The Foundation for Angelman Syndrome Therapeutics (FAST), a patient advocacy organisation working to support the development of transformative treatment options for Angelman syndrome, has entered into an agreement with Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines. Under the agreement, FAST may access TfR1 CapX, Apertura’s blood-brain barrier-crossing capsid technology, in investigational gene therapy programs designed for the treatment of Angelman syndrome.
Angelman syndrome is a rare, nondegenerative neurogenetic disorder that affects an estimated one in 15,000 people. It is caused by the loss of function of the UBE3A gene, which is critical for neuronal function. Angelman syndrome is associated with significant developmental delays, motor challenges, seizures, sleep disruption, and limited or no speech. Individuals living with Angelman syndrome require lifelong support.
“Delivering potential genetic therapies to the central nervous system presents distinct scientific and delivery challenges for Angelman syndrome,” said Julien de Bournet, chief business officer of FAST.
“Addressing those challenges requires exploring multiple scientific approaches and bringing together the right technologies and expertise. Our agreement for access to TfR1 CapX reflects FAST’s commitment to investing in promising delivery technologies that could support future gene therapy programs.”
TfR1 CapX is an AAV capsid designed to target human transferrin receptor 1 (hTfR1), cross the blood-brain barrier after intravenous administration, and enable broad distribution to the brain, addressing one of the major barriers to delivering therapeutics to the central nervous system. Several programmes that use TfR1 CapX are expected to enter clinical trials over the next 12 months.
“This agreement between Apertura and FAST is an example of how biotech companies and patient advocacy organizations can collaborate to develop therapeutic programs that have the potential to help patients living with rare diseases,” said Dave Greenwald, executive chairman of the board at Apertura Gene Therapy and managing director at Deerfield Management.
About FAST (Foundation for Angelman Syndrome Therapeutics)
FAST is an advocacy organisation working to cure Angelman syndrome. As the largest non-governmental funder of Angelman syndrome research in the world, its goal is to drive forward transformative research and development programs to have the greatest impact on those living with Angelman syndrome — regardless of age or genotype.