The critical role of comprehensive RNA sequencing in liquid biopsy for biomarker discovery and clinical trials
Bio Pharma Dive
JUNE 17, 2024
Elevate precision medicine: dose optimization and immune monitoring through advanced liquid biopsies.
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Moderna reports ‘encouraging’ early data for one of its rare disease medicines
Bio Pharma Dive
MAY 19, 2023
The biotech, best known for its vaccine research, said its clinical trial is the first to report results of a messenger RNA therapeutic designed for intracellular protein replacement.
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AIRNA secures $155m to bring RNA-candidate to clinic
Pharmaceutical Technology
APRIL 2, 2025
The Series B funds will support the launch of a Phase I/II clinical trial for AIR-001 to advance its RNA editing pipeline.
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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.
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BioNTech targets infectious diseases for vaccine development
Pharmaceutical Technology
MARCH 28, 2023
There is much interest in the industry around RNA-based therapeutics as their utilisation in indications beyond Covid-19 come into focus. In December 2022, BioNTech initiated a Phase I clinical trial of BNT163 – an HSV vaccine candidate. BNT163 is meant to prevent genital lesions caused by HSV-2 and potentially HSV-1.
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Flamingo gets grant from VLAIO to accelerate RNA-focused oncology portfolio
Pharmaceutical Technology
JUNE 16, 2023
from Flanders Innovation & Entrepreneurship (VLAIO) to further advance its oncology portfolio targeting RNA. The grant will help Flamingo to support its translational research in a Phase II study of its lead clinical programme, danvatirsen, to treat head and neck squamous cell carcinoma.
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Uptick in Clinical Trial Complexity: How Can You Achieve Your Clinical Trial Objectives Faster?
XTalks
JANUARY 8, 2024
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. It’s important to make sure that with this increase in clinical trial complexity, we don’t make our trials overly burdensome to sites or patients,” says Markham.
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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.
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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.
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Codivir Shows Promising Effect Against COVID-19
Pharma Mirror
SEPTEMBER 2, 2021
In-vitro studies conducted at the British virology research laboratory Virology Research Services in London then demonstrated a potent antiviral activity against SARS-CoV-2 and other RNA viruses. The trial was conducted at. The post Codivir Shows Promising Effect Against COVID-19 appeared first on Pharma Mirror Magazine.
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How did pharma develop a vaccine so quickly?
World of DTC Marketing
DECEMBER 29, 2020
Both the Pfizer and Moderna vaccines copied RNA sequence from the virus genome and found a way to manufacture it at scale with high-level processes and quality control. Another consideration is that while in traditional vaccine development, clinical trials are carried out in sequence. Then there are the finances.
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Arrowhead announces push into obesity space with two gene therapies
Pharmaceutical Technology
AUGUST 15, 2024
Clinical trials for the two RNA interference (RNAi) programmes are slated to commence in early 2025.
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BioNTech Founders Predict Cancer Vaccine Is Only Years Away
AuroBlog - Aurous Healthcare Clinical Trials blog
OCTOBER 16, 2022
The husband-and-wife team who co-founded BioNTech, the biotechnology company that partnered with Pfizer to develop an effective messenger-RNA (mRNA) shot against COVID-19, has predicted that a cancer vaccine could be widely available within the next decade.
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Merck and Moderna partner to develop and sell cancer vaccine
Pharmaceutical Technology
OCTOBER 14, 2022
In 2016, the companies entered a strategic partnership to develop novel messenger RNA (mRNA) based PCVs. It is currently being assessed in combination with Merck’s anti-PD-1 therapy, Keytruda, as an adjuvant treatment for high-risk melanoma patients in a Phase II clinical trial being conducted by Moderna.
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Samsung Biologics, GreenLight conclude Covid-19 vaccine engineering run
Pharmaceutical Technology
AUGUST 2, 2022
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. Following the demonstration at Samsung, the clinical trial of GreenLight’s Covid-19 booster vaccine is anticipated to commence this year.
