XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Drug Discovery World

MARCH 21, 2023

UK researchers, from the Wellcome Sanger Institute and collaborators, have used human stem cells and neurons to investigate what features influence how well CRISPR activation works for different sets of genes. CRISPR activation (CRISPRa) is a type of CRISPR gene editing that is used to overexpress certain genes.

Research 52

Research Gene Gene Editing Genome 52

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

DECEMBER 5, 2022

A new partnership between industry and three rare disease foundations has formed with the goal of accelerating the development of precision drugs to treat rare neurodevelopmental disorders. . Understanding rare diseases remains a significant challenge.

Development 52

Development Genome Genomics Drugs 52

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. Advancement in DNA sequencing technologies have resulted in noteworthy developments in various healthcare-related research fields, such as diagnostics and personalized medicine.

Marketing 52

Marketing Gene Sequencing DNA Genome 52

pharmaphorum

SEPTEMBER 9, 2020

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.

Genome 92

Genome Genomics Genetics Genome Project 92

XTalks

NOVEMBER 10, 2020

Expeditious and accurate diagnoses are necessary for patients to access healthcare services and treatment options for rare genetic diseases. Increasing the efficiency of case analysis and interpretation is essential to providing timely care for patients with genetic diseases.

Genome 98

Genome Genomics Medicine Genetic Disease 98

pharmaphorum

SEPTEMBER 9, 2020

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.

Genome 80

Genome Genomics Genetics Genome Project 80

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., 14, 2020 10:00 UTC. BEIJING & CAMBRIDGE, Mass.–( –( BUSINESS WIRE )– EdiGene, Inc. as Head of Business Development. “Our

Business Development 40

Business Development Development Gene Editing In-Vivo 40

Pharmaceutical Technology

MAY 8, 2023

Innovation S-curve for the pharmaceutical industry Zinc-finger nucleases is a key innovation area in pharmaceutical Zinc finger nucleases (ZFNs) are tools used in genetic engineering to cleave a chosen genomic sequence. ZFNs are also used to create a new generation of genetic disease models called isogenic human disease models.

Genetics 130

Genetics DNA Genetic Engineering Antibody 130

The Pharma Data

JANUARY 17, 2021

Developing these drugs commercially would have resulted in exorbitant costs for patients, Crooke explained because most drug platform technologies are not well-suited for creating individualized medicines. Genetic diseases are vastly more common and more complex than we used to think.”. We’re the middleman,” he said.

Genetics 52

Genetics Clinical Trials Clinical Trials Drugs 52

XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). Related: Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data. million (approximately $3.8 million USD).

Gene Therapy 98

Gene Therapy Drugs Gene Gene Silencing 98

Drug Discovery World

MARCH 6, 2024

To find new ways of diagnosing and treating complex diseases we first must understand the mechanisms underpinning their key pathological drivers, how these relate to different patient subgroups, and which drugs might be useful in ameliorating their effects – this is the basis of precision neuroscience.

Genome 64

Genome Genomics Clinical Trials Clinical Trials 64

XTalks

DECEMBER 13, 2023

These efforts encompass programs related to the shingles vaccine, Tdap vaccine and plague vaccine, reinforcing Dynavax’s commitment to advancing the field of vaccines and medicine. The enduring success of Aravive can be attributed to its ongoing investment in R&D and the commercialization of innovative new medicines.

Genetics 111

Genetics Vaccination Vaccine DNA 111

Drug Discovery World

OCTOBER 12, 2023

Dr Michelle Fraser is Head of Cell & Gene Therapy at global health science solutions company, Revvity. When it comes to the technologies driving drug discovery, she thinks that the ability to specifically edit regions of the genome using CRISPR/Cas9 is particularly revolutionary.

Drugs 59

Drugs Life Science Genome Genomics 59

Drug Discovery World

FEBRUARY 28, 2023

Now, given the extraordinary success of the Covid-19 vaccines, the mRNA platform may bring renewed hope as researchers explore its potential to advance medicines for rare diseases. Interestingly, at least 80% of all rare diseases originate from monogenic mutations, meaning that they arise from mutations in a single gene 4.

Genetics 52

Genetics Genetic Analysis Vaccination Vaccine 52