"Truly evil" FDA rejection of gene therapy overturned after Trump official ousted

Gene therapy company UniQure had another FDA meeting after Vinay Prasad's exit.

Ars Technica

Clinic booking ≠ gene therapy screening.

Evaluating advanced therapy review pathways in China requires a review-ready medical dossier—not just a standard clinic booking. Missing pathology records, incomplete treatment history, or poorly translated genetic reports can stop institutional review before it begins. MedBridgeNZ organizes medical records and molecular reports for administrative review-readiness.

#GeneTherapy #RareDisease #MedicalConcierge

The Pacemaker Patch

A pacemaker is implanted to send signals that regulate a patient’s heartbeat, and to do that, you need power. That means they require battery changes, and when the device in question happens …

Hackaday

Exploring advanced therapeutics abroad? Accessing gene therapy pathways in China requires precise record translation and strict institutional screening.

As a specialized medical concierge provider, MedBridgeNZ handles the complex logistics of Medical Tourism China—so you and your doctors can focus on the science.

Read our full guide on navigating these administrative pathways: https://zurl.co/u0AKU

#MedicalTourismChina #GeneTherapy #MedicalConcierge #MedBridgeNZ

international patient access to gene therapy in China

Home > Blog > Advanced Therapies & TechnologyKey Takeaways • Dual-Track Regulatory Framework: China operates a system encompassing both marketed advanced therapies and formal investigator-initiated trials (IITs) guided by hospital ethics committees.   • Protocol-Driven Eligibility: Institutional acceptance into any pathway remains strictly subject to clinician judgment, protocol criteria, and site capacity.   • Mandatory Genotype Confirmation: Accessing specific specialist reviews frequently req

Medbridge Nz
In San Francisco, researchers are preparing the first human trial of #in-utero #GeneTherapy, starting with a rare lysosomal storage disorder.

archive.md/ap00C

#GeneTherapy #HeartDisease

"Gene therapies to fix failing hearts gain steam after years in the doldrums

Treatments aim to bulk up failing hearts to restore their vigour — but can the field move on from past controversy?

The first clinical trial aimed at growing new heart-muscle cells is now under way, and companies are developing at least four other regenerative gene therapies for heart conditions.

'These are the first-in-human studies to take regeneration into the clinic,' says Andrew Baker, a gene-therapy researcher at the University of Edinburgh, UK, who is unaffiliated with the efforts. 'It’s a very exciting time.'

But the excitement is tempered with caution. The mammalian heart is bad at repairing itself and 'is notoriously unwieldy when it comes to efforts to try to get it to regenerate', says Sean Wu, a cardiologist at Stanford University in California. Some scientists are unconvinced that the data underpinning the existing clinical trial show true regeneration in the form of cell division. And efforts to regenerate the heart are still haunted by a controversy that led to the retraction of at least a dozen papers and the closure of a high-profile laboratory.

Developing a gene therapy in this field will be difficult, says Antonio Abbate, a cardiologist at the University of Virginia in Charlottesville. But 'we have to do research because, eventually, we’ll get it right'"

https://archive.ph/UbQtS

@feather1952 @Susan60 @Spoon
This is why I find med research so incredibly hopeful (not unbiased here since my daughter is a human biology researcher). That we can identify genes/proteins which cause deadly and debilitating disorders and take precautions to mitigate their transmission if they cannot be switched off. That and the good fortune of living in a country (never ceded) where body autonomy is still acknowledged and not impinged on by batshit crazy religious zealots. I have reasons to be hopeful more can be done in the future.

#Science #MedicalResearch #Proteonomics #GeneTherapy #Healthcare #HereditaryDisorders

A Phase 1b clinical trial of Eli Lilly’s gene therapy VERVE-102 has shown up to 62% reduction in LDL cholesterol with a single dose, raising hopes for long-term treatment of hypercholesterolaemia and heart disease risk. https://english.mathrubhumi.com/lifestyle/health/one-time-gene-therapy-ldl-cholesterol-lowering-sh8q28ss?utm_source=dlvr.it&utm_medium=mastodon #GeneTherapy #BadCholesterol #Biotechnology #ClinicalTrial