Drug Discovery World

DECEMBER 5, 2022

Scientists have unlocked key insights into virus evolution, revealing new information that could help develop treatments for a wide variety of genetic diseases. . It was led by scientists at the MRC-University of Glasgow Centre for Virus Research (CVR) and University of Massachusetts Chan Medical School. .

Gene Therapy 52

Gene Therapy Gene Genetic Disease Genetics 52

STAT News

FEBRUARY 22, 2023

Moderna is aiming to build a gene editing franchise powered by some of the same technologies used in its COVID-19 vaccines. The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions.

Gene Editing 126

Gene Editing Gene Genetic Disease Gene Therapy 126

Worldwide Clinical Trials

JANUARY 24, 2024

Much of what we discuss I covered in my presentation, “Leveraging Genetics to Support Rare Disease Clinical Trials,” at last year’s World Orphan Drug Congress (WODC) EU. Newborn Sequencing is a research initiative that allows for a baby’s full genome to be sequenced at birth, diagnosing their genetic diseases.

Genetics 184

Genetics Trials Clinical Trials Clinical Trials 184

Delveinsight

FEBRUARY 11, 2021

The scientists analyzed samples from patients with the most common pancreatic cancer, pancreatic ductal adenocarcinoma (PDAC). Avrobio gene therapy eradicates toxic substrate in Fabry patient. Moreover, those that did become ill showed relatively mild disease, from which they recovered quickly.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

Drug Discovery World

JANUARY 11, 2024

Researchers have used messenger RNA (mRNA) to create an effective therapy for a rare liver disease in preclinical studies, demonstrating the technology’s potential therapeutic use in people. Successful industry/academia collaboration Fewer than 5% of rare diseases have approved therapies and most of these treatments use gene therapy.

Research 59

Research Gene Therapy RNA Gene 59

Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. What are the advantages compared to other gene modulating approaches?

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

pharmaphorum

JANUARY 27, 2023

In the morning, I can be talking about gene therapy for hearing loss, for example, [and] in the afternoon, I can be talking about the treatment of a very specific tumour. million people in the UK that have a rare disease,” she continued, “and that’s where a lot of the innovation is, in more of these rare genetic diseases.

Clinical Trials 108

Clinical Trials Clinical Trials Trials Development 108