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.

Vaccination 98

Vaccination Vaccine Gene Therapy RNA 98

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

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

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.

Protein 52

Protein RNA Production Genetics 52

The Pharma Data

DECEMBER 8, 2020

Now poised to advance a robust therapeutics pipeline to clinical development, Nuance will use the funds for ongoing R&D of existing products and business development of potential new assets. Through its Shielded Living Therapeutics platform, the company is developing functional cures for chronic diseases. Sigilon Therapeutics .

RNA 52

RNA Antibody Life Science Protein 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. Due to their size, cells can absorb them where they release products. Langer, now a David H. Still, several established challenges remain.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147

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

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. Oxlumo decreases oxalate production.

Genetic Disease 52

Genetic Disease Antibody Licensing Licensing 52

The Pharma Data

DECEMBER 13, 2020

Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO Dong Wei, CEO of EdiGene. 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

XTalks

JANUARY 3, 2025

By lowering its production, Tryngolza significantly reduces triglyceride levels in the blood. FCS also severely impacts quality of life, causing chronic fatigue and recurrent stomach pain. Tryngolza works by targeting a protein in the liver, apoC-III, which regulates triglyceride metabolism.

FDA Approval 59

FDA Approval Drugs Clinical Trials Clinical Trials 59

Camargo

JULY 27, 2021

Gene therapy is a new therapeutic approach in which genes are used to treat or prevent diseases. It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy. Gene Therapy Definition.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

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

XTalks

APRIL 1, 2025

Management typically involves on-demand treatment or preventive (prophylactic) therapy using FVIII or FIX replacement products, or agents that mimic the function of these factors. Through RNA silencing, it targets the expression of antithrombin, a protein that inhibits blood clotting.

FDA Approval 59

FDA Approval RNA Clinical Trials Clinical Trials 59

Pharma Mirror

APRIL 21, 2024

With the rapid development of biotechnology and molecular medicine, the introduction of mRNA as a vaccine or therapeutic agent enables the production of almost any desired functional protein/peptide within the human body.

In-Vitro 130

In-Vitro Drugs Genetic Disease RNA 130

The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. On November 19, the European Commission granted marketing authorization for Oxlumo for the treatment of PH1 in all age groups, following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP). President of R&D at Alnylam.

FDA Approval 52

FDA Approval Production RNA Medicine 52

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

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. Dynavax boasts two successful commercial products in its portfolio.

Genetics 111

Genetics Vaccination Vaccine DNA 111

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.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 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.

RNA 105

RNA Genetics Genetic Disease Medicine 105

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.

RNA 111

RNA Vaccination Vaccine Pharma Companies 111

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.

Trials 52

Trials Antibody Gene Therapy 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.

Gene Therapy 111

Gene Therapy RNA Gene Editing Gene 111