Partnership to Support Real-World Evidence Studies and Fuel Development of Gene Therapy for Lesch-Nyhan Disease
AllStripes’ Patient-Centric Rare Disease Platform Expands to 20 Rare Diseases
AllStripes (formerly RDMD), a healthcare technology company dedicated to accelerating drug research for patients with rare diseases, today announced a partnership with the Orphan Disease Center at the Perelman School of Medicine at the University of Pennsylvania to fuel the development of gene therapies in Lesch-Nyhan disease and a second rare disease to be named soon.
AllStripes has developed a novel technology platform that generates deep clinical evidence to accelerate rare disease research and drug development, as well as a patient application that empowers patients and families to participate in research from home.
Through this partnership, the Orphan Disease Center will leverage AllStripes’ platform to launch real-world evidence studies aimed at advancing clinical understanding of these diseases through natural history, disease progression and disease severity, with outcomes including quality of life, activities of daily living, health resource utilization and other clinical outcomes. Learnings from these real-world evidence studies will support two rare disease gene therapy programs at the Orphan Disease Center, Lesch-Nyhan disease and another to-be-announced rare disease.
"2020 has been a great year of growth for AllStripes. We recently rebranded from the name RDMD to AllStripes, a change that was inspired by the official symbol for the rare disease community, the zebra. When we come together and learn from each other’s experiences we can push for better treatments across all rare conditions," said Nancy Yu, co-founder and chief executive officer of AllStripes. "This year we have already expanded from 12 diseases to 20, and today we are thrilled to offer people living with Lesch-Nyhan disease a streamlined way to access their medical records and participate in research online. Penn Medicine’s Orphan Disease Center is an esteemed leader in gene therapy and rare disease research, and we are proud to have them as a partner as we create a research resource for the rare disease community that enables the development of novel treatments."
With this partnership, AllStripes has expanded into 20 total rare diseases, including CDKL5 deficiency, chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), Crigler-Najjar syndrome type 1, cystinosis, GM1 gangliosidosis (GM1), GM2 gangliosidosis (GM2), Hunter syndrome (MPS II), inclusion body myositis (IBM), idiopathic thrombocytopenic purpura (ITP), Lesch-Nyhan disease, Myasthenia gravis (MG), neurofibromatosis type 1 (NF1), neurofibromatosis type 2 (NF2), Niemann-Pick disease type C (NPC), progressive supranuclear palsy (PSP), Sanfilippo syndrome (MPS III), SLC6A1 epileptic encephalopathy (SLC6A1), SURF1 Leigh syndrome (SURF1), thymidine kinase type 2 deficiency (TK2d) and Wilson disease.
"We share a common mission – to accelerate therapeutic development for rare diseases in which there are few resources," said James M. Wilson, M.D., Ph.D., Rose H. Weiss Professor and director, Orphan Disease Center. "This partnership will allow Lesch-Nyhan disease and other patients and parents to not only access and own their medical records, but importantly also become active participants in powering multiple research efforts for these life-threatening conditions with limited treatment options."
AllStripes is a healthcare technology company dedicated to accelerating treatments for patients with rare diseases. AllStripes has developed a technology platform that generates FDA-ready evidence to accelerate rare disease research and drug development, as well as a patient application that empowers patients and families to securely participate in drug research online and benefit from their own medical data. AllStripes was founded by CEO Nancy Yu and technology developer Onno Faber, following his diagnosis and journey with the rare disease neurofibromatosis type 2. The company is backed by Lux Capital, Spark Capital, Maveron Capital, Village Global, Garuda Ventures and a number of angel investors. For more information, visit www.allstripes.com.
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Katie Engleman, 1AB