Zirconia thin films unlock new reversible nonpolar-to-polar mechanism

Researchers from National Taiwan University break traditional frameworks by unveiling a new symmetry-transition mechanism in ...
Targeted Oncology

Understanding Holding vs Bridging Therapy for CAR T in Multiple Myeloma

T-cell therapy is the most efficacious approach for many patients with relapsed/refractory multiple myeloma, but it currently comes with 2 periods ...

Nanobody repairs misfolded CFTR inside cells, boosting function in cystic fibrosis

A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers ...
Frontiers

Cell Death Modalities in Neurological Disorders: Mechanisms, Crosstalk, and Translational Perspectives

Cell death research has emerged as a cornerstone for understanding the pathogenesis of neurological disorders. Neurons and glial cells in the central ...
RAPS

Former officials propose changes to FDA’s plausible mechanism pathway

Two former US Food and Drug Administration (FDA) officials have commented on and proposed changes to the agency’s recent draft guidance on the development of a plausible mechanism framework for ...
The American Journal of Managed Care

5 FDA Developments From February: Kinase Inhibitors, GLP-1s, and a New Approval Pathway

Zongertinib gained accelerated approval for unresectable/metastatic HER2 kinase-domain–mutant nonsquamous NSCLC, with 76% ORR and DOR ≥12 months in 44%. AMPLIFY ...
FierceBiotech

UPDATED: FDA illuminates new approval pathway for bespoke gene editing therapies

After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...
STAT

FDA unveils rules for bespoke gene therapies, predicting flood of rare disease applications

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...
MedPage Today

FDA Proposes New Approval Pathway for Rare Disease Gene Therapy

Under the new "plausible mechanism" framework, "one well-controlled clinical investigation supported by confirmatory evidence can support approval," said HHS Secretary Robert F. Kennedy Jr. Photo ...
RAPS

FDA proposes plausible mechanism pathway for ultra-rare disease therapies

The US Food and Drug Administration (FDA) has proposed a new pathway for developing individualized therapies based on a plausible mechanism framework. If finalized, the guidance would lay out a path ...
Healio

FDA provides new approval process for rare disease therapies without large clinical trials

Please provide your email address to receive an email when new articles are posted on . The pathway aims to streamline approval for individualized treatments for rare and ultra-rare diseases. An ...
STAT

FDA’s new ‘plausible mechanism pathway’ for personalized gene editing raises concerns

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...