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: How easy was it to transfer theoretical research into a viable business?

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

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). Formulation Considerations.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

Drug Discovery World

NOVEMBER 7, 2022

One might argue that this all started getting exciting with the launch of the Human Genome Project, which the National Human Genome Research Institute consider to be one of the greatest scientific feats in history 1. With Revio, researchers will be able to access that same great chemistry, but at a much larger scale.” . The origins

Genome 98

Genome Genomics Development Genetics 98

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.

Gene Editing 147

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.

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

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.

Marketing 52

Marketing Gene Sequencing DNA Genome 52

Drug Discovery World

JULY 8, 2024

Beard Distinguished Professor of Experimental Surgery and the Founding Director of the Translational Research Institute at Duke University, explores the rise of RNA therapeutics. With high-throughput RNA sequencing, it is evident the majority of RNAs in human cells are not actually messengers.

RNA 59

RNA Protein Gene Expression Vaccination 59

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

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

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

JANUARY 17, 2021

n-Lorem selects patients based on their genetic mutation and the organ affected rather than their specific diseases. “We It’s the cause of the mutation, not the name of the disease, that matters.”. That’s not the case with RNA-targeted drugs. We focus on the organs we really understand,” he emphasized.

Genetics 52

Genetics Clinical Trials Clinical Trials Drugs 52

Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. Yochi Slonim, CEO of Anima explains further: “The impact of the Covid-19 pandemic on mRNA research has been significant, fundamentally altering the landscape of therapeutics.

RNA 52

RNA Protein Vaccination Vaccine 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. 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 Vaccination Vaccine 98

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 Vaccination Vaccine DNA 111

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 98

RNA Vaccination Vaccine Pharma Companies 98

Drug Discovery World

FEBRUARY 28, 2023

As we turn our focus to new potential applications and disease areas for the platform, scientists and companies must consider the potential life-saving impact on rare, inherited diseases, says Archana Gupta , PhD, staff scientist in genetic sciences at Thermo Fisher Scientific.

Genetics 52

Genetics Genetic Analysis Vaccination Vaccine 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 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 Genome 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 Genome 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 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 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

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

Drug Discovery World

FEBRUARY 22, 2024

Recent advances in biomarkers, research into the central nervous system and breakthroughs in human iPSC cell models, have bolstered neuroscience drug discovery research. ARUK 3 adds that while the UK government increased funding in dementia research from £28.2m to £104.7m per year there are additional issues.

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59

The Pharma Data

SEPTEMBER 22, 2020

All patients deserve a gene therapy designed to address the genetic root cause of their disease with a single dose. 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. .

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52

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