Andelyn Biosciences has announced a strategic partnership with Jaguar Gene Therapy to advance the late-stage Process Performance Qualification (PPQ) manufacturing of JAG201. This investigational gene therapy is specifically designed to address SHANK3 haploinsufficiency and Phelan-McDermid syndrome (PMS), a leading genetic cause of autism spectrum disorder characterized by significant neurodevelopmental impairments.
The collaboration capitalizes on Andelyn’s expertise in adeno-associated virus (AAV) manufacturing, which is critical as Jaguar Gene Therapy aims to prepare JAG201 for commercial readiness. The agreement emphasizes Andelyn’s robust track record in good manufacturing practice (GMP) production and PPQ execution, vital for ensuring the therapeutic’s efficacy and safety.
Focus on Gene Therapy for Neurodevelopmental Disorders
JAG201 utilizes an AAV9 vector to deliver a functional SHANK3 minigene directly to neurons in the central nervous system. The primary objective is to restore synaptic functions essential for learning and memory, alongside the development of cognitive and motor skills. This therapy holds promise for patients suffering from PMS, where treatment options are currently limited.
Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, remarked, “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.” His statement underscores the collaborative commitment to address unmet medical needs in the field of neurodevelopmental disorders.
Rob Mancino, Senior Vice President of Technical Operations at Jaguar Gene Therapy, noted the critical nature of late-stage manufacturing readiness. He pointed out that Andelyn’s proficiency in AAV production is essential for achieving the required purity and potency in therapies targeting SHANK3-related autism and PMS. This partnership signals both companies’ dedication to manufacturing excellence in the evolving field of gene therapy.
Accelerating the Path to Commercial Readiness
The collaboration aligns with Andelyn’s broader mission to expedite gene therapies from preclinical development through to commercial deployment. The company offers contract development and manufacturing organization (CDMO) services across various AAV manufacturing platforms, supported by advanced facilities designed to prioritize patient outcomes.
As the partnership moves forward, it aims to enhance the efficiency of bringing innovative therapies like JAG201 to market, ultimately benefiting patients who are in dire need of effective treatment options. The work of both Andelyn and Jaguar reflects a significant advancement in the biopharmaceutical landscape, particularly in the realm of genetic disorders.
The agreement marks a pivotal step in the journey of JAG201, reinforcing the commitment of both organizations to improve the lives of individuals affected by SHANK3 haploinsufficiency and Phelan-McDermid syndrome.
