Corporate Banner
Satellite Banner
Scientific Communities
Become a Member | Sign in
Home>News>This Article

ReNeuron Receives European and US Orphan Drug Designation

Published: Friday, August 30, 2013
Last Updated: Friday, August 30, 2013
Bookmark and Share
Company has received Orphan Drug Designation for its ReN003 stem cell therapy candidate for retinitis pigmentosa.

ReNeuron Group plc has announced that its ReN003 retinal stem cell therapy candidate for retinitis pigmentosa has achieved a significant regulatory milestone.

The therapy has been granted Orphan Drug Designation in both Europe and the US by the European Commission and the Food and Drug Administration, respectively.

Orphan Drug Designation is typically granted to treatments that provide significant benefit to patients with rare diseases that are life-threatening or chronically debilitating.

Treatments with this designation benefit from significant commercial and regulatory advantages such as market exclusivity for 10 years from approval in the disease concerned, against other treatments offering no greater therapeutic advantage.

ReNeuron is using its proprietary human retinal progenitor cells as the basis of its ReN003 therapeutic candidate targeting retinitis pigmentosa, a group of hereditary diseases of the eye that lead to progressive loss of sight due to cells in the retina becoming damaged and eventually dying.

Retinitis pigmentosa affects approximately 4 in 10,000 people in the EU and US.

The late pre-clinical development of the ReN003 programme also benefits from a recently awarded £0.8 million Biomedical Catalyst grant from the UK Technology Strategy Board.

The Company is targeting an initial Phase I/II clinical trial with ReN003 in the UK and the US, in retinitis pigmentosa patients, with clinical trial applications expected to be filed in the middle part of 2014.

Michael Hunt, Chief Executive Officer of ReNeuron, said: “We are delighted to have received Orphan Drug Designation for our ReN003 stem cell therapy candidate for retinitis pigmentosa from both the European Commission in Europe and the FDA in the US. Orphan status diseases are an increasing area of therapeutic and commercial focus by the mainstream pharmaceutical industry due to the distinct regulatory and market exclusivity advantages that Orphan Drug Designation confers. This fact bodes well for the commercial potential of our ReN003 therapy.”

Further Information
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 2,800+ scientific posters on ePosters
  • More Than 4,000+ 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 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.

Related Content

AGM Trading Update
CTX stem cell therapy candidate currently in a UK Phase II clinical trial.
Friday, September 25, 2015
Preliminary Results for the Year Ended 31 March 2015
Appointment of Olav Hellebø as CEO.
Saturday, July 11, 2015
ReNeuron Announces Research Collaboration with Benitec Biopharma
Collaboration using exosomes as delivery system for gene silencing in cancer therapy.
Tuesday, June 23, 2015
FDA Grants Fast Track Designation to ReNeuron’s RP Cell Therapy Candidate
Approval to commence a Phase I/II clinical trial in the US with stem cell therapy candidate for RP.
Saturday, May 23, 2015
Long Term Clinical Data from PISCES Stroke Trial
Long term data continue to show good safety profile and evidence of sustained improvements in neurological status and limb function.
Saturday, April 18, 2015
ReNeuron Files Application to Commence Phase I/II Clinical Trial in the US
IND application to commence clinical trial for retinitis pigmentosa with hRPC therapy candidate.
Tuesday, April 14, 2015
ReNeuron Reconfigures Board
Company further strengthens executive management for next phase of development.
Wednesday, April 08, 2015
ReNeuron Announces Appointment of Chief Executive Officer
Company appoints Olav Hellebø as new CEO.
Tuesday, September 09, 2014
ReNeuron AGM Trading Update
Results from the Phase I study with ReN009 announced in the first half of 2015.
Tuesday, September 09, 2014
Further Data from First Clinical Trial of ReNeuron’s Stem Cell Therapy to be Presented at Stroke Conference
Long term data continue to show good safety profile and evidence of sustained reductions in neurological impairment and spasticity.
Friday, May 16, 2014
ReNeuron Receives UK Regulatory Approvals
Company receive approvals to commence Phase II clinical trial in stroke and Phase I clinical trial in critical limb ischaemia.
Friday, March 28, 2014
ReNeuron to Receive Support from Foundation Fighting Blindness
Company receive support for its ReN003 retinal stem cell therapy candidate for treatment of retinitis pigmentosa.
Wednesday, March 12, 2014
ReNeuron Announces £33 Million Financing Package
Placing to raise up to £25.35 million, grant package of £7.8 million from Welsh Government and Notice of General Meeting.
Monday, July 22, 2013
ReNeuron Signs License Agreement with Leading US Eye Research Institute to Develop Stem Cell Therapies for Diseases of the Retina
ReNeuron has been collaborating with Schepens in the early development of its human retinal precursor cells (hRPCs).
Monday, June 20, 2011
ReNeuron Scientists Generate Pancreatic Cell Lines for Treatment of Diabetes
ReNeuron has generated data concerning its pancreatic cell lines for the treatment of Type 1 diabetes with its ReN002 diabetes programme.
Tuesday, June 26, 2007
Scientific News
High Throughput Mass Spectrometry-Based Screening Assay Trends
Dr John Comley provides an insight into HT MS-based screening with a focus on future user requirements and preferences.
How a Genetic Locus Protects Adult Blood-Forming Stem Cells
Mammalian imprinted Gtl2 protects adult hematopoietic stem cells by restricting metabolic activity in the cells' mitochondria.
Genetic Basis of Fatal Flu Side Effect Discovered
A group of people with fatal H1N1 flu died after their viral infections triggered a deadly hyperinflammatory disorder in susceptible individuals with gene mutations linked to the overactive immune response, according to a recent study.
New Tech Vastly Improves CRISPR/Cas9 Accuracy
A new CRISPR/Cas9 technology developed by scientists at UMass Medical School is precise enough to surgically edit DNA at nearly any genomic location, while avoiding potentially harmful off-target changes typically seen in standard CRISPR gene editing techniques.
The MaxSignal Colistin ELISA Test Kit from Bioo Scientific
Kit can help prevent the antibiotic apocalypse by keeping last resort drugs out of the food supply.
"Good" Mozzie Virus Might Hold Key to Fighting Human Disease
Australian scientists have discovered a new virus carried by one of the country’s most common pest mosquitoes.
Non-Disease Proteins Kill Brain Cells
Scientists at the forefront of cutting-edge research into neurodegenerative diseases such as Alzheimer’s and Parkinson’s have shown that the mere presence of protein aggregates may be as important as their form and identity in inducing cell death in brain tissue.
Closing the Loop on an HIV Escape Mechanism
Research team finds that protein motions regulate virus infectivity.
New Class of RNA Tumor Suppressors Identified
Two short, “housekeeping” RNA molecules block cancer growth by binding to an important cancer-associated protein called KRAS. More than a quarter of all human cancers are missing these RNAs.
Potential Treatment for Life-Threatening Viral Infections Revealed
The findings point to new therapies for Dengue, West Nile and Ebola.
Scroll Up
Scroll Down
Skyscraper Banner

Skyscraper Banner
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
2,800+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
4,000+ scientific videos