Pharmaceutical Business review

Limelight Bio emerges with $75m to develop next generation gene therapies to overcome limitations of current treatments

Image: Limelight will develop next generation gene therapies to overcome limitations of current treatments. Photo: courtesy of Frauke Feind from Pixabay.

Founded in 2017, Limelight Bio has developed proprietary technologies, advanced targets and leading drug candidates designed to overcome key limitations of current gene therapy and gene editing approaches. Initial areas of focus include severe retinal, hematologic/metabolic, neurologic and hearing loss diseases where no current therapies exist. Limelight Bio is led by CEO Michael Ehlers, MD, PhD, with operations in Philadelphia and Cambridge.

“Gene therapy has made significant advances in recent years, and particularly gene replacement delivered by adeno-associated virus vectors or AAV. Yet, most genetic diseases are not addressed by this approach,” said Dr. Ehlers. “The technology platforms in development at Limelight Bio seek to correct disease at its genetic origin in a way that is broad, versatile, scalable and patient-centered. We are eager to accelerate our science to multiple drug candidates for patients for whom current approaches have fallen short.”

Limelight Bio is building an industry-leading platform to develop technologies, advanced targets, and leading drug candidates designed to treat more diseases for many more patients. The company is pioneering gene correction across unprecedented areas, including proprietary methods that persistently correct large genetic defects independent of gene size or mutation pattern without risk of permanent genomic alteration. Founders include Jean Bennett, MD, PhD of the University of Pennsylvania Perelman School of Medicine and co-director of the CAROT program, and Phil Johnson, MD, former Chief Scientific Officer of the Children’s Hospital of Philadelphia. Dr. Johnson’s work on vaccines, virology, and gene therapy has been foundational for advancing novel therapeutic approaches. Dr. Bennett’s ground-breaking research led to the development of an FDA-approved and marketed gene therapy product and she is a scientific advisor for Limelight Bio.

“There is great potential for Limelight’s technology to transform gene therapy far beyond where it is today,” said Dr. Bennett. “With these advanced tools and technologies, we now have the means to go straight to the origin of the most complex genetic diseases. It could become the foundation for a new class of medicines.”
“We believe Limelight Bio has the potential to leapfrog the prevailing gene therapy and editing approaches to tackle a much broader array of genetic disorders,” said ATP Managing Partner Seth Harrison, MD.

“With Mike Ehlers joining as CEO, we have added fantastic R&D leadership to what looks to us to be break-out technology to create the first gene therapy-based pharmaceutical company.”

Source: Company Press Release