Worldwide Clinical Trials

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow.

Gene Editing 189

Gene Editing Gene Gene Therapy In-Vivo 189

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 17, 2024

We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics. In […]

Genetic Disease 189

Genetic Disease Genetics Gene Editing Medicine 189

Worldwide Clinical Trials

OCTOBER 24, 2023

Global Genes is a rare disease umbrella organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. Roughly 80% of rare diseases are due to a known genetic driver.

Gene 130

Gene Gene Editing Gene Therapy Clinical Trials 130

WCG Clinical

NOVEMBER 17, 2023

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. Since then, however, certain genetic engineering technologies (e.g.,

Genome 52

Genome Genomics Gene Editing Genetic Engineering 52

Trialfacts

SEPTEMBER 12, 2024

About the Research Center: Boston Children’s Hospital The research enterprise at Boston Children’s Hospital, involving over 3,000 researchers, is the largest in the world at a pediatric center. This includes the pathogenesis of Atopic Dermatitis Location What’s Next?

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

Rare Disease Day 2024, which falls on February 29 this year, is an opportunity to unite under a common cause: to bring attention to the challenges faced by those living with rare diseases and to push for advancements in research, treatment and policy.

Research 59

Research Drugs Gene Therapy Clinical Trials 59