pharmaphorum

OCTOBER 16, 2020

By definition, however, trial master files represent a much richer and more detailed source of data on a drug and how it performs. For instance, provided a drug has not failed a trial on safety, the side effects it caused in one population could constitute on-target effects in another.

Clinical Trials 104

Clinical Trials Clinical Trials Trials Life Science 104

XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. Gene-altering and other advanced medicines are radically rewriting the future for people with some of the rarest, and most devastating, diseases on earth. Integrating patients into all trial functions.

Development 98

Development Gene Therapy Clinical Trials Clinical Trials 98

XTalks

AUGUST 29, 2022

CRISPR technology has begun to enter clinical trials due to emerging therapeutic applications, but the technology still has limitations, primarily because it is difficult to safely make large quantities of precisely edited cells. However, researchers are determined to overcome this hurdle.

Gene Editing 98

Gene Editing DNA Gene Genome 98

XTalks

FEBRUARY 28, 2022

In the official Rare Disease Day 2022 video above, patients from all around the world are heard sharing their stories in their native languages about living with rare diseases like Duchenne muscular dystrophy, cystic fibrosis and Gaucher’s disease among others. How is a Rare Disease Defined?

Clinical Trials 98

Clinical Trials Clinical Trials Genetics Trials 98

Delveinsight

FEBRUARY 11, 2021

Verily is partnering with Johnson & Johnson’s Janssen division to witness the body’s earliest immune responses to a coronavirus infection, with people participating in the research from within their homes. . However, they have only shrunk tumors in about half of patients who took them, along with chemotherapy, in the clinical trials.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. Complexities arise while trying to establish a standard, cost-efficient, and expert process for research and manufacturing.

Gene Therapy 52

Gene Therapy Gene Development Production 52

XTalks

NOVEMBER 10, 2023

Related: Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio The FDA approval of Adzynma was based on its demonstrated safety and efficacy in a Phase III global cross-over study, the first of its kind for cTTP. Adzynma’s approval is the second for Takeda this week.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

MARCH 3, 2021

BridgeBio is dedicated to developing therapies for genetic diseases with unmet needs. Today’s action marks the first FDA approval for a therapy to treat this devastating disease,” said Hylton V. Nulibry Trial Results. The approval was granted to BridgeBio Pharma, Inc.

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

XTalks

MAY 12, 2023

What’s been shown in mouse models of Rett syndrome which also have the genetic disease, is that trofinetide helps strengthen those connections between the neurons. So, although it’s not a genetic treatment for Rett syndrome, it does act on the core pathophysiology behind Rett syndrome, which is that synaptic loss.

FDA Approval 97

FDA Approval Genetics Trials Drugs 97

XTalks

APRIL 15, 2021

million people in the US, yet there is no treatment for the most common form of the disease: heart failure with preserved ejection fraction (HFpEF). A lack of efficacy is the greatest reason for clinical trial failures, especially in Phase II and Phase III,” says Dr. Tyl. Heart failure affects about 6.2

Development 98

Development Drugs Genome Genomics 98

XTalks

SEPTEMBER 17, 2020

While the table lists acceptable surrogate endpoints for trials of more common conditions, such as asthma, type 2 diabetes and various cancer types, the document also provides key information to developers of certain rare disease therapies. The traditional approval pathway can be pursued by meeting this surrogate endpoint.

Trials 98

Trials Gene Hormones Clinical Trials 98

XTalks

OCTOBER 1, 2020

WeiQi Lin, MD, PhD, executive vice president of research & development and principal scientist, DURECT Corporation. Lin has made several transitions throughout her career, from being a clinician at an academic center, to academic research, to heading business development in industry. Motivations for Transition. Key Moments.

Scientist 52

Scientist In-Vitro Research Life Science 52

Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. Whilst research into mRNA has been ongoing for decade, interest has sparked in recent years and research has begun to expand into areas of vaccinology, infectious diseases and other indications.

RNA 52

RNA Protein Vaccination Vaccine 52

Drug Discovery World

MARCH 6, 2024

Guiding neurological drug discovery and development to success The 20 years since the Human Genome Project has seen transformational advances in the molecular understanding of cancers and rare genetic diseases, leading to genetically informed, personalised selection of therapies and massively improved outcomes for many patients.

Genome 64

Genome Genomics Clinical Trials Clinical Trials 64