Sep 4 2008, 11:33 AM EST

Repligen Wins $1.125M in Grants to Enhance Preclinical Program in Friedreich's Ataxia

GEN News Highlights

Repligen received $1.125 million in research grants to support the development of new treatments for Friedreich's ataxia. Funding will support preclinical activities until a compound is selected for clinical trials. Repligen will also use the money to develop biomarkers related to these drug candidates, which will be used to augment clinical development.

Repligen obtained $1 million from the Muscular Dystrophy Association (MDA) and $125,000 from the Friedreich's Ataxia Research Foundation (FARA) and the National Ataxia Foundation (NAF).

The MDA grant will partially support the characterization of the lead compounds, which have already been identified. This work includes the evaluation of compounds in preclinical models of disease and the completion of the pharmacology and toxicology.

The FARA and NAF grant will support the development of new methods to quantitate the impact of drug treatment on frataxin gene expression and frataxin protein in preclinical models and in patients.




Thursday, September 4, 2008


Repligen wins $1.12M in research grants

By Mass High Tech Staff


Biopharmaceutical company Repligen Corp.

 reports it has taken in $1.125 million in research grants to develop tools to help create new treatments for the rare neurodegenerative disease Friedreich’s ataxia.

Muscular Dystrophy Association awarded Waltham-based Repligen (Nasdaq: RGEN) $1 million and the Friedreich’s Ataxia Research Foundation and the National Ataxia Foundation combined to give $125,000, according to Repligen officials.

Repligen will use the funds to develop and prepare drug candidates for clinical trials and also develop biomarkers to monitor the effect of the drugs.

Friedreich’s ataxia is a congenital neurodegenerative disease caused by a single gene defect that leads to the body not producing enough of the protein frataxin, leading to progressive damage to the nervous system and loss of muscle function. Repligen launched a development program for Friedreich’s ataxia in April 2007, according to officials.

In April 2008, Repligen settled a patent-infringement lawsuit with drug company
Bristol-Myers Squibb Co. related to Bristol-Myers’ Orencia treatment.

The settlement called for the New York pharmaceutical firm to pay Repligen $5 million initially and tiered royalties on the first $2 billion in sales of Orencia from Jan. 1, 2008 through 2013, that could be worth a potential $117 million, according to Repligen. Under the terms of the deal, Repligen and the
University of Michigan, a co-plaintiff in the suit, granted Bristol-Myers an exclusive license to certain patents related to Orencia and dropped the 2-year old lawsuit.

Repligen reported a $37 million profit for 2008, on revenue of $19 million.

The legacy of Marie Schlau: literature to help cure Friedreich's Ataxia

If you feel like reading an unputdownable novel while collaborating with a just and solidary cause, "The Legacy of Marie Schlau" is your book! 100% of all funds raised will be dedicated to medical research to find a cure for Friedreich's Ataxia, a neurodegenerative disease that affects mostly young people, shortening their life expectancy and confining them to a wheelchair.

The life of Marie Schlau, a German Jewish girl born in 1833 hides great unsolved mysteries: accidents, disappearances, enigmas, unknown diagnoses, disturbing murders, love, tenderness, greed, lies, death ... alternatively a different story unfolds every time and takes us closer to the present. Thus, there are two parallel stories unravelling, each in a different age and place, which surprisingly converge in a revelatory chapter.

Paperback and Kindle versions for "The legacy of Marie Schlau" available for sale at Amazon now!


Research projects currently being financed by BabelFAmily

Currently, BabelFAmily is financing two promising research projects aimed at finding a cure for Friedreich's Ataxia. Whenever you make a donation to us or purchase a copy of "The legacy of Marie Schlau", this is where all funds raised will be devoted to:

1) Gene Therapy for Friedreich's Ataxia research project:

The project is the result of an initiative of Spanish people affected by this rare disease who are grouped in GENEFA in collaboration with the Spanish Federation of Ataxias and the BabelFAmily. The Friedreich’s Ataxia Research Alliance (FARA), one of the main patients’ associations in the United States now joins the endeavour.

2) Frataxin delivery research project:
The associations of patients and families Babel Family and the Asociación Granadina de la Ataxia de Friedreich (ASOGAF) channel 80,000 euros of their donations (50% from each organisation) into a new 18-month project at the Institute for Research in Biomedicine (IRB Barcelona). The project specifically aims to complete a step necessary in order to move towards a future frataxin replacement therapy for the brain, where the reduction of this protein causes the most damage in patients with Friedreich’s Ataxia.

The study is headed by Ernest Giralt, head of the Peptides and Proteins Lab, who has many years of experience and is a recognised expert in peptide chemistry and new systems of through which to delivery drugs to the brain, such as peptide shuttles—molecules that have the capacity to carry the drug across the barrier that surrounds and protects the brain. Since the lab started its relation with these patients’ associations in 2013*, it has been developing another two projects into Friedrich’s Ataxia.



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