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

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

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

Drug Discovery World

APRIL 14, 2023

Carl H June, MD, FAACR – Lifetime Achievement in Cancer Research June, a Fellow of the AACR Academy, is the Richard W Vague Professor in Immunotherapy, Director of the Center for Cellular Immunotherapies, and Director of the Parker Institute for Cancer Immunotherapy in the Perelman School of Medicine at the University of Pennsylvania.

Genetics 52

Genetics Research Medicine Clinical Research 52

Worldwide Clinical Trials

MAY 30, 2024

Authors: Rich Worldwide Clinical Trials Exec. These advancements have fostered increased focus from politicians, policymakers, and the investment communities backing academic and industry-sponsored research projects. Available across the globe Established efficacy in two adequate and well-controlled trials.

Gene 204

Gene Clinical Trials Clinical Trials Gene Therapy 204

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. Sarepta hopes to clarify Elvidys’ effectiveness in older children in a confirmatory trial.

Research 59

Research Drugs Gene Therapy Clinical Trials 59