Worldwide Clinical Trials

JUNE 15, 2022

At the end of May, we hosted a webinar titled “ Changing Times, Changing Therapies: Keeping Up with Advancements in Cell and Gene Therapies ” to provide a quick update on the latest advancements and ongoing in development of these advanced therapeutics. Around 40% of clinical holds are for gene therapy programs.

Gene Therapy 130

Gene Therapy Gene Clinical Trials Clinical Trials 130

XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

Gene Therapy 78

Gene Therapy Gene Trials Clinical Trials 78

XTalks

JANUARY 2, 2024

Filsuvez topical gel is a sterile botanical drug product designed for topical use, containing birch triterpenes within an oil base. For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. Vyjuvek is a gene therapy that employs a herpes simplex virus type 1 (HSV-1) vector.

FDA Approval 102

FDA Approval Gene Therapy Gene Trials 102

Pharmaceutical Technology

OCTOBER 26, 2022

The biotherapeutics market is rapidly growing, with 2021 seeing the highest-ever cell and gene therapy approval number. Gene therapy uses DNA to manipulate cells and correct defective genes, whereas cell therapy is the infusion or transplantation of cells into a patient.

Gene Therapy 130

Gene Therapy Gene Marketing Manufacturing 130

Pharmaceutical Technology

DECEMBER 9, 2022

In regards to the latter, notable examples include the acquisition of Cergentis, a genomics-focused biotechnology company based in the Netherlands. The deal allows the company to provide proprietary technology and knowledge steeped in fast-evolving genomic techniques that support effective decision-making and R&D program design.

Development 130

Development Production Manufacturing Vaccination 130

The Pharma Data

NOVEMBER 3, 2020

LogicBio’s proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Editing Genomics 52

Roots Analysis

MARCH 16, 2023

Further, these can be used to activate both innate and adaptive immune system responses by silencing or upregulating immune-relevant genes. mRNA drugs are considered to offer an effective alternative to gene therapies, specifically for protein replacement, as they avoid the risk of genome integration and offer strong transient expression.

Vaccination 52

Vaccination Vaccine DNA Protein 52