Angelman syndrome is a neurological disorder caused by the loss or malfunction of the maternal copy of the UBE3A gene in neurons found in specific regions of the brain. People inherit two copies of the UBE3A gene, one from their mother and one from their father, but only the mother’s gene is active in certain brain areas.
Replacing the mutated version of the UBE3A gene with a healthy version using gene therapy could, in theory, ease or treat this disease.
For many years, researchers have been experimenting with gene therapy as a cure for genetic diseases. Unfortunately, one of the issues that often is encountered is the incapacity to deliver gene therapy to the correct cells in the body.
Viruses are capable of entering the human body and infect specific cell types. So, scientists have taken advantage of viruses’ natural capacity to enter cells and have engineered viral structures that act as transporters to carry healthy copies of faulty genes to specific cells. These viral vectors are inactivated, but do not cause disease.
StrideBio is focused on the development of adeno-associated viral (AAV)-based gene therapies. The company was founded by Mavis Agbandje-McKenna, PhD, and Aravind Asokan, PhD, leading scientists in the field of AAV-based gene therapy.
StrideBio has an ever-growing collection of new AAV capsids (the outer structure of viruses) that have been evolved in non-human primates; they show reduced seroprevalence (the presence of viral vectors in blood), and have the potential for improved homing to targeted tissues.
As per the agreement, Sarepta now can use StrideBio’s new, structure-driven viral technology for specific targets. This technology will allow for enhanced delivery of gene therapy to the tissues of interest and to escape responses mounted by a patient’s immune system, which is a common concern when using gene therapies.
“With our partnership with StrideBio, Sarepta continues to build on its leadership position in gene therapies to treat rare diseases. We are excited to work with StrideBio and access its innovative AAV platform for next-generation capsids,” Doug Ingram, Sarepta’s president and CEO, said in a press release.
Furthermore, Sarepta and StrideBio plan to devise ways to help address re-dosing challenges in patients who already have received AAV-based gene therapies.
StrideBio will be responsible for leading all the investigational new drug (IND)-enabling research, development and production for four targets, including MECP2 for treating Rett syndrome, SCN1A for Dravet syndrome, NPC1 for Niemann-Pick disease, and UBE3A for Angelman syndrome.
Sarepta will have an exclusive option for up to four other targets based on StrideBio’s capsid technology.
“Our partnership with StrideBio expands our research portfolio by up to eight new targets and, through our strategic partnering approach that has our collaborator lead all IND-enabling research and development, ensures that we gain access to new technology and targets while not distracting Sarepta from its near-term priorities,” Ingram said.
“This partnership will provide significant resources and expertise to enable StrideBio’s continued rapid expansion of our research and manufacturing platform, as well as accelerate the development of AAV gene therapies for multiple rare disease targets. We are looking forward to working together with Sarepta to bring novel treatments to patients as quickly as possible,” said Sapan Shah, PhD, CEO of StrideBio.