The Open Targets Blog

JANUARY 30, 2025

For example, to find drugs associated with the human IL13 gene (Ensembl ID ENSG00000169194*), a cytokine that plays an important role in allergic inflammation and immune response to parasite infection, you can use: # Install gget !pip The gget opentargets module has many applications.

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Bioinformatics Immune Response RNA Packaging 76

Drug Target Review

JUNE 28, 2023

When faced with a viral threat, bacterial cells developed an immune response by capturing and copying DNA fragments of viruses. It was also discovered that the Cas enzyme was responsible for DNA cleavage. Using a synthetic guide RNA (gRNA), scientists can target a specific DNA sequence and employ Cas9 to cut it.

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Engineering DNA Immune Response RNA 98

Alta Sciences

FEBRUARY 19, 2025

These interactions may disrupt protein function, trigger immune responses, or contribute to other toxicological risks. to ligands or linkers) may elicit immune responses. An oligonucleotide is a short strand of nucleotides, the building blocks of DNA or RNA, used in genetic research and therapy.

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Drug Development Clinical Pharmacology Immune Response Drugs 52

Drug Target Review

APRIL 26, 2023

Scientists from the Massachusetts Institute of Technology (MIT) and the University of Massachusetts Medical School (UMass), US, have collaborated to create a novel type of nanoparticle that can deliver messenger RNA that encodes for beneficial proteins to the lungs.

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RNA Medical Schools Engineering Disease 98

DrugBank

MARCH 12, 2025

Introduction Messenger RNA (mRNA) technology has emerged as one of the most significant medical breakthroughs. Once delivered into the body, the mRNA instructs cells to produce these antigens, which are then presented to the immune system. Another challenge is the potential for unintended immune responses.

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Broad Institute

JULY 11, 2024

Treatment with mIDH1 blockers unearths these remnants, triggering an immune response against the tumor cells. IDH1 normally facilitates the activity of enzymes called demethylases, which remove chemical flags called methylation marks from DNA, allowing genes to be transcribed into RNA.

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Treatment Immune Response Drugs Virus 72

The Pharma Data

OCTOBER 25, 2020

German drugmaker CureVac said preclinical studies of its COVID-19 vaccine candidate, CVnCoV, showed the vaccination prompted a successful immune response. The messenger RNA-based vaccine, which is designed to block replication of the coronavirus, was shown to induce neutralizing antibodies and activate T-cells in hamsters and mice.

Vaccine 52

Vaccine Immune Response RNA Clinical Trials 52

Broad Institute

OCTOBER 11, 2023

The drugs work by blocking a protein, PD-1 — which helps keep immune responses in check — and stimulating the immune system to attack tumors. The team studied the immune response to CRISPR and designed a new technology that they published in 2021 — a way of removing immune-triggering molecules from CRISPR-edited cells.

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Research Immune Response Clinical Trials Therapies 137

Drug Target Review

DECEMBER 7, 2023

Researchers from the Laboratory of Bacteriology at The Rockefeller University have now found that bacteria sense phages by a defensive response named CBASS (cyclic oligonucleotide-based antiphage signalling system) which detects viral RNA. In bacteria , cGAS-like cyclases are central parts of the CBASS immune response.

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RNA Immune Response DNA Molecular Biology 59

Drug Target Review

JUNE 6, 2023

Natural killer (NK) cells are another immune cell type that, as the name suggests, also have potent cell-killing activity, and have a well-known role in the anti-tumour immune response. In the context of a tumour microenvironment, Tregs are often present in high numbers, preventing an effective immune response to the tumour.

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Engineering Immune Response Biochemical Pharmacology Clinical Trials 98

PLOS: DNA Science

MAY 16, 2024

As soon as the initial RNA genome sequence of the pathogen that would be named SARS-CoV-2 was published, CDC and other organizations began developing diagnostic tests. • identifies parts of the virus to target to provoke an immune response from a vaccine. Work on vaccines ensued too. • Single-cell sequencing.

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Virus DNA RNA Vaccine 98

The Pharma Data

MARCH 8, 2022

Proteins of SARS-CoV-2 can alter viral ribonucleic acids in a way, that they become indistinguishable from endogenous RNA. Camouflage protects virus from immune system. For example, viral RNAs are masked by the addition of a methyl group. “A robust, early type I IFN production is key to clearing SARS-CoV-2 infection.

