OpenTreatments Foundation Launches Software to Decentralize and Accelerate Drug Development for Rare Genetic Diseases
SEATTLE, March 31, 2021 /PRNewswire/ -- OpenTreatments Foundation, a nonprofit organization with a mission to enable treatments for rare genetic diseases regardless of rarity and geography, today announced the launch of OpenTreatments, an innovative open source software platform that will enable patients to create gene therapies for rare genetic diseases. The platform enables patient-led organizations to develop treatments using the Adeno Associated Virus (AAV) gene therapy technology. It provides a robust roadmap, advice from drug development experts, and research infrastructure (labs, contract research facilities, manufacturing facilities, software tools) to execute every step of the drug development pipeline from planning, design, manufacturing, all the way to clinical trials.
"Rare disease drug development is a complex maze. OpenTreatments software platform empowers patient-led organizations to navigate that maze and create treatments for the diseases they care about. We will enable the hand off of these therapies to commercial, governmental and philanthropic entities to ensure patients get access to the therapies for the years to come", said Sanath Kumar Ramesh, founder and CEO of the OpenTreatments Foundation.
The Linux Foundation, a nonprofit organization enabling mass innovation through open source, is collaborating with OpenTreatments Foundation through RareCamp software project to provide open governance and source code for OpenTreatments platform.
There are 400 million patients worldwide affected by more than 7,000 rare diseases, yet treatments for rare genetic diseases are an underserved area. More than 95 percent of rare diseases do not have an approved treatment, and new treatments are estimated to cost more than $1 billion. "Recently, patient groups have risen as the primary driving force behind drug development for rare diseases. The support provided by OpenTreatments to patient-led organizations will dramatically reduce both the time and cost of drug development, leading to more treatments reaching patients faster.", said Dr. Ashley Winslow, Chief Scientific Officer of Odylia Therapeutics, a non-profit organization working to bring treatments to rare diseases and a collaborator of OpenTreatments Foundation.
OpenTreatments Foundation is a collaborative effort with patients, advocacy and industry organizations. Baylor College of Medicine, Castle IRB, Charles River, Columbus Children's Foundation, GlobalGenes, Odylia Therapeutics, RARE-X, and Turing.com are collaborating with OpenTreatments Foundation to support patient-led organizations with drug development advice and research infrastructure necessary to advance their treatments. Amazon Web Services (AWS) is providing expert advice and support to develop and run the OpenTreatments software platform on the AWS cloud. OpenTreatments Foundation's leadership includes patients and rare disease drug development experts Julia Vitarello, Dr. Plavi Mittal, and Dr. Ethan Perlstein as board of directors and Sanath Kumar Ramesh as the Founder and CEO.
OpenTreatments software platform is launching with three pilot gene therapy programs from patient-led organizations CureCMD, IDefine and CureGPX4. "OpenTreatments will enable IDefine to accelerate our mission by distilling down the necessary steps in the development of a gene therapy program into a concise and approachable plan", said Geoff Rhyne, Co-Founder and CEO of IDefine, a patient-led organization tackling Kleefstra Syndrome caused by mutations in EHMT1 gene.
Patient-led organizations with an interest in building gene therapy can apply to join the platform at OpenTreatments.org.
About OpenTreatments Foundation
SOURCE OpenTreatments Foundation