Clinical Trials, Drugs and RNA - Clinical Research Informer

Beyond Covid-19: the disruptive potential of RNA-based therapeutics

Pharmaceutical Technology

MARCH 1, 2023

Further, the acceptance of new mRNA vaccines has rejuvenated activity within previously established categories of RNA therapeutics including lifesaving antisense technologies. These include antisense oligonucleotides (ASO), RNA interference (RNAi), and RNA aptamers.

RNA

RNA Vaccination Vaccine Genetics

DCGI adds IVD devices for diagnosis of Covid-19, RNA & DNA extraction kits in Class C risk category under MDR-2017

AuroBlog - Aurous Healthcare Clinical Trials blog

NOVEMBER 1, 2023

The Drug Controller General of India (DCGI) has added in-vitro diagnostic (IVD) medical devices including those for diagnosis of Covid-19, ribonucleic acid (RNA) and deoxyribonucleic acid (DNA) extraction kits, among others into the Class C risk category under the Medical Devices Rules (MDR), 2017.

RNA

RNA DNA In-Vitro Drugs

Uptick in Clinical Trial Complexity: How Can You Achieve Your Clinical Trial Objectives Faster?

XTalks

JANUARY 8, 2024

Getting a drug to market used to be a relatively simple and straightforward affair. Whether it’s for a treatment for a chronic ambulatory condition, precision medicine or cell and gene therapy, there is a massive uptick in clinical trial complexity. That’s just not the case anymore.

Clinical Trials

Clinical Trials Clinical Trials Trials Gene Therapy

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

JULY 29, 2022

Last week, CAMP4 Therapeutics announced the close of a $100 million Series B round , which will be used to advance their regulatory RNA (regRNA)-focused programs. CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinical trials go forward for their urea cycle disorder programs late next year.

RNA

RNA Gene Expression Gene Gene Therapy

World RNA Day: What impact RNA it had on drug discovery?

Drug Discovery World

AUGUST 1, 2023

1 August was first chosen as World RNA Day in 2018 as a play on AUG (adenine, uracil and guanine), a triple sequence of RNA (called a codon) that initiates protein synthesis by the cell. Since then, it has been observed to publicise the importance of this molecule in the generation of proteins in the body.

RNA

RNA Drugs Gene Silencing Vaccination

RNA companies to watch over the next 12 months

Drug Discovery World

JANUARY 23, 2023

Following the release of Clarivate’s report ‘RNA Technology Companies to Watch’, DDW’s Diana Spencer speaks to Strand Therapeutics’ Jacob Becraft and HAYA Therapeutics’ Samir Ounzain to find out how this technology could revolutionise disease treatment. DS: How will your work ultimately impact disease treatment?

RNA

RNA Genome Genomics Engineer

Merck makes circular RNA play with $3.5bn Orna alliance

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.

RNA

RNA Vaccination Vaccine Clinical Trials

Webinar: How single-cell RNA sequencing has advanced drug discovery

Drug Discovery World

DECEMBER 5, 2022

The consequence is the identification of drug targets that are not relevant to the biological process under study. . Join DDW for this free event, Broadening the horizon of drug discovery: use of unperturbed, native state cells to uncover true biological targets at single-cell level , which is supported by LevitasBio. Camila Egidio.

RNA

RNA Drugs Clinical Trials Clinical Trials

Exclusive event: An overview of single-cell RNA sequencing as a relevant tool for drug discovery

Drug Discovery World

DECEMBER 1, 2022

The consequence is the identification of drug targets that are not relevant to the biological process under study. . Join DDW for this free event, Broadening the horizon of drug discovery: use of unperturbed, native state cells to uncover true biological targets at single-cell level , which is supported by LevitasBio. Camila Egidio.

RNA

RNA Drugs Clinical Trials Clinical Trials

The future of RNA-based Therapeutics and Vaccines: Introducing advanced, Unrivalled Treatment Approaches

Roots Analysis

NOVEMBER 6, 2023

RNA-based therapeutics have completely revolutionized the healthcare segment, greatly influencing the study and treatment of human diseases by conferring precise targeting ability to therapeutic modalities. The below figure presents the distribution of next generation RNA-based therapeutics and RNA-based vaccines, based on type of molecule.

