XTalks

APRIL 15, 2021

Late-stage clinical trial failures are largely to blame for the lack of effective treatment options for patients with HFpEF, and poor five-year survival rates make this an area of great unmet need. A lack of efficacy is the greatest reason for clinical trial failures, especially in Phase II and Phase III,” says Dr. Tyl.

Development 98

Development Drugs Genomics Genome 98

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. The webinar included operational strategies for these trials from Laura Omoboni, MS, Executive Director of Clinical Trial Management. Rare diseases can often be progressive, chronic and fatal.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

XTalks

JANUARY 21, 2025

In a clinical trial, participants demonstrated a 98% success rate after six months post implantation, with arteries widened successfully and no stent fractures observed. There, plasma is separated, and tumor DNA is identified by detecting genetic mutations, methylation patterns and fragmentation signals.

FDA Approval 105

FDA Approval Manufacturing Development RNA 105

Bioengineer

JANUARY 16, 2024

Another genetic change at the C797 residue in the ATP binding region of EGFR’s exon 20 has emerged as a key mechanism of resistance to osimertinib. However, when osimertinib is used first-line, the T790M mutation is usually absent before or after progression, reducing its significance with increased frontline use.

Genomics 119

Genomics Genome Protein In-Vitro 119

XTalks

FEBRUARY 4, 2021

Through these collaborations, cohort research dataset sharing programs allow for whole-genome sequence and genotype data to be made available for independent research. Now, if all you’ve already got is a bioresource of 20,000 people that have already been genotyped, you can choose those from that sample and focus your effort.”.

Research 98

Research Biobanking Drugs Genomics 98

XTalks

SEPTEMBER 28, 2020

The test is based on the detection of actionable genes – genes with known driver mutations that can be targeted by an approved therapy (or investigational therapies in clinical trials). Based on the genetic biomarker(s) identified, the most effective therapy targeting the specific molecular marker can be selected.

Gene 105

Gene DNA Life Science Genotype 105

The Pharma Data

NOVEMBER 2, 2020

(NASDAQ:ADIL; ADILW) , a clinical-stage biopharmaceutical company focused on the development of treatments for addictions, today provided an update on its landmark ONWARD Phase 3 pivotal trial. 40% of expected Trial patient screenings have been completed and the Trial is more than 25% enrolled relative to planned enrollment.

Trials 52

Trials Genotype Marketing Production 52

XTalks

MARCH 3, 2021

The US Food and Drug Administration (FDA) has granted approval to Nulibry (fosdenopterin) for reducing the risk of death associated with molybdenum cofactor deficiency type A (MoCD Type A), making it the first treatment for the ultra-rare, genetic metabolic disorder. The approval was granted to BridgeBio Pharma, Inc.

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. The safety and efficacy of Oxlumo are also being evaluated in the ongoing ILLUMINATE-C Phase 3 clinical trial in patients of all ages with advanced PH1, including patients on dialysis. Oxlumo should be administered by a healthcare professional.

FDA Approval 52

FDA Approval Production RNA Medicine 52

XTalks

MARCH 26, 2025

XTALKS WEBINAR: Biomarker Solutions for Steatotic Liver Disease: Enabling Better Insights into MASH/MASLD Biology Live and On-Demand: Thursday, May 29, 2025 , at 11am EDT (4pm BST / UK) Register for this free webinar to discover the impact of genetic markers on the severity and treatment of MASH/MASLD. Net loss for Q4 was $20.2

Trials 66

Trials Life Science Clinical Trials Clinical Trials 66