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.

DNA

Drug Target Review

APRIL 17, 2024

HLA are the critical molecules that our immune systems use to present peptides to T cells and facilitate recognition and killing in immune responses to pathogens. HLA-II presents peptides to CD4 T cells thought to be important for indirectly helping immune responses and facilitating antibody production.

Immune Response

DrugBank

MARCH 12, 2025

Once delivered into the body, the mRNA instructs cells to produce these antigens, which are then presented to the immune system. This approach can be personalized for each patient by sequencing the tumor's DNA and RNA to identify unique mutations.

Vaccine

Drug Target Review

SEPTEMBER 8, 2023

The cell’s nucleus, containing compacted DNA, is centrally positioned within the cell. It didn’t have the tools that are important for nuclear receptors, namely the ability to interact with DNA and the ability to translocate from the cytoplasm to the nucleus,” says Cheroutre.

Regulations

DrugBank

JANUARY 29, 2025

Gene Therapy Gene therapy operates on the principle of modulating the DNA blueprint of cells to induce a therapeutic response. These disorders, often characterized by a loss-of-function mutation in a crucial gene, can manifest with a wide range of clinical presentations, depending on the specific gene and its role in cellular physiology.

Therapies

New Drug Approvals

SEPTEMBER 6, 2024

TLR7-9 are among the set that are endosomally located and respond to single-stranded RNA (TLR7and TLR8) or unmethylated single-stranded DNA containing cytosine-phosphate-guanine (CpG) motifs (TLR9). Toll/IL-1 receptor family members are important regulators of inflammation and host resistance. Nature Immunol., 11:373-384 (2010)).

RNA

The Pharma Data

OCTOBER 21, 2020

These findings were presented today at the 2020 Infectious Diseases Society of America (IDSA) IDWeek. Participants identified as impacted by COVID-19 will continue to be followed and data collected from that group is expected to be presented at a future medical meeting. Kimberly Smith, M.D.,

Clinical Development

The Pharma Data

SEPTEMBER 1, 2020

This can help healthcare professionals identify a SARS-CoV-2 infection in people suspected to carry the virus with results typically ready in 15 minutes. The SARS-CoV-2 Rapid Antigen Test is for use in point of care settings for both symptomatic and asymptomatic people.

Virus

The Pharma Data

OCTOBER 14, 2020

The research is part of an ongoing collaboration between Columbia University and Tonix that focuses on T cell and antibody responses to SARS-CoV-2 (CoV-2), the virus that causes COVID-19. The research encompasses two projects. The study led by Dr. Ilya Trakht, Ph.D., The study led by Dr. Sergei Rudchenko, Ph.D., About TNX-1800.

Vaccine

Elrig

FEBRUARY 23, 2023

You’ll get an overview of this info-packed event, the lowdown on why you really can’t afford to miss it , and a sneak peek at the presentation topics from our keynote speakers: Altos Labs’ Dr Ken Raj and Dr Ron Weiss of the Massachusetts Institute of Technology (MIT). You could even get the chance to present in the main speaker sessions.

Research

Drug Target Review

JANUARY 15, 2024

An important limitation of antibodies against tumour antigens is that these agents direct responses to molecular targets present on the surface of the cell. Figure 1: HLA Class I presentation of peptides derived from intracellular antigens. The molecular complex of HLA plus an antigenic peptide is sometimes referred to as “pHLA”.

Engineering

Drug Target Review

JANUARY 26, 2024

These ACE2-coated particles could be tested as an anti-viral therapeutic, and as sensors for detecting virus particles. To find a DNA fragment that contained the intact ACE2 gene, complete with its embedded regulatory information, they searched libraries of cloned DNA fragments generated as part of the Human Genome Project.

Production

Codon

JUNE 25, 2024

However, they plod along as they clone plasmids—the loops of DNA that biologists use to manipulate and study organisms—because propagating them relies, in part, on the pace at which cells grow and divide. Most medicines, including insulin and semaglutide (the weight loss drug), are made using DNA cloning. However, E.

DNA

PLOS: DNA Science

APRIL 20, 2023

DNA is a sleek double helix, with “rungs” consisting of a purine base paired with a smaller pyrimidine base: adenine (A) with thymine (T) and guanine (G) with cytosine (C). . ” DNA encodes amino acid sequences comprising proteins, which impart traits. Watson and F.

DNA

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

PLOS: DNA Science

JUNE 13, 2024

The virus SARS-CoV-2 emerged in Wuhan, home of a premiere research institute that focuses on viruses. Experiments at the Wuhan facility were done at the lowest two of the four standard biosafety levels, which were established at the dawn of recombinant DNA research in the 1970s. An altered virus could have escaped. Coincidence?

Virus

PLOS: DNA Science

AUGUST 22, 2024

The Genetic Explanation Doesn’t Work The narrative returns to the present. ” The explanation continues, and here’s where it ceases to make sense, to anyone who knows anything about DNA, genes, and genomes. A plant genome consists of DNA, and NOT pieces of 2,4-D, which is a completely unrelated organic acid.

DNA

The Pharma Data

SEPTEMBER 28, 2021

Acute respiratory infections are one of the leading causes of death and disability globally, presenting substantial health challenges especially for infants, the elderly and the immunocompromised. TaqMan® DNA probes that hybridise with the target strand are labeled with a fluorescein allowing real-time measurements during the PCR process.

