Repligen HDAC inhibitors progress

5/7/13 - REPLIGEN CORP - 10-Q - MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS . http://www.pharmacychoice.com

Our third clinical development program was targeted at Friedrich's Ataxia and led by RG2833, a class I histone deacetylase ("HDAC") inhibitor. RG2833 has received Orphan Drug designation from the FDA and European Commission. We initiated a single, ascending dose Phase 1 study of RG2833 in Friedreich's Ataxia patients in Italy in the fourth quarter of 2012 and completed the patient dosing in the quarter ended March 31, 2013. We believe the results of this trial, which we are currently analyzing, may be an important consideration for any third-party that may wish to pursue a development or commercialization agreement with us for RG2833. We expect that any additional development activities that we may pursue in the future will be largely supported by sponsors or collaborators.

Fatigue is frequent and multifactorial in Friedreich's ataxia

Fatigue is frequent and multifactorial in Friedreich's ataxia. Cynthia B. da Silva, Camila F. Chevis, Anelyssa D'Abreu, Iscia Lopes-Cendes, Marcondes C. França Jr.; Parkinsonism & Related Disorders, Available online 7 May 2013. http://dx.doi.org/10.1016/j.parkreldis.2013.04.007

Keywords: Friedreich's ataxia, Depression, Fatigue, Sleep disorders.

Non-Viral Gene Therapy Vectors Carrying Genomic Constructs

Non-Viral Gene Therapy Vectors Carrying Genomic Constructs

George Kotzamanis, Hassan Abdulrazzak, Athanassios Kotsinas and Vassilis G. Gorgoulis (2011). Non-Viral Gene Therapy Vectors Carrying Genomic Constructs, Non-Viral Gene Therapy, Prof. Xubo Yuan (Ed.), ISBN: 978-953-307-538-9, InTech, DOI: 10.5772/19225. Available from: http://www.intechopen.com/books/non-viral-gene-therapy/non-viral-gene-therapy-vectors-carrying-genomic-constructs

Good book to understand a little more the basics of this promising technique

This book focuses on recent advancement of gene delivery systems research. With the multidisciplinary contribution in gene delivery, the book covers several aspects in the gene therapy development: various gene delivery systems, methods to enhance delivery, materials with modification and multifunction for the tumor or tissue targeting. This book will help molecular biologists gain a basic knowledge of gene delivery vehicles, while drug delivery scientist will better understand DNA, molecular biology, and DNA manipulation

Entrevista a Javier Díaz-Nido

Radio interview to Javier Diaz-Nido, Spanish Researcher on Friedreich's Ataxia (Spanish)

Manuel Seara ha hablado sobre ataxia con Javier Díaz-Nido en el programa A hombros de gigantes de RNE, profesor titular de Biología Molecular de la Universidad Autónoma de Madrid y experto en la ataxia de Friedreich.

http://www.rtve.es/alacarta/audios/a-hombros-de-gigantes/hombros-gigantes-entrevista-javier-diaz-nido/1803448/

Bioenergetics Reprogrammed in a Cellular Model of Friedreich's Ataxia: Frataxin Deficiency Affects Formation of Respiratory Supercomplexes and Down-regulates Both OXPHOS and Glycolysis.

Bioenergetics Reprogrammed in a Cellular Model of Friedreich's Ataxia: Frataxin Deficiency Affects Formation of Respiratory Supercomplexes and Down-regulates Both OXPHOS and Glycolysis. Van Houten B, Wang T; Journal of Biomolecular Techniques : JBT [2013, 24(Suppl):S54-S55]

Keywords: Friedreich's ataxia (FRDA), glycolysis, OXPHOS, glucose metabolism, fatty acid oxidation, bioenergetics reprogramming.

Radial diffusivity in the cerebellar peduncles correlates with clinical severity in Friedreich ataxia

Radial diffusivity in the cerebellar peduncles correlates with clinical severity in Friedreich ataxia. Christian Clemm von Hohenberg, Michael F. Schocke, Marlene C. Wigand, Wolfgang Nachbauer, Charles RG Guttmann, Marek Kubicki, Martha E. Shenton, Sylvia Boesch, Karl Egger.
Neurological Sciences, May 2013. DOI 10.1007/s10072-013-1402-0

Keywords: Friedreich ataxia, Magnetic resonance imaging, Diffusion-tensor imaging, Tract-based spatial statistics, Cerebellar peduncle, Biomarker.

Heme Levels Are Increased in Human Failing Hearts

Heme Levels Are Increased in Human Failing Hearts. Arineh Khechaduri, MS; Marina Bayeva, PhD; Hsiang-Chun Chang, BA; Hossein Ardehali, MD, PhD.;
J Am Coll Cardiol. 2013;61(18):1884-1893. doi:10.1016/j.jacc.2013.02.012

Similarly, aggregation of iron inside the mitochondria has been observed in the hearts of Friedreich’s ataxia patients, who develop progressive and lethal cardiac dysfunction. Thus, maintenance of iron balance inside the heart appears to be critical for its function, but it remains unknown how iron regulation is altered in failing human hearts.

Triple Therapy with Darbepoetin Alfa, Idebenone, and Riboflavin in Friedreich’s Ataxia: an Open-Label Trial

Triple Therapy with Darbepoetin Alfa, Idebenone, and Riboflavin in Friedreich’s Ataxia: an Open-Label Trial. Javier Arpa, Irene Sanz-Gallego, Francisco J. Rodríguez-de-Rivera, Francisco J. Domínguez-Melcón, Daniel Prefasi, Javier Oliva-Navarro, Mar Moreno-Yangüela, Samuel I. Pascual-Pascual. The Cerebellum April 2013 DOI 10.1007/s12311-013-0482-y



Long-term statistically nonsignificant improvement of LVMI and stability of the echocardiographic parameters could be considered. Triple therapy may slow disease progression of FRDA.

Progress in gene therapy for neurological disorders

Progress in gene therapy for neurological disorders. Michele Simonato, Jean Bennett, Nicholas M. Boulis, Maria G. Castro, David J. Fink, William F. Goins, Steven J. Gray, Pedro R. Lowenstein, Luk H. Vandenberghe, Thomas J. Wilson, John H. Wolfe & Joseph C. Glorioso; Nature Reviews Neurology , | doi:10.1038/nrneurol.2013.56

Standard medical and surgical practice has not proved effective in curing or treating these diseases, and appropriate pharmaceuticals do not exist or are insufficient to slow disease progression. Gene therapy is emerging as a powerful approach with potential to treat and even cure some of the most common diseases of the nervous system.

Erythropoietin receptor (EpoR) agonism to treat a wide range of diseases.

Erythropoietin receptor (EpoR) agonism to treat a wide range of diseases. Sanchis-Gomar F, Perez-Quilis C, Lippi G.; Mol Med. 2013 Apr 11. doi: 10.2119/molmed.2013.00025.

Keywords: Erythropoietin receptor (EpoR), pleitropic actions, heart and cardiovascular diseases, neurodegenerative disorders (Parkinson and Alzheimer), spinal cord injury, stroke, diabetic retinopathy, rare diseases (Friedreich ataxia) side effects, non-hematopoietic EpoR agonists drugs (asialoEpo, Cepo and ARA 290)