Corporate Banner
Satellite Banner
Biologics & Bioprocessing
Scientific Community
 
Become a Member | Sign in
Home>News>This Article
  News
Return

Spark Therapeutics and Genable Technologies Announce Collaboration

Published: Wednesday, March 26, 2014
Last Updated: Wednesday, March 26, 2014
Bookmark and Share
Collaboration to advance a gene therapy treatment for a rare form of retinitis pigmentosa.

Spark Therapeutics and Genable Technologies have announced that they have entered into a collaboration agreement for Genable’s lead therapeutic to treat rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO adRP), GT038.

Under the terms of the collaboration, Genable will license certain adeno-associated virus (AAV) vector manufacturing patents from Spark.

The parties have entered into a broad agreement in which Spark will be the exclusive manufacturer of the product and provide development advice and expertise to Genable to help in the ongoing development of GT038.

Spark will receive milestone payments and royalties on future sales of GT038, as well as near-term revenue from the manufacture and supply of product.

“We are excited to apply our deep expertise in AAV clinical development and manufacturing to augment Genable’s great work, and expand the number of debilitating diseases of the eye that can be addressed through gene therapy,” said Jeffrey D. Marrazzo, Spark Therapeutics co-founder, president and CEO.

Dr. Jason Loveridge, CEO of Genable Technologies commented, “We have chosen Spark as our partner to advise, lend their experience and manufacture GT038 based on their broad expertise in gene therapy. We see them as a world-class organization, and we are excited to be advancing our novel therapy GT038 into the clinic.”

“The collaboration with Spark provides an exciting opportunity to greatly expedite development of Genable's novel therapy targeted towards RHO-adRP,” said Professor Jane Farrar, founder and director, Genable Technologies; professor, Trinity College (Dublin).

GT038 is a potential treatment for rhodopsin (RHO)-linked autosomal dominant retinitis pigmentosa (adRP), an inherited retinal dystrophy that leads to blindness in most cases. There is currently no approved pharmacologic treatment for adRP, which affects an estimated 30,000 patients worldwide.

GT-038 utilizes AAV vectors with an established safety and efficacy profile to deliver RNA interference (RNAi) molecules to suppress the expression of faulty and normal copies of RHO and restore normal gene expression. GT038 has been granted Orphan Drug Designation in both the U.S. and Europe.


Further Information

Join For Free

Access to this exclusive content is for Technology Networks Premium members only.

Join Technology Networks Premium for free access to:

  • Exclusive articles
  • Presentations from international conferences
  • Over 3,200+ scientific posters on ePosters
  • More than 4,600+ scientific videos on LabTube
  • 35 community eNewsletters


Sign In



Forgotten your details? Click Here
If you are not a member you can join here

*Please note: By logging into TechnologyNetworks.com you agree to accept the use of cookies. To find out more about the cookies we use and how to delete them, see our privacy policy.


Scientific News
ASMS 2016: Targeting Mass Spectrometry Tools for the Masses
The expanding application range of MS in life sciences, food, energy, and health sciences research was highlighted at this year's ASMS meeting in San Antonio, Texas.
Long-Term Culturing of Adult Stem Cells
A new procedure developed by Harvard Stem Cell Institute researchers (HSCI) at Massachusetts General Hospital (MGH) may revolutionize the culturing of adult stem cells.
Solutions for Biotherapeutic Characterization
Innovation to speed the routine.
Plant-Based Vaccine Among Front Runners In Search For New Polio Jab
A researcher from Norwich is part of a consortium that has been awarded $1.5 million to develop safer polio vaccines, using a new technique developed at the John Innes Centre.
Harnessing Helpful Microbes
Seeking to further harness microbes’ many uses, the federal government has launched the National Microbiome Initiative (NMI) to “foster the integrated study of microbiomes across different ecosystems.”
What Makes a Good Scientist?
It’s the journey, not just the destination that counts as a scientist when conducting research.
A New Approach to Chemical Synthesis
Communesins, originally found in fungus, could hold potential as cancer drugs.
Better Animal Model to Improve HIV Vaccine Development
Penn study identifies a new tool to produce better HIV vaccine designs.
First Large-Scale Proteogenomic Study of Breast Cancer
The study offers understanding of potential therapeutic targets.
Developing a More Precise Seasonal Flu Vaccine
During the 2014-15 flu season, the poor match between the virus used to make the world’s vaccine stocks and the circulating seasonal virus yielded a vaccine that was less than 20 percent effective.
SELECTBIO

SELECTBIO Market Reports
Go to LabTube
Go to eposters
 
Access to the latest scientific news
Exclusive articles
Upload and share your posters on ePosters
Latest presentations and webinars
View a library of 1,800+ scientific and medical posters
3,200+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
4,600+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FOR FREE!