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. PRISM consists of two key components.

DNA 52

DNA Drugs Small Molecule FDA Approval 52

DrugBank

OCTOBER 17, 2024

By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.

Drug Development 98

Drug Development Metabolic Stability Clinical Trials Drugs 98

DrugBank

JANUARY 9, 2025

Targeted Therapies vs. Traditional Treatments Traditional cancer treatments, such as chemotherapy and radiotherapy, are inherently cytotoxic, targeting both cancerous and healthy cells. The development of imatinib to treat chronic myeloid leukemia (CML) serves as a hallmark for targeted therapies.

Therapies 52

Therapies Disease Treatment Small Molecule 52

Codon

JUNE 11, 2024

And unlike traditional DNA sequencers, which parse genetic material by breaking it up into fragments and interpreting it chunk-by-chunk, a nanopore device unspools a long strand of DNA and reads it all at once. A scientist can isolate DNA and load up a flow cell in fifteen minutes. Nanopore devices work incredibly fast.

DNA 87

DNA Engineering RNA Small Molecule 87

The Pharma Data

JUNE 28, 2021

Broadens company’s oncology platform of Targeted Alpha Therapies / Acquisition includes actinium-225 labeled differentiated PSMA small molecule for the treatment of prostate cancer. It has been demonstrated to inflict difficult to repair damage to tumor cells by inducing DNA double strand breaks.

Small Molecule 52

Small Molecule Licensing Licensing Therapies 52

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. My last stop at Arrakis Therapeutics is with a company targeting RNA with small molecules. I wanted to use molecular biology to create drugs.

Small Molecule 52

Small Molecule RNA Clinical Trials DNA 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 company was founded in 2021 by Stephen Kaldor, Ph.D., Qing Dong, Ph.D.,

Small Molecule 40

Small Molecule DNA Immune Response Science 40

LifeSciVC

DECEMBER 6, 2022

These hair clip-like molecules clamp onto DNA, thereby turning many genes on and off. Darnell’s 2002 paper Transcription Factors as Targets for Cancer Therapy , in which he wrote: “A limited list of transcription factors are overactive in most human cancer cells, which makes them targets for the development of anticancer drugs.

Small Molecule 40

Small Molecule DNA Regulations Science 40

The Pharma Data

DECEMBER 14, 2020

. The current standard of care for GBM consists of de-bulking surgery followed by combined treatments with fractionated ionizing radiation (IR) and the DNA alkylating agent temozolomide (TMZ). At the stage of GBM relapse and recurrence, no effective therapy strategies currently exist. Contact.

Clinical Trials 52

Clinical Trials DNA Drugs Therapies 52

LifeSciVC

MARCH 1, 2023

Augustin et al summarized how novel platforms have more recently been applied to try to solve this question including CRISPR genome editing combined with adoptive cell therapy, siRNA knockdown, DNA encoded library approaches and machine learning algorithms.

Small Molecule 52

Small Molecule Immune Response DNA Therapies 52

The Pharma Data

NOVEMBER 18, 2020

The company’s novel first-in-class AKR1C3 targeted therapy is OBI-3424 (small-molecule prodrug) that selectively releases a potent DNA alkylating agent in the presence of the aldo-keto reductase 1C3 (AKR1C3) enzyme. Additional information can be found at www.obipharma.com.

Vaccine 52

Vaccine Small Molecule Immune Response Hospitals 52

Alta Sciences

APRIL 17, 2024

Psychedelics Psychedelic therapy (or psychedelic-assisted therapy) refers to the use of psychedelic drugs, such as psilocybin, MDMA, LSD, ketamine, and ayahuasca, to treat mental disorders, especially those that have no effective treatments available or are treatment resistant.

Drug Development 52

Drug Development Treatment FDA Approval Drugs 52

The Pharma Data

MARCH 18, 2022

By manipulating the microbes’ DNA, they programmed gene circuits that control the bacteria surface, building a molecular “cloak” that encapsulates the bacteria. They control the iCAP system by giving it an external cue – a small molecule called IPTG – that allows for programmable and dynamic alteration of the E.

