Posts Tagged ‘Gene Therapy’

Double Shot Gives Spark Therapeutics a Jolt

Double Shot Gives Spark Therapeutics a Jolt

Spark Therapeutics is riding a double shot of positive news about its gene therapy treatment for RPE65-mediated inherited retinal disorders that’s given its stock a boost, pushing it near an all-time high. The first shot came when The Lancet published Phase III clinical trial data of voretigene neparvovec, the investigational, potential one-time gene therapy candidate…

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Allergan Makes a Play in Gene Editing

Allergan Makes a Play in Gene Editing

With its licensing agreement to develop genome-editing programs with Editas Medicine to treat retinal diseases, Allergan has made a move to partner with one of the leading players in the CRISPR genome-editing space at a time when the field is poised to take off. That’s not Allergan’s hype. A recent report by San Francisco-based Grand…

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AGTC Pursues Gene Therapy on Multiple Tracks

AGTC is pursuing six different genetic treatments for four different retinal diseases, and expects to have clinical data from its Phase I trial of its lead candidate for treatment of X-linked retinoschisis later in the year, company chief business officer Stephen Potter reported at the “Paying for Breakthrough Eye Therapies” session at OIS@ASRS. “From our…

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Duker Sees “Energy” Being Added to Gene Therapy Field

Duker Sees “Energy” Being Added to Gene Therapy Field

Jay Duker, MD, Director of the New England Eye Center and Chairman of Ophthalmology at Tufts Medical Center and the Tufts University School of Medicine, tells OISTV the approval of gene therapy products including Spark Therapeutics’ SPK-RPE65 would help the sector clear a “real and psychological” hurdle facing gene therapy companies. Speaking With: Jay Duker,…

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Applied Genetic Technologies Corporation (AGTC)

Applied Genetic Technologies Corporation (AGTC)

CEO Mark Shearman, MD, PhD, describes Applied Genetic Technologies Corp., known as AGTC, as a clinical-stage biotechnology company focused on developing its proprietary gene vector technology for treatment of orphan monogenic diseases of the retina. AGTC signed an R&D partnership with Biogen to oversee biological development and commercialization of its platform, which Dr. Shearman described…

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Eleven Biotherapeutics

Eleven Biotherapeutics

Eleven Biotherapeutics is using its proprietary AMP-Rx protein-engineering platform to develop agents for more effective treatment of allergic conjunctivitis as well as to investigate new treatments for patients with diabetic macular edema (DME) who do not respond to anti-VEGF therapy. In October, the company had completed enrollment in its double-masked, randomized, vehicle-controlled Phase III pivotal…

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Spark Therapeutics

Spark Therapeutics

Spark was founded about 2 years ago with the vision of building a fully integrated gene therapy company with research and development, manufacturing, and commercial divisions. The company is the brainchild of several researchers at Children’s Hospital of Philadelphia; at mid-year, Spark had $223 million on hand. The goal of Spark’s development is “one-time, potentially…

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RetroSense Therapeutics

Retrosense - Companies to Watch - 2015

[c RetroSense Therapeutics CEO Sean Ainsworth explains how the company’s optogenetic gene therapeutic can impart light sensitivity to cells in the retina, re-sensitizing it to light. The company is pursuing treatments for Dry AMD and retinitis pigementosa. RetroSense Therapeutics is developing optogenetic gene therapeutic approaches to vision restoration. Optogenetics imparts light sensitivity to cells in…

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Biogen’s High Hopes For Gene Therapy, Ophthalmology

OIS Podcast

In July, Biogen signed a potential $1 billion-plus deal with Applied Genetic Technologies Corp. The deal, which included an equity investment, centered around AGTC’s leading gene therapy programs developing treatments for rare ophthalmic conditions X -Linked Retinoschisis (XLRS) and X-linked Retinitis Pigmentosa (XLRP). In this podcast, Josh Mandel-Brehm, Director, Business Development and M&A at Bio-gen,…

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Can Spark Phase III Results Ignite Gene Therapy?

OIS Podcast

Jeffrey D. Marrazzo, Co-Founder and CEO of Spark Therapeutics, discusses the successful Phase III trial of its lead candidate, SPK-RPE65, was able to improve functional vision in patients with a rare form of a genetic disorder known as RPE65-mediated inherited retinal dystrophies. SPK-RPE65 is intended to treat rare blinding conditions caused by mutations in the…

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Eleven Biotherapeutics

Eleven Biotherapeutics ASCRS 2015

Presenter: Abbie C. Celniker, PhD Dr. Celniker brings more than 20 years of proven protein therapeutic expertise to Eleven Biotherapeutics. Prior to joining Eleven, Abbie was CEO of Taligen Therapeutics, which was acquired by Alexion Pharmaceuticals. View Full Profile

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2014 OIS Innovator Award Presentation

2014 OIS Innovator Award Presentation

The OIS Innovator Award went to Sue Washer, Bill Hauswirth, and Nick Muzyczka for their pioneering role in gene therapy company AGTC. Washer and Hauswirth sit down with OIS Co-Chair Gil Kliman to share the tale of starting and leading the ophthalmology leader. Presenter: Gilbert H. Kliman, MD Dr. Gil Kliman leads the medical device…

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