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Gene therapy and special goggles give some blind people limited sight
Confirmed
In Short: The team followed 15 children, aged from 15 months to 12 years, who were treated with the gene therapy at Great Ormond Street Hospital (GOSH) between 2020 and 2023.
An international research team has reported new results from the first clinical cohort of blind people treated with optogenetic therapy for advanced retinitis pigmentosa, a condition that can lead to blindness.
Optogenetic therapy, based on Nobel prize-winning science, involves turning nerve cells on and off using light. This approach has been shown to be safe and can partly restore sight in blind people.
In a study published in Mirage News, Stefan Futterknecht, second author of the study, stated, “This study represents an important milestone for optogenetic vision restoration. It demonstrates that the effects first observed in a single patient can be reproduced across several patients and provides a foundation for developing more sensitive and effective future therapies.”
The findings build on a 2021 report that the same approach helped one blind man see and count objects. University of California, Berkeley scientists have also inserted a gene for a green-light receptor into the eyes of blind mice, and a month later, the mice were navigating around obstacles as easily as mice with no vision problems.
The researchers noted that they have gone from one patient to 10 patients, seeing improvement in their light sensitivity, and that they can give a stable restoration of some visual function, some light sensitivity, that lasts for four or five years in these patients.
The gene therapy involves replacing the faulty gene in the immune cells with healthy ones, rather than the whole cell, as developed by the UCL team.
By acting as sponsor and manufacturer for this trial, Great Ormond Street Hospital is showing its ongoing commitment to translating innovative science into clinical benefit.
The optogenetic therapy is a significant step forward in treating retinitis pigmentosa, offering hope to those who might otherwise lose their sight by the age of 30.
The research team is optimistic about the future of optogenetic therapy, aiming to develop more sensitive and effective treatments.
The study's success underscores the potential of optogenetic therapy to restore sight in blind individuals, marking a defining moment in the field of gene therapy.
The study's results are a significant advancement in optogenetic therapy, showing consistent improvements across multiple patients.
The gene therapy approach used in this study is different from traditional gene therapies, focusing on replacing faulty genes in immune cells rather than the whole cell.
What's confirmed
- An international research team has reported new results from the first clinical cohort of blind people treated with optogenetic therapy for advanced retinitis pigmentosa, a condition that can lead to blindness.
- Optogenetic therapy, based on Nobel prize-winning science, involves turning nerve cells on and off using light. This approach has been shown to be safe and can partly restore sight in blind people.
- The findings build on a 2021 report that the same approach helped one blind man see and count objects. University of California, Berkeley scientists have also inserted a gene for a green-light receptor into the eyes of blind mice, and a month later, the mice were navigating around obstacles as easily as mice with no vision problems.
- The researchers noted that they have gone from one patient to 10 patients, seeing improvement in their light sensitivity, and that they can give a stable restoration of some visual function, some light sensitivity, that lasts for four or five years in these patients.
- The gene therapy involves replacing the faulty gene in the immune cells with healthy ones, rather than the whole cell, as developed by the UCL team.
- By acting as sponsor and manufacturer for this trial, Great Ormond Street Hospital is showing its ongoing commitment to translating innovative science into clinical benefit.
- The optogenetic therapy is a significant step forward in treating retinitis pigmentosa, offering hope to those who might otherwise lose their sight by the age of 30.
- The research team is optimistic about the future of optogenetic therapy, aiming to develop more sensitive and effective treatments.
- The study's success underscores the potential of optogenetic therapy to restore sight in blind individuals, marking a defining moment in the field of gene therapy.
- The study's results are a significant advancement in optogenetic therapy, showing consistent improvements across multiple patients.
- The gene therapy approach used in this study is different from traditional gene therapies, focusing on replacing faulty genes in immune cells rather than the whole cell.
What's still developing
- Her mother Lisa said the family had been told Saffie would have been "blind by the age of 30" without treatment.
- "It was such a rollercoaster of a journey, but we were so relieved and grateful when we heard there was a treatment available on the NHS."
- Lisa said she set her expectations "quite low" ahead of the surgery but could never have imagined the results.
- Like Beam’s other therapies, the AATD treatment BEAM-302 is built on “base editing” technology that precisely changes a specific nucleotide, or letter, in genetic code.
- “Today’s FDA approval of Isembyld marks a defining moment for the SMA community,” said David Hallal, CEO of Scholar Rock, in a press release.
- “After decades of failed industry-wide efforts to unlock the potential of myostatin inhibition, Scholar Rock has delivered a therapeutic breakthrough.” To read the rest of this story subscribe to STAT+.
- Gene Hamilton explains what the ruling allows the administration to do next, when states could return to court and how federal agencies may move to implement Trump’s mail-voting order ahead of the midterms.
- "But applying the rule in the 2026 elections would be arbitrary and capricious in violation of the Administrative Procedure Act because state and local election officials do not have sufficient time to reasonably implement the rule before the elections."
- In a press release, the drug’s maker, Elevar, said lirafugratinib “selectively targets FGFR2, differentiating it from existing pan-FGFR inhibitors that broadly inhibit FGFR1, FGFR3, and/or FGFR4 in addition to FGFR2. Consistent with its high selectivity, the rates of hyperphosphatemia and diarrhea – adverse events commonly associated with non-selective FGFR inhibitors – were 20.7% and 21.6%, respectively,” in testing.
- The FDA has approved lirafugratinib (Lyrfigtu, Elevar Therapeutics) for adults with previously treated unresectable, locally advanced, or metastatic cholangiocarcinoma harboring a fibroblast growth factor receptor 2 (FGFR2) gene fusion or other rearrangement.
- The entire field of optogenetics traces back to light-seeking algae.
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