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. Since then, the drug has been used off-label in breast cancer and other types of cancer. Langer, now a David H.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147

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 97

RNA Genetics Dermatology Antibody 97

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

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.

Research 59

Research Gene Therapy RNA Gene 59

Drug Discovery World

FEBRUARY 22, 2024

Lu Rahman looks at neuroscience drug discovery – advances and challenges – and how breakthroughs in this field are helping address areas of unmet need. Recent advances in biomarkers, research into the central nervous system and breakthroughs in human iPSC cell models, have bolstered neuroscience drug discovery research.

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59

Camargo

JULY 27, 2021

When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing. Gene Therapy Definition.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

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 103

RNA Vaccination Vaccine Pharma Companies 103

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.

Immune Response 52

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.

Vaccination 98

Vaccination Vaccine Gene Therapy RNA 98

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.

Marketing 52

Marketing Gene Sequencing DNA Genome 52

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 90

Genetic Disease Protein RNA Doctors 90

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

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.

Genome 98

Genome Genomics Development Genetics 98

The Pharma Data

DECEMBER 8, 2020

AbCellera’s AI-powered antibody discovery platform speeds the otherwise lengthy and grueling process by analyzing the database of natural immune systems to find antibodies that can be developed into drugs. Tackle the toughest problems in drug development.” The promise to partners is to “move quickly. Reduce cost. Remix Therapeutics .

RNA 52

RNA Antibody Life Science Protein 52

The Pharma Data

NOVEMBER 29, 2020

Food and Drug Administration (FDA). The drug was developed by researchers at Memorial Sloan Kettering Cancer Center and exclusively licensed to Y-mAbs. The FDA approved the drug on November 24. It is an RNA interference (RNAi) therapeutic. The drug hit the primary endpoint, percent change in 24-hour urinary oxalate.

Genetic Disease 52

Genetic Disease Antibody Licensing Licensing 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. This substantial investment is accelerating the growth of their new drug pipeline.

Genetics 111

Genetics Vaccination Vaccine DNA 111

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US. 14, 2020 10:00 UTC.

Business Development 40

Business Development Development Gene Editing In-Vivo 40

Drug Discovery World

FEBRUARY 28, 2023

Given its recent success, I believe we will see continued innovation in the field that extends into mRNA-based therapeutics for several diseases, especially those that have had limited or ineffective drug options so far. Rare diseases often have few, if any, therapeutic options for their condition.

Genetics 52

Genetics Genetic Analysis Vaccination Vaccine 52

The Pharma Data

JANUARY 17, 2021

n-Lorem Foundation has taken on a challenge that many nations consider too great: treating patients with ultra-rare diseases (which affect 30 or fewer people) for free, for life. That’s not the case with RNA-targeted drugs. Antisense drugs are versatile, too, he pointed out. they can help many patients.”.

Genetics 52

Genetics Clinical Trials Clinical Trials Drugs 52

The Pharma Data

DECEMBER 27, 2020

The drug, Yeliva (opaganib) is a first-in-class, oral spingosine kinase-2 (SK2) selective inhibitor with triple-action effect on the pathophysiological processes linked to COVID-19. Galidesivir is a broad-spectrum antiviral, an adenosine nucleoside analog that blocks viral RNA polymerase. Non-COVID-19-Related.

Trials 52

Trials Antibody Gene Therapy Clinical Trials 52

The Pharma Data

NOVEMBER 24, 2020

Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. PH1 is an ultra-rare genetic disease characterized by oxalate overproduction.

FDA Approval 52

FDA Approval Production RNA Medicine 52

The Pharma Data

SEPTEMBER 22, 2020

Basel, September 23 , 2020 – Novartis Gene Therapies recently received feedback from the US Food and Drug Administration (FDA) following their review of data from the STRONG study of the intrathecal (IT) formulation of AVXS-101 in older patients with spinal muscular atrophy (SMA). More than 30% of patients with SMA Type 2 will die by age 25.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52