pharmaphorum

AUGUST 16, 2022

Merck & Co has ramped up its involvement in the RNA category, partnering with US biotech Orna Therapeutics in a deal valued at up to $3.5 Now, Merck has made its own play, partnering with Orna on its proprietary ‘oRNA’ technology, which stands for circular RNA. billion, including $150 million upfront.

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Drug Discovery World

DECEMBER 13, 2022

Japanese researchers propose using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama muscular dystrophy. . The genetic anomaly blocks the chemical glycosylation of a biologically important protein. Modifying RNA function. Image credit: Mariko Taniguchi-Ikeda from Fujita Health University

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Drug Discovery World

JANUARY 10, 2023

CircRNAs represent an emerging, powerful mechanism for delivering therapeutics and vaccines due to their protein-coding potential and improved stability in comparison to their linear mRNA counterparts. . The post Alliance to develop circular RNA-based therapeutics appeared first on Drug Discovery World (DDW).

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Drug Discovery World

APRIL 18, 2024

Nutcracker Therapeutics’ prostate cancer mRNA drug candidate NTX-470 successfully engaged CD3 T cells, while retaining low bystander activity and reduced off-tumour binding, according to data presented at the 2024 AACR Annual Meeting.

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pharmaphorum

JANUARY 6, 2022

Roivant is the latest pharma group to take a position in the emerging field of therapies targeted at RNA splicing, licensing exclusive rights to a drug developed by a unit of Japanese drugmaker Eisai. . The post Roivant joins RNA splicing push with Eisai deal appeared first on.

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Pharmaceutical Technology

JUNE 23, 2022

Researchers at the University of New Hampshire (UNH) have found that a repurposed drug, CCG-50014, could hinder the activity of a key enzyme of the SARS-CoV-2 virus, which causes Covid-19. Utilising a currently available drug compound in a new way is called drug repurposing.

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The Pharma Data

JANUARY 5, 2021

Durham, North Carolina-based Ribometrix announced a strategic collaboration deal with Genentech , a Roche company, to identify and advance novel RNA-targeted small molecule therapeutics. Targeting RNA is believed to be a way to develop therapeutics for so-called undruggable proteins. The first identifies the 3D RNA motifs.

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Drug Discovery World

MARCH 21, 2024

A project focused on developing a small RNA-based platform technology for treating monogenic diseases, smartRNA, has joined the BioInnovation Institute’s (BII) Bio Studio programme. The technology has platform potential, as it leverages a universal small RNA pathway to target generic mRNA features.

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pharmaphorum

MAY 14, 2021

Biogen has made a further push into RNA-based drug discovery via a collaboration with Envisagenics, which applies artificial intelligence RNA sequencing data to discover new drug targets and therapeutics. It’s not the first partnership for Biogen in the RNA splicing area.

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XTalks

JUNE 20, 2022

Alnylam Pharmaceuticals, a leading RNA interference (RNAi) therapeutics biopharmaceutical company, announced it received approval from the US Food and Drug Administration (FDA) for its RNAi therapeutic Amvuttra (vutrisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis in adults.

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Pharmaceutical Technology

JUNE 6, 2023

The drug’s revenue forecasts along with estimated costs are used to measure the value of an investment opportunity in that drug, otherwise known as net present value (NPV). The rNPV model is a more conservative valuation measure that accounts for the risk of a drug in clinical development failing to progress.

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Pharmaceutical Technology

JANUARY 19, 2023

The US Food and Drug Administration (FDA) has granted Fast Track designation for Avidity Biosciences’ AOC 1020 to treat facioscapulohumeral muscular dystrophy (FSHD). This DUX4 protein abnormal expression leads to modifications in gene expression in muscle cells which are associated with progressive muscle function loss in FSHD patients.

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Drug Discovery World

OCTOBER 26, 2023

The technologies cover the whole range of biomolecules from genes to protein to metabolites. As one key element for PanOmics-driven drug discovery we have established leading high-throughput and high-resolution transcriptomics data generation capabilities.

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Drug Discovery World

MARCH 26, 2024

For example, progress made in developing gene mutant-specific inhibitors, antibody-drug conjugates (ADC), or cellular therapies. The post Conversations from ESMO Targeted Anticancer Therapies Congress 2024 appeared first on Drug Discovery World (DDW).

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Drug Discovery World

MARCH 2, 2023

A new paper has solved the 20-year mystery of how epigenetic modifications act as traffic lights to control gene expression and could ultimately speed up the development of a new class of epigenetic cancer drugs. When it gives the green light, H3K4me3 allows RNA polymerase II to move along DNA, transcribing it into RNA as it moves.

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The Pharma Data

MARCH 23, 2022

Polymerase is a viral protein that directs how Ebola virus replicates its genome as it infects new hosts. Drugs that target polymerase could potentially treat Ebola virus infections and save lives. The researchers discovered that Ebola virus polymerase hijacks a cellular protein called GSPT1. Catching polymerase in the act.

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Drug Discovery World

AUGUST 4, 2023

The pace of innovation in cancer drug discovery showed no signs of slowing this week, with several pretty momentous discoveries boosting research in the sector. The post This week in drug discovery (31 July – 4 August) appeared first on Drug Discovery World (DDW).

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pharmaphorum

OCTOBER 31, 2022

Verge Genomics has joined a select group of biotechs who have taken a drug discovered and developed using artificial intelligence into human testing. The post Verge Genomics takes AI-sourced drug for ALS into clinic appeared first on.

