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

Pharmaceutical Technology

JANUARY 12, 2023

Solid Biosciences has entered a strategic research collaboration with Phlox Therapeutics to expedite the development of new therapies for rare cardiac diseases. Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM).

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RNA Gene Therapy Genetics Genetic Disease 130

Pharmaceutical Technology

APRIL 24, 2023

Robert Langer was part of the first cohort of researchers to begin nanoparticle research , as his lab at Cornell University developed tiny particles to deliver large molecules for angiogenesis. The next wave came from early research into liposomes and lipid nanoparticles, both of which are used for drug delivery.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147

Camargo

JULY 27, 2021

The technology of gene therapy is possible due to extensive DNA research and our resulting understanding of many diseases on the genetic level, and it encompasses several mechanisms such as introducing new genes (gene addition) and inactivating or replacing mutated genes (gene editing). Route of Administration Considerations.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

XTalks

APRIL 1, 2025

Through RNA silencing, it targets the expression of antithrombin, a protein that inhibits blood clotting. Qfitlia is the second RNA interference therapy to receive a highly anticipated FDA approval recently. However, in 2018, the companies concluded the research and option phase of their partnership.

FDA Approval 59

FDA Approval RNA Clinical Trials Clinical Trials 59

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. percent, according to a report by BCC Research. RNA technology is not new nor has its potential been surprising. RNA in the Making. RNA Therapeutics.

Life Science 98

Life Science RNA Vaccine Vaccination 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 breakthrough was hailed as a revolution for disease treatment and particularly a major success against the pandemic. Where next?

RNA 111

RNA Vaccine Vaccination Pharma Companies 111

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Under the deal, both companies will partner on the research and preclinical studies, which will be funded by Moderna.

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Gene Editing Gene In-Vivo Gene Therapy 147

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

XTalks

AUGUST 29, 2022

However, researchers are determined to overcome this hurdle. In a press release last week, it was announced that novel CRISPR research completed by a group at the Gladstone Institutes and UC San Francisco had been published in Nature Biotechnology. Harnessing the Cellular Engineering Potential of CRISPR.

Gene Editing 98

Gene Editing DNA Gene Genomics 98

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. The research was published earlier this month in the journal Cell.

DNA 98

DNA Gene Gene Silencing Genomics 98

Roots Analysis

FEBRUARY 21, 2022

Advancement in DNA sequencing technologies have resulted in noteworthy developments in various healthcare-related research fields, such as diagnostics and personalized medicine. During our research, we came across more than 55 players, which claim to offer 280+ NGS library preparation kits. Concluding Remarks.

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Marketing Gene Sequencing DNA Genomics 52

Roots Analysis

AUGUST 12, 2024

Molecular diagnostic tests refer to tests intended to detect specific sequences in human genomic samples, such as DNA or RNA, in order to diagnose a particular disease. Further, such tests also aim to identify single nucleotide polymorphisms, deletions, rearrangements, and insertions in genetic sequences.

Genetics 40

Genetics Gene Expression RNA DNA 40

XTalks

DECEMBER 13, 2023

In 2023, biotechnology and pharmaceutical companies leverage cutting-edge technology and significant investments in research and development (R&D) to continue pushing the boundaries of innovation in the healthcare sector, transcending the challenges posed by the pandemic.

Genetics 111

Genetics Vaccine Vaccination DNA 111

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

The Pharma Data

NOVEMBER 29, 2020

The drug was developed by researchers at Memorial Sloan Kettering Cancer Center and exclusively licensed to Y-mAbs. It is an RNA interference (RNAi) therapeutic. It works by degrading HAO1 mRNA and decreasing the synthesis of glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1.

Genetic Disease 52

Genetic Disease Antibody Licensing Licensing 52

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. The research was published earlier this month in the journal Cell. pyogenes dCas9.

DNA 52

DNA Gene Gene Silencing Genomics 52

The Pharma Data

DECEMBER 13, 2020

Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US. Prior to that, he held various oncology research and development positions at Merrimack Pharmaceuticals and Archemix. Dr. Zhang completed his postdoctoral fellowship at Harvard Medical School/Boston Children’s Hospital.

Business Development 40

Business Development Development Gene Editing In-Vivo 40

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.

FDA Approval 52

FDA Approval Production RNA Medicine 52

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.

Genetics 52

Genetics Clinical Trials Clinical Trials Drugs 52

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. . In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52

XTalks

JANUARY 6, 2025

From leveraging artificial intelligence (AI) to streamline diagnostics and treatments to exploring the untapped potential of RNA-based therapeutics, biotechnology is shaping the future of healthcare and beyond. As of January 31, 2024, approximately 131 unique RNA-based therapies are in clinical development across various therapeutic areas.

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Gene Therapy RNA Gene Editing Gene 111