Gene Therapy, Genetic Disease and Genomics - Clinical Research Informer

Abu Dhabi DoH signs new deal to accelerate development in life sciences

Pharmaceutical Technology

JUNE 16, 2023

The Abu Dhabi Department of Health (DoH) in the UAE has made a declaration of collaboration with Mass General Brigham’s (MGB) International Center for Genetic Disease (iCGD) to advance life sciences.

Life Science

Life Science Genetic Disease Genome Genomics

Ancient viruses may hold key to gene therapy treatments

Drug Discovery World

DECEMBER 5, 2022

Scientists have unlocked key insights into virus evolution, revealing new information that could help develop treatments for a wide variety of genetic diseases. . Many distinct types of parvoviruses infect mammals – including humans – without causing disease. Parvovirus gene therapy is a cutting-edge biomedical technology.

Gene Therapy

Gene Therapy Gene Genetic Disease Genetics

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

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. How Can Study Protocols Be More Effective?

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

Leveraging Genetic Testing for Enrolling Rare Disease Trials

Worldwide Clinical Trials

JANUARY 24, 2024

Much of what we discuss I covered in my presentation, “Leveraging Genetics to Support Rare Disease Clinical Trials,” at last year’s World Orphan Drug Congress (WODC) EU. Newborn Sequencing is a research initiative that allows for a baby’s full genome to be sequenced at birth, diagnosing their genetic diseases.

Genetics

Genetics Trials Clinical Trials Clinical Trials

Gene therapy for ‘childhood dementia’ shows promise

Drug Discovery World

FEBRUARY 27, 2023

An investigational gene therapy for Sanfilippo syndrome – which leads to a form of childhood dementia – has shown promising early results in a proof-of-concept study. Patients with MPS-IIIA have a mutation in the SGSH gene, causing them to lack an enzyme which normally breaks down large sugar molecules.

Gene Therapy

Gene Therapy Gene Genetics Genetic Disease

STAT+: Moderna expands CRISPR gene editing research with ElevateBio partnership

STAT News

FEBRUARY 22, 2023

The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions. Life Edit is the North Carolina subsidiary of ElevateBio, a cell and gene therapy manufacturing firm in Waltham.

Gene Editing

Gene Editing Gene Genetic Disease Gene Therapy

Moderna partners with Life Edit for mRNA gene editing therapies

Pharmaceutical Technology

FEBRUARY 23, 2023

The collaboration will combine the mRNA platform of Moderna with the gene editing technologies suite, including the base editing capabilities of Life Edit for the development of curative therapies to treat challenging genetic diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Editing

Gene Editing Gene In-Vivo Gene Therapy

AstraZeneca pays record 660% premium for gene editing company LogicBio

pharmaphorum

OCTOBER 4, 2022

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. per share, a rare 660% premium on LogicBio’s share price. .

Gene Editing

Gene Editing Gene Genome Genomics

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

JULY 29, 2022

Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression. This treatment activates the OTC gene so that ammonia can be removed from the blood. This gene therapy will give the instructions to produce the enzyme arginine so that it can be hydrolysed to ornithine.

RNA

RNA Gene Expression Gene Gene Therapy

Turning science into business: Amplifying mRNA by targeting regRNAs

Drug Discovery World

APRIL 17, 2024

As for gene-modulating approaches, I want to point out that our therapeutic candidates do not in any way modulate the gene itself. Increasing gene expression via protein replacement or gene therapy has been shown to have limitations and drawbacks. What diseases are you currently targeting?

Gene Expression

Gene Expression Gene Genetic Disease RNA

Next Generation Sequencing (NGS) Library Preparation Kits Market: Current Scenario and Future Potential

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. Moreover, the manual protocols require extensive manipulation, costly reagents and long duration of skilled genomic library production.

Marketing

Marketing Gene Sequencing DNA Genome

EdiGene Expands Management Team by Appointment of Head of US Subsidiary Dr. Bo Zhang and Head of Business Development Dr. Kehua Fan

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., Prior to joining EdiGene, he was Vice President of KLUS Pharma and focused on cell therapy and new technologies. 14, 2020 10:00 UTC.

Business Development

Business Development Development Gene Editing In-Vivo

UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD

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). Bone marrow transplantation is also used in some infantile-onset cases to help delay disease progression. It is one of five European sites that will deliver the treatment.

Gene Therapy

Gene Therapy Drugs Gene Gene Silencing

Researchers discover treatment for rare muscle disease

Drug Discovery World

MARCH 24, 2023

Israeli researchers have developed a drug that is effective in treating a rare genetic muscle disease. The team demonstrated that the disease is caused by a mutation in the gene encoding HMG CoA reductase, inhibiting the enzyme’s activity.

Research

Research Genetics Gene Licensing

Meet the industry: Who’s at Drug Discovery 2023?

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

Drugs Life Science Genome Genomics

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

MAY 4, 2021

The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. Nearly one third of human genes lack CGIs, which would limit the use of the tool. Epigenetic Editing with CRISPR.

DNA

DNA Gene Gene Silencing Genome

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

XTalks

MAY 4, 2021

The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. Nearly one third of human genes lack CGIs, which would limit the use of the tool. Epigenome Editing with CRISPR.

DNA

DNA Gene Gene Silencing Genome

The future outlook for mRNA therapies

Drug Discovery World

APRIL 30, 2024

Rare diseases are largely treated using gene therapies which employ modified viruses to bring the therapy to the disease cells. However, these therapies can cause severe adverse effects. We believe it can now do the same for rare diseases.” “We

RNA

RNA Protein Vaccination Vaccine

Clinical Research Informer

Abu Dhabi DoH signs new deal to accelerate development in life sciences

Ancient viruses may hold key to gene therapy treatments

Trending Sources

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

Leveraging Genetic Testing for Enrolling Rare Disease Trials

Gene therapy for ‘childhood dementia’ shows promise

STAT+: Moderna expands CRISPR gene editing research with ElevateBio partnership

Moderna partners with Life Edit for mRNA gene editing therapies

AstraZeneca pays record 660% premium for gene editing company LogicBio

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Turning science into business: Amplifying mRNA by targeting regRNAs

Next Generation Sequencing (NGS) Library Preparation Kits Market: Current Scenario and Future Potential

EdiGene Expands Management Team by Appointment of Head of US Subsidiary Dr. Bo Zhang and Head of Business Development Dr. Kehua Fan

UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD

Researchers discover treatment for rare muscle disease

Meet the industry: Who’s at Drug Discovery 2023?

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

The future outlook for mRNA therapies

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