Posts Tagged ‘Orphan Drugs’

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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AGTC Makes Moves with Cash in Hand

AGTC Makes Moves with Cash in Hand

As Applied Genetic Technologies Corporation (AGTC) heads into the summer with encouraging results for two of its lead candidates using the adeno-associated virus-based gene technology for treatment of X-linked retinoschisis (XLRS) and achromatopsia, the company finds itself well positioned to fund the programs along with five others it has in the works as its cash…

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Dempsey on Building the Shire Brand in Ophthalmology

Dempsey on Building the Shire Brand in Ophthalmology - OIS Podcast - Eye On Innovation

Robert Dempsey, vice president of ophthalmics at Shire, was charged with building a brand in ophthalmology with no new drugs. Two years later, Shire has – in the words of one physician – one of “the best kept secrets” in eye care. Dempsey explains how Shire came to build something from nothing. Podcast Guest Robert…

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Aura’s CEO Describes the Challenges of Ocular Melanoma

Aura’s CEO Describes the Challenges of Ocular Melanoma

Elisabet “Eli” de los Pinos founded Aura Biosciences in 2009 to chase down innovative treatments for cancer. AURA, which made its first presentation at OIS in 2015, is reporting promising progress with AU-O11, a drug delivery technology that can deliver cancer-killing drugs to the surface of the eye. Speaking With: Elisabet de los Pinos, PhD…

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Aura Biosciences Offers Hope for Ocular Melanoma

OIS Podcast

Elisabet “Eli” de los Pinos was enjoying her job at Eli Lilly’s Oncology Business Unit where she was part of the leadership team responsible for Alimta’s market launch in Europe, a new drug for the treatment of Lung Cancer. But she took the leap, leaving corporate comfort to build Aura Biosciences from the ground up…

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Aura Biosciences

Aura Biosciences

Patients with ocular melanoma have very few treatment options, and those that exist – removal of the eye or plaque radiation – leave them blind within two years. Aura Biosciences has received orphan drug designation from the Food and Drug Administration for its class of intravitreal therapies that target ocular melanoma, founder and CEO Elisabet…

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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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How Could the 21st Century Cures Act Aid Innovation?

How Could the 21st Century Cures Act Aid Innovation?

There’s more to the 21st Century Cures Act, which passed the full House of Representatives with overwhelming bipartisan support, than $9 billion in new funding for the National Institutes of Health (NIH) to pick up the pace of medical research. The legislation also includes a number of provisions that would speed up the Food and…

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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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