Andelyn Biosciences and Tern Therapeutics announce collaboration for gene therapy production.
In a significant development for the medical world, a collaboration between Andelyn Biosciences and Tern Therapeutics is working towards a potential breakthrough in treating vision loss in children with CLN2 Batten disease, a rare pediatric neuro-degenerative disorder.
CLN2 Batten disease, with an estimated incidence of 0.5 per 100,000 live births, is a condition that affects young children, often causing seizures, language, motor, and cognitive decline between ages two and four. Vision loss follows shortly after and can lead to total loss of vision as early as age seven. Currently, there are no effective treatment options for the ocular manifestations of this disease.
The goal of the collaboration is to bring TTX-381, a one-time, subretinal AAV gene therapy, to patients as soon as possible. TTX-381 is an investigational gene therapy targeting vision loss in children with CLN2 Batten disease. It 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.
Matt Niloff, CCO at Andelyn, highlights their deep experience in AAV process development and manufacturing, while Alex Bailey, CEO at Tern, emphasizes their shared aim to deliver a therapy that has the potential to change the lives of children and families facing CLN2 Batten disease.
Andelyn Biosciences' commitment to enabling breakthrough therapies for rare and devastating diseases is underscored by their partnership with Tern Therapeutics on the TTX-381 program. Their experience includes regulatory alignment and proven execution of PPQ batches, positioning them to help bring TTX-381 to patients efficiently and safely.
The institutions involved in the development of a consent process for the TTX-381 program, a gene-based therapy for the rare disease CLN2 Batten disease, include Torch Therapeutics and regulatory and clinical research bodies collaborating on the therapy's clinical trials.
Andelyn Biosciences has partnered with Tern Therapeutics for late-stage manufacturing of the TTX-381 program. The goal of the collaboration is not just to bring a potential treatment to market, but to profoundly improve the quality of life and independence of children affected by CLN2 Batten disease.
With TTX-381, there is hope for a future where children with this rare disease can preserve their vision and live more independently. The collaboration between Andelyn Biosciences and Tern Therapeutics is a step towards making this hope a reality.
