Andelyn Biosciences and Tern Therapeutics Partner for Late-Stage Process Performance Qualification (PPQ) Manufacturing of AAV Gene Therapy for the Treatment of CLN2 Batten Disease

COLUMBUS, Ohio, Sept. 3, 2025 /PRNewswire/ -- Andelyn Biosciences, Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has partnered with Tern Therapeutics (Tern) to perform late-stage Process Performance Qualification (PPQ) manufacturing of Tern's TTX-381 program. TTX-381 is an investigational gene therapy targeting vision loss in children with CLN2 Batten disease, an ultra-rare pediatric neuro-degenerative disorder with no available treatment for the ocular manifestations of the disease.

CLN2, a form of Batten disease caused by TPP1 protein deficiency, is a pediatric neurodegenerative disorder with an estimated incidence of 0.5 per 100,000 live births. Most affected children begin experiencing seizures and language, motor, and cognitive decline between ages 2 and 4, with vision loss beginning soon afterwards that rapidly leads to total loss of vision as early as age 7.

TTX-381 is a one-time, subretinal AAV gene therapy that is designed to deliver a functional copy of the TPP1 gene directly to the retinal cells to produce the missing TPP1 enzyme and preserve patient vision, with the potential to profoundly improve patient quality of life and independence. Andelyn Biosciences is honored for the opportunity to leverage deep expertise in AAV vector manufacturing and demonstrated success in PPQ batch production toward the advancement of TTX-381 and accelerating access to this much needed therapy.

"Partnering with Tern Therapeutics on this transformative program underscores Andelyn's commitment to enabling breakthrough therapies for rare and devastating diseases," said Matt Niloff, Chief Commercial Officer at Andelyn, "Our deep experience in AAV process development and manufacturing, coupled with regulatory alignment and proven execution of PPQ batches, positions us to help bring this therapy to patients as efficiently and safely as possible."

"We're excited to partner with Andelyn and leverage their long history with AAV technologies, strong late-stage manufacturing capability, and a shared dedication to rare disease patients," said Alex Bailey, Chief Executive Officer at Tern. "Together, we aim to deliver a therapy that has the potential to change the lives of children and families facing CLN2 Batten disease, providing hope where today there are no effective treatment options."

This collaboration reflects Andelyn's broader mission to accelerate gene therapies from preclinical through commercial readiness. With industry-leading 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, visit andelynbio.com.

About Tern Therapeutics

TERN THERAPEUTICS is a privately held biotechnology company founded in 2023 with a new vision for speeding the development of transformative, one-time gene therapy medicines for rare diseases. Guided by a team of leading physicians, scientists, and business leaders and in collaboration with patient communities, Tern is driven to deliver transformative treatments with urgency to those living around the world with rare diseases. For more information about Tern, please visit www.terntx.com.

SOURCE Andelyn Biosciences