Pharmaceutical Technology

AUGUST 18, 2022

The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia 0 genotypes. thalassemia in adult and paediatric patients.

Gene Therapy 246

Gene Therapy Gene Genotype In-Vivo 246

Worldwide Clinical Trials

FEBRUARY 6, 2023

When using genetic screening to identify clinical trial volunteers, a sponsor’s obligations for further testing and disclosure of results to patients and families are unclear, especially when the results have no impact on medical management. Guidance in this area is much needed. Read the full article!

Genetics 130

Genetics Clinical Trials Clinical Trials Genotype 130

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. For rare disease studies where there is little or no clinical trial experience, outcome assessments would have to be validated within a clinical development program.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

The Pharma Data

DECEMBER 9, 2020

The aim of the research will be to create a TGS IVD platform by optimizing a multi-layer approach encompassing the initial sample and library preparation, state-of-the-art sequencing technologies and improved genotyping procedures. Amolyt has also acquired an option to license the identified candidates for future clinical development.

In-Vivo 52

In-Vivo In-Vitro Antibody Development 52

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

Delveinsight

DECEMBER 16, 2020

As per the WHO, more than 100 types of HPV are found worldwide, out of them at least 13 known HPV genotypes can cause cancer of the cervix. However many companies at the global level are experimenting with their therapies at different stages of clinical development. The Chlamydia can be cured with antibiotics. Trichomoniasis.

Bacteria 52

Bacteria Development Genotype Vaccination 52