RNA

RNA Vaccination Vaccine Clinical Trials

Opinion: As it turns 40, the Orphan Drug Act for rare diseases needs a refresh

STAT News

JANUARY 30, 2023

This month marks the 40th anniversary of the Orphan Drug Act. That said, a lot has changed in science and drug development since 1983. It is now possible to treat diseases with gene therapy, antisense oligonucleotides, messenger RNA (mRNA), noncoding RNA (known as small interfering RNA, or siRNA), and other gene-based modalities.

RNA

RNA Gene Therapy Drugs Gene

STAT+: Arrowhead RNA treatment improves liver scarring in patients, but so does a placebo, clouding study results

STAT News

JANUARY 9, 2023

An experimental RNA treatment reduced liver scarring in half of patients with an inherited disease called alpha-1 antitrypsin deficiency, or AATD, according to results from a mid-stage clinical trial reported Monday by its maker Arrowhead Pharmaceuticals.

RNA

RNA Clinical Trials Clinical Trials Trials

RNA aptamer reopens blocked arteries

Drug Discovery World

FEBRUARY 15, 2023

Shahid M Nimjee, co-founder and Chief Medical Officer of Basking, and Associate Professor and practicing neurosurgeon at the Ohio State University Wexner Medical Center, said: “BB-031 is an RNA aptamer that targets vWF.

RNA

RNA Clinical Trials Clinical Trials Trials

RNA interference method could treat muscular dystrophy

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. . Using RNA interference, researchers restored the normal biological function of FKTN in patient-derived cells, offering hope for a new therapy. . Modifying RNA function.

RNA

RNA Genetics Gene In-Vitro

CAMP4 raises $100m to take lead RNA drugs into clinic

pharmaphorum

JULY 20, 2022

Armed with a $100 million second-round financing, CAMP4 Therapeutics is preparing to start the first clinical trial of a drug targeting regulatory RNA (regRNA) molecules that can be used to fine-tune the expression of genes. ” The post CAMP4 raises $100m to take lead RNA drugs into clinic appeared first on. .

RNA

RNA Genetic Disease Drugs Protein

RNA-based immunotherapy eradicates melanoma tumours

Drug Discovery World

AUGUST 1, 2023

Investigators at the Icahn School of Medicine at Mount Sinai have designed an RNA-based strategy to activate dendritic cells that eradicated tumours and prevented their recurrence in mouse models of melanoma. However, these have not been as successful in clinical trials as hoped.

RNA

RNA Bioassay Genome Genomics

FDA grants orphan drug designation to SiSaf’s siRNA therapy for ADO2

Pharmaceutical Technology

MAY 15, 2023

The US Food and Drug Administration (FDA) has given orphan drug designation to SiSaf’s siRNA [a double-stranded RNA molecule that is non-coding] therapeutic, SIS-101-ADO, for the treatment of autosomal dominant osteopetrosis type 2 (ADO2), a rare and serious skeletal disorder in children.

Gene Therapy

Gene Therapy Drugs RNA Clinical Trials

DDW readers’ top drug discovery developments of 2023

Drug Discovery World

DECEMBER 26, 2023

Representing a dominant field of drug discovery, it isn’t surprising that breakthroughs in cancer research were popular with DDW readers in 2023, particularly strategies to overcome treatment resistance and prevent the spread of disease. The drug works by targeting proprotein convertase subtilisin/kexin type 9 serine protease (PCSK9).

Development

Development Clinical Trials Clinical Trials Drugs

Present Treatments & Hopeful Future Directions for ALS Pharmacotherapies

Worldwide Clinical Trials

MAY 30, 2024

Authors: Rich Worldwide Clinical Trials Exec. This trend is on the rise despite recent disappointments with clinical trial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. Director, Therapeutic Area Medical Lead.

Gene

Gene Clinical Trials Clinical Trials Gene Therapy

mRNA universal influenza vaccine enters clinical trials

Drug Discovery World

MAY 18, 2023

A clinical trial of an experimental universal influenza vaccine developed by researchers at the National Institute of Allergy and Infectious Diseases’ (NIAID) Vaccine Research Center (VRC) has begun enrolling volunteers at Duke University in Durham, North Carolina.

