Nav: Home

Katherine High talks gene therapy progress for hemophilia & inherited retinopathies

January 04, 2017

New Rochelle, NY, January 4, 2017--Gene therapy has shown some of its most promising early results in treating patients with hemophilia and inherited retinal disorders that cause vision loss and blindness, both important research and drug development targets during the career of Katherine High, MD, President and Chief Scientific Officer of Spark Therapeutics (Philadelphia, PA). James M. Wilson, MD, PhD, Editor of Human Gene Therapy Clinical Development, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers recently spoke with Dr. High and the absorbing interview is published in the Journal. The article is available free on the Human Gene Therapy Clinical Development website until February 4, 2017.

The article "Interview with Katherine A. High, MD" explores a range of topics, including Dr. High's long career in gene therapy and her decision to study hemophilia as a model for in vivo and clinical gene therapy. Dr. Wilson expands the conversation to focus on the work underway at Spark Therapeutics, which is focusing on not only the clinical development of gene therapy for hematologic disorders, but also for inherited retinal diseases and neurodegenerative diseases. Dr. High discusses the progress in each of these programs and comments on key scientific, regulatory, and commercial challenges.

"In this interview, Kathy provided fascinating insight into her career and the key decisions she made in advancing in vivo gene therapy across multiple fronts," says Human Gene Therapy Clinical Development Editor James M. Wilson, MD, PhD, Director of the Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA. "Her leadership in both the academic and business domains has been critical to the current success that the field is realizing."
-end-


Mary Ann Liebert, Inc./Genetic Engineering News

Related Gene Therapy Articles:

Mysterious gene transcripts after cancer therapy
Drugs that are used in cancer therapy to erase epigenetic alterations in cancer cells simultaneously promote the production of countless mysterious gene transcripts, scientists from the German Cancer Research Center now report in Nature Genetics.
Gene therapy could 'turn off' severe allergies
A single treatment giving life-long protection from severe allergies such as asthma could be made possible by immunology research at The University of Queensland.
Lipid nanoparticles for gene therapy
Twenty-five years have passed since the publication of the first work on solid lipid nanoparticles (SLNs) and nanostructured lipid carriers (NLCs) as a system for delivering drugs.
New gene therapy for pseudarthrosis trialed at Kazan University
A team headed by Professor Albert Rizvanov, director of the Gene and Cell Technologies Open Lab, created a gene therapy drug that encodes growth factors for the stimulation of blood vessel and bone formation.
WSU researcher develops safer gene therapy
A Washington State University researcher has developed a way to reduce the development of cancer cells that are an infrequent but dangerous byproduct of gene therapy.
New gene therapy prevents muscle wasting associated with cancer
A new gene therapy could be used to prevent the loss of muscle mass and physical strength associated with advanced cancer
On the path to controlled gene therapy
The ability to switch disease-causing genes on and off remains a dream for many physicians, research scientists and patients.
Gene therapy against brain cancer
A team from the International School for Advanced Studies (SISSA) in Trieste has obtained very promising results by applying gene therapy to glioblastoma.
First gene therapy successful against human aging
Elizabeth Parrish, CEO of Bioviva USA Inc. has become the first human being to be successfully rejuvenated by gene therapy, after her own company's experimental therapies reversed 20 years of normal telomere shortening.
Designing gene therapy
Scientists in the Barabas group at EMBL have increased the efficiency of a genome-engineering tool called Sleeping Beauty, which is showing promise in clinical trials for leukemia and lymphoma immunotherapies.

Related Gene Therapy Reading:

Best Science Podcasts 2019

We have hand picked the best science podcasts for 2019. Sit back and enjoy new science podcasts updated daily from your favorite science news services and scientists.
Now Playing: TED Radio Hour

Anthropomorphic
Do animals grieve? Do they have language or consciousness? For a long time, scientists resisted the urge to look for human qualities in animals. This hour, TED speakers explore how that is changing. Guests include biological anthropologist Barbara King, dolphin researcher Denise Herzing, primatologist Frans de Waal, and ecologist Carl Safina.
Now Playing: Science for the People

#532 A Class Conversation
This week we take a look at the sociology of class. What factors create and impact class? How do we try and study it? How does class play out differently in different countries like the US and the UK? How does it impact the political system? We talk with Daniel Laurison, Assistant Professor of Sociology at Swarthmore College and coauthor of the book "The Class Ceiling: Why it Pays to be Privileged", about class and its impacts on people and our systems.