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

NOVEMBER 27, 2023

Throughout the course of extensive clinical trials, Pombiliti and Opfolda have consistently exhibited a favorable safety profile, and patients have generally tolerated the treatment well.

FDA Approval 104

FDA Approval Clinical Trials Clinical Trials Trials 104

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. The complexities of sample management can significantly extend the timelines for clinical trials. Conclusion.

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

DECEMBER 13, 2023

This approval also marks bluebird’s third ex vivo gene therapy approved by the FDA for a rare genetic disease and second FDA approval for an inherited hemoglobin disorder, cementing our position as a gene therapy leader.”

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

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

The Pharma Data

DECEMBER 8, 2020

December is bringing frost in the North and plenty of cold hard cash for these life sciences companies. . European venture capital firm Forbion rounded up $545 million for its fifth life sciences fund. Cornering the untapped Chinese CNS market, SciNeuro launched with $100 million in their pocket.

RNA 52

RNA Antibody Life Science Protein 52

XTalks

SEPTEMBER 17, 2020

Acromegaly Surrogate Endpoint: Serum growth hormone and serum insulin-like growth factor 1 (IGF-1) are acceptable surrogate endpoints for acromegaly clinical trials involving somatostatin analogs such as octreotide, lanreotide and pasireotide. The marketing rights to Thiola were acquired by Retrophin in 2014 from Mission Pharmacol.

Trials 98

Trials Gene Hormones Clinical Trials 98

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

Drug Discovery World

APRIL 30, 2024

The biggest growth driver for mRNA therapeutics came from the Covid-19 pandemic, when mRNA-based vaccines proved effective in treating SARS-CoV-2, and pharmaceutical companies such as Pfizer/BioNTech and Moderna were successful in bringing these treatments to market. We believe it can now do the same for rare diseases.” “We

RNA 52

RNA Protein Vaccination Vaccine 52