Clinical Pharmacology 52

Clinical Pharmacology Immune Response RNA Virus 52

Codon

NOVEMBER 3, 2024

If present before vaccination, memory immune cells primed to destroy the environmental relatives might cross-react with and neutralize the BCG strain before it has a chance to set up an immune response against itself and M. tuberculosis. A Peace Corps Volunteer administers the BCG vaccine to a six-year-old Korean boy.

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Vaccine Trials Therapies Treatment 102

The Pharma Data

DECEMBER 14, 2020

Supported by promising phase 1 data, CureVac announced Monday that it has enrolled the first participant in the pivotal phase 2b/3 study of its messenger-RNA based coronavirus vaccine, CVnCoV. The company announced positive phase 1 trial results in November that demonstrated its candidate elicited immune responses and was well-tolerated.

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Vaccine Trials Immune Response RNA 52

The Pharma Data

DECEMBER 3, 2020

Strong Th1 cell-mediated immune responses were also observed for the vaccine candidates with either adjuvant. We are encouraged by the high level of neutralizing antibodies in combination with the strong Th1 response which we believe could play an important role in controlling infection.

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DrugBank

MAY 15, 2024

Another promising avenue is the use of technologies like RNA interference and gene editing, which allow scientists to turn off the production of certain proteins altogether. These methods could bypass the need to directly target these proteins with traditional drugs.

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Small Molecule Molecular Biology Drugs Disease 98

Broad Institute

JUNE 27, 2024

CRISPRoff silences the targeted gene by adding methyl groups, chemical tags that prevent the gene from being transcribed or read into RNA and so from being expressed as protein. ZFPs are also common in human cells, meaning they are less likely to trigger an immune response against themselves than the bacterial Cas9.

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Disease Therapies Clinical Trials Engineering 142

Drug Target Review

FEBRUARY 7, 2024

We had built a machine learning classifier that has been trained to identify amino acid sequences that have the potential to mimic ‘host defense peptides’ from the human innate immune system. Such peptides play a critical role in the machinery for amplifying innate immune responses.

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RNA Virus Immune Response Engineering 69

Drug Target Review

MARCH 21, 2024

AAV-based therapies often can’t be dosed more than once due to concerns about an immune response. Additionally, the COVID-19 RNA vaccines have already demonstrated how safe RNA can be, and we look forward to seeing additional safety data on RNA approaches as the field continues to grow.

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Therapies RNA Immune Response Treatment 64

Drug Target Review

OCTOBER 2, 2024

RNA-based therapies, including mRNA and RNA interference (RNAi), are being explored to modulate gene expression in heart cells. While commonly used, AAVs have limitations, including immune reactions and difficulties with re-dosing due to pre-existing antibodies. Allogeneic cell therapies must avoid immune rejection.

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The Pharma Data

DECEMBER 8, 2020

. “In future analyses, with more data included as it becomes available, we will investigate differences in key subgroups such as older adults, various ethnicities, doses, timing of booster vaccines, and we will determine which immune responses equate to protection from infection or disease.” © 2020 HealthDay.

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Vaccine Trials Immune Response RNA 52

The Pharma Data

JULY 18, 2021

Clinical Data Supporting Approval Demonstrated Non-Inferior Immune Responses for the Serotypes Shared with PCV13 (1, 3, 4, 5, 6A, 6B, 7F, 9V, 14, 18C, 19A, 19F and 23F). VAXNEUVANCE Elicited Superior Immune Responses for Serotypes 3, 22F and 33F Compared to PCV13, Which Are Major Causes of Disease.

Disease 52

Disease Immune Response Vaccine FDA Approval 52

The Pharma Data

JUNE 26, 2021

BNT111 is the lead product candidate from BioNTech’s FixVac platform that targets a fixed combination of mRNA-encoded, tumor-associated antigens with the objective of triggering a strong and precise immune response against cancer and is fully owned by BioNTech.

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Clinical Trials Trials Immune Response Vaccine 52

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 104

The Pharma Data

MAY 17, 2021

Sanofi and GlaxoSmithKline (GSK) have reemerged as COVID-19 vaccine contenders after their reformulated candidate delivered a strong immune response across all age groups in a phase 2 study. 14, 2020 ). The mRNA candidate is currently being assessed in a phase 1/2 study.

