Fierce Biotech

UPDATED: Senior FDA official refutes uniQure's claims amid controversy over Huntington's gene therapy

In a strikingly blunt briefing, a senior FDA official accused uniQure of pushing “distorted” data to mask a “failed” therapy ...
BioSpace

Why Industry Is Questioning FDA’s Data-Sharing Push in Cell and Gene Therapy

After the FDA’s first-ever public listening meeting on data-sharing in the cell and gene therapy space, new draft guidance ...
BioSpace

UniQure’s Path for Huntington’s Gene Therapy Clouded by Ethical Questions as Potential Phase 3 Looms

While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be ...
Health AffairsOpinion

The FDA Removed REMS For CAR-T Cell Therapy—Now Community Providers Can Expand Access

Consider the case of patient WB, a 79-year-old male with recurrent diffuse large B cell lymphoma. He was referred for ...
Clinical Trials Arena on MSN

Ultragenyx’s gene therapy meets co-primary endpoint in Phase III trial

Ultragenyx said that the trial of the gene therapy will continue while data from the second primary endpoint is collected.
Fierce Biotech

FDA details rationale for rejecting rare disease gene therapy from Regenxbio

RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ...
MPR

Gene Therapy Fast Tracked for BAG3-Associated Dilated Cardiomyopathy

AFTX-201 uses a proprietary capsid to deliver a human BAG3 transgene, aiming to restore cardiac function with lower doses than conventional gene therapies.
Zacks Investment Research on MSN

SRPT stock up as enrollment begins in DMD gene therapy safety study

Shares of Sarepta Therapeutics SRPT rose about 2% yesterday after it announced that screening and enrollment are underway in ...

Intellia gets FDA nod to resume second gene therapy trial after safety pause

By Christy Santhosh March 2 (Reuters) - Intellia Therapeutics said on Monday the U.S. Food and Drug Administration has lifted the clinical hold on a late-stage trial of its gene therapy for a heart ...
MedPage Today on MSN

Cardiologist's Fake Cancer; Gene Therapy Trial Resumes; More Impella Woes

Recent developments of interest in cardiovascular medicine ...

Locus Cell, Taiwan-Based Cell and Gene Therapy CDMO, Signs MoU with Charles River at APAC Biomanufacturing Leadership Summit

The MoU establishes a framework for the Parties to explore potential collaboration in CMC support, analytical testing, technical development, and related services for advanced therapy developers in ...