Engineering 52

Engineering Drugs Small Molecule Therapies 52

The Pharma Data

APRIL 11, 2023

Bayer is advancing its oncology R&D efforts in three scientific areas that have the potential to address unmet needs in cancer patients: next-generation Immuno-Oncology, Targeted Radionuclide Therapies and Precision Molecular Oncology. Data from all three areas of scientific focus will be showcased during this year’s meeting.

Research 40

Research Clinical Trials Small Molecule Trials 40

Drug Target Review

MAY 4, 2023

A cure for sickle cell anaemia A recent scientific breakthrough in the treatment of sickle cell anaemia —a genetic disorder marked by episodes of intense pain—illustrates how combining GWAS with cutting-edge molecular tools like gene editing can identify causal variants and lead to innovative therapies.

Disease 52

Disease Small Molecule Protein Expression Research 52

Vial

FEBRUARY 19, 2024

Explore the groundbreaking VersAptx bioconjugation platform, designed to elevate cancer therapies and uncover the delicate balance between clinical excellence and commercial viability in the dynamic landscape of pharmaceuticals. Can you elaborate on how the platform allows your company to tailor therapy? Ahmed Hamdy: Absolutely.

Small Molecule 52

Small Molecule Therapies Treatment Science 52

The Pharma Data

OCTOBER 14, 2020

Assistant Professor of Medical Sciences at Columbia University Vagelos College of Physicians and Surgeons, is designed to generate DNA aptamer-based anti-idiotypes to selected monoclonal antibodies identified in Dr. Ilya Trakht’s study. The study led by Dr. Sergei Rudchenko, Ph.D., 1 Noyce RS, et al. 2018) PLoS One. 13(1):e0188453.

Vaccine 40

Vaccine Pharmaceuticals Small Molecule Virus 40

KIF1A

MAY 31, 2024

Dr. Kaur now oversees 5 KAND research projects that utilize our patient-derived cell lines to screen for small molecule therapeutics and test potential gene therapies. This would allow a single gene therapy to replace an area of KIF1A that includes multiple disease-causing mutations, and treat multiple mutations at once.

Treatment 52

Treatment Small Molecule International Research 52

The Pharma Data

DECEMBER 27, 2020

NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today provided a year-end review and an outline of its plans for 2021. CARLSBAD, Calif.–( –( BUSINESS WIRE )– Lineage Cell Therapeutics, Inc.

Therapies 40

Therapies Clinical Trials Production Treatment 40

New Drug Approvals

APRIL 18, 2023

H]Cl.NCCCCN(CC1=NC2=CC=CC=C2N1)[C@@H]3C4=NC=CC=C4CCC3 AMD-070 is a small molecule drug candidate that belongs to a new investigational class of anti-HIV drugs known as entry (fusion) inhibitors. Molecular Weight 349.47 Appearance Solid Formula C 21 H 27 N 5 CAS No. Appearance Solid Formula C 21 H 30 Cl 3 N 5 CAS No. Skerlj RT, et al.

Clinical Trials 52

Clinical Trials Virus Small Molecule Trials 52

Drug Target Review

APRIL 16, 2024

Can you explain how Radioligand Therapy (RLT) is applicable in the treatment of both chemotherapy-resistant tumours and in naïve patients who have not undergone prior cancer treatment? By delivering radiation directly to the tumour cells, RLT can induce DNA damage, leading to cell death and tumour regression.

Small Molecule 59

Small Molecule Therapies Treatment Pharmaceutical Companies 59

New Drug Approvals

SEPTEMBER 23, 2024

Target: DNA/RNA Synthesis Pathway: Cell Cycle/DNA Damage Huntington’s disease (HD) is a progressive, autosomal dominant neurodegenerative disorder of the brain, having symptoms characterized by involuntary movements, cognitive impairment, and mental deterioration. SCHEME PATENT PTC Therapeutics Inc.,

Small Molecule 57

Small Molecule RNA DNA Disease 57

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 52

DNA RNA Engineering Licensing 52

PLOS: DNA Science

SEPTEMBER 5, 2024

Gleevec is a small molecule that interferes with entry of an enzyme – a tyrosine kinase – that enables growth signals to enter specific cells and trigger division. A name ending in “nib” means a small molecule that inhibits an enzyme called a kinase, and is short for “inhibit.”

