Stop Sanfilippo Fundacion
Stop Sanfilippo is an independent non-profit foundation based in Madrid, Spain, established in 2012. The organization is committed to advancing research and development focused on Sanfilippo Syndrome, a rare genetic disorder that affects children. Its mission encompasses promoting scientific research to discover effective treatments or cures, enhancing the quality of life for affected children and their families, and increasing awareness of the condition to facilitate early diagnosis. Stop Sanfilippo is a registered member of several notable organizations, including Federación Española de Enfermedades Raras (FEDER) and Organización Europea de Enfermedades Raras (EURORDIS), which further supports its advocacy and outreach efforts.
Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing gene and cell therapies for severe, life-threatening rare genetic diseases. The company's primary programs include EB-101, an autologous cell therapy for recessive dystrophic epidermolysis bullosa, and AAV-based gene therapies for Sanfilippo syndrome types A and B, designated as ABO-102 and ABO-101, respectively. Additionally, Abeona is advancing therapies for Batten disease with ABO-201 and ABO-202, and exploring treatments for cystic fibrosis and genetic eye disorders. The company has a strategic partnership for the development of gene therapies targeting MPS III and other conditions. Abeona also operates a fully-functional manufacturing facility to support its clinical and preclinical endeavors and is developing the AIM Vector Platform, which includes over 100 next-generation AAV capsids designed for efficient gene delivery. Founded in 1974 and headquartered in New York, Abeona Therapeutics is committed to addressing unmet medical needs in rare diseases.
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