Clinical Trials

Clinical Trials Clinical Trials Vaccination Vaccine

The Utility of Liquid Biopsy in Oncology Clinical Trials

XTalks

NOVEMBER 7, 2022

Gleich, MD, FACS, Senior Vice President, Medical Department, and Dr. Christopher Huth, PhD, Senior Clinical Trial Manager, Clinical Trial Management. Liquid Biopsy Use in Oncology Clinical Trials. Also, exosomes are mostly being used for the detection of circulating RNA and microRNA. “An

Clinical Trials

Clinical Trials Clinical Trials Trials FDA Approval

Receptor Occupancy Assays by Flow Cytometry: Benefits for Clinical Trials

XTalks

APRIL 22, 2022

Flow cytometric receptor occupancy assays are being increasingly used in preclinical and clinical studies. Both the areas of drug development and clinical trials are increasingly using in vitro assays to help determine the efficacy of an investigational therapeutic. What is Flow Cytometry?

Clinical Trials

Clinical Trials Clinical Trials Trials Antibody

This week in drug discovery (13-16 May)

Drug Discovery World

MAY 17, 2024

From a weight loss drug that prevents heart attacks and a gene therapy that restores hearing, to a vaccine that can treat viruses that don’t exist yet, our chosen news stories this week all represent potential breakthroughs in their respective fields. News round-up for 13-16 May by DDW Senior Digital Content Editor Diana Spencer.

Gene Therapy

Gene Therapy Gene Drugs In-Vivo

How transcriptomics is driving drug discovery

Drug Discovery World

FEBRUARY 13, 2023

Dr Stephen Barat, Senior Vice President and Therapeutics Division Head at HTG Molecular Diagnostics tells DDW how transcriptomics is driving drug discovery and precision medicines. SB: Transcriptomics is the study of genomic RNA transcripts in a biological system. DDW: Could you explain the process of transcriptomics?

RNA

RNA Drugs Gene Expression Gene

Samsung Biologics, GreenLight conclude Covid-19 vaccine engineering run

Pharmaceutical Technology

AUGUST 2, 2022

GreenLight’s process from formulation of the drug substance and lipid nanoparticle to bulk drug product can be carried out in this facility. The RNA platform of Samsung Biologics permitted GreenLight to move from mRNA vaccine conceptualisation to the delivery of released clinical trial material in under two years.

Engineer

Engineer Vaccination Vaccine Clinical Trials

Broadening drug discovery: use of unperturbed, native state cells to uncover true biological targets at single-cell level

Drug Discovery World

NOVEMBER 25, 2022

The consequence is identification of drug targets that are not relevant to the biological process under study. Based on Nakhoda and Olszanski, 2022 (Addressing Recent Failures in Immuno-Oncology Trials to Guide Novel Immunotherapeutic Treatment Strategies), lack of an optimal biomarker is one of the main challenges to clinical trial success.

RNA

RNA Drugs Clinical Trials Clinical Trials

Recipharm acquires GenIbet to bolster Biologics offering

Pharma Mirror

FEBRUARY 2, 2022

Global contract development and manufacturing organisation (CDMO), Recipharm, has today announced the acquisition of GenIbet, a Portuguese CDMO, specialising in the manufacture of biological clinical trial material and novel modalities such as viral vectors, RNA and microbiome.

RNA

RNA Manufacturing Clinical Trials Clinical Trials

Why native state cells are needed to uncover true drug targets

Drug Discovery World

DECEMBER 8, 2022

The consequence is the identification of drug targets that are not relevant to the biological process under study. . Join DDW for this free event, Broadening the horizon of drug discovery: use of unperturbed, native state cells to uncover true biological targets at single-cell level , which is supported by LevitasBio. Camila Egidio.

RNA

RNA Drugs Clinical Trials Clinical Trials

How next-generation sequencing is informing oncology drug discovery

Drug Discovery World

APRIL 3, 2023

Hosted by DDW and supported by Tecan, “How next-generation sequencing is informing oncology drug discovery” will be free to attend on April 24 at 7AM PST / 10AM EST / 3PM BST / 4PM CET. Join this free webinar to learn about the biggest challenges and opportunities facing the NGS oncology space. You will hear from Darrell Green, a lecturer in (..)

