BioSpace

JUNE 20, 2022

Therorna brought in $42 million to advance its circular RNA platform, while Carbon Biosciences banked $38 million to develop therapies for genetic diseases, beginning with cystic fibrosis.

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Genetic Disease RNA Genetics Development 97

Scienmag

JULY 1, 2022

Therapeutics based on messenger RNA, or mRNA, can potentially treat a wide range of maladies, including cancer, genetic diseases, and as the world has learned in recent years, deadly viruses.

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Genetic Disease RNA Genetics 67

BioPharma Reporter

JULY 21, 2022

US biotech, CAMP4 Therapeutics, has secured US$100m in a Series B financing round that it says will be used to accelerate expansion of its regulatory RNA (regRNA) platform.

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Genetic Disease RNA Genetics Regulation 52

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.

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Development Genetic Disease Genetics Gene Therapy 130

Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. DS: You describe your therapeutics as RNA Amplifiers. How do they work?

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Gene Expression Gene Genetic Disease RNA 59

Pharmaceutical Technology

JANUARY 12, 2023

The collaboration will use the vector biology, manufacturing capabilities and the drug development experience of Solid Biosciences and Phlox Therapeutics’ knowledge in genetic cardiomyopathies and RNA therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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RNA Gene Therapy Genetics Genetic Disease 130

XTalks

JANUARY 5, 2022

These include the continued promise of RNA-based vaccines as well as therapeutics based on RNA editing approaches for the treatment of various conditions including rare genetic diseases. Ayesha also discussed some of the predicted trends in the life sciences for 2022.

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Life Science RNA Vaccination Vaccine 52

Drug Discovery World

JANUARY 11, 2024

Researchers have used messenger RNA (mRNA) to create an effective therapy for a rare liver disease in preclinical studies, demonstrating the technology’s potential therapeutic use in people. Patients affected by the disease are found to also experience an imbalance of glutathione regulation, which is important for liver detoxification.

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Research Gene Therapy RNA Gene 59

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.

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RNA Genetics Dermatology Antibody 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.

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Gene Editing Gene In-Vivo Gene Therapy 147

Drug Discovery World

FEBRUARY 15, 2024

The strengths of etherna’s lipid based nanoparticle (LNP) platform have been showcased in a peer-reviewed paper in Advanced Functional Materials. etherna’s Vice President, Technology & Innovation, Stefaan De Koker described the paper as a “landmark” for the company and its technology.

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Immune Response In-Vivo Vaccination Vaccine 52

Delveinsight

AUGUST 26, 2021

Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of gene therapies for Alzheimer’s and Parkinson’s disease. Shape’s RNA editing technologies can modify the RNA sequence, which makes the body’s protein building blocks.

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Vaccination Vaccine Gene Therapy RNA 98

Camargo

JULY 27, 2021

Research and development in the area is currently growing at a fast rate, and the National Institute of Health reports hundreds of clinical trials to test gene therapies for different genetic diseases, immune system disorders, oncology treatments, neurogenerative diseases, infectious diseases, and more.

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Gene Therapy Gene Gene Editing Genetics 238

Roots Analysis

FEBRUARY 21, 2022

These kits require less input sample, produce sufficient DNA / RNA libraries with minimum errors and are compatible with a broad range of input samples, including blood, cells, plasma and saliva. As per our analysis, majority of the kits use purified RNA / DNA as the input sample. Diverse Landscape of NGS Library Preparation Kits.

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Marketing Gene Sequencing DNA Genome 52

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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RNA Genetics Genetic Disease Medicine 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. .

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Genetic Disease Protein RNA Doctors 104

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.

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In-Vitro Drugs Genetic Disease RNA 130

Drug Discovery World

NOVEMBER 7, 2022

WGS and Dante’s genomic reports will provide information about predisposition to genetic diseases, genetic carrier information and more. In April 2022, PacBio expanded a research collaboration with Children’s Mercy Kansas City in an effort to improve scientists’ understanding of the genetic underpinnings of rare diseases.

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Genome Genomics Development Genetics 98

The Pharma Data

DECEMBER 8, 2020

The impact of CNS diseases extends beyond patients—to their families and society as well.” The assumption is SciNeuro will start with Parkinson’s disease and pain in addition to rare genetic diseases, after scaling up staffing. . Laying down a new track for RNA processing, Remix launched with $81 million in financing.

