pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The post Vertex, CRISPR prep filing for gene-editing blood disorder therapy appeared first on.

Gene Editing 59

Gene Editing Gene In-Vivo Clinical Trials 59

XTalks

DECEMBER 13, 2023

Casgevy’s approval by the FDA is momentous: it is the first CRISPR-based gene-editing therapy to be approved in the US. In 2022, bluebird won approvals for two gene therapies — Skysona for the treatment of the rare neurological disorder cerebral adrenoleukodystrophy (CALD) and Zynteglo for beta-thalassemia.

Gene Therapy 98

Gene Therapy Gene FDA Approval In-Vivo 98

pharmaphorum

JANUARY 10, 2022

With the two approvals acting as a validation of the technology, larger companies are prepared to make larger investments to ensure that they are at the forefront of developments in RNA-focused therapies.

RNA 111

RNA Vaccine Vaccination Pharma Companies 111

XTalks

DECEMBER 22, 2021

The gene-editing tool CRISPR edits DNA using specialized strands of guide RNA and endogenous cellular mechanisms of RNA interference. Non-invasive or minimally invasive medical devices that can diagnose, monitor and treat disease is entering a promising era, and will be a key life science trend next year.

Life Science 98

Life Science RNA Vaccine Vaccination 98

XTalks

DECEMBER 29, 2022

As of December 16, 2022, there are seven US Food and Drug Administration (FDA) approved gene therapies. In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. In 2023, a number of gene therapies are expected to get the FDA green light.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

XTalks

JANUARY 6, 2025

Notably, the ASO drug Vitravene (fomivirsen) received US Food and Drug Administration (FDA) approval for treating cytomegalovirus (CMV) retinitis in AIDS patients. RNA-based therapies hold the potential to offer new treatment options for diseases with limited or inadequate therapeutic alternatives.

Gene Therapy 111

Gene Therapy RNA Gene Editing Gene 111