XTalks

DECEMBER 4, 2024

Conventional resveratrol products often face challenges like poor absorption and significant gastrointestinal side effects. 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

Medical Xpress

NOVEMBER 8, 2022

Researchers at the University Hospital Bonn (UKB) and the Technical University (TU) Dresden have investigated an underlying mechanism and deciphered the driving force of uncontrolled interferon production through studying the rare autoimmune disease Aicardi-Goutières syndrome.

RNA 98

RNA Genetics Medicine Production 98

Scienmag

NOVEMBER 13, 2020

In a screening for a functional impact to the neuronal differentiation process, Danish researchers identified a specific circular RNA, circZNF827, which surprisingly “taps the brake” on neurogenesis.

RNA 93

RNA Regulation Protein Production 93

Pharmaceutical Technology

MAY 25, 2023

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.

Gene Therapy 243

Gene Therapy Gene Gene Editing Development 243

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

Scienmag

AUGUST 4, 2020

Led by Boyce Thompson Institute’s Andrew Nelson, four partners will identify RNA modifications and develop resources that may lead to hardier crops Credit: Photo credit: Anna Nelson Dittrich ITHACA, NY, August 4, 2020 — RNA perform a variety of functions in cells, helping with everything from regulating genes to building proteins.

RNA 74

RNA Bioinformatics Protein Gene 74

Pharmaceutical Technology

MARCH 28, 2023

The system comprises the Cas9 enzyme and a guide RNA. The company will also be eligible to receive research and development milestones of up to an additional $230m, along with royalties on any future products resulting from the deal.

Gene Editing 235

Gene Editing Gene DNA Gene Therapy 235

Pharmaceutical Technology

APRIL 26, 2023

MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. The new deal excludes oncology and other therapeutic areas outside the scope of genetic disease.

Development 130

Development Genetic Disease Genetics Gene Therapy 130

Pharmaceutical Technology

NOVEMBER 10, 2022

According to the deal, Royalty Pharma will acquire Arrowhead’s complete royalty interest in olpasiran, which amounts to a royalty of up to the low double digits on global net product sales. . The siRNA therapy is created to reduce lipoprotein(a) [Lp(a)] levels, which is a genetically decided, independent cardiovascular disease risk factor.

RNA 130

RNA Sales Clinical Trials Clinical Trials 130

Medical Xpress

FEBRUARY 9, 2023

When cells copy DNA to produce RNA transcripts, they include only some chunks of genetic material known as exons and throw out the rest. The resulting product is a fully-mature RNA molecule, which can be used as a template to build a protein.

RNA 75

RNA DNA Gene Genetics 75

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

Scienmag

DECEMBER 16, 2020

New research from the Whitehead Institute suggests that the products of transcription — RNA molecules — regulate their own production through a feedback loop Credit: Jon Henninger/Whitehead Institute At any given moment in the human body, in about 30 trillion cells, DNA is being “read” into molecules of messenger RNA, the intermediary (..)

RNA 52

RNA DNA Regulation Production 52

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

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

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

Scienmag

JANUARY 5, 2021

Review of the life histories of RNA viruses, their genetics, and the compounds that could disrupt them offers hope for curtailment of future pandemics Credit: Illustration: Jennifer Matthews/Scripps Oceanography Researchers at Scripps Institution of Oceanography and Skaggs School of Pharmacy and Pharmaceutical Sciences at the University of California (..)

Production 52

Production RNA Genomics Genome 52

Worldwide Clinical Trials

APRIL 15, 2025

Mutations in genes such as SOD1, C9orf72, FUS, and TARDBP further support the role of proteinopathy and RNA dysregulation in ALS pathogenesis. Integration of fluid biomarkers, imaging, and genetic profiling enhances diagnostic precision and provides valuable tools for clinical trials in ALS.

Clinical Trials 100

Clinical Trials Clinical Trials Trials Protein 100

XTalks

JANUARY 21, 2025

There, plasma is separated, and tumor DNA is identified by detecting genetic mutations, methylation patterns and fragmentation signals. ColoSense is a laboratory test that detects eight different RNA biomarkers and trace amounts of hemoglobin (as a measure of blood) in human stool that is not visible to the naked eye (fecal occult blood).

FDA Approval 105

FDA Approval Manufacturing Development RNA 105

Pharmaceutical Technology

FEBRUARY 23, 2023

The collaboration will combine the mRNA platform of Moderna with the gene editing technologies suite, including the base editing capabilities of Life Edit for the development of curative therapies to treat challenging genetic diseases. Life Edit will get an upfront payment and is eligible for potential milestone payments.

