Walter’s summary of the status of the HDACI program was that the FDA has requested that additional tests be conducted in animals to demonstrate that any lesions that resulted from the “worst case scenario – highest doses” in the male dogs used in the first tests, were redressed over time following cessation of the dosing.  He said that Repligen has these additional tests under way and plans to complete them over the "next few months." 
Walter went on to say that, if the results of the additional tests are not sufficient to resolve the FDA questions entirely, Repligen will make a decision about which of two alternative plans to pursue:
1)      Advance an HDACI trial in FA patients in Europe (face-to-face meetings already held with the European regulators indicated these regulators are receptive to a trial in patients rather than healthy volunteers, recognizing that the single ascending doses planned for patients are much lower than those that seemed to have caused FDA’s concerns with the male dogs), or
2)      Advance the follow-on HDACI for FDA approval for clinical trial. Walter said Repligen is identifying follow-on HDACI candidates that should  cross the blood-brain barrier more efficiently and be more stable so that the minimum effective dose could be achieved at much lower dosing levels, greatly reducing the toxicity profile.
The Repligen team later explained to us that these two options could even be combined.  So, if the FDA were not to lift the hold on a U.S. trial of the lead candidate even after seeing the new animal data, Repligen might even pursue a Phase I clinical trial of the lead candidate in Europe while pursuing FDA approval of a Phase I clinical trial of the follow-on candidate in the United States. 
Walter also said that numerous drug companies, some of them the large ones, have approached Repligen regarding partnerships on the HDACI program for FA.  He said there is a great deal of interest, now, including from the large pharmaceutical companies, in pursuing treatments for rare diseases and many of them see Repligen’s HDACI program for FA as very well positioned in this space.
So, the HDACI program continues to look very promising for FA.  We should hear more by very early in the New Year.
In hopes we all have a very Happy New Year,

Ronald J. Bartek
Friedreich's Ataxia Research Alliance (FARA)

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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