Ascidian and Exegenesis Bio Enter Strategic Agreement to Accelerate Development of RNA Exon Editors with Next-Generation AAV Capsids

Combines RNA exon editing with AI-enabled capsid discovery to
support targeted delivery across multiple tissue types

Flexible, non-exclusive framework enables program-specific
capsid selection and preserves manufacturing options

BOSTON and PHILADELPHIA, Nov. 12, 2026 /PRNewswire/ -- Ascidian Therapeutics, a biotechnology company seeking to treat human diseases by rewriting RNA, and Exegenesis Bio, a global gene therapy company with proprietary AI-enabled optimized AAV capsid discovery capabilities, today announced a strategic agreement combining Ascidian's RNA exon editing technology with Exegenesis Bio's capsid engineering and discovery workflows. The partnership enables novel delivery approaches tailored to the specific clinical needs of individual programs. Collaborative scientific work underway will accelerate in vivo data generation and progression toward clinical development for a suite of RNA exon editors targeting devastating diseases.

Achieving effective delivery at a tolerable dose remains a major bottleneck in the development of genetic therapies. Engineered, tissue-specific AAV capsids can direct therapeutic payloads to target cell types and minimize exposure to non-target cells, improving patient safety, reducing cost of goods, and maximizing therapeutic benefit. To identify the right delivery capabilities for its RNA exon editing platform, Ascidian conducted a global search for AAV capsid engineering partners. The resulting agreement brings together complementary technologies to evaluate combinations of RNA exon editors and next-generation capsids suited to the needs of individual programs.

"The right delivery approach is essential to realizing the power of RNA exon editing," said Daniel Rosan, President, Chief Financial and Business Officer of Ascidian Therapeutics. "Exegenesis Bio's capsid engineering and AI-enabled discovery capabilities give Ascidian flexibility to match delivery strategies to the needs of each patient population and develop RNA exon editors across a broader range of devastating diseases."

"Pairing advanced capsid engineering with innovative therapeutic modalities creates opportunities to address complex clinical challenges," said Zhenhua Wu, Ph.D., Chief Executive Officer of Exegenesis Bio. "Powered by AAVarta^®, our AI-assisted capsid engineering platform, we have established innovative capsid engineering technologies that allow us to identify and optimize capsids with distinct delivery profiles, bringing additional options to novel payload developers, such as Ascidian, and expanding the reach of RNA exon editing across multiple tissue types."

A Flexible Framework to Scale Program-Specific Delivery

Under the agreement, the companies will evaluate Exegenesis Bio's AAV capsids and capsid discovery capabilities against the delivery requirements of individual RNA exon editing programs. Rigorous scientific considerations and defined target capsid profiles will guide the selection of complementary RNA exon editor-capsid combinations for in vivo and clinical development. Ascidian will carry out further execution of resulting RNA exon editor-capsid combinations.

The non-exclusive framework allows Ascidian to work with other capsid providers as needed and to select clinical and commercial manufacturing partners based on program needs. Exegenesis Bio retains the ability to independently, or with partners, further develop the relevant capsids outside of Ascidian's RNA exon editor programs.

Specific target indications, program details, and financial terms are not being disclosed.

About Ascidian Therapeutics

Ascidian Therapeutics is redefining the treatment of disease by rewriting RNA. By editing exons at the RNA level, Ascidian therapies enable precise post-transcriptional editing of genes, resulting in full-length, functional proteins at the right levels, in the right cells, at the right time. With partnered and wholly owned programs in retinal, renal, neurological, neuromuscular, and genetically defined diseases, Ascidian's approach has the potential to treat patients with one dose of an RNA exon editor, opening new therapeutic possibilities for patients and their families who are seeking breakthroughs. Learn more at Ascidian.com, or for Ascidian's currently enrolling clinical trials in Stargardt disease, visit AscidianClinicalTrials.com. 

About Exegenesis Bio

Exegenesis Bio is a clinical-stage biopharmaceutical company focused on discovering and developing gene therapies for neurological and ophthalmic diseases. Exegenesis Bio's pipeline also includes oligonucleotide-based therapeutics. Built upon AAVarta^®, an AI-enabled capsid discovery platform, the company has established optimized AAV capsid engineering capabilities. By seamlessly integrating AI algorithms with high-throughput experimental screening, the company continuously drives innovation in delivery vectors, improving tissue specificity and delivery efficiency, and thereby enhancing the safety and efficacy of genetic medicines. For more information, please visit https://exegenesisbio.com.

SOURCE Ascidian Therapeutics