Diorasis Therapeutics and Northway Biotech Partner to Advance Glaucoma Gene Therapy Manufacturing
Boston, MA and Vilnius, Lithuania – July 29, 2025 – A significant collaboration has been announced in the field of ophthalmic gene therapy, as Diorasis Therapeutics (DTx), a preclinical-stage biotechnology company, has partnered with Northway Biotech (NBT), a contract development and manufacturing organization (CDMO). This alliance aims to establish a scalable cGMP (current Good Manufacturing Practice) production process for DTx's lead AAV-based gene therapy candidate for open-angle glaucoma. While this news directly addresses glaucoma, a condition distinct from inherited retinal diseases (IRDs), advancements in gene therapy manufacturing are crucial for the entire ocular gene therapy landscape, including future treatments for IRDs.
The partnership is a critical step for Diorasis Therapeutics as it works to advance its lead gene therapy candidate toward clinical trials. Northway Biotech will leverage its two decades of experience in biologics manufacturing to develop a robust and compliant process for producing materials needed for both preclinical and clinical studies. This collaboration encompasses AAV process development, analytical method development and qualification, and the manufacturing of cGMP drug substance and drug product.
Diorasis Therapeutics is focused on developing disease-modifying gene therapies for ophthalmic diseases, specifically targeting intraocular pressure (IOP) lowering for glaucoma and neuroprotection. Their lead candidate, DT003, is designed to address the dysfunctional outflow of aqueous humor in glaucoma through a single, in-office injection. This approach aims to restore the eye's natural drainage system by delivering a modified tissue plasminogen activator (tPA) gene directly to trabecular meshwork cells. Unlike gene therapies that target specific mutations, DT003 offers a gene-independent approach, making it potentially applicable to a broad range of open-angle glaucoma patients, including both adult and juvenile forms. Preclinical models have shown DT003 to achieve durable IOP reduction to normal levels. The company's mission is to transform glaucoma care with a long-lasting treatment that addresses the root cause of elevated IOP, which can lead to irreversible blindness.
For patients and families within the IRD community, this partnership, while focused on glaucoma, signifies the broader progress being made in ocular gene therapy. The development of efficient and scalable manufacturing processes for AAV (adeno-associated virus) gene therapies is a shared challenge across all ophthalmic gene therapy programs. Improved manufacturing capabilities mean that once a gene therapy for an IRD is proven safe and effective, the path to widespread availability could be smoother and faster. It underscores the ongoing commitment within the biotech industry to develop innovative, long-term solutions for various blinding eye conditions.
Diorasis Therapeutics' lead program in open-angle glaucoma is expected to enter clinical development following the completion of toxicology studies and regulatory submissions. This collaboration with Northway Biotech represents a crucial step in bringing this potential treatment closer to patients.