"Building the Road to a Cure"
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YBRP Funded Research

Funded Research Projects:

Natural History Study (NHS): 2018- present, YBRP has provided approximately 110K in funding towards the NHS. Dr. Bain and her team are actively recruiting HNRNPH2 patients for the crucial Natural History Study.  The greater the number of participants, the deeper the understanding of the disorder.  This study is fundamental to future research into potential treatments and/or a cure and will be key in developing a "road map" and plan of care for doctors, clinicians, parents, and care-givers to be able to give the proper care to a patient throughout the course of their development. 

Andlit Research:  2022-2023, YBRP has provided 100k in funding for ongoing research for a treatment of HNRNPH2.

Andlit Research: 2020-2022, YBRP provided 420K in funding for research for a treatment of HNRNPH2.

 

YBRP's First Funded Research Project

Yellow Brick Road Project Logo        Andlit Therapeutics Logo

 

The Yellow Brick Road Project is very excited to announce that we have signed an agreement with Andlit Therapeutics to fund research into HNRNPH2 mutations and potential treatments.

 Andlit Therapeutics is a biotech spinout from Medison Ventures, corporate Venture arm of Medison Pharma, one of the world's largest commercial partners of leading global biotech companies. Medison's mission is to provide patients with access to life saving and life altering therapeutics as early as possible post FDA or EMA approval. Andlit has been formed specifically to develop therapeutic treatment for HNRNPH2 mutations.

Dr Yael Gruenbaum-Cohen
Dr Yael Gruenbaum-Cohen
Dr Rotem KarniProfessor Rotem Karni

The project that will be funded by the Yellow Brick Road Project will last for two years and consist of two main parts:


1) researching the expression of the disorder at the cellular level including analysis of variants to learn if there is a gain or loss of function

2) exploring possible avenues for treatment, targeting therapies based on the results of the stage 1 analysis

Andlit's CEO, Dr Yael Gruenbaum-Cohen, is a Director at Medison Ventures, with over 10 years' experience in rare disease biotech development and research. Dr. Gruenbaum-Cohen also leads the pro-bono arm's activities in rare diseases. She has assembled an exceptional team of researchers and scientific advisors which will be overseen by Andlit's CTO, Professor Rotem Karni. Professor Karni works in the Faculty of Medicine at the Hebrew University of Jerusalem where his lab is involved in genetic research.

Please visit the Andlit website for more information.

 

Our Fundraising Goal - Last Updated February 2021

 

This cutting-edge research would not be possible without the support of our donors. We are so grateful for all the donations received so far and will be launching several new initiatives in the coming year to continue to fund this important work.

 

  • The total cost of the 2-year project is $420,000

     

  • Through the generosity of our donors, the Yellow Brick Road Project made the initial installment of the grant in the amount of $150,000.

     

  • We've since raised additional funds through the support of our generous donors and need your help to raise the remaining $110,000 to fully fund this project.

     

  • Our goal is to reach the total of $420,000 by July 1, 2021!

     

  • Please visit our donations page to find out how you can help.