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 ...
BioSpace

Intellia’s CRISPR Gene Therapy Free To Move Forward After FDA Fully Lifts Hold

The FDA last October paused Intellia Therapeutics’ late-stage CRISPR studies after detecting life-threatening enzyme ...

FDA releases hold on second of Intellia’s ATTR-CM gene therapy trials

Intellia is now able to continue both Phase III trials of nex-z. Credit: Piotr Swat / Shutterstock.com (Piotr Swat / Shutterstock.com) The US Food and Drug Administration (FDA) has released the ...

Jaguar Gene Therapy completes Cohort I patient dosing in JAG201 clinical trial

Clinical-stage biotechnology company Jaguar Gene Therapy has completed dosing in Cohort I of its first-in-human clinical ...
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 ...
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, ...

Hope And Hurdles For Sickle Cell Gene Therapy

CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...
Star Local Media

Frisco mother leads gene therapy breakthrough for her son

A Frisco mom’s yearslong fight led to a gene therapy breakthrough for the rare SLC6A1 disorder. Her son Maxwell is the first patient treated and is already showing promising progress.
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 ability to properly define a patient population, the use of a natural history ...
European Medical Journal

Gene Therapy Breakthrough Restores Key Function in Cystic Fibrosis

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new avenues for CF therapy.
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 ...

After a baby became the world's first CRISPR gene editing therapy patient, CHOP wants to expand treatment to others

One year after receiving CRISPR gene editing therapy at CHOP, a Delaware County baby is thriving, and doctors want to expand ...