Scienmag

JULY 2, 2021

Studies using human cell lines and tumors grown in mice provide early evidence that inhibiting RNA-binding proteins, a previously overlooked family of molecules, might provide a new approach for treating some cancers Credit: UC San Diego Health Sciences In cancer research, it’s a common goal to find something about cancer cells — some sort (..)

RNA 87

RNA Protein Drugs Research 87

pharmaphorum

NOVEMBER 1, 2022

14th Annual RNA Therapeutics. Investigating the next generation of genetic medicine through RNA based therapies. RNA therapeutics is a rapidly expanding industry with increasingly growing potential for immunotherapy, personalised medicines, and treatment of genetic, infectious, and chronic diseases.

RNA 52

RNA Genetics Gene Editing In-Vitro 52

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. At least 370 genetic disorders are known to derive from errors introduced during this splicing process.

RNA 60

RNA Licensing Licensing Genetics 60

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 52

RNA Genetic Disease Drugs Protein 52

Worldwide Clinical Trials

AUGUST 14, 2024

Historically, these indications have challenged the one-size-fits-all treatment approach due to patient variability, such as genetic differences in drug metabolism and underlying health conditions. This resistance may occur due to genetic mutations, epigenetic changes, or a tumor’s microenvironment.

Genetics 195

Genetics Protein Gene Genotype 195

Pharmaceutical Technology

JANUARY 12, 2023

Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM). We believe the advanced RNA technology and human disease models of Phlox Therapeutics perfectly partner with the delivery tools Solid Biosciences brings to this collaboration.”.

RNA 130

RNA Gene Therapy Genetics Genetic Disease 130

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.

RNA 105

RNA Genetics Dermatology Antibody 105

XTalks

AUGUST 5, 2020

In recent webinars by Genuity Science, formerly known as WuXi NextCODE, experts from the biotech and pharma industries spoke about leveraging the power of single cell RNA sequencing platforms and solutions in conjunction with machine learning technologies such as AI in cell biology and disease research. The Power of Single Cell Technology.

Gene Expression 112

Gene Expression Development RNA Drugs 112

pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. In three more patients treated at 0.1mg/kg levels fell by 52%.

In-Vivo 105

In-Vivo Gene Editing Trials Drugs 105

XTalks

JANUARY 21, 2025

Agent drug-coated balloon. The balloons outer surface is coated with the drug paclitaxel, a safe and effective measure to prevent the arteries from narrowing again. There, plasma is separated, and tumor DNA is identified by detecting genetic mutations, methylation patterns and fragmentation signals.

FDA Approval 105

FDA Approval Manufacturing Development RNA 105

Pharmaceutical Technology

DECEMBER 14, 2022

GSK has entered a strategic partnership with Wave Life Sciences to progress the discovery and development of oligonucleotide therapies for new genetic targets. The alliance will merge the PRISM oligonucleotide platform of Wave and the capabilities of GSK in genetics and genomics.

Life Science 162

Life Science Development Gene Therapy Genetics 162

Pharmaceutical Technology

APRIL 24, 2023

Since then, the field of nanomedicine has steadily progressed to reach high points such as the successful use of nanotechnology to deliver messenger RNA (mRNA)-based Covid-19 vaccines. Since then, the drug has been used off-label in breast cancer and other types of cancer. Langer, now a David H.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147

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.

Genome 70

Genome Genomics RNA Protein 70

Scienmag

FEBRUARY 3, 2022

In a Perspective, Ronald Swanstrom and Raymond Schinazi argue that antiviral drugs that lead RNA viruses to produce genetic mutations so fast that they cannot remove deleterious ones – a strategy used in antivirals like the recently approved COVID-19 drug molnupiravir – may be viable, but also present unknown risks to the host.

RNA 66

RNA Genetics Drugs 66

Pharmaceutical Technology

JULY 5, 2022

It was also found in lab experiments that ZCCHC14, a protein that interacts with zinc, attaches to certain part of HAV’s ribonucleic acid (RNA), enabling the virus to recruit TENT4 from the human cell. Subsequently, Lemon’s lab tested RG7834 and found the precise effects of the oral drug on HAV in liver and faeces.

