UCSF Benioff Children's Hospitals Become the First US Qualified Treatment Center Contracted to Administer Individualized Gene Therapy WASKYRA®
WASKYRA®, the first gene therapy approved in the US to treat Wiskott-Aldrich Syndrome, is now commercially available, with more centers expected to be added in the coming months.
How A Nonprofit Ecosystem Is Rescuing Orphaned Cell And Gene Therapies
Nonprofit organizations are building genuine commercial infrastructure — not just advocacy — to rescue clinically viable cell and gene therapies abandoned by for-profit sponsors.
Building a nonprofit pathway for lifesaving gene therapies
Fondazione Telethon and Orphan Therapies are piloting a cost-plus, nonprofit model to sustain access to an ultra-rare gene therapy.
Alternative Commercialisation Models Offer Lifeline to Future of Gene Therapies
Nonprofit founder and CEO, Craig Martin, notes that the industry will likely see more alternative gene therapy commercialisation models in the future.
Fondazione Telethon and Orphan Therapeutics Accelerator Sign Memorandum of Understanding to Pioneer Non-Profit Commercial Access Model for Ultra-Rare Disease Gene Therapy in the US
Collaboration Aims to Establish Sustainable Access to a Newly Approved Therapy Targeting a Rare Immunodeficiency, Filling Urgent Gap Left by For-Profit Efforts
Orphan Therapeutics Accelerator Launches Orphan Therapies, a Non-Profit Biotech Dedicated to Commercializing Ultra-Rare Disease Treatments
Orphan Therapeutics Accelerator (OTXL), a non-profit organization focused on advancing shelved ultra-rare disease treatments through clinical trials, today announced the launch of its wholly owned subsidiary, Orphan Therapies (OT) LLC. Orphan Therapies will focus on commercializing and providing sustained, equitable patient access to approved therapies for conditions with very small patient populations.