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

Roots Analysis

FEBRUARY 23, 2022

Over the past few years, several companies have started offering a diverse range of genome sequencing products and services using various second and third generation sequencing technologies. Currently, close to 60 industry players are actively offering NGS kits for preparing DNA / RNA libraries. Players Engaged in the Domain.

Genetics 52

Genetics DNA RNA Reagent 52

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 Vaccine Vaccination Development 40

XTalks

OCTOBER 26, 2021

AHP is a life-threatening genetic condition caused by an inherited deficiency in the metabolic enzyme hydroxymethylbilane synthase (HMBS), also known as porphobilinogen deaminase (PBGD). Givlaari reduces ALAS1 levels through RNA interference-mediated gene silencing, which targets ALAS1 mRNA. “This is a huge development for patients.”.

Gene Silencing 98

Gene Silencing Gene Clinical Trials Clinical Trials 98

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

XTalks

AUGUST 10, 2021

A porous membrane was engineered to capture RNA on its surface. A CRISPR-based reaction generates a fluorescent signal in the presence of SARS-CoV-2 viral RNA in a saliva sample. The device costs about $15, but mass production would bring the housing costs down to about $3. Image source: Wyss Institute at Harvard University.

RNA 105

RNA Containment DNA Engineer 105

Scienmag

MARCH 4, 2021

Findings explain how plants use RNA to defend against fungal invaders Credit: Nicole Ward Gauthier/University of Kentucky New research reveals an essential step in scientists’ quest to create targeted, more eco-friendly fungicides that protect food crops.

Protein 95

Protein RNA Scientist Manufacturing 95

pharmaphorum

DECEMBER 1, 2020

The FDA and the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) have already begun their reviews and similar rolling submissions have begun in countries including Australia, Canada and Japan. The EMA has also confirmed it has received a filing from Moderna, which is producing a rival vaccine based on similar RNA technology.

Vaccine 88

Vaccine Vaccination RNA Clinical Trials 88

pharmaphorum

AUGUST 10, 2021

The partners will also examine the transcriptomes of around 60 participants – in other words the messenger RNA coded for by genes that in turn is used to produce proteins – and carry out bioinformatic analyses to tease out new pathways involved in asthma.

Gene Sequencing 116

Gene Sequencing Gene Bioinformatics Genome 116

Scienmag

JULY 1, 2021

Almost twenty years ago, the process of RNA silencing was discovered in plants, whereby small fragments of RNA inactivate a portion of a gene during protein synthesis.

RNA 60

RNA Protein Research Gene 60

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.

Vaccine 98

Vaccine Vaccination Gene Therapy RNA 98

Scienmag

NOVEMBER 1, 2020

Scientists from an international group led by the RIKEN Center for Integrative Medical Sciences and Yokohama City University have discovered that a pair of proteins play a key role in allowing an important type of functional non-coding RNA, known as SINEUPs, to act to promote their target messenger RNA.SINEUPs are a recently discovered type of […]. (..)

Protein 45

Protein Production RNA Research 45

pharmaphorum

JUNE 28, 2021

Intellia and Regeneron’s in vivo approach involves administration of a gene-editing drug – in the form of a CRISPR mRNA and guide RNA packaged in a lipid nanoparticle – directly into the body. . — 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

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 Doctor 104

The Pharma Data

JANUARY 19, 2021

A range of companies are exploring potential gene editing therapies based on a CRISPR-Cas9 system, and the ability to tune the CRISPR cut could prevent off-target editing or control the degree of a target protein’s production. The authors found that tracr-L redirects Cas9 in S. The authors suggest natural tracr-L regulates autoimmunity.

Bacteria 52

Bacteria Gene Editing Research RNA 52

pharmaphorum

OCTOBER 21, 2021

Givlaari (givosiran) has been recommended as a treatment option for people aged 12 and over with AHP, a rare life-threatening genetic condition that causes attacks of severe abdominal pain, vomiting and nervous system disorders, such as seizures, depression and anxiety. It was approved by the European Medicines Agency (EMA) last year.

Gene Silencing 98

Gene Silencing Gene RNA Drugs 98

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

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. If approved, it could become a $500 million-a-year product, according to the company. billion in cash.

Gene Silencing 52

Gene Silencing RNA Protein Gene 52

Roots Analysis

FEBRUARY 21, 2022

However, the conventional nucleotide library preparation process has several challenges, including requirement of huge amount of starting materials, inadequate throughput and deteriorated quality of the product. As per our analysis, majority of the kits use purified RNA / DNA as the input sample. Product Competitiveness Analysis.

Marketing 52

Marketing Gene Sequencing DNA Genome 52