Protein 130

Protein RNA Genome Genomics 130

Roots Analysis

OCTOBER 30, 2023

In the past few years, Next Generation RNA therapeutics have emerged as one of the key therapeutic modalities in the modern healthcare industry. These RNA based therapeutics play a crucial role in protein production and regulation of gene functions.

RNA 40

RNA Vaccination Vaccine Development 40

XTalks

OCTOBER 26, 2021

This latest data may have finally won over NICE to reverse its decision and grant approval to the drug. Givlaari was approved by the European Medicines Agency (EMA) in March 2020 and by the US Food and Drug Administration ( FDA ) in 2019. The treatment will be offered through the UK’s National Health Service (NHS).

Gene Silencing 98

Gene Silencing Gene Clinical Trials Clinical Trials 98

Roots Analysis

FEBRUARY 23, 2022

Further, NGS library preparation kits offer several benefits, such as low requirement of starting material, generation of longer RNA / DNA sequences and production of superior quality nucleotide libraries, thereby, catering to existing unmet needs of the research domain. Next Generation Sequencing (NGS) Library Preparation Kits. Web: [link].

Genetics 52

Genetics DNA RNA Reagent 52

pharmaphorum

SEPTEMBER 21, 2020

A group of rare genetic disorders, FCS affects between 55 and 110 people in England and causes very high levels of triglyceride fat in the blood. It is the first and only drug available to treat FCS since being approved by the EMA last year. This news is a real step change for patients with FCS.”.

Drugs 80

Drugs RNA Medicine Genetics 80

pharmaphorum

AUGUST 10, 2021

Oxford University and UK clinical artificial intelligence (AI) company Sensyne Health will partner on a project to find new drug targets for people with hard-to-treat asthma. Dr Timothy Hinks. According to Hinks, “we still don’t understand the causes of asthma, because in the past the scientific tools just didn’t exist.

Gene Sequencing 116

Gene Sequencing Gene Bioinformatics Genome 116

Delveinsight

AUGUST 26, 2021

Shape’s RNA editing technologies can modify the RNA sequence, which makes the body’s protein building blocks. This system is designed to deliver RNA editing technology or other payloads directly to particular body areas, such as the nervous system or muscle. Datavant inks real-world data partnership with Real Chemistry.

Vaccination 98

Vaccination Vaccine Gene Therapy RNA 98

BioTech 365

NOVEMBER 23, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Folding of SARS-CoV2 genome reveals drug targets—preparation for SARS-CoV3.The The genetic code of the SARS-CoV2 virus is exactly 29,902 characters long, strung through a long RNA molecule.

Genome 40

Genome Genomics RNA Drugs 40

pharmaphorum

OCTOBER 21, 2021

UK cost-effectiveness watchdog NICE is set to recommend NHS use of Alnylam’s gene-silencing therapy Givlaari in England and Wales for the rare disease acute hepatic porphyria (AHP), after its advisors issued a positive verdict on the drug. It was approved by the European Medicines Agency (EMA) last year.

Gene Silencing 98

Gene Silencing Gene RNA Drugs 98

pharmaphorum

NOVEMBER 7, 2022

Final results of the OCEAN(a)-DOSE trial – published in the New England Journal of Medicine – showed that the small, interfering RNA (siRNA) drug reduced Lp(a) levels compared to placebo at all four doses tested, with doses above 75 mg given every 12 weeks cutting levels by 95% or more after six months.

Drugs 59

Drugs RNA Medicine Genetics 59

pharmaphorum

JUNE 1, 2022

The US biotech has just reported phase 2 trial results with the small, interfering RNA (siRNA) gene-silencing drug showing that it an cause a 90% or greater reduction in Lp(a) levels – a risk factor for cardiovascular disease – that was sustained over 48 weeks of follow-up.

Gene Silencing 59

Gene Silencing Drugs Medicine RNA 59

Scienmag

SEPTEMBER 30, 2020

Researchers engineer RNA-targeting compounds that disable the pandemic coronavirus’ replication engine Credit: Scott Wiseman for Scripps Research JUPITER, FL – Sept.

