Worldwide Clinical Trials

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.

Genetics 190

Genetics Trials Clinical Trials Clinical Trials 190

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies. How Can Study Protocols Be More Effective?

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

Gene Therapy 52

Gene Therapy Gene Production Clinical Trials 52

Pharmaceutical Technology

DECEMBER 14, 2022

GSK has entered a strategic partnership with Wave Life Sciences to progress the discovery and development of oligonucleotide therapies for new genetic targets. The alliance will merge the PRISM oligonucleotide platform of Wave and the capabilities of GSK in genetics and genomics. By Cytiva Thematic.

Life Science 162

Life Science Development Gene Therapy Genetics 162

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

Drug Discovery World

OCTOBER 4, 2022

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. Cell and gene therapies. billion, compared to $19.9

Gene Therapy 52

Gene Therapy Drugs Gene Manufacturing 52