Drug Discovery World

OCTOBER 4, 2022

The pandemic might have affected the number of clinical trials taking place for cell and gene therapies, with activity decreasing by around 15% compared to 2020, but major therapies such as Novartis’ gene therapy drug for spinal muscular atrophy (SMA) Zolgensma still made it to market. . billion, compared to $19.9

Gene Therapy 52

Gene Therapy Drugs Gene Manufacturing 52

Pharmaceutical Technology

FEBRUARY 20, 2023

Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. Other lines of research look at the genetic overlap between FTD and amyotrophic lateral sclerosis (ALS), which could be used in the development of treatments for both conditions.

Development 246

Development Gene Therapy Protein Gene 246

The Pharma Data

NOVEMBER 24, 2020

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. Until today, there were no approved pharmaceutical therapies for PH1.

FDA Approval 52

FDA Approval Production RNA Medicine 52