#USA #Columbus #Andelyn Biosciences #AAV Gene Therapy #CLN2 Batten Disease

Andelyn Biosciences Partners with Tern Therapeutics

Andelyn Biosciences, a leader in the field of cell and gene therapy, has announced an exciting partnership with Tern Therapeutics. This collaboration focuses on the late-stage manufacturing process performance qualification (PPQ) of AAV gene therapy, specifically targeting the treatment of CLN2 Batten disease. This condition is a severe neurological disorder that affects young children, leading to rapid vision loss and various cognitive impairments.

Understanding CLN2 Batten Disease

CLN2 Batten disease is an ultra-rare neurodegenerative condition caused by a deficiency in the TPP1 protein. The severity of this disease is particularly troubling, as affected children typically start showing symptoms like seizures, cognitive decline, and vision loss from a very early age, often between two to four years old. Unfortunately, total loss of vision can occur as quickly as age seven. Given the staggering estimated incidence of 0.5 cases per 100,000 live births, the need for effective therapies is dire.

TTX-381: A Promising Therapeutic Approach

The investigational therapy at the center of this collaboration is known as TTX-381. This one-time subretinal AAV gene therapy aims to deliver a functional copy of the TPP1 gene directly into retinal cells. By doing this, the therapy seeks to produce the missing TPP1 enzyme that is crucial for preserving vision in these young patients. Successful administration could significantly enhance their quality of life and independence, where previously no effective treatments existed.

The Role of Andelyn Biosciences

Andelyn Biosciences is well-equipped to support the manufacturing needs of Tern's TTX-381 program. With a wealth of experience in AAV vector production, Andelyn is delighted to apply its expertise in process performance qualification batch production to this life-changing project. Matt Niloff, the Chief Commercial Officer at Andelyn, emphasized the importance of this partnership, stating that it highlights the company's commitment to enabling innovative therapies for serious and rare diseases. The organization's proven track record, regulatory alignment, and depth of AAV development and manufacturing processes position it as a key player in bringing this transformative therapy to those in need as quickly and safely as possible.

The Vision for the Future

Tern Therapeutics, on the other hand, brings together a team of dedicated professionals—including leading scientists and physicians—eager to expedite the treatment process for patients battling rare diseases like CLN2 Batten disease. Alex Bailey, the CEO of Tern, expressed excitement at the collaboration, noting that combining their efforts with Andelyn's strengths could make a profound difference for children and families affected by this condition.

A Broader Mission

This partnership exemplifies Andelyn Biosciences' overarching mission to accelerate the journey of gene therapies from preclinical stages to commercial readiness. Their advanced capabilities in AAV manufacture, along with state-of-the-art facilities, enable them to efficiently develop and deliver high-quality products. By maintaining a patient-first approach, Andelyn continues to be a preferred contract development and manufacturing organization (CDMO) for those pioneering significant advancements in gene therapy.

Conclusion

Through the collaboration between Andelyn Biosciences and Tern Therapeutics, there is hope on the horizon for families affected by CLN2 Batten disease. As research and development in gene therapy continue to advance, partnerships like these are essential in creating effective treatments, which not only ease devastating health conditions but also enhance the overall quality of life for patients. Together, they embody a commitment to enriching lives and providing solutions in the face of challenging medical conditions.

For more information about Andelyn Biosciences and their extensive capabilities, visit andelynbio.com. To learn more about Tern Therapeutics and their mission, check out terntx.com.