Wave sees RNA editing validation in early trial results
Bio Pharma Dive
OCTOBER 16, 2024
The results provide the first clinical evidence of RNA editing, a burgeoning field that's drawn interest from biotechs and pharmaceutical companies alike.
BioNTech’s RNA vaccine sparks potential in pancreatic cancer
Pharmaceutical Technology
MAY 11, 2023
The vaccine, developed by BioNTech, led to half of the patients with pancreatic cancer in the Phase I trial remaining cancer-free 18 months later. A global randomised follow-up trial is planned. In 2016, BioNTech and Genentech, a part of Roche signed an agreement to develop personalised mRNA therapies in oncology.
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Pfizer begins plan to challenge Shingrix with an mRNA vaccine
Bio Pharma Dive
FEBRUARY 10, 2023
The pharma and partner BioNTech have kicked off the first human trial of a messenger RNA shot for shingles, believing there’s room to clear the high bar set by GSK’s rival vaccine.
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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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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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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. It will also support the firm’s preclinical effort on the long non-coding RNA (LncRNA) programme, FTX-001, that targets MALAT-1. Flamingo Therapeutics has received a research grant of €1.7m
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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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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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RNA editing biotech Airna adds $155M in funding
Bio Pharma Dive
APRIL 1, 2025
Airna will use the Series B round to launch a Phase 1/2 trial of its lead program for alpha-1 antitrypsin deficiency, a disease targeted by several other firms.
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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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Novo Nordisk agrees $1.1bn takeover of RNA biotech Cardior
pharmaphorum
MARCH 25, 2024
billion, adding expertise in RNA therapeutics and a heart failure therapy in mid-stage trials. Novo Nordisk has agreed to buy Cardior Pharmaceuticals for $1.1
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RNA glioblastoma vaccine shows promise in first human trial
pharmaphorum
MAY 2, 2024
A personalised vaccine for the aggressive brain cancer glioblastoma developed has shown encouraging signs of efficacy in its first human trial.
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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. In 2022, the company also started five first-in-human clinical trials from its oncology programme.
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Eli Lilly Pursues RNA Editing in New Partnership with ProQR
XTalks
SEPTEMBER 13, 2021
With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.
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Circular RNA: Vaccines, therapeutics and biomarkers could be revolutionised
Pharmaceutical Technology
MAY 15, 2024
CircRNA is still in early days of development, but could be in trials as vaccines, therapeutics and biomarkers in the next few years.
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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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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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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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Alnylam Files Lawsuits Against Pfizer and Moderna Over Patent Infringement of RNA Delivery Technology
XTalks
MARCH 21, 2022
Credited as a pioneering RNA interference (RNAi) therapeutics company, Alnylam Pharmaceuticals is taking Pfizer and Moderna to court, claiming that the companies’ use of the lipid nanoparticle (LNP)-based RNA delivery technology in their mRNA COVID-19 vaccines infringes on a patented technology.
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Shifting focus, ProQR to seek partners for stalled eye drugs
Bio Pharma Dive
AUGUST 11, 2022
The company will wind down five trials involving two drug candidates and lay off more staff, prioritizing instead its RNA-editing technology.
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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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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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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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Alnylam CEO touts 'big-win scenario' for Amvuttra in closely watched heart disease trial
Fierce Pharma
JUNE 24, 2024
In one of the most closely watched biotech trial readouts of the year, Alnylam Pharmaceuticals said its RNA interference drug reduced the risk of death or recurrent cardiovascular events in patient | In one of the most closely watched biotech trial readouts of the year, Alnylam Pharmaceuticals said its RNA interference drug reduced the risk of death (..)
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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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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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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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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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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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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. Both companies will assess the new AAV vectors’ functional effects in rodent and NHP models.
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RNA therapy firm Avidity files $400m placement
pharmaphorum
MARCH 3, 2024
Avidity Biosciences files $400m private placement as it prepares for phase 3 trial of lead antibody oligonucleotide conjugate AOC 1001 in rare disease DM1
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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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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. 1455NO-HEALTH-CORONAVIRUS_VACCINES_PFIZER_O_.
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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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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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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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HIV vaccines suffer setbacks but new approaches could turn the tide
Pharmaceutical Technology
FEBRUARY 3, 2023
Only a few weeks into the new year, the prospect of getting a successful advanced HIV vaccine shrank after the discontinuation of yet another late-stage trial. This marks the second time one of Janssen’s HIV vaccines failed after another showed disappointing results in the Phase IIb Imbokodo trial in August 2021.
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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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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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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. Read the rest…
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Alnylam changes analysis plan for key Amvuttra heart trial, jolting investors
Fierce Pharma
FEBRUARY 15, 2024
Changing a clinical trial’s statistical analysis plan on the cusp of a readout? Changing a clinical trial’s statistical analysis plan on the cusp of a readout? That’s exactly what Alnylam just did for a closely watched study of its next-generation RNA interference therapy Amvuttra in a rare heart disease.
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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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FDA grants EUAs for Moderna and Pfizer-BioNTech’s bivalent Covid-19 boosters
Pharmaceutical Technology
SEPTEMBER 1, 2022
These 'updated boosters' comprise the SARS-CoV-2 virus’ two messenger RNA (mRNA) components, one from the initial strain and the other one common between the BA.4 According to the amended EUA, the vaccines are indicated to be administered at a minimum of two months after the initial or booster dose. 5 lineage of the Omicron variant.
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BioSig abandons COVID-19 drug trial on safety concerns
pharmaphorum
OCTOBER 27, 2020
US biotech BioSig Technologies has abandoned a phase 2 trial of its antiviral drug merimepodib with Gilead’s Veklury in severe COVID-19 patients, after concluding the safety of the combination was in doubt. However the Grade 3 group had “markedly different outcomes… making it unlikely that the trial would meet its primary safety endpoints.”.
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