Drug intelligence / Profile preview

SamPro-2

Development stage
Preclinical
Lead developer
Progeria Research Foundation
Modality
Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
01

Overview

SamPro-2 is an investigational gene therapy developed by the Progeria Research Foundation (PRF) for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS), a rare and fatal genetic disorder characterized by accelerated aging. HGPS is caused by a point mutation in the LMNA gene, which results in the production of a truncated, toxic protein called progerin that accumulates in cells and causes systemic damage. SamPro-2 is designed to address the underlying genetic cause of the disease. In 2023, the Progeria Research Foundation partnered with Forge Biologics, a contract development and manufacturing organization (CDMO), to provide process development and cGMP manufacturing services to support IND-enabling studies, moving the therapy toward its first clinical trials.

02

Targets

LMNA (Lamin A)

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