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Investor Outlook: Focus on Upcoming LCA2 Gene Therapy Phase III Results.

Human gene therapy. Clinical development - 1 Sept 2015

Schimmer Joshua, Breazzano Steven

Abstract excerpt

Investor interest in gene therapy has increased substantially over the past few years, and the next major catalyst for the field is likely to be Spark Therapeutics's phase III trial for the treatment of visual impairment caused by RPE65 gene mutations (often referred to as Leber congenital amaurosis type 2, or LCA2, but may include other retinal disorders). Analysis of the approach from the basic genetics,...

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Source

Identifiers

PMID: 26390089
DOI: 10.1089/humc.2015.29001.sch

Genes

RPE65

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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