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

Advarra

NOVEMBER 29, 2022

The Bespoke Gene Therapy Consortium (BGTC) 2 aims to overcome major obstacles and streamline the development process for small-batch gene therapies. The BGTC will act as a traffic light for these therapies, providing information on basic and clinical research, manufacturing, production, and regulatory requirements.

Gene Therapy 52

Gene Therapy Gene Production Clinical Trials 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

XTalks

SEPTEMBER 4, 2024

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. The MAGNITUDE trial uses CRISPR/Cas9 technology to target the genetic source of the disease, potentially offering a one-time treatment.

Trials 52

Trials RNA Protein Gene Editing 52

Worldwide Clinical Trials

MAY 30, 2024

Additional research is underway using gene editing via CRISPR, primarily investigating genes with known relationships to ALS, such as SOD1 and C9orf72. However, work is needed before CRISPR-mediated treatments become possible, including a better understanding of the genes involved in ALS.

Gene 204

Gene Clinical Trials Clinical Trials Gene Therapy 204

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. Gene therapy, while in its infancy, could be a gamechanger for cancer treatment and certain rare diseases and advancements in gene-editing technology relies on knowledge of viral factors.

Research 130

Research Clinical Trials Clinical Trials Trials 130

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

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