In Progeria mouse models, lonafarnib extended lifespan by 25%; the addition of progerinin extended it to 50%. The Phase 2A trial enrolled 10 children who each made four week-long trips to Boston Children's Hospital (spaced four months apart). Eight children received progerinin combined with lonafarnib; two remained on lonafarnib alone as a comparison group. The results of the trial are currently under evaluation. Thereafter, a larger, Phase 2B trial to evaluate efficacy will be considered.

View study details: Randomized, Open-Label Study to Determine the Optimal Dose and Evaluate the Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients with HGPS.

PRF entered into manufacturing partnerships with Forge Biologics and PureSyn to produce SamPro-2, an investigational gene-editing therapy designed to correct the gene mutation that causes Progeria. This is a defining step toward Progeria gene therapy clinical trials.

For more information about SamPro-2, click here.

Darrien is responsible for managing all non-medical logistics for clinical trials, including travel and lodging, while also serving as the primary point of contact for Progeria families. She plays a key role in facilitating communication between PRF and the coordinators from participating Boston hospitals.

In addition, she assists the Patient Programs Coordinator with PRF’s other research related programs.

Please contact Darrien at [email protected].