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RNA Antibody Life Science Protein 52

The Pharma Data

NOVEMBER 29, 2020

PH1 is an ultra-rare genetic disease marked by overproduction of oxalate, which causes deposits of calcium oxalate crystals in the kidneys and urinary tract and can lead to painful and recurrent kidney stones, nephrocalcinosis, progression to kidney failure, and system organ dysfunction. It is an RNA interference (RNAi) therapeutic.

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Genetic Disease Antibody Licensing Licensing 52

The Pharma Data

DECEMBER 13, 2020

About EdiGene, Inc EdiGene is a biotechnology company focused on leveraging the cutting-edge genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of genetic diseases and cancer.

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Business Development Development Gene Editing In-Vivo 40

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?

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Life Science RNA Vaccination Vaccine 98

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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The Pharma Data

JANUARY 17, 2021

That’s not the case with RNA-targeted drugs. They’re basically the same,” but each one uses a particular nucleotide to bind to specific genetic sequence. The Undiagnosed Disease Network (a consortium of tertiary care institutions for that purpose) directs a large number of patients to n-Lorem, but most come from physicians.

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Genetics Clinical Trials Clinical Trials Drugs 52

XTalks

DECEMBER 13, 2023

Related: Top 30 Pharma Companies in 2023: Statistics and Trends 1) Moderna Compound annual growth rate: 415 percent Moderna, headquartered in Massachusetts, is a prominent biotechnology company specializing in RNA therapeutics, particularly mRNA vaccines.

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Genetics Vaccination Vaccine DNA 111

Drug Discovery World

FEBRUARY 28, 2023

Recent progress on mRNA therapeutics – with at least five programmes in clinical studies – is encouraging, and I hope that we will only see more scientific advancement in the years to come.

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Genetics Genetic Analysis Vaccination Vaccine 52

The Pharma Data

OCTOBER 14, 2020

is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA.

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Protein RNA Production Genetics 52

XTalks

MAY 4, 2021

The CRISPR gene editing system consists of the Cas9 enzyme, which serves as molecular scissors to cleave double-stranded DNA, and a guide RNA template targeted to a specific genomic sequence, which allows for precise editing. Epigenetic Editing with CRISPR.

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DNA Gene Gene Silencing Genome 98

The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. HAO1 encodes glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

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FDA Approval Production RNA Medicine 52

XTalks

AUGUST 29, 2022

The CRISPR-Cas9 system works by having “spacer” sequences transcribed into short RNA sequences that can guide the system to the matching target in the DNA sequence of a cell. The results demonstrate potential as a novel, safe and effective approach to cell therapies. Harnessing the Cellular Engineering Potential of CRISPR.

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Gene Editing DNA Gene Genome 98

XTalks

MAY 4, 2021

The CRISPR gene editing system consists of the Cas9 enzyme, which serves as molecular scissors to cleave double-stranded DNA, and a guide RNA template targeted to a specific genomic sequence, which allows for precise editing. Epigenome Editing with CRISPR.

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DNA Gene Gene Silencing Genome 52

The Pharma Data

DECEMBER 27, 2020

Galidesivir is a broad-spectrum antiviral, an adenosine nucleoside analog that blocks viral RNA polymerase. STP705 is a small interfering RNA (siRNA) therapy that leverages a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to knock down both TGF-beta1 and COX-2 gene expression.

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Trials Antibody Gene Therapy Clinical Trials 52

Drug Discovery World

FEBRUARY 22, 2024

Indeed, we have shown that the causative mutations of some leukodystrophies affect the subunits of an important cellular enzyme, RNA polymerase III, preventing its normal assembly – it turned out that Riluzole can counteract this assembly defect,” says Maxime Pinard, the researcher responsible for the project in Coulombe’s lab. ‘’For

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Drugs Gene Clinical Trials Clinical Trials 59

The Pharma Data

SEPTEMBER 22, 2020

The company’s commitment to SMA extends beyond gene therapy to branaplam (LMI070), an oral, once-weekly RNA splicing modulator also currently under development, to expand the treatment options for SMA patients. . More than 30% of patients with SMA Type 2 will die by age 25.

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Clinical Development Gene Therapy Development Gene 52

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. Langer, now a David H. These could feature in the next four to five years. Free Report How is the Biopharmaceutical industry evolving?

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Drug Delivery Vaccination Vaccine Research 147