WCG Clinical

NOVEMBER 17, 2023

It also establishes the role of the Institutional Biosafety Committee (IBC) in providing local oversight of such research. At the time this change was implemented, methods used to deliver rsNA to cells or research participants typically involved the use of viral vectors that were subject to the NIH Guidelines.

Genomics 52

Genomics Genome Gene Editing Genetic Engineering 52

WCG Clinical

OCTOBER 21, 2024

In a recent episode of WCG Talks Trials, WCG experts explored topics like the importance of community engagement, the latest developments in gene and cell therapies, and the challenges of clinical research in rare and chronic diseases like sickle cell disease and melanoma.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Clinical Research 52

XTalks

SEPTEMBER 4, 2024

Vutrisiran works by silencing the gene responsible for producing TTR, reducing both mutant and wild-type TTR proteins. The HELIOS-B study’s findings are timely as new gene-editing therapies, like the one being studied in MedStar Health’s Phase III MAGNITUDE trial for ATTR-CM, are also being explored.

Trials 52

Trials RNA Protein Gene Editing 52

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

The Pharma Data

JANUARY 10, 2021

EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. Previously, the Company received Rare Pediatric Disease designation from the FDA for EDIT-301. EDIT-301 is the first experimental medicine in development generated using CRISPR/Cas12a gene editing.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

Trialfacts

SEPTEMBER 12, 2024

Their work is driven by a profound understanding of disease biology and supported by advanced discovery platforms such as genetics and genomics, gene editing, bioinformatics, proteomics, bioengineering, image analysis, biobanks, disease-specific stem cell lines, and various animal models.

Research 52

Research Gene Editing Biobanking Bioinformatics 52

The Pharma Data

JANUARY 24, 2021

It will be run through the NIAID-supported Infectious Diseases Clinical Research Consortium (IDCRC), with support from the Bill & Melinda Gates Foundation. CYAD-101 is the company’s allogeneic NKG2D-receptor and T cell receptor inhibitory molecule-based, non-gene edited CAR-T candidate.

Trials 52

Trials Allergies In-Vitro Antibody 52

Pharmaceutical Technology

FEBRUARY 20, 2023

This level of homogeneity within the clinical trial population may prevent the identification of variable therapy responses within population subgroups. In addition, safety oncology and immunotherapy, which is a focus area for Cerba Research has close ties to virology. The technology platforms are almost the same.

Research 130

Research Clinical Trials Clinical Trials Trials 130

XTalks

FEBRUARY 11, 2025

Gene therapy has shown promise in treating cancers that are particularly difficult to manage such as neuroblastoma and Wilms tumor using CAR-T cell therapy, CRISPR-Cas9 gene editing and RNA-based interventions to target genetic drivers of disease.

Gene Therapy 66

Gene Therapy Clinical Trials Clinical Trials Trials 66