THE TRUTH ABOUT: Gene therapy - The Untold Story

Cell and Gene Therapy Makers Should Prove Every Batch Works as FDA Replaces Its 2011 Potency Guidance

The FDA's final guidance urges cell and gene therapy makers to use broader potency assurance strategies, combining manufacturing controls and testing to help ensure each batch delivers its intended effect.

Medical Daily · 11h ago

Arizona parents donate adventure van to launch charity for son with rare disease; research grants now reach $6.1 million

After their son Luke was diagnosed with Bardet-Biedl syndrome, an Arizona couple established A Race Against Blindness. The charity has since raised over $6.1 million for research into inherited retinal diseases. They also organized a sweepstakes featuring a custom adventure van, significantly increasing awareness. Their contributions support groundbreaking gene therapy…

The Times of India · Oct 31

Custom Gene-Editing Therapies for Rare Diseases Could Win Faster Approvals Under FDA Draft Plan, Not Yet Final

The FDA's draft guidance could streamline approval of gene-editing therapies tailored to individual mutations, while families and experts weigh safety, affordability, evidence requirements, and access challenges.

Medical Daily · Oct 30

Gene therapy can partly restore sight in blind people, researchers reveal

Optogenetic therapy, based on Nobel prize-winning science, involves turning nerve cells on and off using light

The Guardian - UK · Oct 7

Huntington's Gene Therapy Still Appears to Slow Daily Function Decline at Four Years, but Main Score Misses Significance

Four-year AMT-130 data show a lasting daily function signal, but the main Huntington's score lost significance. What families should know.

Medical Daily · Oct 1

Gene therapy: The breakthrough that inspired Spider-Man | Euronews Tech Talks

Although an inspiration for science fiction movies, gene therapy is real. Here is how it works and what could be improved about it.

Euronews · Sep 30

FDA Clears First Gene Therapy for Sanfilippo Syndrome Type A with Shipments to Treatment Centers Within 60 Days

The FDA approved Fayuvi, the first gene therapy for Sanfilippo syndrome type A. What families should know about access, cost and safety checks.

Medical Daily · Sep 18

uniQure Files for US Approval of Its Huntington's Gene Therapy and Asks Regulators for Faster Review

uniQure filed for FDA approval of AMT-130, a one-time Huntington's gene therapy, and requested priority review. Here is what it means.

Medical Daily · Sep 3

Hunter Syndrome Gene Therapy Halted by FDA After Spine Scans Show Small Masses in Five Children

FDA placed REGENXBIO's RGX-121 on clinical hold after spine MRIs showed nodules or cysts in five Hunter syndrome trial participants.

Medical Daily · Aug 25

Gene Therapy for Glycogen Storage Disease Type Ia Wins Accelerated Approval Priced at 2.7 Million Dollars

FDA granted accelerated approval to Genglycos for glycogen storage disease type Ia. It reduces cornstarch use, and the long-term benefit is unproven.

Medical Daily · Aug 21