PET PET
  • My Account
  • Subscribe
Become a Friend Donate
  • About Us
    • People
    • Press Office
    • Our History
  • Get Involved
    • Become a Friend of PET
    • Volunteer
    • Campaigns
    • Writing Scheme
    • Partnership and Sponsorship
    • Advertise with Us
  • Donate
    • Become a Friend of PET
  • BioNews
    • News
    • Comment
    • Reviews
    • Elsewhere
    • Topics
    • Glossary
    • Newsletters
  • Events
    • Upcoming Events
    • Previous Events
  • Engagement
    • Policy and Projects
      • Resources
    • Education
  • Jobs & Opportunities
  • Contact Us
  • About Us
    • People
    • Press Office
    • Our History
  • Get Involved
    • Become a Friend of PET
    • Volunteer
    • Campaigns
    • Writing Scheme
    • Partnership and Sponsorship
    • Advertise with Us
  • Donate
    • Become a Friend of PET
  • BioNews
    • News
    • Comment
    • Reviews
    • Elsewhere
    • Topics
    • Glossary
    • Newsletters
  • Events
    • Upcoming Events
    • Previous Events
  • Engagement
    • Policy and Projects
      • Resources
    • Education
  • Jobs & Opportunities
  • Contact Us
  • My Account
  • Subscribe
  • Privacy Statement
  • Advertising Policy
  • Thanks and Acknowledgements
PETBioNewsNewsFDA advisors back gene therapy for rare inherited blindness

BioNews

FDA advisors back gene therapy for rare inherited blindness

Published 16 October 2017 posted in News and appears in BioNews 922

Author

Rikita Patel

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.

The US Food Drug and Administration advisory committee has backed the use of gene therapy to treat a hereditary disease for the first time...

The US Food Drug and Administration (FDA) advisory committee has backed the use of gene therapy to treat a hereditary disease for the first time.

Luxturna has been tested to treat patients who have inherited retinal dystrophy, a disease caused when both copies of the RPE65 gene carry a mutation. The eyes' ability to respond to light is increasingly impaired and patients eventually become blind.

'This would be the first approved treatment of any sort for this condition and the first approved gene therapy treatment for the eye, in general,' Dr Paul Yang of Oregon Health and Science University told National Public Radio. 'So, on multiple fronts, it's a first and ushers in a new era of gene therapy', he added.

In a clinical trial of 31 people, sponsored by Spark Therapeutics and led by Professor Albert Maguire of the University of Pennsylvania, Luxturna significantly improved the visual outcome in people aged between four to 44 years old who received the drug as a one-time sub-retinal injection, compared to a control group.

The injection consisted of a genetically engineered adeno-associated viral vector, which contained the normal copy of the RPE65 gene. When injected into the retinal cells, the gene expressed the RPE65 protein: a vital enzyme for normal retinal function.

The effectiveness of the therapy was determined by using a novel approach: multi-luminance mobility testing, which assesses how well a person is able to navigate and move under different light levels. At one year post-treatment, those who had received treatment showed improved visual acuity, compared to the control group.

However, there were a few complications, including events related to the procedure and permanent retinal damage caused by use of prednisone medication. Taking into consideration, all 16 members of The Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) decided the efficacy data outweighed the safety risks, and gave favourable recommendation to the FDA to approve Luxturna.

In most cases, the FDA follows the recommendations set by advisory committee but there is currently a series of unanswered questions, as outlined in the FDA briefing document. These include: 'what additional data, if any, would be necessary to support such repeat administration?' and, 'at what stage of clinical presentation do the benefits of therapy outweigh the risks?'

Furthermore, the long-term effects of the treatment are unknown. Speaking to Nature, Professor Mark Kay, a geneticist at Stanford University, California, said: 'I think we still need to have major improvements in the technology before we're going to be able to cure these diseases, but along the way there may be treatments that help make improvements'.

A decision from the FDA on Luxturna is expected by early next year.

