Andelyn Biosciences has partnered with Tern Therapeutics (Tern) to perform late-stage process performance qualification (PPQ) manufacturing of Tern’s TTX-381 programme.
TTX-381 is an investigational gene therapy targeting vision loss in children with CLN2 Batten disease — an ultra-rare pediatric neurodegenerative 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 paediatric 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 designed to deliver a functional copy of the TPP1 gene directly to retinal cells, thereby producing the missing TPP1 enzyme and preserving patient vision. This approach has the potential to improve patient quality of life and independence.
Andelyn Biosciences is honoured 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 programme 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.”
