Eli Lilly reached an agreement to acquire Prevail Therapeutics, including its clinical program for PR001, an experimental one-time gene therapy for neuronopathic Gaucher disease. Prevail has a portfolio…
A bone marrow transplant successfully reduced the severe lung involvement that developed in a 5-year-old boy with Gaucher disease who was receiving enzyme replacement therapy (ERT), a study reported. The boy was described in a previous report as the youngest patient…
The U.S. Patent and Trademark Office has issued a patent related to PR001, Prevail Therapeutics‘ investigational gene therapy for neuronopathic Gaucher disease. The composition of matter patent grants coverage until Oct. 3, 2038, excluding potential extensions or additional related patent filings. Specifically, it concerns the modified,…
To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…
The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…
A single infusion of AVR-RD-02, Avrobio’s experimental gene therapy for Gaucher disease type 1, was able to lower the levels of two disease biomarkers within three months in the first patient dosed in an ongoing Phase 1/2 trial, the company said. The investigational therapy also was safe…
Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…
Note: This story was updated Nov. 10, 2020, to clarify that Foggin volunteers with the International Gaucher Alliance and does not hold a formal position with Prevail. In addition, the story has been corrected to note that the same PR001 gene therapy is being tested in both Gaucher and Parkinson’s…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is central to NORD’s mission and history — community…
The U.S. Food and Drug Administration (FDA) has given fast track designation to PR001, Prevail Therapeutics‘ investigational gene therapy for neuronopathic Gaucher disease. The FDA previously granted the therapy — now in an enrolling clinical trial in the U.S. — orphan drug and rare pediatric disease status; all aim…
