- Andelyn Biosciences has partnered with Jaguar Gene Therapy to perform late-stage PPQ manufacturing of JAG201, an investigational therapy targeting SHANK3 haploinsufficiency and Phelan-McDermid syndrome.
- The collaboration leverages Andelyn’s AAV manufacturing expertise to support Jaguar’s programme as it moves toward commercial readiness.
Andelyn Biosciences has entered a partnership with Jaguar Gene Therapy to conduct late-stage Process Performance Qualification (PPQ) manufacturing for JAG201, an investigational gene therapy designed for SHANK3 haploinsufficiency and clinically diagnosed Phelan-McDermid syndrome (PMS). PMS is a leading monogenic cause of autism spectrum disorder and is characterised by severe neurodevelopmental impairment across behavioural, motor, social and cognitive functions.
The agreement outlines how Jaguar will rely on Andelyn’s experience in AAV vector production and its record in GMP manufacturing and PPQ execution. JAG201 is a gene replacement therapy using an AAV9 vector to deliver a functional SHANK3 minigene to neurons in the central nervous system. The therapeutic goal is to restore synaptic function necessary for learning, memory and the development of core cognitive and motor skills.
“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.”
Matt Niloff, Chief Commercial Officer at Andelyn
Rob Mancino, Senior Vice President of Technical Operations at Jaguar Gene Therapy, highlighted the importance of late-stage readiness and AAV manufacturing expertise in ensuring purity and potency for programmes targeting SHANK3-related autism and PMS. The partnership reflects Jaguar’s commitment to manufacturing excellence.
The collaboration aligns with Andelyn’s broader mission to accelerate gene therapies from preclinical development through to commercial readiness. The company provides CDMO services across suspension and adherent AAV manufacturing platforms, supported by advanced facilities and a patient-first operating model aimed at enabling the delivery of next-generation therapeutic programmes.
