3, 2024

One of the oldest and most successful immunotherapies is simpler: a tamed version of a classic vaccine, against the infectious disease tuberculosis (TB). Their report appears in Disease Models and Mechanisms. Eventually, a strain emerged that seemingly protected against a host developing the associated infectious lung disease.

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DrugBank

MAY 15, 2024

Today, we're able to identify and target specific molecules involved in disease processes—a method that's much more like using a sniper rifle than throwing darts blindfolded. Among these targets are proteins, receptors, and enzymes that are fundamental to disease mechanisms.

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Small Molecule Molecular Biology Drugs Disease 81

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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Regulations Research DNA Immune Response 98

PLOS: DNA Science

SEPTEMBER 5, 2024

This removes the cancer’s blocking of the immune response, so that T cells can fight the cancer. Keytruda’s classification based on mechanism, rather than its structure, is immune checkpoint inhibitor. Analyzing DNA patterns using AI may replace time-consuming process DNA and genomic sequencing tests, Yu said.

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Treatment FDA Approval DNA Small Molecule 71

Codon

FEBRUARY 13, 2024

alone, according to the Centers for Disease Control and Prevention. Such contaminants could have the same toxic effects as the bacterial infection, or provoke a severe immune response in the patient, and therefore the phage mixtures must go through extensive purification and testing procedures.

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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. This approach can be highly targeted, with the potential to correct mutations that lead to specific diseases or deliver therapeutic genes to improve heart function.

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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.

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Regulations Clinical Trials Engineering Science 91

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.

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DNA Immune Response Therapies Treatment 52

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.

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Engineering Virus Therapies Immune Response 105

PLOS: DNA Science

JANUARY 4, 2024

The end-of-year FDA approval of the first CRISPR-based therapy , for sickle cell disease, came a mere dozen years after Jennifer Doudna and Emmanuelle Charpentier introduced the technology. The microbes deploy them to dismantle the genetic material of infecting viruses, a little like an immune response. Perhaps one of these.

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FDA Approval DNA Therapies Clinical Trials 52

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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Science Clinical Development Treatment DNA 105

Drug Target Review

DECEMBER 13, 2024

These types differ in their etiology, natural history, and present distinct challenges in disease management. 7,8 The discovery of these biomarkers provides valuable insights into prognosis and disease progression, while also guiding the clinical development of new targeted immunotherapies. percent of total cancer fatalities.

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Therapies Treatment Clinical Trials Drugs 98

Codon

DECEMBER 12, 2024

DNA and RNA molecules are also built from exclusively right-handed nucleic acids. Across the tree of life, organisms strictly require exactly one of the two chiral forms of their molecular building blocks — amino acids, nucleotides of RNA and DNA. 4 As far as we know, right-handed proteins never occur naturally.

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DNA RNA Vaccine Virus 102

The Pharma Data

JANUARY 18, 2021

Next Generation AAVs enhance gene therapies by increasing transduction efficiency and specificity while reducing immune responses and cost. Our ATHENA platform is designed to select the optimal AAV vector for different cells and diseases,” said Daozhan Yu, Ph.D., WUHAN, China and NEWARK, DE. ,

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Drug Target Review

DECEMBER 5, 2024

2 Since then, the FDA has approved four more AAV-based gene therapies—Zolgensma, Hemgenix, Elevidys and Rocktavian—for treating various diseases. 2,3 Engineering both the capsid and DNA cargo improves the safety and efficacy of rAAV vectors and expands their clinical applications. Signal Transduct Target Ther. 2024;9(1):78.

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Therapies Virus DNA Compliance 52

The Pharma Data

MAY 15, 2023

Through the acquisition, Gilead gains rights to a portfolio of small molecule inhibitors targeting PARP1 for oncology and MK2 for inflammatory diseases that could enter clinical trials later this year. Executive Vice President, Research, Gilead Sciences. “The Executive Vice President, Research, Gilead Sciences. Qing Dong, Ph.D.,

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Small Molecule DNA Immune Response Science 40

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. ” Presentation number: 397P / Poster: ID 680. Additional information can be found at www.obipharma.com. Forward-Looking Statements.

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Vaccine Small Molecule Immune Response Hospitals 52

The Pharma Data

JANUARY 20, 2021

Nasdaq: INO), a biotechnology company focused on bringing to market precisely designed DNA medicines to treat and protect people from infectious diseases, including COVID-19, cancer and HPV-associated diseases, today announced the pricing of an underwritten public offering of 17,700,000 shares of its common stock at a public offering price of $8.50

DNA 40

DNA Immune Response Disease Clinical Trials 40

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.

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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.

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Vaccine DNA Engineering RNA 52

Codon

JULY 27, 2023

CNGA3-achromatopsia is a hereditary disease that causes people to see in shades of gray. Their cone cells, which are responsible for color vision, don’t work like normal. They’re talking about a patient who has a rare genetic disease. Transcription factors are proteins that bind to DNA and control gene expression.

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Therapies RNA DNA Engineering 52

Codon

JULY 27, 2023

CNGA3-achromatopsia is a hereditary disease that causes people to see in shades of gray. Their cone cells, which are responsible for color vision, don’t work like normal. They’re talking about a patient who has a rare genetic disease. Transcription factors are proteins that bind to DNA and control gene expression.

Therapies 52

Therapies RNA DNA Engineering 52

The Pharma Data

DECEMBER 20, 2020

Pfizer and BioNTech announced additional data on neutralizing antibody and T-cell responses from their Phase I/II trial of their COVID-19 vaccine conducted in Germany. GM1 gangliosidosis is a life-limiting disease caused by GLB1 gene mutations that impair beta-galactosidase enzyme activity.

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Dark Matter Blog

JULY 16, 2021

As soon as I learned about DNA and RNA, I wanted to be a molecular biologist. Last stops at RNA My last roles in biotech were where my original passion began: DNA and RNA. Delivering the IFN-β gene into tumors caused cancer cell apoptosis and activated both innate and adaptive anti-tumor immune responses.

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Small Molecule RNA Clinical Trials DNA 52

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.

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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.

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Vaccine Pharmaceuticals Small Molecule Virus 40

The Pharma Data

NOVEMBER 29, 2020

Early interim data show Cytomegalovirus (CMV)-negative kidney transplant recipients vaccinated with three doses of HB-101 had reduced incidence of CMV viremia, reduced antiviral use and no CMV disease. Observed CMV-neutralizing antibody responses and tolerability profile are consistent with previous interim results.

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