Genetic Disease, Medicine, Protein and RNA - Clinical Research Informer

Merck and Orna partner for RNA technology-based vaccines and therapies

Pharmaceutical Technology

AUGUST 17, 2022

Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. These programmes will include therapies and vaccines in infectious disease and oncology areas.

RNA

RNA Vaccination Vaccine In-Vivo

Rare genetic disease caused by mutations in protein that controls RNA metabolism

Scienmag

MAY 7, 2021

Credit: UPMC PITTSBURGH, May 7, 2021 – In a paper published today in Nature Communications, an international group of collaborators led by researchers at UPMC Children’s Hospital of Pittsburgh have identified a genetic cause of a rare neurological disorder marked by developmental delay and loss of coordination, or ataxia.

Genetics

Genetics Genetic Disease RNA Protein

CAMP4 raises $100m to take lead RNA drugs into clinic

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

RNA Genetic Disease Drugs Protein

MiNA and BioMarin partner to speed development of RNAa therapies

Pharmaceutical Technology

APRIL 26, 2023

MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. The new deal excludes oncology and other therapeutic areas outside the scope of genetic disease.

Development

Development Genetic Disease Genetics Gene Therapy

Shape Therapeutics-Roche’s Deal; AllStripes Raises $50M; Datavant-Real Chemistry’s Partnership; BlueWillow’s Nasal Vaccine

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

Vaccination Vaccine Gene Therapy RNA

General Design Methods for mRNA Drugs

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

In-Vitro Drugs Genetic Disease RNA

Biopharma Money on the Move: December 2 – 8

The Pharma Data

DECEMBER 8, 2020

Through its Shielded Living Therapeutics platform, the company is developing functional cures for chronic diseases. The assumption is SciNeuro will start with Parkinson’s disease and pain in addition to rare genetic diseases, after scaling up staffing. . Pear Therapeutics.

RNA

RNA Antibody Life Science Protein

Key Trends in the Life Sciences to Look Forward to in 2022

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

Life Science RNA Vaccination Vaccine

Beyond the pandemic: the potential of mRNA technology

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

RNA Vaccination Vaccine Pharma Companies

Top 10 Fastest Growing Biotech Companies in 2023

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. Alpine Immune Sciences has recently announced an augmentation in its R&D investment.

Genetics

Genetics Vaccination Vaccine DNA

Unlocking the potential of mRNA for the future treatment of rare diseases

Drug Discovery World

FEBRUARY 28, 2023

This presents a unique opportunity for mRNA-based therapies to potentially restore normal cell function by supplying copies of the functional transcript for the cell to read. mRNA enables the delivery of a transiently expressed genetic molecule that is translated into a target protein using the machinery of the host cell.

Genetics

Genetics Genetic Analysis Vaccination Vaccine

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

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

FDA Approval Production RNA Medicine

Advances in neuroscience drug discovery

Drug Discovery World

FEBRUARY 22, 2024

With the breadth and range of central nervous system disorders – for example Parkinson’s disease and multiple sclerosis – facing the global population there is an unmet need within this area of drug discovery. They focused on miRNAs, which regulate multiple target proteins controlling the cellular processes in the amygdala.

Drugs

Drugs Gene Clinical Trials Clinical Trials

Novartis Provides Update on AVXS-101 Intrathecal Clinical Development Program

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. . About Novartis Novartis is reimagining medicine to improve and extend people’s lives.

Clinical Development

Clinical Development Gene Therapy Development Gene

Clinical Research Informer

Merck and Orna partner for RNA technology-based vaccines and therapies

Rare genetic disease caused by mutations in protein that controls RNA metabolism

Trending Sources

CAMP4 raises $100m to take lead RNA drugs into clinic

MiNA and BioMarin partner to speed development of RNAa therapies

Shape Therapeutics-Roche’s Deal; AllStripes Raises $50M; Datavant-Real Chemistry’s Partnership; BlueWillow’s Nasal Vaccine

General Design Methods for mRNA Drugs

Biopharma Money on the Move: December 2 – 8

Key Trends in the Life Sciences to Look Forward to in 2022

Beyond the pandemic: the potential of mRNA technology

Top 10 Fastest Growing Biotech Companies in 2023

Unlocking the potential of mRNA for the future treatment of rare diseases

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

Advances in neuroscience drug discovery

Novartis Provides Update on AVXS-101 Intrathecal Clinical Development Program

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