Pharma Mirror

APRIL 21, 2024

This represents an increasingly promising field of precision medicine, holding significant prospects for preventing and treating numerous challenging or genetic diseases. Messenger RNA (mRNA) is a single-stranded ribonucleic acid transcribed from the DNA chain, carrying the encoding information for protein synthesis.

In-Vitro 130

In-Vitro Drugs Genetic Disease RNA 130

XTalks

DECEMBER 4, 2024

The company has secured Orphan Drug designations for Friedreich’s ataxia, offering benefits like market exclusivity and eligibility for Priority Review vouchers. CAMP4’s RNA-based therapies focus on genetic diseases like urea cycle disorders, while Rapport’s small molecules aim to address epilepsy, pain and bipolar disorder.

Bioavailability 111

Bioavailability Genetic Disease Marketing RNA 111

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. These vaccines were developed using different “platform technologies” that involve slotting genetic material from the virus into a tried and tested delivery package.

Vaccine 307

Vaccine Vaccination Development Clinical Trials 307

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.

Trials 59

Trials RNA Allergies Drugs 59

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

pharmaphorum

JANUARY 26, 2023

We are already seeing an increase in projects exploring population genomics in Africa, the Middle East, and Asia, with initiatives including the GenomeAsia100K Project and the Genome Aggregation Database focusing on capturing genetic data of non-European individuals. Just one mutation can lead to a complete change in protein formation.

Genome 129

Genome Genomics Genome Project Genetics 129

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

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.

FDA Approval 98

FDA Approval Protein Genetics RNA 98

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

APRIL 1, 2025

Hemophilia A and B are rare genetic bleeding disorders caused by a deficiency or dysfunction of clotting factors VIII (FVIII) and IX (FIX), respectively. Through RNA silencing, it targets the expression of antithrombin, a protein that inhibits blood clotting. The frequent intravenous infusions can be challenging for patients.

FDA Approval 59

FDA Approval RNA Clinical Trials Clinical Trials 59

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

pharmaphorum

JANUARY 22, 2021

According to the US biopharma company, genetic analyses of publicly available sequences for the new variants of SARS-CoV-2 suggest the mutations in the viral spike protein that have made them more transmissible shouldn’t affect the way Veklury (remdesivir) works. “We

In-Vitro 141

In-Vitro RNA Licensing Licensing 141

XTalks

DECEMBER 22, 2021

The RNA Revolution: From mRNA Vaccines to RNA Editing. The age of RNA is officially here, and it’s here to stay as more than a passing life science trend. RNA technology is not new nor has its potential been surprising. RNA in the Making. So why did this perceived RNA ‘revolution’ take so long?

Life Science 98

Life Science RNA Vaccination Vaccine 98

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.

Genomics 70

Genomics Genome RNA Protein 70

XTalks

OCTOBER 8, 2024

In a statement , the Nobel Assembly said the laureates discovered a new class of non-coding RNA molecules that play a crucial role in gene regulation. To activate these instructions, cells create a copy called messenger RNA (mRNA), which exits the nucleus and directs the cell’s machinery to produce a specific protein.

Regulation 52

Regulation Genetics Scientist RNA 52

Roots Analysis

OCTOBER 22, 2023

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers.

RNA 40

RNA Protein Genetics Gene Expression 40

Worldwide Clinical Trials

MAY 30, 2024

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. The shared placebo design is another approach to accelerating drug development in ALS research.

Gene 204

Gene Clinical Trials Clinical Trials Gene Therapy 204

STAT News

JANUARY 25, 2023

From Covid-19 vaccines to therapies against a range of deadly diseases, every RNA molecule has a complex 3D shape that controls its function. But the startup already has big ambitions for how its AI-based approach can both help biotechs identify RNA-targeting drugs and design RNA to be stable, compact, or to have other desired properties.

RNA 52

RNA Vaccination Vaccine Development 52

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.

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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.

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Protein RNA Genome Genomics 130

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, another antiviral drug candidate, was originally developed to treat influenza.

RNA 52

RNA Drugs Genome Genomics 52

pharmaphorum

JANUARY 10, 2022

Previously, messenger RNA (mRNA) therapies were a niche part of the global R&D pipeline, now a wide section of the public is at least familiar with the name of this type of therapy. The company has also stated, at the same time as the study was announced, that it will look at developing mRNA technology in oncology and genetic diseases.

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RNA Vaccination Vaccine Pharma Companies 111

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.

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RNA Vaccine Vaccination 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

pharmaphorum

DECEMBER 13, 2021

One of its other scientific legacies was the specific development and approval of mRNA vaccines, the first in that drug class on the market. Dormitzer comes from Pfizer, where he was chief scientific officer for RNA and viral vaccines for more than six years. For instance, in March, Belgian immunotherapy firm eTheRNA received a €6.9

RNA 105

RNA Vaccination Vaccine Development 105

Delveinsight

SEPTEMBER 23, 2021

Boehringer Ingelheim acquires Abexxa Biologics for precision most cancers drug pipeline. 858 Therapeutics closes USD 60 Million series A round to drug RNA modulation. Now, they are ready for their next venture, which is launching RNA biotech 858 Therapeutics with a USD 60 million Series A fundraising. Stafford and co.

RNA 61

RNA Gene Therapy Gene Protein 61

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

Scienmag

SEPTEMBER 30, 2020

Scientists are fighting back with multiple strategies, including vaccines, repurposed drugs developed for other diseases and brand-new therapies. Now, researchers reporting in ACS Central Science have identified small molecules that target a structure within the RNA genome […].

RNA 40

RNA Genomics Genome Branding 40