隶属于 founders combined charity income common investment fund
相关临床试验
15
5 进行中
药物批准
1
批准总数
监管机构
1
监管机构数
成立时间
1990
进行中（未招募）
3
20.0%
已完成
6
40.0%
Enrolling By Invitation
1
6.7%
尚未招募
1
6.7%
招募中
3
20.0%
撤回
1
6.7%
- UCSF Benioff Children's Hospitals has been designated the first US Qualified Treatment Center contracted to administer WASKYRA (etuvetidigene autotemcel) to eligible patients with Wiskott-Aldrich Syndrome. - WASKYRA, approved by the FDA in December 2025, is the first gene therapy available in the US for Wiskott-Aldrich Syndrome and the first advanced therapy commercialized entirely through a non-profit collaboration. - The autologous hematopoietic stem cell-based therapy is indicated for patients aged six months and older with a WAS gene mutation and no suitable HLA-matched related stem cell donor. - Fondazione Telethon developed and submitted the therapy for approval, while Orphan Therapies serves as the exclusive US commercialization partner, with more treatment centers expected to be added.
- Fondazione Telethon will manage a €30 million ERDERA funding call supporting Phase I, I/II, and II clinical trials for rare diseases with limited or no therapeutic options. - The call requires international consortia of at least three partners from three different countries, with mandatory patient organization involvement in trial design and conduct. - Applications open with a mandatory Expression of Interest phase from July 1 to September 10, 2026, with the full selection process expected to conclude by February 2028. - The initiative spans over 170 organizations across 37 countries and includes the UK Ministry of Research and Innovation's financial contribution managed through Fondazione Telethon.
- Fondazione Telethon has named Graciana Diez-Roux as its new Scientific Director to lead research strategy for rare genetic diseases. - Diez-Roux brings over two decades of experience at TIGEM and has coordinated European projects worth approximately €70 million focused on rare disease therapies. - She emphasizes investing in young researchers and fostering collaborative, impact-oriented environments to accelerate translation of discoveries into clinical applications. - Her leadership will focus on defining research priorities, overseeing scientific evaluation integrity, and integrating intramural and extramural research efforts.
- Cytiva has launched a multi-year research collaboration with San Raffaele Telethon Institute for Gene Therapy (SRTIGET) to advance next-generation genomic medicine platforms, focusing on lentiviral gene transfer and targeted gene editing of hematopoietic stem cells. - The partnership represents the first Danaher Beacon in the European Union and aims to address persistent bottlenecks in cell and gene therapy manufacturing, targeting high-impact areas where improvements can accelerate translation from discovery to delivery. - With 2,130 cell and gene therapy clinical trials in progress in 2025, the collaboration seeks to optimize processes, improve consistency, reduce costs, and enhance scalability for genomic medicine platforms. - The initiative combines SRTIGET's scientific expertise in hematopoietic stem cell gene therapies with Cytiva's manufacturing capabilities, spanning from process development through commercialization.
- The FDA has approved Waskyra, the first gene therapy for Wiskott-Aldrich syndrome (WAS), a rare genetic immunodeficiency affecting 1 in 250,000 male births. - Fondazione Telethon becomes the first non-profit organization worldwide to receive FDA authorization to distribute a therapy developed from its own research. - Clinical trials demonstrated substantial benefits including a 93% reduction in severe infections and 60% decrease in bleeding events following treatment. - The approval establishes a new patient-centered model for developing treatments where pharmaceutical companies withdraw due to limited commercial viability.
- The FDA has approved etuvetidigene autotemcel (Waskyra), the first cell-based gene therapy for treating Wiskott-Aldrich syndrome in patients six months and older. - Clinical trials demonstrated a 93% reduction in severe infections and 60% decrease in moderate to severe bleeding events following treatment. - Waskyra represents the first approved genetic treatment from a non-profit sponsor, developed by Italy-based Fondazione Telethon ETS. - The therapy uses patients' own genetically corrected hematopoietic stem cells to restore functional WAS protein expression and address the underlying disease cause.
- The European Medicines Agency's Committee for Medicinal Products for Human Use issued a positive opinion recommending marketing authorization for Waskyra, an ex vivo gene therapy for Wiskott-Aldrich syndrome. - Fondazione Telethon becomes the first non-profit organization to successfully lead the complete pathway from laboratory research to regulatory approval for a gene therapy. - The therapy consists of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the WAS gene, offering a potential curative option for patients without compatible donors. - Clinical data demonstrates that Waskyra reduces the frequency of severe and moderate bleeding events and serious infections in patients with this rare primary immunodeficiency.
- Can-Fite BioPharma has completed the design of a Phase II clinical trial for Piclidenoson in Lowe Syndrome, a rare genetic disease affecting approximately 1 in 500,000 people with no approved treatments. - The open-label study will enroll five patients who will receive 3mg of Piclidenoson twice daily for 12 months, with the primary endpoint measuring improvements in renal uptake. - The development follows successful preclinical research by Dr. Antonella De Matteis, who found Piclidenoson significantly decreased urinary protein loss in Lowe Syndrome models after testing thousands of compounds.