Engineer 68

Engineer RNA Research Drugs 68

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. These adverse effects were observed in approximately 5 percent of patients receiving treatment with Wainua.

FDA Approval 105

FDA Approval Protein RNA Clinical Trials 105

pharmaphorum

MAY 6, 2021

Thalassaemia is a severe genetic disease that is characterised by significantly reduced production of functional beta-globin, a component of haemoglobin, the oxygen-carrying protein in the blood. Severely-affected patients need regular blood transfusions to maintain their haemoglobin levels. .

Genetic Disease 104

Genetic Disease Protein RNA Doctors 104

XTalks

JANUARY 3, 2025

Ionis Pharmaceuticals recently announced that its drug, Tryngolza (olezarsen), has been approved as an adjunct to diet for the reduction of triglyceride levels in FCS patients, who have long faced a condition with no existing pharmacological solutions. In the Balance study, patients who received the drug showed a 42.5

FDA Approval 59

FDA Approval Drugs Clinical Trials Clinical Trials 59

pharmaphorum

NOVEMBER 24, 2020

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. Alnylam is hoping that revenue will stream from its new products and payments from Novartis, which owns the rights to cholesterol lowering drug inclisiran.

FDA Approval 52

FDA Approval Gene Silencing Drugs RNA 52

The Pharma Data

NOVEMBER 17, 2021

Mount Sinai researchers have developed a new model that uses DNA and RNA sequencing data from hundreds of patients to identify specific genes and genetic alterations responsible for never-before-defined subtypes of a blood cancer called multiple myeloma. doi: 10.1126/sciadv.abg9551. Source link: [link].

Scientist 52

Scientist RNA DNA Genetics 52

pharmaphorum

JULY 28, 2022

The RNA interference specialist says its researchers and collaborators have identified mutations in a gene called INHBE that are associated with protection against abdominal obesity and metabolic syndrome – a condition impacting more than 20% of adults worldwide. . Image by Bruno /Germany from Pixabay .

Biobanking 52

Biobanking Gene Gene Silencing Drugs 52

Roots Analysis

OCTOBER 13, 2023

Over the last two decades the pharmaceutical industry has observed a paradigm shift from conventional drug delivery strategies to more enhanced, potent, and targeted therapeutics. This led the researchers to find alternative pathways and drug delivery strategies to enhance the potent delivery of the drug to the site of action.

Drugs 40

Drugs Antibody RNA Genetics 40

pharmaphorum

NOVEMBER 18, 2021

The two companies have been working together since 2019 to develop multiple RNA interference (RNAi) candidates for disorders such as non-alcoholic steatohepatitis (NASH), type 2 diabetes, obesity and rare diseases. Dicerna has been a hot tip for takeover for a couple of years, as the list of approved drugs that work via RNAi has lengthened.

Gene Silencing 52

Gene Silencing RNA Protein Gene 52

Roots Analysis

MARCH 16, 2023

It is important in the process of protein synthesis because mRNA is responsible for transferring genetic information from DNA to ribosomes, which then decodes the genetic information into a protein. In the last few years, researchers have become interested in using in vitro transcribed (IVT) mRNA as a drug delivery agent.

Vaccination 52

Vaccination Vaccine DNA Protein 52

Roots Analysis

AUGUST 12, 2024

These diagnostic solutions are essential for detecting and monitoring diseases, identifying genetic abnormalities, and guiding personalized treatment plans. Molecular diagnostic solutions are pivotal across various medical fields, including oncological disorders, infectious diseases, genetic testing, and personalized medicine.

Genetics 40

Genetics Gene Expression RNA DNA 40

The Pharma Data

MARCH 28, 2022

“C9orf72-associated ALS is a complex genetic form of ALS and there are multiple mechanisms by which the scientific community believes the C9orf72 gene causes disease. Today, the company has a pipeline of several investigational drugs being evaluated in ALS, including tofersen, BIIB105 and BIIB100. About Ionis Pharmaceuticals, Inc.

Drugs 52

Drugs Genetics RNA Development 52