News

InnovationRx: Sarepta Blinks In Showdown With FDA
In this week’s edition of InnovationRx, we look at Moderna’s use of quantum computers, a new top drug regulator at the FDA, ...
Sarepta falls after gene therapy maker pauses Elevidys shipments in US
By Sriparna Roy (Reuters) -Shares of Sarepta Therapeutics fell as much as 8% on Tuesday after the company agreed to comply with the U.S. health regulator's request to pause all shipments of its gene ...
BioSpace7h
Sarepta’s Wild Week, CDER’s New Leader, FDA Rejections, Manufacturing Billions
Sarepta Therapeutics faces serious FDA action after news broke of a third patient death, the FDA gets a new top drug ...
Sarepta Therapeutics laying off dozens in Ohio, won't comply with FDA request to halt therapy
After the FDA request, Cambridge, Massachusetts-based Sarepta said in a statement that it will continue to ship the therapy ...
TMEM63A gene linked to brain myelin defects in rare leukodystrophy
Researchers at Oregon Health & Science University have identified a gene that plays a crucial role in the brain's ability to ...
BioSpace1d
Lack of Transparency Tarnishes Sarepta’s Sheen as Patient Deaths Trigger FDA Battle
Sarepta Therapeutics’ stock has dropped precipitously as questions swirl around the safety of its gene therapies. Meanwhile, the Duchenne patient community fears losing access to Elevidys while the ...
Fierce Biotech2d
Sarepta LGMD trials all hit by FDA hold amid newly surfaced safety concerns over gene therapy
The Sarepta saga continues, with the FDA slapping a clinical hold across all of the company’s investigational limb-girdle ...
STAT2d
Pharmalittle: We’re reading about Sarepta resisting FDA over Elevidys, agency naming top drug regulator, and more
Sarepta Therapeutics refused a FDA request to halt shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy ...
Fierce Pharma2d
Sarepta stands behind Elevidys after FDA requests gene therapy be pulled from market
As the controversy over the safety of Sarepta Therapeutics’ gene therapies comes to a head, the biotech is standing firm ...
News-Medical.Net on MSN1d
Blocking a single gene disrupts vitamin D metabolism and cancer pathways in new cell study
Researchers identified SDR42E1 as a key regulator of vitamin D absorption and sterol metabolism in colorectal cancer cells.
STAT1d
Replimune skin cancer therapy rejected by FDA, a sign of regulators’ hardened stance
The FDA on Tuesday rejected a skin therapy from Replimune Group, suggesting a hardened stance on drug approvals under new agency officials ...
The Scientist1y
A Master Regulator of Gene Expression | The Scientist
To assess whether gene expression changes induced by BATF3 associated with positive clinical outcomes, the team compared the transcriptional signatures of T cells with or without BATF3 overexpression ...