Top Executives from AveXis Join New Gene Therapy Venture — This Time Targeting Tens of Millions More Patients – PRNewswire

MADISON, Wis., March 29, 2021 /PRNewswire/ --A pair of top C-level Executives from blockbuster gene therapy company AveXis have joined ENDSULIN, a new venture that could reach millions more than any gene therapy to date. Spearheaded by one of the disease's most accomplished pioneers, the company aims to upend a multi-billion dollar treatment industry.

Both Brian Kaspar and Thomas Dee, the founding Chief Science and Chief Finance Officers, respectively, of AveXis (developer of Zolgensma, bought by Novartis for $8.7 Billionin May of 2018) have accepted board roles at ENDSULIN. While AveXis sought to "transform rare diseases," ENDSULIN is solely targeting one of the most prolific Type 1 diabetes (T1D) with what they hope will be a one-time treatment to free patients from daily insulin injections by more precisely regulating their glycemic control.

"The science itself is remarkable, but the potential to change millions of lives is unprecedented," Kaspar said. "We've proven gene therapy can revolutionize medicine with lasting, durable treatments. But now we need players like ENDSULIN with the right vision and focus who can harness that momentum to give patients the relief they deserve."

The former AveXis leaders round out a team of pioneering experts at the forefront of diabetes, gene therapy and drug development, strategically brought together to accelerate ENDSULIN's undivided focus: FDA authorization to begin human clinical trials.

Founder Hans Sollinger's previous discoveries have shaped modern pancreatic transplantation treatment for T1D. But because of the complexity of the disease, while the science of management has advanced, insulin shots have been the enduring standard of care since the 1920s almost a full century.

"25 years ago, I had performed more kidney-pancreas transplants than anyone in the world, but I'd only helped 500 people," Sollinger said. "The most important aspect of my search for a cure became its reach, in terms of both scale and accessibility. As I advanced, it became more and more obvious that gene therapy was the way to achieve it all, with no compromises."

Through decades of research at the UW Hospitals and Clinics, Sollinger's team developed a highly unique and targeted expression system that addresses one of the greatest challenges in treating T1D: precise regulation of insulin in response to rapidly fluctuating glucose levels. Several publications over the last 10 years have demonstratedlong-lasting euglycemia control in hundreds of mice and rats, indicating a durable cure may be within reach.

"We've focused everything we are doing around our mission to get this treatment to patients," said Eric Spyra, Interim CEO and Board Member. "Using existing commercial infrastructure and following the path of other liver-directed AAV gene therapies, we're putting all the pieces in place now that will accelerate this therapy through every stage."

ENDSULIN has already initiated a pilot study in autoimmune-modeled naturally diabetic companion dogs, while executing their IND-enabling plan and preclinical studies. The company is preparing for a Series A funding round this year.

ABOUT ENDSULINENDSULIN is reshaping the way we approach a cure for diabetes. They are working to free patients from daily injections and 24/7 management using the most cutting-edge gene therapy technology, developed from decades of research by noted diabetes leader Hans Sollinger, MD, PhD, Dr hc, at the University of Wisconsin Hospitals and Clinics. Their sole focus is to get a durable, one-time treatment to the millions of people who need it.

SOURCE ENDSULIN

http://www.endsulin.com

Read the original here:
Top Executives from AveXis Join New Gene Therapy Venture -- This Time Targeting Tens of Millions More Patients - PRNewswire