Current challenges to health are complex and diverse. They require a variety of strategies to create meaningful change. At Scriptr Global, we have multiple platforms with the potential to expand the reach of gene editing and gene therapy. Each platform represents an opportunity to significantly impact outcomes.
To efficiently leverage our diverse platforms for the greatest good, we incorporate a model of advancing programs internally through a pre-clinical or validation stage. We then transition successful programs into either Scriptr Global affiliate companies or into agreements with industry partners for commercial advancement.
Seamless, Scarless Dual AAV Approach for Large Gene Replacement
Stable Genome Modification with Broad Applicability
Targeted Nuclear RNA for Degradation
Targeted Nuclear RNA for Alternative Polyadenylation
We create the best health solutions for those we serve by levering our diverse scientific technologies to address underlying disease. The below pipeline demonstrates our ongoing commitments.
Over his thirty-year career in biopharmaceuticals, Keith has launched a variety of treatments predominately in rare diseases. His more recent positions have included CEO and Co-Founder of Speragen, a privately held biopharmaceutical corporation engaged in early-stage development of a treatment for SSADH Deficiency and a variety of leadership roles for Sanofi/Genzyme focusing on rare diseases.
Doug’s research focuses on the discovery and role of Long noncoding RNAs (lncRNAs) and micropeptides as novel regulators of muscle development and disease. Doug performed his postdoctoral training in the prestigious laboratory of Dr. Eric Olson at UT Southwestern Medical Center, where he made the discovery that many long noncoding RNAs encode small bioactive micropeptides. Doug published this work in outstanding high-impact papers in Cell, Nature, Science, PNAS and Science Signaling, and was awarded UT Southwestern’s Postdoc of the Year. Doug is currently Assistant Professor of Medicine in the Cardiovascular Research Institute and Center for RNA Biology at the University of Rochester Medical Center. Basic research discoveries in his lab have led to the development of the novel DNA and RNA-editing technologies which have broad therapeutic potential for treating many human monogenic diseases.
In addition to the founders, Scriptr Global engages with a team of industry and academic experts to accomplish the mission.