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Gene Therapy

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November 29, 2022
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Eikonoklastes, Forge announce partnership to develop ALS gene therapy

Eikonoklastes, Forge announce partnership to develop ALS gene therapy

Preclinical biotech company Eikonoklastes Therapeutics and gene therapy manufacturer Forge Biologics announced a partnership that will advance Eikonoklastes’ ET-101 gene therapy into clinical trials for patients with ALS.

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November 25, 2022
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VIDEO: Several options for gene therapy in treatment of geographic atrophy

VIDEO: Several options for gene therapy in treatment of geographic atrophy

NEW YORK — In this video, David Eichenbaum, MD, director of research at Retina Vitreous Associates of Florida, discussed the current pipeline of treatments for geographic atrophy at OSN New York Retina.

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November 22, 2022
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FDA approves Hemgenix as first gene therapy for treatment of hemophilia B

FDA approves Hemgenix as first gene therapy for treatment of hemophilia B

The FDA approved etranacogene dezaparvovec-drlb, a single-dose gene therapy for treatment of adults with hemophilia B.

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November 21, 2022
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Phase 2/3 study of gene therapy for young children with MPS IIIA yields mixed results

Phase 2/3 study of gene therapy for young children with MPS IIIA yields mixed results

Lysogene has announced topline results from its phase 2/3 study evaluating LYS-SAF302, an investigational gene therapy for the treatment of mucopolysaccharidosis type IIIA, or Sanfilippo syndrome.

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November 17, 2022
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VIDEO: Gene therapy shows growing promise in treatment of retinal diseases

VIDEO: Gene therapy shows growing promise in treatment of retinal diseases

NEW YORK — In this Healio Video Perspective from OSN New York Retina, Lejla Vajzovic, MD, FASRS, discusses the growing role of intravitreal and subretinal gene therapy in treating retinal diseases.

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October 25, 2022
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VIDEO: Gene therapy potential treatment target for Duchenne muscular dystrophy

VIDEO: Gene therapy potential treatment target for Duchenne muscular dystrophy

CHICAGO – Jeffrey Chamberlain, PhD, professor of neurology at the University of Washington, discussed types of gene therapy as a possible treatment for muscular dystrophy at ANA 2022.

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October 24, 2022
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VIDEO: Gene therapy shows positive safety, tolerability results for geographic atrophy

VIDEO: Gene therapy shows positive safety, tolerability results for geographic atrophy

CHICAGO — In this Healio Video Perspective from the AAO meeting, Michael Nathan Cohen, MD, discusses phase 1 data investigating JNJ-81201887 gene therapy from Janssen for the treatment of geographic atrophy.

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October 02, 2022
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VIDEO: Gene therapy for X-linked retinitis pigmentosa shows positive safety, efficacy data

VIDEO: Gene therapy for X-linked retinitis pigmentosa shows positive safety, efficacy data

CHICAGO — In this Healio Video Perspective from Retina Subspecialty Day at the AAO meeting, Michel Michaelides, MD, discusses study results from a gene therapy trial for RPGR-associated X-linked retinitis pigmentosa.

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October 01, 2022
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VIDEO: Opus Genetics CEO discusses IND application for retinal degeneration gene therapy

VIDEO: Opus Genetics CEO discusses IND application for retinal degeneration gene therapy

CHICAGO — In this Healio Video Perspective from Eyecelerator@AAO, Opus Genetics CEO Ben Yerxa discusses an upcoming investigational new drug application for OPGx-001.

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September 19, 2022
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FDA grants accelerated approval for gene therapy to treat cerebral adrenoleukodystrophy

FDA grants accelerated approval for gene therapy to treat cerebral adrenoleukodystrophy

The FDA granted accelerated approval to bluebird bio Inc for elivaldogene autotemcel, its gene therapy that aims to slow progression of neurologic dysfunction in boys aged 4 to 17 with early, active cerebral adrenoleukodystrophy, or CALD.

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