Related Articles

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
5 September 2019 • 2 minutes read

NHS to provide gene therapy for rare eye disease

by Jen Willows

An in-body gene therapy treatment has been approved to treat a rare genetic form of blindness...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
12 July 2019 • 2 minutes read

NHS approves gene-silencing drug for rare hereditary condition

by Charlotte Spicer

The first RNA-based therapy has been approved for NHS use in England...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
31 August 2018 • 2 minutes read

Genome editing relieves muscular dystrophy in dogs

by Dr Charlott Repschläger

Dogs with Duchenne muscular dystrophy (DMD) have been treated using genome editing, a study in Science reports...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
26 February 2018 • 2 minutes read

FDA issues warning on DIY gene-therapy kits

by Theofanis Michailidis

The US Food and Drug Administration (FDA) is taking an active stance against the use of 'do it yourself' gene therapy kits...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
4 December 2017 • 2 minutes read

FDA to speed up gene therapy and regenerative medicine approvals

by Martha Henriques

The US Food and Drug Administration has announced a fast-track review process for gene therapies and other regenerative medicine treatments...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
19 October 2017 • 2 minutes read

Gene therapy restores vision in blind mice

by Dr Kimberley Bryon-Dodd

Gene therapy has restored some vision in mice blinded by retinitis pigmentosa, an inherited degenerative eye disease...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
14 September 2016 • 2 minutes read

Gene therapy reverses inherited blindness

by Sarah Gregory

A trial of gene therapy for choroideremia, a rare form of inherited blindness, has partially restored the vision of several patients...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
21 August 2015 • 2 minutes read

Gene therapy restores sight in mice

by Dr Antony Starza-Allen

An experimental form of gene therapy to treat blindness has been showed to restore some vision in mice....

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
15 December 2014 • 2 minutes read

Gene therapy partially restores sight in mice and dogs

by Dr Rosie Morley

Scientists have used gene therapy to restore some sight to animals with visual impairment similar to retinosa pigmentosa, a type of human blindness...

Leave a Reply Cancel reply

You must be logged in to post a comment.

« Epigenetics of addiction relapse probed using rats

Data-Label The UK's Leading Supplier Of Medical Labels & Asset Labels

RetiringDentist.co.uk The UK's Leading M&A Company.

Find out how you can advertise here
easyfundraising
amazon

This month in BioNews

  • Popular
  • Recent
8 August 2022 • 2 minutes read

Placenta and organ formation observed in mouse embryo models

8 August 2022 • 2 minutes read

Lower hormone doses may improve IVF egg quality

8 August 2022 • 2 minutes read

Boosting muscle cell production of gene therapy proteins

1 August 2022 • 2 minutes read

First UK medical guidelines issued for trans fertility preservation

1 August 2022 • 2 minutes read

Male age has more impact on IVF birth rate than previously thought

8 August 2022 • 2 minutes read

Placenta and organ formation observed in mouse embryo models

8 August 2022 • 2 minutes read

Complex structures of the human heart bioengineered

8 August 2022 • 1 minute read

Brain tumour gene also linked to childhood cancers

8 August 2022 • 2 minutes read

Lower hormone doses may improve IVF egg quality

8 August 2022 • 2 minutes read

Boosting muscle cell production of gene therapy proteins

Subscribe to BioNews and other PET updates for free.

Subscribe
  • Twitter
  • Facebook
  • Instagram
  • LinkedIn
  • YouTube
  • RSS
Wellcome
Website redevelopment supported by Wellcome.

Website by Impact Media Impact Media

  • Privacy Statement
  • Advertising Policy
  • Thanks and Acknowledgements

© 1992 - 2022 Progress Educational Trust. All rights reserved.

Limited company registered in England and Wales no 07405980 • Registered charity no 1139856

Subscribe to BioNews and other PET updates for free.

Subscribe
PET PET

PET is an independent charity that improves choices for people affected by infertility and genetic conditions.

  • Twitter
  • Facebook
  • Instagram
  • LinkedIn
  • YouTube
  • RSS
Wellcome
Website redevelopment supported by Wellcome.

Navigation

  • About Us
  • Get Involved
  • Donate
  • BioNews
  • Events
  • Engagement
  • Jobs & Opportunities
  • Contact Us

BioNews

  • News
  • Comment
  • Reviews
  • Elsewhere
  • Topics
  • Glossary
  • Newsletters

Other

  • My Account
  • Subscribe

Website by Impact Media Impact Media

  • Privacy Statement
  • Advertising Policy
  • Thanks and Acknowledgements

© 1992 - 2022 Progress Educational Trust. All rights reserved.

Limited company registered in England and Wales no 07405980 • Registered charity no 1139856