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Cyagen and Neurophth partner to develop gene therapy vectors
Pharmaceutical Technology
NOVEMBER 8, 2022
Additionally, Neurophth will oversee the clinical trials and marketing of gene therapy products developed leveraging the new AAV capsids of Cyagen. Under the collaboration, Cyagen is entitled to get research phase and clinical phase milestone payments and sales-based royalty payments totalling over $140m.
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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.
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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.
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Donidalorsen Shows Sustained HAE Attack Reduction According to New Trial Data
XTalks
FEBRUARY 24, 2025
Carlsbad, California-based Ionis Pharmaceuticals announced it will be sharing promising new trial data for its RNA-targeted prophylactic treatment donidalorsen for hereditary angioedema (HAE) at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI)/World Allergy Organization (WAO) Joint Congress in San Diego, California.
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Jupiter Neurosciences Makes Way in CNS Treatments with $11M IPO
XTalks
DECEMBER 4, 2024
Proceeds from the IPO will propel key initiatives, including the Phase II clinical trial of Jotrol in Parkinson’s disease. While Jupiter faces financial uncertainties and challenges in scaling operations in competitive markets, it remains committed to refining grant applications for proof-of-concept trials.
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Health Canada grants approval for Moderna’s Covid-19 vaccine in children
Pharmaceutical Technology
JULY 15, 2022
Health Canada has granted approval for the usage of Moderna’s messenger RNA (mRNA) Covid-19 vaccine, Spikevax, in a 25µg two-dose regimen for active immunisation to prevent Covid-19 in children aged six months to five years. The KidCOVE trial was carried out at eight Canadian trial sites involving 414 children aged below five years.
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US FDA grants Fast Track status for Avidity’s AOC 1020 to treat FSHD
Pharmaceutical Technology
JANUARY 19, 2023
“We look forward to working collaboratively with the FDA to bring the first RNA therapy directly targeting DUX4 to patients as quickly as possible.” AOC 1020 is currently being studied in the double-blind, randomised, placebo-controlled Phase I/II FORTITUDE clinical trial in nearly 70 FSHD adult patients.
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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
Biology opens up new frontiers in medicine to cure genetic diseases: Kiran Mazumdar Shaw
AuroBlog - Aurous Healthcare Clinical Trials blog
SEPTEMBER 17, 2024
We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics. Biology is opening up new frontiers in medicine.
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Radical Study Proposes a Single Cause to Explain Alzheimer’s Disease
AuroBlog - Aurous Healthcare Clinical Trials blog
FEBRUARY 11, 2025
Researchers from Arizona State University suggest that stress granules protein and RNA clumps that form around cells in stressful […]
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HDT Bio receives Emergency Use Approval for Covid-19 vaccine in India
Pharmaceutical Technology
JUNE 30, 2022
The vaccine leverages self-amplifying RNA (saRNA), which can replicate itself after administration and could be effective at very low doses. At present, the vaccine is being analysed in clinical trials in the US, Brazil and South Korea. HDT Bio CEO Steve Reed said: “Our saRNA vaccine is a game-changer.
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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.
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Myeloid raises funds to support lead cell therapy programme
Pharmaceutical Technology
MAY 19, 2023
Myeloid Therapeutics has raised $73m to support the continued clinical development of its lead cell therapy programme, MT-101, in Phase I/II trials for T cell lymphoma. The financing will help to fast-track the development of other in vivo programming candidates into clinical trials.
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Sanofi’s Qfitlia Gets FDA Approval as First siRNA Therapy for Hemophilia
XTalks
APRIL 1, 2025
XTALKS WEBINAR: Keys to Success in Clinical Trials: A Strategic Guide for Biotechs and Startups Live and On-Demand: Thursday, May 22, 2025 , at 11am EDT (5pm CEST / EU-Central) Register for this free webinar to learn how biotechs can navigate endpoint challenges in clinical trials and accelerate their path to drug approval.
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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? What is a Receptor Occupancy Assay?
