Good Things Come in 3’s!

Niccolo, Alessandro and Sammy are among nearly 100 children and young adults with Progeria across the world who are benefitting from the life-extending, FDA-approved lonafarnib drug. PRF will continue to devote funds to ensure access to lonafarnib for all, and to study its long-term effects.

But we won’t stop there.

PRF is exploring 3 other therapeutic pathways

to treat and cure Progeria, attacking this disease:

at the Protein, at the RNA, and at the DNA levels!

SMALL MOLECULE DRUGS

HOW does it work? This treatment reduces the level of the toxic progerin protein that causes Progeria.

WHAT is PRF’s role? PRF is collaborating with the biotech company PRGS&T to begin a new clinical trial exploring whether Ionafarnib, in combination with a new drug called progerinin, is more effective than Ionafarnib alone.

WHY is it promising? This new drug increased lifespan in a Progeria mouse model by 50%.

RNA THERAPEUTICS

HOW does it work? This treatment blocks the production of progerin at the RNA level, before the toxic protein is made by the body’s cells.

WHAT is PRF’s role? PRF is the drug developer (or “sponsor”). This means PRF assumes responsibility and costs for drug manufacturing, pre-trial animal and cellular studies, and clinical trials conducted by world-class scientific experts.

WHY is it promising? The RNA therapeutic resulted in a 62% increase in Progeria mouse lifespan.

DNA BASE EDITING

HOW does it work? This drug will enter the body’s cells, find the Progeria mutation, and fix it permanently. The edited cell will no longer make any progerin – WOW!

WHAT is PRF’s role? PRF will fund drug manufacturing, pre-trial animal and cellular studies, and clinical trials conducted by world-class scientific experts.

WHY is it promising? In the Progeria mouse model, lifespan was extended by 140%, approaching what is normally old age for healthy mice. This is a one-time injection that could be our pathway to the CURE!

Science is a costly endeavor, and with your ongoing support, we will continue to

push the boundaries of science toward the cure.

Donate today and be ONE to make the cure POSSIBLE!