News

BioPharma Dive1d
FDA rejects Ultragenyx gene therapy over manufacturing concerns
While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather ...
Precision Medicine Online1d
FDA Declines to Approve Ultragenyx's Gene Therapy for Sanfilippo Syndrome Type A
The firm said the agency cited chemistry, manufacturing, and controls issues in its complete response letter, but had no concerns about product quality.
STAT1d
FDA declines to approve Ultragenyx gene therapy
In his first 100 days as commissioner of the Food and Drug Administration, Marty Makary has made a handful of splashy ...
Rewriting Genetic Fate: Universal Newborn Genomic Screening Arrives
As predicted the era of universal newborn genomic screening is dawning. The UK and Florida are leading the way.
Crispr Therapeutics' Bright Future Is Coming Into Focus (Rating Upgrade)
CRISPR Therapeutics is revolutionizing medicine with gene editing. Learn more about CRSP's breakthroughs, stock rally, and ...
US FDA declines to approve Ultragenyx's gene therapy for rare genetic disorder
The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...
Promising leap for cancer therapies: Gene-editing breakthrough rewires T cells to attack tumors safely
T cell-based immunotherapy, a form of cancer treatment, harnesses a patient’s own T cells — white blood cells that help fight disease — by modifying or enhancing them to target and destroy cancer ...
The Manila Times6d
Purespring Therapeutics announce FDA IND clearance for Phase I/II clinical trial for primary IgA nephropathy (IgAN)
PS-002, Purespring’s lead precision nephrology programme, targets the complement pathway known to be a driver of IgA ...