Moderna’s Covid-19 shot gets Australian TGA provisional approval for kids
Pharmaceutical Technology
JULY 18, 2022
Moderna has received provisional registration from the Australian Therapeutic Goods Administration (TGA) for its messenger RNA (mRNA) Covid-19 vaccine, Spikevax, for kids aged six months to five years. The 25µg two-dose vaccine regimen is indicated for active immunisation for the prevention of Covid-19.
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Sartorius upscales gene therapy prowess with $2.6bn Polyplus acquisition
Pharmaceutical Technology
APRIL 3, 2023
GlobalData, the parent company of Pharmaceutical Technology, outlines a rise in gene therapy trials in 2023, in line with the growing market Fáber describes. According to GlobalData’s Clinical Trials Database, gene therapy trials represent a 0.91% share of all planned trials in 2023, up from just 0.25% in 2014.
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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.
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US FDA grants Fast Track status for Pfizer-BioNTech’s combination vaccine
Pharmaceutical Technology
DECEMBER 11, 2022
In November, the companies announced plans for a Phase I clinical trial of the combined vaccine candidate in healthy adult subjects. The first subject was dosed in the trial, which is designed to evaluate the safety, tolerability and immunogenicity of a nucleoside-modified RNA-based combination vaccine approach.
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Opinion: As it turns 40, the Orphan Drug Act for rare diseases needs a refresh
STAT News
JANUARY 30, 2023
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. New ways of conducting clinical trials have also emerged. The human genome was sequenced in 2003.
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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.
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Highlight Therapeutics trials second-line melanoma combo after immunotherapy fails
pharmaphorum
SEPTEMBER 2, 2020
Spain’s Highlight Therapeutics is attempting to get patients with melanoma to respond to Keytruda by combining with its RNA-based cancer therapy in patients with melanoma who have progressed despite treatment with PD-1 therapies such as Keytruda or BMS’ rival Opdivo (nivolumab). A total of 40 patients are planned to be enrolled.
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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.
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?5 Things You Need to Know from Our Recent Gene Therapy Webinar
Worldwide Clinical Trials
JUNE 15, 2022
To discuss your study and how to reduce the chances of receiving a clinical hold, talk with us. . The pandemic has had a lasting impact on the ability of sites to participate in trials, particularly in the US. We need to consider how to use diverse populations in gene therapy trials.
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LIB Therapeutics’ Cholesterol Lowering Med Lerodalcibep Receives FDA BLA Acceptance
XTalks
FEBRUARY 12, 2025
Current PCSK9 inhibitors on the market include Regenerons monoclonal antibody Praluent (alirocumab), Amgens Repatha (evolocumab) and Novartiss Leqvio (inclisiran), which is a small RNA interference (siRNA) therapy designed to lower LDL-C. Results from the trials demonstrated that lerodalcibep achieved significant reductions in LDL-C levels.
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Royalty Pharma acquires royalty interest in olpasiran for $250m
Pharmaceutical Technology
NOVEMBER 10, 2022
Olpasiran is entering a Phase III clinical trial to evaluate if the siRNA therapy can reduce cardiovascular event risk in atherosclerotic cardiovascular disease patients with increased Lp(a) levels. . The post Royalty Pharma acquires royalty interest in olpasiran for $250m appeared first on Pharmaceutical Technology.
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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.
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Sanofi and Translate Bio initiate Phase 1 clinical trial of mRNA influenza vaccine
The Pharma Data
JUNE 26, 2021
Sanofi and Translate Bio initiate Phase 1 clinical trial of mRNA influenza vaccine. The trial will evaluate the safety and immunogenicity of a monovalent flu vaccine candidate coding for the hemagglutinin protein of the A/H3N2 strain of the influenza virus. JUNE 22 , 2021.
The siRNA STP705 by Sirnaomics Has Promise for Treating in situ Squamous Cell Carcinoma (isSCC)
XTalks
JUNE 20, 2022
Small interfering RNAs (siRNAs) are a promising class of therapeutic modalities that involve a double-stranded RNA molecule (20 to 25 bp long) which can target and cease the translation of a disease-related mRNA. Clinical Trial Outcomes of isSCC Treatment with STP705. Sirnaomics’ Current Research.
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