Managed Healthcare Executive

A Q&A with Zahra Mahmoudjafari on building successful cell and gene therapy programs

Zahra Mahmoudjafari explains how cell and gene therapy programs succeed with strong infrastructure, pharmacy leadership, ...
Fierce Biotech

UniQure shares crash 40% as FDA rejects early approval path for Huntington's gene therapy

Shares of uniQure plummeted more than 40% in premarket trading Monday after the company confirmed the FDA has rejected its current data package for AMT-130, a highly anticipated gene therapy for ...

US FDA lifts clinical hold on Intellia's heart disease gene therapy trial

March 2 (Reuters) - Intellia Therapeutics said on Monday the U.S. Food and Drug Administration has removed a clinical hold on ...
BioSpace

UniQure Eyes Phase 3 for Huntington’s Gene Therapy Despite Makary’s Apparent Criticism

Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed ...
Fierce Biotech

FDA fully releases clinical hold on Intellia CRISPR gene therapy trials

The FDA has liberated the second of two Intellia Therapeutics clinical trials that were put on hold last October following a report of serious liver toxicity that later led to death.

UniQure Down in Premarket as FDA Says Huntington's Gene-Therapy Data Falls Short

Huntington's disease is an inherited and progressive neurodegenerative disorder that is caused by faulty genes triggering the breakdown of nerve cells in the brain. UniQure said the regulator is ...

FDA tells uniQure to conduct another study of Huntington’s gene therapy

Qure's stock has dropped by more than 40% after being asked to conduct another study of its Huntington’s disease gene therapy. During a Type A meeting, the US Food and Drug Administration (FDA) told ...

UniQure Falls as FDA Seeks Controversial Gene Therapy Study

UniQure NV’s shares plunged after US regulators said the company should conduct another study of its Huntington’s disease ...

Gene therapy is transforming lives, but for many Americans it's hard to reach

Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

First successes in the development of a gene therapy for incurable LAMA2-related muscular dystrophy

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...
Fierce Pharma

BioMarin pulls hemophilia gene therapy Roctavian, taking $240M hit after divestiture efforts flounder

Still, broad uptake has long been an uphill battle for the gene therapy as it competes with a variety of treatments including, Roche’s blockbuster Hemlibra, Sanofi’s newer sales driver Altuviiio and ...

UniQure says FDA wants another study of Huntington’s gene therapy

Agency staff "strongly recommended" a sham surgery-controlled trial be conducted before an approval filing, a stance one ...