Navigation Links
Gene Therapy Developed for ALS Treatment: New Biotech Company Neuralgene Enters Evaluation Phase
Date:4/12/2013

BOGOTA, Colombia, April 12, 2013 /PRNewswire/ -- Startup biotechnology company Neuralgene (http://neuralgene.com) has announced that it will begin animal studies in May to evaluate the efficacy of PRCN-829, its new gene therapy agent for the treatment of amyotrophic lateral sclerosis (ALS). PRCN-829 is the first gene therapy for sporadic ALS.

(Photo: http://photos.prnewswire.com/prnh/20130412/PH93428-a )

(Photo: http://photos.prnewswire.com/prnh/20130412/PH93428-b )

Neuralgene's neurotropic AAV-based gene therapy platform for the treatment of neurodegenerative diseases is based on the stem cell work performed by Jason Williams , M.D., founder and CEO of Neuralgene. "This technology addresses several key aspects of the underlying pathology of ALS," said Leonardo Gonzalez , M.D., clinical researcher for Neuralgene. "In his stem cell work, Dr. Williams had identified that production of Factor H by fat-derived mesenchymal stem cells may be a key mode of action."

The gene therapy is based on Dr. Williams' discovery that certain proteins produced by stem cells inhibit the attack of ALS. During the development of the gene therapy, he added new targets: neural growth factors and a protein implicated in ALS named TDP-43. "When Dr. Williams demonstrated the concept behind stem cells and how to address the treatment of ALS using gene therapy, we immediately knew that this was a revolutionary new concept," said Dr. Gonzalez.

The PRCN-829 gene therapy is designed to not only target gene delivery to the brain and spinal cord, but also to genetically engineer stem cells. The AAV9 viral vector delivers multiple genes, which include Factor H (a regulator of complement activity), neural growth factors and regulators of TDP-43, to the neural cells. Initial animal studies have demonstrated the safety of the gene therapy platform.

"The problem with stem cell therapy for ALS is that the results are generally partial and temporary," stated Dr. Williams. "This is because the stem cells produce the growth factors and other proteins for a short period, but then cease. Several stem cell studies have confirmed this. Now with gene therapy, we can increase those factors by a millionfold or greater so that recuperation lasts for many years or maybe is even lifelong."

"ALS is a complex disease with many different underlying causes," continued Dr. Williams. "Our gene therapy will target several of the main underlying mechanisms related to ALS with the hopes of getting a good response in a larger group of patients. However, our platform is versatile, allowing us to change and add different target genes. We expect that soon we will be able to perform a detailed genetic analysis of the patient, identifying their exact underlying cause of ALS. Then we will be able to tailor the therapy to each individual patient."

The company has partnered with several labs for the development of its patent-pending neurotropic AAV-based gene therapy for the treatment of neurodegenerative diseases such as ALS. In fact, Neuralgene partnered with Dr. Williams' imaging and image guided treatment facility, Precision StemCell in Bogota, to begin studies using image-guided administration of gene therapy to the spinal cord.

"This is a completely new therapy for ALS, and the groundwork for this technology will lead to the treatment of many other diseases," said Dr. Williams. Neuralgene has several other AAV gene therapies in its research and development pipeline for the treatment of neurodegenerative diseases such as Parkinson's and Multiple Sclerosis (MS). After initial testing of PRCN-829 in Colombia, Neuralgene plans to seek approval from the FDA for trials in the United States.

For more information, contact Dr. Leonardo Gonzalez at email.

About Neuralgene

Neuralgene is a startup biotech company focused on AAV gene therapy, which produces multiple proteins for the treatment of neurodegenerative disorders such as ALS and Parkinson's. PRCN-829 is a patent-pending gene therapy designed to transmit genes of interest into neural and glial cells. The technology is based on discoveries made by Dr. Jason Williams while using autologous adipose derived stem cells in the treatment of ALS. Information constitutes "forward looking statements."

Contact:

Dr. Leonardo Gonzalez
Colombia: 57 (311) 231-3684
Neuralgene
Email

This press release was issued through eReleases® Press Release Distribution. For more information, visit http://www.ereleases.com

 


'/>"/>
SOURCE Neuralgene
Copyright©2012 PR Newswire.
All rights reserved

Related biology technology :

1. Gene Therapy - Technologies, Markets and Companies
2. Studies Assess Hydroxyurea Therapy and Pre-Operative Transfusions for Patients with Sickle Cell Disease
3. TrovaGene, Inc. Obtains Exclusive License to Novel Mutations Associated with Prognosis and Chemotherapy Response in Leukemia
4. Amsterdam Molecular Therapeutics Receives US Orphan Designation for Hemophilia B Gene Therapy
5. Squamous Cell Cancer Responds to SPDT Therapy
6. Stem cell therapy reverses diabetes
7. Islet Sciences, Inc. Selects NeoStems Manufacturing Subsidiary Progenitor Cell Therapy for Product Manufacturing
8. UH Case Medical Center Offers New Therapy for Gynecologic Cancer Patients
9. Senior Management of NeoStem and Its Subsidiaries Invited to Speak at Seventh International Conference on Cell Therapy for Cardiovascular Disease
10. Aethlon Medical Establishes Extracorporeal Therapy Scientific Advisory Board
11. BioRestorative Therapies to Present at 7th International Conference on Cell Therapy for Cardiovascular Disease
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:12/8/2016)... , ... December 08, 2016 , ... ... FrontPanel SDK that provide essential device-to-computer interconnect using USB or PCI Express, announced ... FrontPanel support. The FOMD-ACV-A4 is a small, thin, SODIMM-style module that fits a ...
(Date:12/8/2016)... , ... December 08, 2016 ... ... the commercial launch of flexible packaging for their exceptionally efficient human mesenchymal ... system extends RoosterBio’s portfolio of bioprocess media products engineered to radically streamline ...
(Date:12/8/2016)... Fla. , Dec. 8, 2016  HedgePath ... company that discovers, develops and plans to commercialize ... its shares of common stock were approved for ... will begin trading on the OTCQX, effective today, ... qualify for the OTCQX market, companies must meet ...
(Date:12/7/2016)... , ... December 07, 2016 , ... ... its phase I/II dose escalation and expansion clinical trial for its lead drug ... Austria. The purpose of the trial was to determine the safety, antitumor activity, ...
Breaking Biology Technology:
(Date:11/29/2016)... Nearly one billion matches per second with DERMALOG,s high-speed AFIS    ... ... DERMALOG is Germany's largest Multi-Biometric supplier: The company's Fingerprint ... ... Multi-Biometric supplier: The company's Fingerprint Identification System is part of an efficient ...
(Date:11/21/2016)... VILNIUS, Lithuania , Nov. 21, 2016 /PRNewswire/ ... identification and object recognition technologies, today announced that ... for smart cards was submitted for the ... and successfully passed all the mandatory steps of ... III evaluation is a continuing test of fingerprint ...
(Date:11/15/2016)... , Nov. 15, 2016  Synthetic Biologics, Inc. ... therapeutics focused on the gut microbiome, today announced ... 25,000,000 shares of its common stock and warrants ... at a price to the public of $1.00 ... Synthetic Biologics from the offering, excluding the proceeds, ...
Breaking Biology News(10 mins):