How one family gave a critical boost to gene therapy research

A family affected by Sanfilippo syndrome raised millions of dollars for research. The Food and Drug Administration just ...
BioSpace

Plug-and-play gene therapy: FDA executes on vision that’s been in the works for years

Former biologics boss Peter Marks had a vision. With the FDA’s latest draft guidance, it’s coming to fruition. Speaking with BioSpace, current CBER leader Karim Mikhail said the “adapted pathway” is ...
Tech Times on MSN

UK clears Casgevy gene therapy for children as young as five; NHS funding unresolved

Casgevy gene therapy for UK children was extended to patients aged five and over by the MHRA on October 29, 2026; a milestone ...
Healio

Positive outcomes seen with intravitreal gene therapy for wet AMD

In a phase 2 study, SAR402663 gene therapy for wet age-related macular degeneration showed positive efficacy and safety ...
STAT

FDA reassures rare disease advocates that ‘being flexible’ is plan for gene therapy

Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77. What will the Trump administration mean for ...
The American Bazaar

Vishal and Anubha Suri’s gift to launch UVA gene therapy center

Indian American couple’s gift will help combine genetic research with nanotechnology to develop more effective gene therapies ...
Nature

Gene therapy that led to boy’s death had limited efficacy

Therapy pioneered by biotechnology Chinese firm HuidaGene showed promise in animals but caused a fatal reaction in a child.

Q&A: Gene editing is coming for common diseases. Cardiologists discuss if we are ready

Gene therapy is no longer the future of medicine—it's reality. Treatments that replace, silence or edit genes are already ...
RTTNews

Pelthos To Merge With Castle Creek, Expanding Cell And Gene Therapy Pipeline

Pelthos currently markets three FDA-approved dermatology products: ZELSUVMI (berdazimer) for molluscum contagiosum, XEPI (ozenoxacin) for impetigo and XEGLYZE (abametapir) for head lice.
Forbes

A Turning Point For Gene Therapy: What One Tragic Event Reveals About The Promise And Peril Of Genetic Medicine

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

Next-Generation Adeno-Associated Virus (AAV) Gene Therapy Delivery Market Report 2026 | Capitalize on $11.91B Market as Novartis, Voyager and Affinia Accelerate Engineered Capsids

Players: Pfizer, Novartis, Bayer, Astellas and Sarepta. Profile: next-gen AAV delivery, including engineered capsids, targeting, safety and scalable manufacturing.Dublin, Oct. 30, 2026 (GLOBE NEWSWIRE ...
BioSpace

Neurogene Presents New Data at ESGCT Congress that Reinforce Key Differentiators of NGN-401 Gene Therapy for Rett Syndrome

New data showing rapid and durable full-length MECP2 expression in vivo with single-stranded AAV support improvements ...