Marketing

Codon

APRIL 23, 2023

This newsletter’s format has shifted again, as I try to figure out the most enticing way to present this stuff. Most people would take the two CRISPR gene-editing components (a Cas9 protein and guide RNA), package them up inside of a virus, and then inject the viruses into the skulls of mice. Please send me your feedback.

Vaccine

Codon

APRIL 23, 2023

This newsletter’s format has shifted again, as I try to figure out the most enticing way to present this stuff. Most people would take the two CRISPR gene-editing components (a Cas9 protein and guide RNA), package them up inside of a virus, and then inject the viruses into the skulls of mice. Please send me your feedback.

Vaccine

Drug Target Review

OCTOBER 2, 2024

For example, integrating gene therapies that alter the DNA of cardiac cells pose risks of unintended genetic modifications, which could lead to tumorigenesis. Still, the high cost of CGTs, driven by their complexity and individualised nature, presents barriers to widespread adoption in cardiology, limiting patient access.

Therapies

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? DNA vectors in mouse models? Circular RNA (circRNA) has two major advantages versus mRNA in a vector-expression context.

Therapies

Codon

APRIL 2, 2023

But now, by studying DNA extracted from microbes in the blood of almost 10,000 healthy people, this paper shows that there is no such thing. Read Transcription factors bind to DNA and control gene expression. Read Switchable hydrophobic pockets in DNA protocells enhance chemical conversion. Nature Microbiology. Meeussen J.V.W.

DNA

The Pharma Data

AUGUST 4, 2020

Accurate monitoring of Epstein-Barr virus DNA levels is critical, as they are associated with a range of life threatening-diseases including cancer in transplant patients. This is the first quantitative in vitro diagnostic test for Epstein-Barr virus (EBV) DNA in the United States.

Virus

PLOS: DNA Science

OCTOBER 12, 2023

million DNA bases. The DMD gene therapy delivers a shortened version of the dystrophin gene, just 4,558 DNA bases. First, adeno-associated virus (AAV) delivers the genes, rather than the adenovirus (AV) that had entered cells in Jesse’s liver that weren’t the targets. Two other design strategies add precision.

Therapies

The Pharma Data

DECEMBER 3, 2020

Consequently, performing an immunoassay in the early stages of an infection will be negative because the antibodies cannot be produced yet, even though the disease is present. The amount of DNA produced is much higher, and a positive test result can be viewed visually without the need for an additional analysis step,” the study reported.

RNA

PLOS: DNA Science

FEBRUARY 15, 2024

A Five-Generation Saga The tale alternates between ancestors and the present generation farming the same land. The narrative is a bit confusing because Aki is the only ancestor named, and there’s no family tree to link present-day Jake to the past. But dirt is dead, depleted of life. The soil microbiome and gut microbiome?”

Science

The Pharma Data

JANUARY 21, 2021

Food and Drug Administration (FDA) has approved CABENUVA (consisting of Janssen’s rilpivirine and ViiV Healthcare’s cabotegravir), the first and only once-monthly, long-acting regimen for the treatment of human immunodeficiency virus type 1 (HIV-1) infection in adults.

FDA Approval

The Pharma Data

DECEMBER 5, 2020

The first of the new therapies, developed at Harvard Medical School by a team led by Dr. David Williams, uses a virus to introduce RNA into the extracted bone marrow that turns off BCL11A. The CRISPR gene editing method won the 2020 Nobel Prize in Chemistry, and uses chemicals to open up and directly edit DNA sequences, Hsu said.

Therapies

The Pharma Data

JUNE 30, 2021

Key data on lenacapavir will be presented during the 11th International AIDS Society (IAS) Conference on HIV Science in July 2021. These data were previously presented at the virtual 28th Conference on Retroviruses and Opportunistic Infections ( virtual CROI 2021). log 10 copies/mL vs. -0.29 log 10 copies/mL, p<0.0001).

Treatment

Codon

JULY 27, 2023

They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. “The first printout of the human genome to be presented as a series of books, displayed in the 'Medicine Now' room at the Wellcome Collection, London. It fills up an entire bookcase ! From Ling B et al.

Therapies

Codon

JULY 27, 2023

They’ve just finished sequencing the patient’s genome, but they don’t have “DNA sorting” software. “The first printout of the human genome to be presented as a series of books, displayed in the 'Medicine Now' room at the Wellcome Collection, London. It fills up an entire bookcase ! From Ling B et al.

Therapies

Codon

JUNE 26, 2023

DNA sequences are designed on a computer, and it takes a dozen or more clicks to change a single nucleotide. DNA sequences are also checked by hand, so it’s easy to make a mistake. The tool outputs a DNA sequence that encodes all the required enzymes. A phage is a virus that infects bacteria. Paper by Sha G.

DNA

Codon

JUNE 26, 2023

DNA sequences are designed on a computer, and it takes a dozen or more clicks to change a single nucleotide. DNA sequences are also checked by hand, so it’s easy to make a mistake. The tool outputs a DNA sequence that encodes all the required enzymes. A phage is a virus that infects bacteria. Paper by Sha G.

DNA

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

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

Drug Target Review

DECEMBER 28, 2023

During the process of transformation from a normal cell into a cancer cell, a cell acquires a series of changes, or mutations, in its DNA. But DNA mutations can also result in changes to the proteins that are displayed on the surface of the cancer cell. These mutations are called clonal mutations or clonal neoantigens.

Bioinformatics