Partnership will Support Advancement of Jaguar's JAG201 Clinical-Stage Investigational Gene Therapy Targeting a Monogenic Cause of Autism Spectrum Disorder Known as Phelan-McDermid Syndrome
COLUMBUS, Ohio, Dec. 8, 2025 /PRNewswire/ -- Andelyn Biosciences, a leading and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), 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."
This relationship reflects Andelyn's broader mission to accelerate gene therapies from preclinical through commercial readiness. With industry-leading commercial capabilities across suspension and adherent AAV manufacture, state-of-the-art facilities, and a patient-first approach, Andelyn continues to serve as the CDMO partner of choice for organizations advancing life-changing gene therapies.
About Andelyn Biosciences, Inc.
Andelyn Biosciences is a full-service cell and gene therapy CDMO focused on the development, characterization, and production of viral vectors for gene therapy. With more than 20 years of experience, Andelyn's deep scientific expertise has resulted in the production of cGMP material for more than 450 clinical batches and 75 global clinical trials. Operating out of its development and manufacturing facilities in Columbus, Ohio, Andelyn supports its clients in developing cell and gene therapies from concept through plasmid engineering and manufacturing, process and analytical development, and cGMP clinical and commercial manufacturing. Andelyn can accelerate programs and deliver high-quality products by developing and manufacturing processes on its configurable, data-driven AAV Curator® Platform or tech transfer in an established client program. Capabilities include cGMP manufacturing for suspension processes up to a 2,000-liter and adherent processes. A rigorous quality system, regulatory support, and supply chain vertical integration further advantages clients in bringing their critical therapies to market. For more information, please visit andelynbio.com and follow Andelyn Biosciences on LinkedIn.
About Jaguar Gene Therapy
Jaguar Gene Therapy, LLC is a clinical-stage biotechnology company dedicated to accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases including those that impact sizeable patient populations. The company is made up of a proven team of experts who have first-hand experience in bringing novel gene therapy treatments to patients and their families. Jaguar is rapidly advancing an initial pipeline of three programs. The lead program targets SHANK3 haploinsufficiency, a leading monogenic cause of autism spectrum disorder that is clinically diagnosed as Phelan-McDermid syndrome (PMS). Individuals with SHANK3-related autism or PMS present with lifelong and severe neurobehavioral, motor, social, communicative, and cognitive impairments characterized by delayed or absent developmental milestones. A clinical trial in pediatric patients is currently underway. The second pipeline program targets Type 1 galactosemia and the third targets Type 1 diabetes. Jaguar's key investors include Deerfield Management Company, ARCH Venture Partners and Eli Lilly and Company. For more information, please visit www.jaguargenetherapy.com and follow Jaguar Gene Therapy on LinkedIn.
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SOURCE Andelyn Biosciences