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The Pharma Data

JANUARY 31, 2021

Adjuvanted S-Trimer COVID-19 vaccine candidates demonstrated favorable safety and tolerability profiles and strong neutralizing immune responses in a phase 1 trial. CHENGDU, China , Feb. These findings give confidence that Clover’s COVID-19 vaccine candidates are suitable for further clinical development.”

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Clinical Trials Vaccine Trials Virus 52

The Pharma Data

SEPTEMBER 30, 2021

. “We used NanoSTING because the adjuvant for intranasal vaccination and single-cell RNA-sequencing to verify the nasal-associated lymphatic tissue as an inductive site upon vaccination. A fundamental limitation of intramuscular vaccines is that they’re not designed to elicit mucosal immunity.

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

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Small Molecule RNA Clinical Trials DNA 52

The Pharma Data

JUNE 26, 2021

Sanofi Pasteur, the vaccines global business unit of Sanofi and Translate Bio (NASDAQ: TBIO), a clinical-stage messenger RNA (mRNA) therapeutics company, have initiated a Phase 1 clinical trial evaluating an mRNA-based investigational vaccine against seasonal influenza. JUNE 22 , 2021.

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Clinical Trials Vaccine Trials Immune Response 52

The Pharma Data

JUNE 25, 2021

Phase 1 interim data reported in November 2020 showed that CVnCoV was generally well tolerated across all tested doses and induced strong antibody responses in addition to first indication of T cell activation. A pivotal Phase 2b/3, the HERALD study, with a 12µg dose of CVnCoV was initiated in December 2020. About CureVac.

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Vaccine Trials Immune Response Virus 52

The Pharma Data

JANUARY 21, 2021

Therefore, it’s not likely that a mutation would be able to evade the complex immune response created by a vaccine, even if the mutation makes the spike protein more effective at infecting unvaccinated people, the experts said. “That process is done so efficiently by the [U.S.

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Vaccine Virus Immune Response Disease 52

LifeSciVC

JULY 1, 2024

The central dogma of molecular biology is that information generally (with few exceptions) flows from DNA to RNA to Protein. Build your social immunity. The adaptive immune response identifies and responds to highly specific antigens associated with infections and cancer. Remember life’s Central Dogma.

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Science Immune Response Molecular Biology RNA 86

The Pharma Data

SEPTEMBER 9, 2020

Immunization of non-human primates (rhesus macaques) with BNT162b2, a nucleoside-modified messenger RNA (modRNA) candidate that expresses the SARS-CoV-2 spike glycoprotein, resulted in strong anti-viral effects against an infectious SARS-CoV-2 challenge. producing CD8+ T cell responses, which is thought to promote an anti-viral effect.

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Vaccine Clinical Trials RNA Trials 52

The Pharma Data

MARCH 11, 2021

Clinical trial to assess safety, immune response and reactogenicity, after preclinical data showed high neutralizing antibody levels. Expected to enroll 415 participants; interim results expected in Q3 2021. In parallel, preclinical studies are underway to evaluate additional mRNA candidates against emerging SARS-CoV-2 variants.

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Clinical Trials Vaccine Trials Immune Response 52

The Pharma Data

DECEMBER 16, 2020

The Moderna vaccine is similar to Pfizer and BioNTech’s vaccine in the sense that they both use an approach utilizing messenger RNA (mRNA). Instead, it uses a genetic code, which tricks the body into developing an immune response to the virus.

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Vaccine Immune Response FDA FDA Approval 52

The Pharma Data

JANUARY 31, 2021

Clover’s S-Trimer antigen adjuvanted with CpG 1018 plus alum demonstrated low reactogenicity while providing high levels of neutralizing antibodies and a strong Th1-biased cell-mediated immune response. Dynavax developed CpG 1018 to provide an increased vaccine immune response, which has been demonstrated in HEPLISAV-B.

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Vaccine Trials Virus RNA 52

Drug Target Review

JULY 11, 2023

In simple terms, our immune system is divided into innate and adaptive responses – the innate being the first line of defence, and the adaptive responds to specific challenges by modifying the body’s immune response in a tailored fashion.

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