pharmaphorum

JULY 20, 2022

Armed with a $100 million second-round financing, CAMP4 Therapeutics is preparing to start the first clinical trial of a drug targeting regulatory RNA (regRNA) molecules that can be used to fine-tune the expression of genes. ” The post CAMP4 raises $100m to take lead RNA drugs into clinic appeared first on.

RNA 52

RNA Genetic Disease Drugs Protein 52

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?

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

Pharmaceutical Technology

JANUARY 12, 2023

Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM). Our pipeline of programmes focused on neuromuscular and cardiac rare disease gives us deep insight into the diverse and complex nature of dilated cardiomyopathy.”.

RNA 130

RNA Gene Therapy Genetics Genetic Disease 130

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

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

Delveinsight

AUGUST 26, 2021

Shape Therapeutics inks gene therapy deal with Roche worth up to USD 3 Billion. 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. was swooped up by Real Chemistry in January.

Vaccination 98

Vaccination Vaccine Gene Therapy RNA 98

Roots Analysis

FEBRUARY 21, 2022

Since the introduction of a gene sequencing method by Frederick Sanger in 1977, the field of genomic data collection and analysis has evolved significantly. 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 104

Genetic Disease Protein RNA Doctors 104

Drug Discovery World

MAY 15, 2024

In vivo proof-of-concept for Circio Holding’s differentiated circVec platform approach to gene therapy has been demonstrated in two posters at the American Society of Gene & Cell Therapy (ASGCT) 2024 annual meeting. AATD is a challenging genetic disease to treat. This substantially outperforms mRNA vector expression.

RNA 52

RNA In-Vivo Gene Therapy Genetic Disease 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 Prior to joining EdiGene, he was Vice President of KLUS Pharma and focused on cell therapy and new technologies.

Business Development 40

Business Development Development Gene Editing In-Vivo 40

The Pharma Data

NOVEMBER 29, 2020

In rare cases it is caused by a point mutation in the RAI1 gene in the deleted region. 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. The most common is a severe sleep disorder.

Genetic Disease 52

Genetic Disease Antibody Licensing Licensing 52

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. mRNA is a molecule made up of a single strand of ribonucleic acid (RNA). We believe it can now do the same for rare diseases.” “We

RNA 52

RNA Protein Vaccination Vaccine 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.

Genetics 111

Genetics Vaccination Vaccine DNA 111

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

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

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. It’s a great tool for controlling gene expression.”.

DNA 98

DNA Gene Gene Silencing Genome 98

XTalks

AUGUST 29, 2022

Now a common gene editing tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. From the Bench to Patient Therapeutics.

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. These modifications regulate gene expression without altering the sequence or structure of DNA.

DNA 52

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.

Trials 52

Trials Antibody Gene Therapy Clinical Trials 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

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

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59

The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.

Clinical Development 52

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. Conde says the use of nanoparticle delivery for gene therapies has become more common over recent years. Langer, now a David H.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147