Andelyn Biosciences has partnered with Jaguar Gene Therapy to perform late-stage Process Performance Qualification (PPQ) manufacturing of JAG201, an investigational gene therapy targeting SHANK3 haploinsufficiency which is clinically diagnosed as Phelan-McDermid syndrome (PMS), a leading monogenic cause of autism spectrum disorder (ASD). Individuals with PMS or SHANK3-related autism present with lifelong and severe neurobehavioral, motor, social, communicative and cognitive impairments characterized by delayed or absent developmental milestones. Almost all activities of daily living, including being able to communicate their needs, ability to self-care, including dressing, bathing and toileting, and socialization with family or peers are absent or severely impaired, requiring 24-hour care and/or supervision. There is an overwhelming unmet need in PMS and SHANK3-related autism with no effective or approved medications that treat the underlying disease. Available treatments only address individual symptoms. Standard-of-care includes early initiation of speech, occupational, physical and behavioral therapies.
Andelyn will be applying its deep expertise in AAV vector manufacturing and proven track records in GMP production and PPQ execution to support the JAG201 program. As a gene replacement therapy, JAG201 delivers a functional SHANK3 minigene via the AAV9 vector to target neurons in the central nervous system. The therapy is designed to transduce haploinsufficient neurons to provide proper SHANK3 levels and to durably restore the synaptic function required for learning and memory, which underlie appropriate neurodevelopment and maintenance of cognitive, communicative, social and motor skills.
Matt Niloff, Chief Commercial Officer at Andelyn, said, "Partnering with Jaguar on this program highlights our shared passion to accelerate the delivery of safe and effective gene therapies to patients with few or no therapeutic options. Andelyn was established to bring novel therapies like JAG201 to commercial readiness, and we are honored that Jaguar is leveraging our expertise and capabilities to fulfill this purpose."
Rob Mancino, Senior Vice President of Technical Operations at Jaguar Gene Therapy, said, "We are pleased to partner with Andelyn on the Process Performance Qualification (PPQ) manufacturing of JAG201, which aims to treat individuals with SHANK3-related autism and Phelan-McDermid syndrome, and benefit from the company's deep expertise in AAV manufacturing and late-stage readiness. Through this partnership, we continue to demonstrate Jaguar's commitment to ensuring excellence in manufacturing, which enables the highest levels of purity and potency for our therapies."