RNA

RNA Drugs Genome Genomics

Free webinar today: How is NGS informing oncology drug discovery?

Drug Discovery World

APRIL 24, 2023

The post Free webinar today: How is NGS informing oncology drug discovery? appeared first on Drug Discovery World (DDW). Join this free webinar to learn about the biggest challenges and opportunities facing the next-generation sequencing (NGS) oncology space. Register now.

RNA

RNA Drugs Genome Genomics

Webinar: Find out how NGS is informing oncology drug discovery

Drug Discovery World

APRIL 11, 2023

The post Webinar: Find out how NGS is informing oncology drug discovery appeared first on Drug Discovery World (DDW). Join this free webinar to learn about the biggest challenges and opportunities facing the next-generation sequencing (NGS) oncology space. Register now.

RNA

RNA Drugs Genome Genomics

Register now to learn how NGS is informing oncology drug discovery

Drug Discovery World

APRIL 18, 2023

Can NGS increase the chances of an oncology drug making it to market? Can NGS identify previously undruggable targets in cancer? How can the technology best be implemented in research labs? Will NGS help drive personalised cancer therapies? Can NGS technology help direct patients towards suitable clinical trials?

RNA

RNA Drugs Genome Genomics

How Can You Manage the Complexity of Early Phase Clinical Development?

XTalks

JUNE 4, 2024

In the ever-evolving landscape of pharmaceutical development, the complexity of early phase clinical trials is increasing. This surge in complexity is driven by escalating drug development costs, heightened competition and advanced scientific innovations. This is the storm that we find ourselves in the midst of.”

Clinical Development

Clinical Development Development Clinical Trials Clinical Trials

Gilead’s Veklury receives WHO recommendation to treat severe Covid-19

Pharmaceutical Technology

SEPTEMBER 16, 2022

The latest conditional recommendation is based on the final data from the SOLIDARITY clinical trial, which was sponsored by the WHO. These results are in line with those obtained from the placebo-controlled, double-blind ACTT-1 trial of the National Institute of Allergy and Infectious Diseases.

Allergies

Allergies RNA Clinical Trials Clinical Trials

Positive preclinical data for siRNA therapy for osteopetrosis

Drug Discovery World

SEPTEMBER 25, 2023

RNA therapeutics company SiSaf has published positive safety and efficacy data for its siRNA therapy for Autosomal Dominant Osteopetrosis 2 (ADO2). The company says these results could have significant translational impact on bone disease therapies and open the path to human trials of it’s potentially curative treatment for ADO2.

RNA

RNA In-Vivo Clinical Trials Clinical Trials

STAT+: The biotech scorecard for the fourth quarter: 14 stock-moving events to watch

STAT News

SEPTEMBER 30, 2022

Participants in the study have undergone liver biopsies at baseline and one year to determine if fazirsiran improves disease symptoms, including liver fibrosis.

RNA

RNA Clinical Trials Clinical Trials Trials

Risk adjusted net present value: What is the current valuation of Moderna’s MRNA-0184?

Pharmaceutical Technology

JUNE 6, 2023

According to Globaldata, it is involved in 1 clinical trial, which is ongoing. 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 drug candidate is administered by intravenous route.

RNA

RNA Protein Antibody Vaccination

Next-generation sequencing and oncology drug discovery

Drug Discovery World

APRIL 4, 2023

The post Next-generation sequencing and oncology drug discovery appeared first on Drug Discovery World (DDW). Register now.

Drugs

Drugs RNA Genome Genomics

This week in drug discovery (31 July – 4 August)

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).

Drugs

Drugs RNA Gene Scientist

US FDA grants Fast Track status for Avidity’s AOC 1020 to treat FSHD

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). We look forward to working collaboratively with the FDA to bring the first RNA therapy directly targeting DUX4 to patients as quickly as possible.”

Gene Expression

Gene Expression Gene Protein RNA

Verge Genomics takes AI-sourced drug for ALS into clinic

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.

