This designation may also qualify Repligen to benefit from certain tax credits and a waiver of the company's obligation to pay the FDA application user fees for this product as required by the Prescription Drug User Fee Act. The U.S. Orphan Drug Act provides incentives for companies developing and marketing therapies for rare diseases, defined as those affecting fewer than 200,000 Americans. There are approximately 15,000 people worldwide with Friedreich's ataxia. “We are very pleased to receive Orphan Drug Designation for our Friedreich’s ataxia program,” stated Walter C. Herlihy, President and Chief Executive Officer of Repligen. “RG2833 is the first compound that targets activation of the defective gene responsible for Friedreich’s ataxia. If this approach is successful, it has the potential to significantly impact patients’ lives.” Earlier this month, we filed an Investigational New Drug Application with the FDA for a Phase 1 study of RG2833 to evaluate the pharmacokinetic and safety profile of RG2833 in up to 40 healthy volunteers. This study will also evaluate the pharmacodynamic response of various biomarkers in peripheral blood to RG2833. RG2833 is a new chemical entity, which is the subject of a composition of matter patent application, which if allowed, will remain in force until 2029 prior to any patent term extensions. RG2833 has been developed in collaboration with scientists from The Scripps Research Institute and a broad network of international scientific thought leaders. Repligen’s research efforts have been partially funded with grants from the Muscular Dystrophy Association, the Friedreich’s Ataxia Research Alliance, GoFAR and the National Ataxia Foundation. We are also evaluating HDAC-3 inhibitors in animal models of Huntington’s disease and cognition. Source

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!

https://www.amazon.com/Legacy-Marie-Schlau-collective-Friedreichs-ebook/dp/B01N28AFWZ

 

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:

https://www.irbbarcelona.org/en/news/international-patient-advocates-partner-to-fund-spanish-gene-therapy-project-to-treat

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:

https://www.irbbarcelona.org/en/news/new-research-front-to-tackle-friedreichs-ataxia
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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