Sentynl Therapeutics to License Experimental Progeria Drug

Rare Daily Staff

Sentynl Therapeutics has entered into an agreement to license an experimental drug for Hutchinson-Gilford Progeria Syndrome, a rare genetic disorder that causes rapid aging in children.

The company, a U.S.-based subsidiary of India’s Zydus Lifesciences, reached a deal with South Korea’s PRG S&T to license Progerinin, an investigational small-molecule drug also known as SLC-D011. The agreement allows Sentynl to begin working with PRG S&T immediately to advance the drug’s clinical development.

If certain milestones are met, Sentynl will acquire full rights to the therapy for progeria. The drug program is completing a phase 2A clinical trial, with data expected in the first half of 2026. The U.S. Food and Drug Administration has granted Progerinin orphan drug designation.

Hutchinson-Gilford Progeria Syndrome, often called progeria, is an ultra-rare genetic disease that causes dramatic and rapid aging beginning in early childhood. The condition is caused by a mutation in the LMNA gene that leads to production of an abnormal protein called progerin. The buildup of progerin damages cells and accelerates aging throughout the body. Children with the disease develop symptoms such as severe growth failure, hair loss, joint problems, and hardening of the arteries. Most patients die from heart disease or stroke at an average age of about 14.

Progerinin is designed to target the underlying biology of the disease by blocking harmful interactions caused by the progerin protein inside cells. By interfering with those effects, researchers hope the drug can improve the structure of the cell nucleus and reduce cellular damage linked to premature aging. The drug is taken orally and remains experimental, meaning it has not yet been approved by the FDA or other regulatory agencies.

In early research using mouse models of progeria, Progerinin extended lifespan and improved body weight compared with untreated animals. Scientists are studying whether the drug can slow disease progression and improve survival in patients.

The Progeria Research Foundation helped fund early research that contributed to the development of Progerinin.

“PRF is proud to have funded the foundational research that led to the development of Progerinin, and we are happy to see this potential path moving forward,” said Leslie Gordon, the foundation’s medical director.

Currently, lonafarnib, marketed as Zokinvy, is the only approved treatment for progeria and certain related disorders in the United States and several other countries.

Photo: Leslie Gordon, medical director of Progeria Research Foundation