Treatment 68

Treatment FDA Approval DNA Small Molecule 68

DrugBank

FEBRUARY 26, 2025

This stagnation has created a perfect storm, threatening the effectiveness of current therapies and limiting the pipeline of new drugs. Targeting Novel Bacterial Pathways Traditional antibiotics often target essential bacterial processes such as cell wall synthesis or DNA replication.

Therapies 52

Therapies Pharmaceutical Companies Pharmacokinetics Treatment 52

The Pharma Data

OCTOBER 14, 2020

Food and Drug Administration granted Breakthrough Therapy designation to France-based Inventiva’s experimental NASH treatment lanifibranor. has expanded its product portfolio to include Blueprint, a self-service, scientific visualization and analytics application for small molecule discovery. Dolmatics – U.K.-based

Clinical Trials 52

Clinical Trials Trials Pharmaceuticals Therapies 52

The Pharma Data

MARCH 7, 2022

The new technique controls gene activity without altering the DNA sequence of the genome by targeting chemical modifications that help package genes in our chromosomes and regulate their activity. In this experiment, however, the cutting function of the Cas9 protein is disabled so the genomic DNA sequence is unaltered.

RNA 52

RNA DNA Packaging Small Molecule 52

Codon

MARCH 19, 2024

Most cells (excluding T-cells and B cells after V(D)J recombination) in our body contain the same DNA but appear and behave in distinct ways: A neuron looks and acts very differently from a hepatocyte. While DNA is relatively stable , the epigenome is not; it has to orchestrate changes in cell state, cell type, and more.

DNA 57

DNA Small Molecule Regulations Laboratories 57

Codon

MARCH 20, 2023

Signal Transduction and Targeted Therapy. Marine biofilm engineered to produce current in response to small molecules. Digital nanoreactors to control absolute stoichiometry and spatiotemporal behavior of DNA receptors within lipid bilayers. CRISPR-induced DNA reorganization for multiplexed nucleic acid detection.

Virus 52

Virus Engineering RNA Small Molecule 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

KIF1A

AUGUST 20, 2024

Susannah is being treated with an Antisense Oligonucleotide (ASO) : To break down the term, this is a molecule designed with a sequence of 20 (oligo, “few”) letters of DNA (nucleotide) that match a sequence on the mutant KIF1A RNA (Antisense) and knock it down. Will this _ KAND?

Science 52

Science Treatment Therapies Small Molecule 52

The Pharma Data

AUGUST 3, 2021

The company announced donanemab received Breakthrough Therapy designation for treatment of Alzheimer’s disease and its intention to submit a biologics license application (BLA) for donanemab under the accelerated approval pathway later this year based on data from TRAILBLAZER-ALZ. Loxo Oncology at Lilly and Kumquat Biosciences Inc.

Production 52

Production Marketing Research DNA 52

The Pharma Data

DECEMBER 21, 2020

Tagrisso was recently granted Breakthrough Therapy Designation for patients in the early-stage disease setting by the US FDA. At two years, 89% of patients treated with Tagrisso remained alive and disease free versus 52% on placebo after surgery, the current standard of care. J Clin Oncol. 2011:29;2121-27. Ellison G, et al.

Treatment 52

Treatment Trials Disease FDA 52

Agency IQ

APRIL 19, 2024

Patients frequently receive up to four therapies at once and go through multiple prolonged courses of treatment to keep recurrence and progression at bay. Commonly combined treatment options include antineoplastics (chemotherapies), radiation therapy, corticosteroids, immunomodulators, proteasome inhibitors, and monoclonal antibodies.

FDA 40

FDA Disease Trials Clinical Trials 40

Drug Target Review

JULY 18, 2024

We believe liquid biopsy is very helpful for oncology drug development because the reduction and clearance of circulating DNA occurs quickly before radiographic imaging is available. The initial techniques were able to detect circulating tumour DNA in patients who already had metastatic spread.

Drug Development 59

Drug Development Clinical Trials Drugs Vaccine 59

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.

Therapies 98

Therapies Treatment Clinical Trials Drugs 98