Hogan Lovells supports Riaan Research Initiative in securing FDA clearance of IND for experimental gene therapy to treat Cockayne syndrome

Press releases | 11 June 2026

New York - Global law firm Hogan Lovells provided pro bono legal counsel to the Riaan Research Initiative (RRI), a rare disease patient advocacy organization based in New York City, in securing U.S. Food and Drug Administration (FDA) clearance for an Investigational New Drug (IND) study to provide 6-year-old Riaan Singh Digeorge an experimental gene therapy for Cockayne syndrome, a devastating genetic disorder that causes premature aging and early death.

This groundbreaking milestone reflects the critical role of parent-led efforts in developing treatments for pediatric rare diseases when traditional pathways remain limited. It is the product of a nearly five-year effort by Riaan’s parents, Jo Kaur and Richard Digeorge—who founded RRI in June 2021, shortly after their son was diagnosed—and the dedication of the broader Cockayne syndrome community. Hogan Lovells began providing pro bono support to RRI shortly after the organization was founded.

Cockayne syndrome is a severe DNA repair and transcription disorder with no FDA-approved treatments. It causes significant brain atrophy, growth failure, vision and hearing loss, developmental disabilities, and early death. Children who are more severely affected, like Riaan, have a life expectancy of five to seven years. The disease is estimated to affect approximately one to three individuals per million people and is one of many ultra-rare conditions where urgent patient needs often remain unmet under conventional drug development models.

The novel gene therapy uses an adeno-associated viral vector, AAV9, to deliver a functional copy of the ERCC8/CSA transgene directly to the brain. Following promising preclinical results in mice, including an 8.5-fold increase in lifespan, the FDA cleared the IND application sponsored by UMass Chan Medical School.

In addition to supporting the IND application, Hogan Lovells advised RRI on the underlying nonclinical and manufacturing development, as well as contractual agreements related to product development and funding. The firm also provided strategic guidance on FDA regulatory strategy and communications with the FDA review team.

Blake Wilson, a Hogan Lovells partner, led the pro bono engagement and was supported by partners Mike Druckman, Sally Gu, and Barry Burgdorf; counsel Erkang Ai, Ph.D.; and associates Bryan Walsh, Ashley Grey, Lauren Massie, and Noah Fisher. Former Hogan Lovells partner Lowell Zeta, also provided strategic advice on this matter while employed at the firm.