Our Process to Get Results

Our team is committed to focused, directed research.

 

Step 1: Build a Great Team

We have built an amazing team by aligning with researchers at Weill Cornell Medicine, and Ionis Pharmaceuticals. This team is dedicated to medical and scientific research directly targeted toward developing treatments for Genetic ALS.

 

Step 2: Collaborative Directed Research

Our team has been instrumental in developing systems of collaboration between different academic and research institutions all around the country. These efforts have allowed us to access existing scientific data that has already been developed. For example, The team at Weil Cornell University had been able to coordinate directly with the preFALS study in Miami to obtain biological data and samples, allowing the team to jump start their research efforts.

In addition, Dr. Giovanni Manfredi and Dr. Hibiki Kawamata Fujita from Weill Cornell Medicine have already been collaborating with Ionis Pharmaceuticals toward testing and developing treatments for Genetic forms of ALS. So far, the team has made a series ASOs targeted toward specific ALS genes that they are now testing to see which ones knock down the gene most effectively. After that they will test the most successful ones for tolerability, and if all goes well we will be testing the top choices at Weill Cornell by this summer!

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Giovanni Manfredi , M.D., Ph.D.

Finbar and Marianne Kenny Professor in Clinical and Research Neurology

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Hibiki Kawamata Fujita , Ph.D.

Assistant Professor

Step 3: Apply Results

At NextGen ALS, we believe in results driven research. We understand that research without the goal of treatment, and maybe even a cure, is not an effective approach. This is why we believe in targeted, results driven research, with a goal of medical application. We believe that all forms of genetic ALS can be treated through targeted research, and hopefully, someday these results may even be applied to sporadic ALS and other neurodegenerative diseases!