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.
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.
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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.
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.
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.
Drug Discovery World
FEBRUARY 22, 2024
For diseases as serious and debilitating for patients and their families as leukodystrophies, learning about such advances in knowledge carries a great deal of hope, which IRCM warmly welcomes,” adds Dr Jean-François Côté, the IRCM’s President and Scientific Director.
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