Goggles and a gene injection give some retinitis pigmentosa patients limited object detection
Four of eight patients tested consistently detected a notebook through optogenetic goggles after one gene injection, a 10-person NEJM trial reports. The therapy targets surviving ganglion cells, so it need not match any of 100-plus disease genes, but ScienceAlert's account does not break results out by gene.
Reality
- Evidence45
- Adoption
- Not scored
- Hype gap+20
- Incentives55
- Confidence50
Optogenetic gene therapy was safe in 10 blind people with advanced retinitis pigmentosa, six of whom gained clinically meaningful light sensitivity. The gains were measured within each patient in a small dose-escalation study, so the result supports safety and shows the approach works in people while sharp vision stays years away.
Reality
- Evidence60
- Adoption
- Not scored
- Hype gap+15
- Incentives
- Not scored
- Confidence65
Optogenetic gene therapy restored some vision to people blinded by retinitis pigmentosa in a small NEJM trial. Seven of 10 participants improved in light sensitivity, Science News reported, though the goggle-aided sight let none of them read words or see faces.
Reality
- Evidence50
- Adoption
- Not scored
- Hype gap+15
- Incentives
- Not scored
- Confidence55
IEEE poll of 166 experts expects gene therapy to be used frequently by 2028
IEEE's 2030 Technology Megatrends Report, built from 166 experts in 38 countries, forecasts frequent use of gene therapy by 2028. The dates are graded expert opinion, good for seeing where specialists expect medicine and energy to go and a weaker guide to timing than clinical trials.
Reality
- Evidence30
- Adoption
- Not scored
- Hype gap+35
- Incentives55
- Confidence40
Bird-genome search yields 159 RNA-only tools for inserting genes in human cells
Researchers found 159 R2 retrotransposons in 1,139 avian genomes and engineered variants that reached up to 60% gene insertion in human primary cells. These are RNA-only tools, able to add a gene to the genome without delivering any DNA to the cell.
Reality
- Evidence58
- Adoption
- Not scored
- Hype gap+10
- Incentives50
- Confidence55
Single-dose gene therapy rebuilt retinal wiring in adult whippets with CaBP4 vision loss
Michigan State researchers say one dose of CaBP4 gene therapy improved whippets' vision and rebuilt retinal synapses, with gains lasting up to three years. It shows adult retinal wiring can still be remodelled, so far only in dogs.
Reality
- Evidence45
- Adoption
- Not scored
- Hype gap+35
- Incentives
- Not scored
- Confidence40
Regeneron's Otarmeni becomes the first US-approved gene therapy for hearing loss
Regeneron's Otarmeni won FDA approval in April as the first gene therapy for hearing loss, with 42 percent of 24 treated children since regaining hearing. The deafness it treats leaves the rest of the ear intact. How far the approach can reach beyond this one gene is still unknown.
Reality
- Evidence58
- Adoption
- Not scored
- Hype gap+10
- Incentives
- Not scored
- Confidence52
Sickle cell went 113 years from first case report to approved gene-editing treatment
Kevin Davies's Curved Air follows a single point mutation from its 1910 case report to the two gene therapies approved in December 2023, and Davies calls the disease underfunded and forgotten by much of medicine and pharma.
Reality
- Evidence58
- Adoption24
- Hype gap+14
- Incentives68
- Confidence62