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Delveinsight

SEPTEMBER 23, 2021

Boehringer Ingelheim acquires Abexxa Biologics for precision most cancers drug pipeline. Boehringer stated that the Abexxa’s generation aims at cancer-specific proteins situated throughout the mobile versus proteins at the mobile membrane broadening the selection of possible cancer antigen objectives. Jeffrey Stafford, Ph.D.,

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Drug Discovery World

APRIL 17, 2024

DS: CAMP4 leverages research from co-founder Richard Young on how regulatory RNA (regRNA) molecules control the expression of genes. What are the challenges of drug discovery in this area? JMB: Regulatory RNAs (or regRNAS) are molecules that directly control the expression of nearby protein-coding genes. How do they work?

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Gene Expression Gene Genetic Disease RNA 59

STAT News

AUGUST 17, 2022

On Target is a recurring feature from STAT that dives deep into the most promising drug targets in oncology.    The hunt for cancer cures has, to a large degree, been a hunt for biomarkers — DNA, peptides, RNA, proteins or more — that might set tumor cells apart from healthy tissue.

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Scienmag

NOVEMBER 20, 2020

Scientists at Goethe University within the international consortium COVID19-NMR refine previous 2D models The genetic code of the SARS-CoV2 virus is exactly 29,902 characters long, strung through a long RNA molecule. It contains the information for the production of 27 proteins.

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Pharmaceutical Technology

JULY 5, 2022

Researchers at the UNC School of Medicine have discovered that hepatitis A virus (HAV) replication needs particular interactions between the human protein ZCCHC14 and TENT4 poly(A) polymerases, a group of enzymes. Subsequently, Lemon’s lab tested RG7834 and found the precise effects of the oral drug on HAV in liver and faeces.

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Drug Discovery World

NOVEMBER 1, 2022

Researchers say they have successfully used a cell’s natural process for making proteins to ‘slide’ genetic instructions into a cell and produce critical proteins missing from those cells. . Two methods currently used to deliver protein-making packages into cells, ‘mini promoters’ and serotype-mediated gene expression, vary widely.

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pharmaphorum

OCTOBER 5, 2022

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule. billion deal in 2018.

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XTalks

JANUARY 3, 2024

Wainua is the only FDA-approved drug for the treatment of ATTRv-PN that can be self-administered via an auto-injector. In individuals affected by ATTR, which includes both hereditary and wild-type (non-hereditary) variants, the TTR protein forms fibrils that accumulate in various tissues.

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pharmaphorum

JANUARY 26, 2023

Understanding individual drug reactions and metabolism Pharmacogenomics is emerging as an important area. Genomic insights will increasingly be used to reduce the hundreds of millions of pounds wasted administering drugs that are ineffective due to an individual’s genetic makeup. So, when it comes to proteomics, accuracy is vital.

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Drug Discovery World

FEBRUARY 19, 2024

“Achieving a broad therapeutic window is an important step-up from other existing mRNA and srRNA approaches, which allows us to expand applications of our self-replicating RNA to complex infectious diseases, off-the-shelf cancer vaccines, and as a platform for protein production.”

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Drug Discovery World

MARCH 21, 2023

Diana Spencer explores the different ways the drug discovery sector is tackling colorectal cancer. So, what are the recent developments and how is the drug discovery sector tackling this common cancer? Improved diagnostics Diagnostics and screening are essential to the early treatment of colorectal cancer.

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Drug Discovery World

OCTOBER 24, 2022

New research suggests messenger RNA can be effectively used as a ‘universal’ therapy against different coronaviruses. . Rather than messenger RNA as a vaccine, this shows that mRNA can be used as a universal therapy against different coronaviruses,” said Gaurav Sahay, OSU and the Texas Biomedical Research Institute.

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Drug Discovery World

APRIL 11, 2023

Here, we take a look at the latest drug discovery efforts to meet this unmet need. The molecule, known as lissodendoric acid A, appears to counteract other molecules that can damage DNA, RNA and proteins and even destroy whole cells.

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pharmaphorum

SEPTEMBER 14, 2021

Repeat sequences aren’t involved in expressing proteins so have been generally under-studied by researchers, but there is increasing evidence that some human traits and diseases may be associated with mutations within them. Most drug discovery programmes target the roughly 2% of the human genome which encodes for protein.

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DNA Immune Response Genome Genomics 52

Drug Discovery World

JANUARY 23, 2023

Researchers have discovered a clinical drug that sensitises glioblastoma (GBM) cells to radiotherapy and could replace the current standard of care. As an FDA-approved oral drug, the side-effects of cladribine are quite manageable and it has been well evaluated for pharmacokinetics.

FDA Approval 52

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Drug Discovery World

MAY 13, 2024

MA: Regarding vaccine development specifically, and the role my team plays in the development of cryogenic electron microscopy (cryo-EM) technology, we see the study and dissemination of virus structures, and the proteins that comprise them, as vital to vaccine design. For example, there are global monitoring efforts for new Covid strains.

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Drug Discovery World

MARCH 3, 2023

Professor Achim Aigner, Clinical Pharmacology in the Faculty of Medicine at Leipzig University, said “Due to their parallel, selective effects on multiple defined targets, miRNAs offer exceptional opportunities for the development of novel drugs that show enhanced efficacy while avoiding tumour cell resistance.

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Drug Discovery World

JANUARY 20, 2023

Precision medicine uses information about an individual’s genes, proteins, and environment to prevent, diagnose, and treat diseases, including cancer. Differences in DNA, RNA, and protein can be identified in individual tissue samples, and even in individual cells.

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pharmaphorum

NOVEMBER 9, 2020

Sanofi’s management has highlighted fitusiran, a potential competitor to Roche’s haemophilia A drug Hemlibra, as one of the most important drugs in its pipeline. Fitusiran is being developed for haemophilia A and B in partnership with RNA interference (RNAi) specialist Alnylam – but has already been affected by safety issues.

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