On July 27, 2021, Knowledge Ecology International (KEI) commented on the “Prospective Grant of an Exclusive Patent License: RP2 AAV-Based Gene Human Therapy for Ocular Diseases and Disorders Including XLRP” (86 FR 36565). The National Institutes of Health (NIH) intends to grant the exclusive license to PTC Therapeutics GT, Inc., a company based in Middlesex, New Jersey.
The geographic scope of the license is to be worldwide, while the field of use is the “development and commercialization of RP2 AAV-based gene human therapy for any ocular disease, disorder or condition, including human X-linked retinitis pigmentosa (XLRP).”
In our comments, KEI urges the NIH to consider the impact of the terms of this exclusive license on patients. Thus far, the gene therapies for inherited genetic disorders that have received FDA approval are some of the highest-priced treatments on the market, including Luxturna, a gene therapy for an inherited form of blindness, which is offered at $850,000. These prices restrict access and harm health outcomes, particularly in low-income countries. Narrowing the term of exclusivity and field of use, as the NIH is required to do, would ensure that the licensee receives adequate remuneration for its investment in the technology, while balancing that interest against the requirement that the license serve the public interest. Patients do not benefit from gene therapies that they cannot afford, and it is a policy of the NIH to promote broad access to NIH-owned inventions in developing countries.
KEI’s full comments regarding the prospective license to PTC Therapeutics are available here: KEI-Comments-NIH-License-PTC-27July2021