KEI Comments Regarding NIH Exclusive License to Progeria Research Foundation for Ultra-Rare Disease Treatment

Knowledge Ecology International (KEI) filed comments on July 27, 2021 regarding the National Institutes of Health’s (NIH) “Prospective Grant of an Exclusive Patent License: Oligonucleotides Analogues Targeting
Human LMNA “lamin A” Gene” (86 FR 36563). The inventions are currently co-owned by the Government of the United States of America, the University of Maryland, Sarepta Therapeutics, Inc, and the Progeria Research Foundation, and with this exclusive license, will be consolidated under the Progeria Research Foundation (PRF). PRF is based in Peabody, Massachusetts.

The proposed license will be worldwide, exclusive, and sublicensable. The inventions to be licensed involve “modified oligonucleotides (called phosphorodiamidate morpholino oligonucleotides or PMOs) targeted to pre-mRNA of human LMNA Lamin A gene. These PMOs can be used to correct aberrant splicing of LMNA gene.” This aberrant splicing of the LMNA gene is known to be related to Hutchinson-Gilford Progeria Syndrome (HGPS), among other related laminopathies. HGPS is an ultra-rare, fatal, genetic disorder, with a prevalence of 1 in 20 million.

It is important that the terms of the license, and any sublicense, promote access to the treatment for patients. Patients with rare diseases often face high priced treatments, and it is critical that the NIH ensures that these patients are not held hostage to high prices.

A PDF of KEI’s full comments is available here: KEI-Comments-NIH-License-PRF-27July2021