Rare Daily Staff

Limelight Bio has raised $75 million in new financing from Apple Tree Partners to advance development of proprietary technologies, targets, and therapeutic candidates designed to overcome limitations of current gene therapy and gene editing approaches.

The biotech is working on eight gene therapy programs that focus on severe inherited retinal dystrophies, genetic mutations that cause hematologic and metabolic disorders, and neurologic and hearing loss diseases where no current therapies exist.  

“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 Michael Ehlers, CEO of Limelight, and former executive vice president for R&D at Biogen. “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.”

Founded in 2017, 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, of the University of Pennsylvania Perelman School of Medicine and co-director of the CAROT program, with which Limelight has an exclusive partnership, and Phil Johnson, former chief scientific officer of the Children’s Hospital of Philadelphia. Johnson’s work on vaccines, virology, and gene therapy has been foundational for advancing novel therapeutic approaches. Bennett’s 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 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.”

Photo: Michael Ehlers, CEO of Limelight

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