Genome

Genome Genomics Drugs RNA

€10M financing to support disease-modifying obesity drug

Drug Discovery World

JANUARY 9, 2024

The company intends to use the proceeds to initiate and complete the first-in-human Phase I clinical trial and reach Phase II readiness for its lead program, RES-010, in obesity. The post €10M financing to support disease-modifying obesity drug appeared first on Drug Discovery World (DDW).

Drugs

Drugs Life Science RNA Clinical Trials

Molecular mechanisms of corona drug candidate Molnupiravir unraveled

The Pharma Data

AUGUST 16, 2021

The antiviral agent incorporates RNA-like building blocks into the RNA genome of the virus. If this genetic material is further replicated, defective RNA copies are produced and the pathogen can no longer spread. Molnupiravir is currently being tested in clinical trials. Molnupiravir has a lot of potential.”

RNA

RNA Drugs Genome Genomics

The future outlook for mRNA therapies

Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. mRNA is a molecule made up of a single strand of ribonucleic acid (RNA). As such, teams are now researching a variety of medical applications for mRNA-based drugs.

RNA

RNA Protein Vaccination Vaccine

Beyond Covid-19: the disruptive potential of RNA-based therapeutics

DCGI adds IVD devices for diagnosis of Covid-19, RNA & DNA extraction kits in Class C risk category under MDR-2017

Trending Sources

Uptick in Clinical Trial Complexity: How Can You Achieve Your Clinical Trial Objectives Faster?

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

World RNA Day: What impact RNA it had on drug discovery?

RNA companies to watch over the next 12 months

Merck makes circular RNA play with $3.5bn Orna alliance

Webinar: How single-cell RNA sequencing has advanced drug discovery

Exclusive event: An overview of single-cell RNA sequencing as a relevant tool for drug discovery

The future of RNA-based Therapeutics and Vaccines: Introducing advanced, Unrivalled Treatment Approaches

Opinion: As it turns 40, the Orphan Drug Act for rare diseases needs a refresh

STAT+: Arrowhead RNA treatment improves liver scarring in patients, but so does a placebo, clouding study results

RNA aptamer reopens blocked arteries

RNA interference method could treat muscular dystrophy

CAMP4 raises $100m to take lead RNA drugs into clinic

RNA-based immunotherapy eradicates melanoma tumours

FDA grants orphan drug designation to SiSaf’s siRNA therapy for ADO2

DDW readers’ top drug discovery developments of 2023

Present Treatments & Hopeful Future Directions for ALS Pharmacotherapies

mRNA universal influenza vaccine enters clinical trials

The Utility of Liquid Biopsy in Oncology Clinical Trials

Receptor Occupancy Assays by Flow Cytometry: Benefits for Clinical Trials

This week in drug discovery (13-16 May)

How transcriptomics is driving drug discovery

Samsung Biologics, GreenLight conclude Covid-19 vaccine engineering run

Broadening drug discovery: use of unperturbed, native state cells to uncover true biological targets at single-cell level

Recipharm acquires GenIbet to bolster Biologics offering

Why native state cells are needed to uncover true drug targets

How next-generation sequencing is informing oncology drug discovery

Free webinar today: How is NGS informing oncology drug discovery?

Webinar: Find out how NGS is informing oncology drug discovery

Register now to learn how NGS is informing oncology drug discovery

How Can You Manage the Complexity of Early Phase Clinical Development?

Gilead’s Veklury receives WHO recommendation to treat severe Covid-19

Positive preclinical data for siRNA therapy for osteopetrosis

STAT+: The biotech scorecard for the fourth quarter: 14 stock-moving events to watch

Risk adjusted net present value: What is the current valuation of Moderna’s MRNA-0184?

Next-generation sequencing and oncology drug discovery

This week in drug discovery (31 July – 4 August)

US FDA grants Fast Track status for Avidity’s AOC 1020 to treat FSHD

Verge Genomics takes AI-sourced drug for ALS into clinic

€10M financing to support disease-modifying obesity drug

Molecular mechanisms of corona drug candidate Molnupiravir unraveled

The future outlook for mRNA therapies

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