On the last day of February, millions of people came together globally to honor Rare Disease Day. It’s a day dedicated to raising awareness of rare diseases, the impact they have on people’s lives and the importance of developing treatments to improve quality of life for patients and families around the world.
The National Institutes of Health defines a rare disorder as any disease that impacts fewer than 200,000 people in the U.S. According to EURORDIS and the National Organization for Rare Disorders (NORD), one in 20 people will live with a rare disease at some point in their life.
The 2021 theme of Rare Disease Day – “Rare is Many. Rare is Strong. Rare is Proud.” – underscores an important truth of the rare disease community: The diseases themselves are rare, but the amount of people affected is staggering.
Advancing the treatment of rare diseases through cell and gene therapy
That truth also gets to the core of our mission at Be The Match BioTherapies: saving lives through cellular therapy. Our team is committed to supporting the development of life-saving cell and gene therapies to treat a variety of diseases, including liquid and solid tumors, primary immune deficiencies and rare diseases.
Our global cell and gene therapy partners work daily to advance therapies that will have a positive impact on the lives of patients. Some of our partners are advancing programs to address the needs of rare and underserved patient populations as part of their research pipeline.
They include Gamida Cell, a company developing curative therapies for hematologic cancers as well as rare blood disorders, and Orchard Therapeutics, a company focused on treating rare, inherited disorders that have an immense burden on children, families and caregivers.
Supporting the EU commercial launch of a gene therapy for MLD
While there is work to do, we are encouraged by the progress we are seeing in our industry with regard to rare diseases. On March 3, we announced an extension of our long-term collaboration with Orchard to support the commercial launch of LibmeldyTM. The gene therapy developed by Orchard treats early-onset metachromatic leukodystrophy (MLD).
Libmeldy is the first gene therapy to receive full EU marketing authorization for eligible MLD patients. It has been shown to preserve motor and cognitive function for patients through a single treatment. As Orchard moves to make the therapy available to more MLD patients, our team will provide support across the Libmeldy gene therapy supply chain. This is a complex and delicate process that requires an experienced and dedicated team.
Our partnership will span every step of Libmeldy’s commercial development, including:
- Support of the onboarding and training of apheresis centers
- Oversight of the autologous cell collection process
- Delivery of both harvested cells to the manufacturing site and gene-corrected cells back to the qualified treatment center
Creating a brighter future for patients with rare diseases and their families
This story and video on Orchard’s website illustrate the perspective of one family with MLD and underscore just how transformative a treatment can be. It’s stories like these shared by the community that are the heart of Rare Disease Day. And it’s the work of our team, and that of our partners, that will help create a brighter future for that community.
As we reflect on Rare Disease Day 2021 and look toward the next steps for our organization, we remain deeply committed to playing a part in that important effort and driving continued progress in making a difference for the many people affected by rare diseases around the world.