[New York, New York, February 25, 2025] – In the lead up to Rare Disease Day, Jack’s Tomorrow is delighted to announce the addition of Andrew Steinsapir, MBA, to its Scientific Advisory Board.

For 17 years, Rare Disease Day has raised awareness every February for the approximately 300 million people worldwide living with rare diseases. Jack’s Tomorrow remains steadfast in its mission to fund rigorous research to unlock mysteries about PURA Syndrome, a rare neurodevelopmental disorder caused by mutations in the PURA gene. The condition leads to global developmental delays, hypotonia, epilepsy, and other health challenges. To date, 750 individuals have been diagnosed, although it is likely that number will grow with increased access to genetic testing.   

By expanding its Scientific Advisory Board to include Steinsapir, a top gene therapy expert, Jack’s Tomorrow increases visibility about rare diseases and accelerates research to find treatments, and ultimately a cure, for PURA Syndrome. Steinsapir currently serves as Director, Gene Therapy Program Lead at Deerfield Discovery and Development, where he has played a pivotal role since joining in 2021. Steinsapir is also Acting Chief Technology Officer for Apertura Gene Therapy, a Deerfield portfolio company. Prior to his tenure at Deerfield, Steinsapir spent more than three years as a Consultant at Dark Horse Consulting Group, offering strategic and tactical expertise to more than 30 cell and gene therapy firms, pharmaceutical companies, and investment firms.

Earlier in his career, Steinsapir held research and process development roles at 4D Molecular Therapeutics. Mr. Steinsapir has a B.S. in Chemical Engineering with an emphasis on Biotechnology and an MBA, both from the University of California, Berkeley.

“We are thrilled to welcome Andrew to our Scientific Advisory Board,” said Kyle Czepiel, Co-Founder, Jack's Tomorrow. “His expertise in gene therapy and biotechnology innovation will be instrumental to guide our research strategy and speed our search for transformative treatments for children with rare genetic disorders like PURA Syndrome.”

Jack’s Tomorrow’s well-rounded research portfolio funds cutting-edge studies aimed at understanding the genetic and molecular mechanisms of PURA Syndrome and finding targeted therapies:

The Jackson Laboratory 

• PURA mouse model and induced pluripotent stem cell (iPSC) lines to test PURA Syndrome therapeutics

• Gene overexpression studies in preparation for potential PURA Syndrome gene therapies

   

Yale School of Medicine 

• Determining molecular control mechanisms of PURA activity in human and mouse cells

• Study of existing mouse models of PURA Syndrome

• Testing currently approved drugs that might be repurposed to treat PURA Syndrome

The Hospital for Sick Children

• Model organism research with zebrafish and worms toward large-scale screening of potential drugs for PURA Syndrome